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- 2025-12-17 14:30:15
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- "Trodelvy for TNBC secures evidence for expanded indication"
- by Son, Hyung Min Nov 27, 2025 06:14am
- The therapeutic paradigm for Triple-Negative Breast Cancer (TNBC) is entering a new phase. Gilead's Trodelvy (sacituzumab govitecan), a Trop-2 targeted Antibody-Drug Conjugate (ADC), demonstrated significant results in the Phase 3 ASCENT-03 study, which evaluated the efficacy of this drug as a first-line monotherapy. The study proves Trodelvy's potential to expand beyond its current second-line indication. DailyPharm met with the authors for the ASCENT-03 trial conducted in South Korea, Professor Joohyuk Sohn (Division of Medical Oncology at Yonsei Cancer Center) and Professor Yeon Hee Park (Department of Hematology- Oncology at Samsung Medical Center), to discuss the evolving TNBC treatment landscape and the clinical value of Trodelvy. (from left) Professor Yeon Hee Park of the Department of Hematology- Oncology at Samsung Medical Center , Professor Joohyuk Sohn of the Division of Medical Oncology at Yonsei Cancer Center At the European Society for Medical Oncology (ESMO 2025) Annual Congress held last month in Berlin, the Phase 3 study evaluating Trodelvy's efficacy was unveiled. The trial enrolled patients with unresectable locally advanced or metastatic TNBC who were either PD-L1 negative (CPS below 10) or PD-L1 positive (CPS over 10) but ineligible for immune checkpoint inhibitor therapy. In the trial, Trodelvy recorded a median Progression-Free Survival (PFS) of 9.7 months, which was significantly longer than the 6.9 months with chemotherapy. Furthermore, Trodelvy reduced the risk of disease progression or death by 38% compared to the control group. While Overall Survival (OS) data were not yet mature at the time of the primary analysis, the widening gap in PFS2 (time from randomization to second progression) between the treatment and control groups suggests a potential for future OS improvement. There has been lack of distinct first-line options in untreated metastatic TNBC, besides the immune checkpoint inhibitor 'Keytruda (pembrolizumab)'. Notably, for the PD-L1 negative patient group who are ineligible for pembrolizumab, no viable alternatives to conventional chemotherapy existed. This is why analysis indicates the potential for greater use of Trodelvy. A particularly noteworthy aspect of this study was the design for allowance of crossover from the control group. Control patients whose cancer progressed during the trial were permitted to receive Trodelvy as a salvage chemotherapy. Offering crossover to an agent already proven to improve OS in a subsequent-line setting typically makes it statistically much harder to demonstrate a difference in OS for the primary outcome. Despite this challenge, the investigators chose this patient-centric design, which was evaluated at ESMO as 'a decision reflecting confidence in the drug's efficacy and the study results.' Experts assert that these results reaffirm the clinical value of Trop-2 targeted ADCs, anticipating this will be a pivotal turning point that significantly expands treatment options in the previously limited TNBC space. Q. What was the first-line treatment landscape for TNBC like before the introduction of ADCs? Prof. Park: TNBC is characterized by a lack of traditional targets, and treatment typically stratifies based on PD-L1 expression. In the PD-L1 positive patient group, immune checkpoint inhibitors combined with chemotherapy have shown good results and become the standard of care, leading to PFS improvement, despiting having been less than a year. In contrast, the PD-L1 negative patient group, which comprises the majority (about 60%) has seen virtually no new treatment options for the past 20 years. When clinical trials succeeded for the first time in PD-L1 positive patients, we had patients requesting the combination, but the evidence was lacking for the negative group. Patients with aggressive progression, young age, or recurrence within a year of early-stage adjuvant therapy had to rely on chemotherapy, despite its toxicity. Professor Joohyuk Sohn of the Division of Medical Oncology at Yonsei Cancer CenterProf. Sohn: From an oncologist's view, TNBC is challenging. For nearly 20 years, standard treatment has been limited to conventional chemotherapy agents like taxanes or Xeloda (capecitabine). It is concerning because this cancer has a higher incidence in younger patients, and the restricted options have been a constant source of clinical difficulty. The prognosis for TNBC is notoriously poor. Patients who appear healthy at initial diagnosis can unfortunately pass away in as little as 18 months, a devastating reality. While the introduction of immunotherapies and targeted agents has been helpful, their use is restricted to specific patient groups, underscoring the desperate need for better options. Q. Please elaborate on the key results and significance of the ASCENT-03 study.. Prof. Sohn: PD-L1 This study is highly encouraging because it demonstrated a statistically and clinically meaningful extension of PFS compared with standard-of-care chemotherapy in the first-line treatment of PD-L1-negative TNBC. We anticipate these results will position Trodelvy as a new standard of care in first-line TNBC, offering tangible benefits to many patients. Prof. Park: ASCENT-03 garnered significant attention for presenting the first data in the first-line PD-L1-negative TNBC setting in approximately 20 years. Presenting a new possibility for this patient group makes the results highly meaningful. The primary endpoint of ASCENT-03 was PFS. While the control group, despite utilizing best efforts with existing standard treatments, only achieved 6.9 months, the Trodelvy group reached 9.7 months, which is highly encouraging. Q. What were the unique aspects of the study design? Professor Yeon Hee Park of the Department of Hematology- Oncology at Samsung Medical Center Prof. Park: Several points make this study design stand out. First, the control group included not only single-agent chemotherapies (paclitaxel, albumin-bound paclitaxel) but also combination chemotherapy (gemcitabine-carboplatin). Second, the study registered a significant proportion of patients with a relatively poor prognosis. Trials typically exclude patients who relapse within one year of completing adjuvant therapy. This study, however, included patients who relapsed between 6 and 12 months after adjuvant therapy. Furthermore, some patients had previously received adjuvant immunotherapy. These are patients for whom selecting treatment choices are tough after relapse and metastasis. The most notable feature was the allowance for crossover from the control group. Trodelvy has already demonstrated OS improvement in the subsequent-line setting and is used in clinical practice. It is rare for a new trial to permit crossover to an agent with known survival benefits for ethical and statistical reasons, as it complicates OS analysis and makes it difficult to prove the drug's effect. This was essentially a high-stakes decision, reflecting the research team's confidence in the drug's efficacy. The choice to prioritize the patient perspective by allowing crossover, despite the statistical risk, was highly evaluated. While OS data are incomplete, the study showed a significant effect in the surrogate endpoint, PFS2. Even with the crossover allowance in the control group, the PFS2 was markedly longer in the Trodelvy treatment group. This result clearly demonstrates the critical impact of the initial treatment choice on the patient's overall therapeutic course. The study successfully achieved both meticulous design and uncompromising evidence of efficacy. Q. On the day of presentation of ASCENT-03 data, TROPION-Breast02 data for the competing drug Dato-DXd have also been presented. How do you project the OS outcome for ASCENT-03, given the incomplete data? Prof. Park: ESMO 2025 was a major stage for breast cancer research. Unlike Hormone Receptor-positive or HER2-positive breast cancers, where prognosis has rapidly improved, TNBC has lacked attention. However, this year saw the release of significant data across all three subtypes, with ASCENT-03 and TROPION-Breast02 at the center. There is a strong possibility of an OS improvement with Trodelvy in TNBC. The basis for this is the PFS2 data. Despite the fact that most patients (82%) in the control group who received subsequent therapy crossed over to Trodelvy, the difference in PFS2 between the Trodelvy-treated group and the control group continues to widen. This trend suggests a positive OS outcome is anticipated. A direct comparison between ASCENT-03 and TROPION-Breast02 is challenging due to entirely different trial designs. First, the statistical design and analytical structure differ based on the primary and secondary endpoints. ASCENT-03 set the primary endpoint to PFS alone, while TROPION-Breast02 set it to both PFS and OS. Furthermore, ASCENT-03 allowed crossover to a standard treatment known to improve OS, making the interpretation of the results necessarily more complex. Prof. Sohn: Given the crossover design, there is a possibility that a statistically significant OS difference may not yield even with long-term follow-up. However, since the primary endpoint, PFS, has already been met, there may not be any issues with regulatory approval or clinical use. The fact that Dato-DXd achieved a significant improvement in OS is certainly encouraging. However, direct comparison is difficult due to the design differences. It's also inappropriate to definitively assess superiority or determine which drug is necessary in clinical practice solely on the basis of the presence or absence of OS data. The important part is that both agents met their primary endpoints, diversifying the available first-line treatment options for TNBC. Q. We understand the ASCENT-04 study, evaluating the efficacy and safety of Trodelvy + Keytruda combination therapy, is also underway. Can you share those clinical results? Prof. Sohn: Similar to ASCENT-03, the PFS for patients treated with the Trodelvy + Keytruda combination was statistically significantly longer (11.2 months vs. 7.8 months) than for the chemotherapy + Keytruda combination group. Taken together, these results indicate a high probability that Trodelvy will be established as a new standard of care in the first-line setting, regardless of PD-L1 expression. Prof. Park: This study was also conducted on a large scale. The ability to enroll many patients stemmed from the opportunity for control group patients to access the Keytruda combination therapy, which is not covered by national health insurance. The ASCENT-04 results are also highly encouraging, showing a PFS of nearly 1 year with the Trodelvy + Keytruda combination group. Despite these significant results, the lack of FDA approval at this time is surprising. Q. Q. What is your view on the positive performance of Trop-2 targeted ADCs recently? Prof. Sohn: Oncology research often begins with breast cancer. Breast cancer has the highest incidence among female cancers and carries significant symbolic weight. Furthermore, most research is conducted using cell lines, and breast cancer cell lines have historically constituted the largest proportion. The biological characteristics of breast cancer are well-understood, leading to active clinical trials for new drugs. The high expression rate of Trop-2 in breast cancer makes it particularly responsive to Trop-2-targeted ADCs. While research is ongoing in other cancer types, the response has not been as pronounced as in breast cancer. Prof. Park: The efficacy is due to the high Trop-2 expression in breast cancer, combined with the characteristics of ADCs, where the cytotoxic payload is broadly distributed within the tumor cell and has a sustained effect. Trop-2 serves as the guide that helps direct the payload to the cancer cell. ADCs enhance therapeutic effects by enabling targeted cancer cell killing while retaining the mechanisms and cytotoxicity of traditional chemotherapy. While conventional chemotherapy is highly toxic and difficult to use long-term, ADCs can be regarded as a treatment option that can be used safely and stably for a relatively longer period.
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- Primary biliary cholangitis drug Livdelzi’s launch imminent
- by Eo, Yun-Ho Nov 27, 2025 06:14am
- A new treatment for primary biliary cholangitis (PBC), Livdelzi, may soon enter the Korean market. Gilead Sciences Korea recently submitted a marketing authorization application for Livdelzi (seladelpar), an oral therapy for adults with PBC who show inadequate response or intolerance to ursodeoxycholic acid (UDCA). The application is currently under review by the Ministry of Food and Drug Safety. Livdelzi was designated as a Global Innovative product on Fast Track (GIFT) in June and as an orphan drug in August in Korea. PBC is a rare, intractable autoimmune disease characterized by chronic inflammation and destruction of intrahepatic bile ducts. This leads to cholestasis and liver damage, which ultimately progresses to cirrhosis or liver failure. While ursodeoxycholic acid (UDCA) is currently used as the first-line treatment, there has been a persistent need for new therapeutic strategies for patients who have an insufficient response to or intolerance to UDCA. Seladelpar is an orally administered drug that selectively acts on the peroxisome proliferator-activated receptor (PPAR) delta. The drug demonstrated efficacy in the Phase III RESPONSE trial. The RESPONSE trial was a randomized, double-blind, placebo-controlled trial evaluating the efficacy and safety of seladelpar versus placebo in adult PBC patients with an inadequate response to or intolerance of UDCA. Results showed that 62% of the seladelpar group achieved the primary endpoint of composite biochemical response (improvement in ALP and total bilirubin levels) at 12 months, demonstrating statistically significant superiority compared to 20% in the placebo group. Notably, 25% of the seladelpar treatment group achieved normalization of ALP, a meaningful clinical finding. Seladelpar also showed benefit in the key secondary endpoint of pruritus improvement. Among patients with moderate-to-severe pruritus at baseline, seladelpar reduced itch scores by 3.2 points at month 6, compared with 1.7 points in the placebo group, again demonstrating a statistically significant improvement. Livdelzi received accelerated approval from the U.S. FDA in August last year and was also approved in Europe in February this year.
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- Korea’s US pharma exports up 43% this year
- by Kim, Jin-Gu Nov 27, 2025 06:13am
- Korea’s cumulative pharmaceutical exports have surpassed KRW 10 trillion. Exports through October increased 18% year-on-year, continuing their strong performance. Industry observers expect this export momentum to continue following the final resolution of the U.S.-Korea pharmaceutical tariff negotiations, which had been the biggest source of uncertainty. October cumulative pharmaceutical exports up 18%... hints at record high performance According to the Korea Customs Service, as of the 17th, cumulative pharmaceutical exports for the year to date reached USD 7.30631 billion (approximately KRW 10.65 trillion). This represents an 18% increase compared to the USD 6.20242 billion (approximately KRW 9.04 trillion) recorded during the same period last year. Korea surpassed the KRW 10 trillion export milestone 1 month earlier than the previous year. If this trend continues, exports are expected to exceed the previous all-time high set in 2021. At that time, annual pharmaceutical exports soared to USD 8.12125 billion, largely driven by the COVID-19 pandemic-related surge. During this period, exports surged significantly as exports of domestically produced COVID-19 vaccines began to be exported in earnest. Annual pharmaceutical exports (Unit: USD 1 million, Source: Korea Customs Service) Meanwhile, pharmaceutical imports fell 1%, from USD 7.672 billion to USD 7.614 billion. With exports increasing substantially and imports decreasing slightly, the pharmaceutical trade balance improved significantly. The cumulative pharmaceutical trade deficit for October stands at a deficit of USD 308.24 million. This represents a reduction to one-fifth of the USD 1.4698 billion deficit recorded in the same period last year. US exports surge 43% year-on-year... Favorable trend expected to continue following tariff agreement Export performance to the U.S. showed strong results. Cumulative exports of domestic pharmaceuticals to the US in October reached USD 1.56555 billion (approximately KRW 2.28 trillion), a 43% increase compared to USD 1.09767 billion (approximately KRW 1.6 trillion) in the same period last year. The US share of total pharmaceutical exports also expanded by 3 percentage points compared to the same period last year. The US's export share, which was around 18% in October last year, rose to 21% this year. This is interpreted as a result of domestic pharmaceutical and biotech companies actively responding to concerns over potential U.S. government tariffs. Companies like Samsung Biologics and Celltrion secured large inventories by preemptively exporting pharmaceuticals to their US subsidiaries in preparation for tariff concerns. In the mid-to-long term, they plan to secure production facilities locally in the U.S. Monthly Korean pharmaceutical exports to the US(Unit: USD 1 million, Source: Korea Customs Service) Exports to Canada, major European countries, Japan, and China also increased significantly. Cumulative exports to Canada in October rose 81% year-on-year, from USD 34.59 million to USD 62.43 million. Exports to Switzerland surged 141% to USD 870.78 million, Germany rose 35% to USD 703.95 million, the Netherlands jumped 239% to USD 462.17 million, and France soared 473% to USD 91.56 million. Exports to Japan rose 16% from USD 261.52 million to USD 302.51 million, while exports to China increased 36% from USD 132.63 million to USD 180.49 million. With the recent resolution of the pharmaceutical tariff negotiations with the US, the industry's biggest variable, the pharmaceutical export boom, particularly the US-bound exports, is expected to continue. On October 29, at the APEC Summit in Gyeongju, the two countries agreed to apply Most-Favored-Nation (MFN) tariff treatment to pharmaceuticals. The core of the agreement is that Korean pharmaceuticals in the US will receive MFN treatment, similar to Japan and the EU, with a maximum tariff rate of 15% On the 14th, the details of the agreement reached in the follow-up tariff negotiations were disclosed. The White House released a Korea-US Joint Fact Sheet (JFS) from the summit, stating that the two countries agreed tariffs on Korean pharmaceuticals would not exceed 15%. Also, generic drugs will be fully exempt from tariffs. However, industry assessments indicate that tariff uncertainties remain. The fact sheet did not precisely specify the tariff scope for biosimilars. It remains undetermined whether biosimilars will be included in the generic category to receive zero tariffs or be subject to separate assessment.
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- ADC MM drug Blenrep set for commercialization in Korea
- by Eo, Yun-Ho Nov 26, 2025 06:09am
- The new multiple myeloma drug Blenrep is expected to soon become commercially available in Korea. The Ministry of Food and Drug Safety (MFDS) is currently conducting the final review for the approval of GSK Korea’s Blenrep (belantamab mafodotin), the first anti-BCMA (B-cell maturation antigen) antibody-drug conjugate (ADC) in its class, developed by GSK Korea. Industry expects the approval could come as early as this year. Blenrep was approved in Europe last July and in the United States last October. The anticipated approved indications for Blenrep are ‘in combination with bortezomib and dexamethasone (BVd) in adult patients with relapsed or refractory multiple myeloma who have received one or more prior therapies,’ and ‘pomalidomide plus dexamethasone (BPd) in patients who have received one or more prior therapies, including lenalidomide (Revlimid).’ This drug demonstrated efficacy through the Phase III DREAMM-7 trial. In patients who had received at least 2 prior therapies, Blenrep combination therapy reduced the risk of death by 51% compared to the Darzalex (daratumumab)-based triple regimen (DVd). The median progression-free survival (PFS) was 31.3 months for the Blenrep combination therapy group versus 10.4 months for the Darzalex-based triple regimen group, representing approximately a threefold improvement in the Blenrep combination therapy group. The safety and tolerability profile of the Blenrep combination was generally consistent with what is known for the individual agents. However, the Blenrep arm showed a higher incidence of ocular toxicity, such as keratopathy and vision changes. Due to this safety concern, the FDA advisory committee voted against Blenrep’s reapproval in July. Meanwhile, GSK is conducting a clinical program to demonstrate Blenrep’s potential benefit in earlier-line treatment settings. Overall survival follow-up for the DREAMM-7 and DREAMM-8 trials, which include patients who received at least one prior line of therapy, is ongoing. Results from these studies are expected to be released in 2028.
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- Bispecific antibody opens new era of DLBCL treatment
- by Son, Hyung Min Nov 26, 2025 06:09am
- The treatment landscape for diffuse large B-cell lymphoma (DLBCL) is changing quickly. Roche's bispecific antibody, which was previously a third-line option, received an expanded indication for second-line treatment, broadening the therapeutic strategy for DLBCL. Given the aggressive nature of DLBCL, where prognosis rapidly worsens after first-line failure, there is significant attention on whether a more potent combination treatment and a new mechanism of action can increase patients' chances of survival. On November 25, Roche Korea held an event at its headquarters in Gangnam-gu, Seoul, to discuss the unmet needs in the DLBCL treatment landscape and the clinical value of its new drug. Roche has already launched products in this field, including Polivy (polatuzumab vedotin) and the bispecific antibody 'Columvi (glofitamab)'. Currently, Polivy can be used as a first-line treatment in combination with cyclophosphamide, doxorubicin, and prednisone (R-CHOP), and Columvi can be used for subsequent lines of treatment. In July, Columvi received an expanded indication from third-line to second-line treatment. The specific indication is for adult patients with relapsed or refractory DLBCL not otherwise specified (DLBCBL NOS) who are ineligible for autologous stem cell transplantation (ASCT), in combination with gemcitabine and oxaliplatin. Professor Seok Jin Kim of the Department of Hematology-Oncology at Samsung Medical CenterColumvi has a 2:1 structure, in which two binding domains target the CD20 surface antigen expressed on malignant B cells, and one domain targets the CD3 antigen expressed on immune T cells. This structure allows for a more robust and stable binding. The efficacy of Columvi was confirmed through the Phase 3 STARGLO study. The trial included patients with relapsed or refractory DLBCL who were ineligible for ASCT after one or more prior systemic therapies, or who had a history of two or more prior systemic therapies. The two-year follow-up results demonstrated that the Columvi + gemcitabine + oxaliplatin combination therapy reduced the risk of death by 41% compared with rituximab + gemcitabine + oxaliplatin combination therapy. The Columvi combination group's Progression-Free Survival (PFS) was 13.8 months, approximately a four-fold increase from 3.6 months in the rituximab combination group. The rate of patients achieving Complete Response (CR) was higher in the Columvi combination group (58.5%) than the control group (25.3%). Professor Seok Jin Kim (President of the Korean Society of Hematology) of the Department of Hematology-Oncology at Samsung Medical Center commented, "While remission can be reached, maintaining it is not easy. Patients in the Columvi combination group survived without disease progression for even one year after the end of treatment. This is a significant finding." Still high unmet needs in DLBCL…"more treatment options must be secured" DLBCL is a disease in which B cells, which protect the body, grow or multiply uncontrollably. It is the most common subtype, accounting for about 40% of Non-Hodgkin Lymphomas. The disease is characterized by its aggressive nature, with rapid progression through stages. The number of DLBCL patients in South Korea was 14,183 as of last year, a 36% increase from 10,428 in 2018. Up to 15% of DLBCL patients fail to respond to the first-line standard treatment, and among those who achieve CR, 25% experience relapse within 18 months. Patients with relapsed/refractory DLBCL face a rapidly deteriorating prognosis as the line of treatment increases. Seunghun Lee, Medical Lead at RochePolivy combination therapy is known to be effective in about two-thirds of patients when used as first-line treatment. However, this means that approximately one-third of patients do not benefit from first-line treatment. Chimeric Antigen Receptor T-cell (CAR-T) therapies and bispecific antibodies are now available for subsequent treatment lines. Currently, second-line treatment options include Gilead's CAR-T drug 'Yescarta (axicabtagene ciloleucel)' and the antibody drug Columvi. For the third line, Novartis's ''Kymriah (tisagenlecleucel)' and AbbVie's 'Epkinly (epcoritamab)' are utilized. However, all options except Kymriah are currently non-reimbursed options. Professor Kim stressed, "CAR-T and bispecific antibodies are not to be compared directly. Patients who can tolerate the side effects choose CAR-T, and those who cannot choose the bispecific antibody. If all new drugs were reimbursed, prescribing would align with global guidelines. It is not appropriate to evaluate which drug is superior," pointing out, "The problem is that even when effective treatment options exist, patients are forced to repeat the same treatment due to regulatory approval and reimbursement hurdles." Seunghun Lee, Medical Lead at Roche, said, "The reason the U.S. FDA rejected Columvi for second-line approval was that it did not meet the criteria for the number of enrolled patients, but since it has been approved in major countries in Europe and Asia, we believe the racial differences are not significant," and emphasized, "Roche is currently strengthening its leadership by launching a variety of treatments for hematologic malignancies. We will continue to expand the range of choices in the DLBCL area to enable personalized treatment strategies for different patient groups."
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- Bio investments hit 3yr high...investor sentiment recovers
- by Cha, Jihyun Nov 26, 2025 06:09am
- Venture capital (VC) new investments in the biotech and medical sectors during the third quarter of this year reached their highest level in 3 years, both in scale and proportion. Quarterly investment volume increased by 56% compared to the previous quarter, and the bio/medical VC investment share within the industry rose to 20%. This is interpreted as a clear sign of recovery of the once-frozen biotech investment market. According to the Korea Venture Capital Association on the 25th, new investments in the bio-medical sector during the third quarter totaled at KRW 396.9 billion. This is a sharp 56% increase from the previous quarter and a 20% rise compared to the same period last year. While overall new VC investments expanded by 33% quarter-on-quarter during the same period, the bio/medical sector's investment amount showed a much steeper growth trajectory, effectively driving the market’s rebound. The trend of increased investment in the bio-medical sector is also evident in the share indicators. The bio/medical sector accounted for 20% of all new VC investments in Q3, up 3 percentage points from the previous quarter. The fact that the share of bio-medical investments expanded despite the overall new investment pie growing larger quarter-on-quarter indicates a strong shift in capital toward biotechnology over other industries. As a result, new investments in the bio/medical sector this quarter reached their highest level in the past 11 quarters, both in absolute investment and share. This signals a full revival of previously subdued investment sentiment, with analysts suggesting the bio/medical investment trend has moved beyond a short-term rebound and entered a phase of structural recovery. (Source: Korea Venture Capital Association) Investment in the biotech/medical sector peaked at KRW 1.677 trillion in 2021 during the COVID-19 pandemic and has been on a steep decline since. Affected by interest rate hikes and economic uncertainty, investment fell to KRW 1.1058 trillion in 2022 and further declined to KRW 884.4 billion in 2023, marking two consecutive years of contraction. The bio investment market began showing signs of a rebound last year. New investment in the bio/medical sector in 2024 reached KRW 1.0695 trillion, a 20% increase year-on-year, marking a shift in the trend. Looking at quarterly figures, the market's recovery became clearly evident last year when KRW 331.6 billion poured in during the third quarter. This recovery became even more pronounced this year. Quarterly investments have continued to rise, clearly showing that the market is in a full restoration phase. Particularly significant is this third quarter's investment volume, which significantly surpassed the record set in Q3 2024 that drove last year's rebound, but also represents the largest quarterly figure since the downturn began in 2023. The biotech industry attributes this surge in investment to: ▲ the completion of valuation adjustments, ▲ an increase in companies delivering clinical results, and ▲ expectations of interest rate cuts. Specifically, it is analyzed that VCs, who maintained a conservative stance until the first half of the year, began betting on ‘high-quality companies’ with solid technological capabilities in the second half, driven by pressure to deplete their dry powder (uncommitted investment funds). Some view the first-quarter investment cliff as having actually heightened valuation attractiveness, creating bargain-buying opportunities for companies with solid technology foundations. Looking ahead, projections suggest VC funding will enter a more pronounced phase of ‘separating the wheat from the chaff,’ focusing squarely on technological prowess. Unlike the past when investments were based solely on growth potential, the trend of capital concentrating on companies with verifiable substance—such as clinical data and commercialization feasibility—is expected to continue for the foreseeable future.
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- Biogen attempts Qalsody reimb listing in Korea
- by Eo, Yun-Ho Nov 25, 2025 06:14am
- The new ALS drug Qalsody (tofersen) has begun the process of applying for reimbursement coverage in Korea. Since receiving domestic approval in August, the company has been moving rapidly to seek its reimbursement. According to industry sources, Biogen Korea’s Qalsody, a treatment for amyotrophic lateral sclerosis (ALS) associated with SOD1 (Superoxide Dismutase 1) gene mutation, is scheduled to be reviewed by the Drug Reimbursement Standard Subcommittee. Before approval, Qalsody was designated a GIFT (Global Innovative Products on Fast Track) item and as an orphan drug by the Ministry of Food and Drug Safety/ However, the road ahead is expected to be rocky due to its extremely high price. In the U.S., Qalsody costs USD 15,097 per vial (approx. KRW 22.03 million), and the annual treatment cost reaches USD 211,358 (approx. KRW 308.5 million). Biogen submitted a reimbursement application immediately after approval in August, but faced its first hurdle when reviewers requested additional data. It now awaits the Reimbursement Standards Subcommittee review on the 28th. Should the subcommittee require further review or additional data, the reimbursement schedule could face further delays due to re-review. ALS, particularly SOD1 gene mutation ALS, falls into the category of ultra-rare diseases, with fewer than 100 patients worldwide. Qalsody is the first targeted therapy developed specifically for this patient group. Because ALS presents widely variable symptom onset patterns among patients, clinical trial design and endpoint determination are extremely challenging. Experts note that the emergence of the first treatment option showing potential for improving disease progression, even in just one subset of patients, holds significant meaning in ALS, a field where drug development is exceptionally difficult. Given the U.S. Trump administration’s MFN drug-pricing stance and Korea’s historically restrictive approach to rare-disease reimbursement, experts warn that if reimbursement criteria become too narrow, the possibility of the drug's domestic launch being canceled cannot be ruled out. It remains to be seen whether Qalsody will become a reimbursed treatment option in an area with virtually no therapeutic alternatives. Meanwhile, in the Phase III VALOR study, Qalsody did not meet the primary endpoint, the ALSFRS-R functional score. However, it demonstrated reductions in the secondary endpoints: a 26-38% decrease in total SOD1 protein cerebrospinal fluid and a 48-67% decrease in plasma neurofilament light (Nfl) concentration.
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- "Disparity btwn innovation speed and policy… Economic Eval"
- by Son, Hyung Min Nov 24, 2025 06:19am
- Forum As the South Korean positive list system for pharmaceutical reimbursement marks its 20th anniversary, discussion has begun on how to reflect the value of innovative new drugs within the existing economic evaluation system. With the acceleration of technological development and the surge of high-cost innovative treatments for ultra-rare diseases, critics argue that the current framework, which focuses on cost-effectiveness, can no longer sustain the balance between patient access and financial sustainability. At the forum, '20 Years of the Positive List System for Pharmaceutical Reimbursement: Value-Based System Where Innovation Meets Regulation,' held in the National Assembly on November 21, academia, government, industry, and the media all agreed on the necessity of 'modernization of economic evaluation.' The event was jointly hosted by Democratic Party Rep. Seo Young-seok and Rep. Kim Yoon, and Reform Party Rep. Lee Joo-young, and organized by the ISPOR Korea Chapter. The system focused on cost-effectiveness limits evaluation of innovative new drugs Korea's economic evaluation system was introduced in 2006 with the government's announcement of the Drug Expenditure Rationalization Plan (DERP). The PLS, introduced the following year, established the principle that all drugs must be jointly verified for clinical usefulness and economic value through the economic evaluation process, with only those drugs proven to be cost-effective included in the reimbursement list. Although the Economic Evaluation Exemption track was established in 2015 to provide an exceptional pathway, the principle that cost-effectiveness proof is mandatory for new drug reimbursement has persisted for 20 years. However, the industry points out that this cost-effectiveness-centric structure clashes with the rapidly evolving paradigm of innovative new drugs, creating a growing gap between regulation and technology that cannot be ignored. BaeChan Kim, Unit Head at Boehringer Ingelheim Korea, directly criticized the current economic evaluation framework, stating that it has failed to keep pace with changes in treatment paradigms over the last decade. Kim said, "The problem is that as the speed of innovation has accelerated, the complexity of clinical evidence has increased exponentially," and added, "The expansion of areas where standard Randomized Controlled Trials (RCTs) are practically impossible, such as ultra-rare disease treatments, has led to an increase in innovative new drugs approved based only on single-group trials. However, the current economic evaluation and reimbursement system is still aligned with the RCT standard from 20 years ago. Economic evaluation requires evolution, not reinforcement." Kim highlighted Korea's position by citing international comparisons. According to data from the U.S. Pharmaceutical Research and Manufacturers of America (PhRMA), analyzing reimbursement rates for innovative new drugs across nine developed countries, Australia, Korea, and Canada fall behind significantly, with reimbursement rates about half the average. Kim pointed out, "It is a contradictory structure where countries called 'innovation leaders' are actually the furthest behind in access to innovative new drugs." There was also criticism that limiting economic evaluation to a cost-effectiveness-centric structure is no longer realistic. Lee Sang-il, Head of the Department of Policy Planning at the National Evidence-based Healthcare Collaborating Agency (NECA), assessed, "The challenges of securing both patient access and financial sustainability have become incomparably difficult compared to the past, due to accelerated aging, the emergence of ultra-high-cost innovative drugs, and increased uncertainty in clinical evidence," and said, "We are in an era where cost-effectiveness alone cannot explain even half of the decision-making process." Lee particularly cited the absence of entry evaluations and the failure to establish a post-market evaluation system as the biggest structural problems. Lee said, "For high-cost drugs where there is a disparity between RCT data and real-world clinical data, the insurer needs to conduct a health-economic evaluation directly or through objective verification by a third party. The sustainability of drugs entering via the Risk-Sharing Agreement (RSA) or economic evaluation exemption tracks cannot be guaranteed without post-market management." He emphasized the need to establish a Korean-style Managed Entry Agreement (MEA) that defines the post-market evaluation system concurrently with the listing decision. The event was jointly hosted by Democratic Party Rep. Seo Young-seok and Rep. Kim Yoon, and Reform Party Rep. Lee Joo-young, and organized by the ISPOR Korea Chapter. It has been confirmed that such a problem exists not only in the industry but also in the media covering the regulatory sector. This suggests that the difficulties in the field are repeatedly exposed in official discussions and in practical, real-world cases. Yun-Ho Eo, a journalist at DailyPharm, pointed out, "The core message from the field is that the reason for the increase in economic evaluation exemptions is not that companies want the exemption, but that the current health-economic evaluation itself has become impossibly difficult to handle." Eo added, "Companies are mobilizing law firms, economic evaluation expert agencies, and external consultants because they cannot manage the process using only in-house personnel," and stated, "This shows that Korea's economic evaluation procedures are extremely complicated and rigid on a global scale. Korea's low drug prices and slow listing speed are recognized as a policy reality by the industry." Agrees on modernizing the economic evaluation system...Has proposed directions to strengthen review and evaluation In response to concerns raised by industry and academia, the government acknowledged the limitations of the existing economic evaluation framework and agreed on the need for institutional improvement. However, it clarified that what is needed is not the reinforcement of the system, but its refined modernization. The government highlighted securing evaluation flexibility and expertise, and establishing a post-market management system that reflects actual clinical outcomes after listing, as the core directions for reform. (from left) Sook-hyun Lee, Head of HIRAYeon-sook Kim, Director of Pharmaceutical Management Division at MOHW, said, "Korea was the first country in Asia to adopt the economic evaluation system and has steadily expanded access through institutional evolution, including the Risk-Sharing Agreement (RSA) and Economic Evaluation Exemption." Kim noted, "However, the pace of change recently is fundamentally different from when the system was designed." Kim said, "In an era where innovative new drugs based on uncertainty are surging, the current evaluation method is no longer manageable. I realized the limitations firsthand after working in the field for five months," and added, "We cannot be satisfied with the current system." Kim then presented two tasks: first, the need to define 'innovativeness' more clearly and precisely in the guidelines adopted by HIRA; and second, the need to establish a structure that systematically evaluates actual clinical outcomes after listing and feeds that back into reimbursement. Kim said, "Post-market evaluation is fundamental to all healthcare programs, but it is not yet fully established in Korea. We must create a structure that confirms actual efficacy while minimizing the burden on the clinical and industrial sectors," and added, "Our system lacks flexibility and expertise compared to those of developed countries." We will develop the system in the most practical way possible." The Health Insurance Review & Assessment Service (HIRA) also acknowledged the conflict between technological innovation and the existing evaluation framework, stating that an adjustment to the evaluation structure is unavoidable. Sook-hyun Lee, Head of HIRA's New DRG Review Division, said, "Ultra-high-cost innovative new drugs like cell and gene therapies, ADCs, and one-shot treatments are entering the actual listing stage," and added, "The question is whether the cost-effectiveness evaluation alone is sufficient to determine the appropriate price for these drugs." Lee stated, "We cannot process all drugs through the economic evaluation system, nor can we exempt all drugs from it," explaining the currently operating hybrid management system. She emphasized that various forms of conditional reimbursement models have been introduced, including the refund-type RSA, the pre-listing/post-evaluation model, and the requirement for post-listing submission of clinical data. Regarding the recent increase in economic evaluation exemptions, Lee stressed that this should be understood not as a hurdle-free passage, but as a conditional system intended to expand access. Lee explained, "The uncertainty of ultra-high-cost/ultra-rare drugs cannot be resolved simply by increasing the ICER threshold or reinforcing the economic evaluation system," and concluded, "Given the variance in RWD quality, the gap in evidence levels, and the difficulty of quantitative evaluation, a comprehensive adjustment of the evaluation structure is necessary." Lee then added, "Economic evaluation and its exemption cannot be viewed as a simple dichotomy. We will expand post-market management and value-based evaluation to match technological changes." The government largely agrees with industry and academia on the problems and has repeatedly emphasized that the time calls for a more sophisticated evaluation method, not for strengthening regulations. The assessment is that the core task for the next 20 years will be precisely balancing the conflicting policy goals of access, sustainability, innovation, uncertainty, and pre- and post-market evaluation.
- Company
- Cosentyx gains reimbursement for hidradenitis suppurativa
- by Eo, Yun-Ho Nov 21, 2025 06:12am
- Cosentyx, the first new treatment option for hidradenitis suppurativa in 10 years since Humira, has succeeded in expanding its reimbursement one year after reapplying for reimbursement in Korea. The Ministry of Health and Welfare recently announced that it will expand the reimbursment criteria for Novartis Korea's interleukin (IL)-17A inhibitor Cosentyx (secukinumab) for hidradenitis suppurativa (HS) starting next month (December), through a partial amendment to the ‘Detailed Rules on the Standards and Methods for Applying Health Insurance Benefits.’ Specifically, hidradenitis suppurativa patients can use Cosentyx with reimbursement if they satisfy one of the following criteria: ▲ Adults with a diagnosis of over 1 year ▲ Lesions in two or more distinct areas, with a total of three or more abscesses and inflammatory nodules ▲ Insufficient therapeutic effect or adverse effects despite at least 3 months of antibiotic treatment. The process of expanding Cosentyx’s reimbursement was not smooth. Novartis originally submitted a reimbursement application after expanding its indication for Cosentyx to hidradenitis suppurativa in 2023, but voluntarily withdrew the application due to a lack of progress. Subsequently, in the European Hidradenitis Suppurativa Foundation (EHSF) practice guidelines presented at the 2024 European Academy of Dermatology and Venereology (EADV) Annual Congress, the EHSF recommended Cosentyx as a first-line biologic treatment option for patients with moderate-to-severe hidradenitis suppurativa (HS) and those with an inadequate response to prior systemic therapies. This led to the resubmission of the reimbursement expansion application in November of the same year, and the company gained success 1 year after resubmitting the application. Until now, AbbVie Korea’s Humira (adalimumab) has been virtually the only treatment option available for hidradenitis suppurativa in Korea. The introduction of Cosentyx, an IL inhibitor with a different mechanism of action that is also recommended as a first-line therapy, can become a viable option in managing hidradenitis suppurativa. Meanwhile, Cosentyx has demonstrated its clinical utility through the Phase III SUNNY (SUNSHINE, SUNRISE) trial, which was conducted on 1,084 patients with moderate-to-severe hidradenitis suppurativa. Results showed that the HiSCR achievement rate in the Cosentyx group was 45% in the SUNSHINE study and 42% in the SUNRISE study when the drug was administered every 2 weeks at 16 weeks of treatment, which was a significant improvement compared to the placebo group’s 34% and 31%, respectively. In the SUNRISE study, even when the drug was administered every 4 weeks, the HiSCR achievement rate for Cosentyx was 46%, which was significantly higher than the 31% in the placebo group, and the HiSCR achievement rate of Cosentyx improved steadily until the 52nd week of treatment. Cosentyx also showed significant effects in improving patients' pain. According to the results of the SUNNY study, the NRS30 achievement rate at Week 16 of treatment was 39% in the group that received Cosentyx every 2 weeks and 36% in the group that received it every 4 weeks. On the other hand, only 27% of the placebo group achieved NRS30. Also, by week 52 of treatment, 79.6% of the Cosentyx arm, who were administered Cosentyx every 2 weeks, and 72.7% of those who were administered every 4 weeks, did not experience disease exacerbation.
- Company
- "Yescarta leads DLBCL Tx…CAR-T therapy in earlier lines"
- by Son, Hyung Min Nov 21, 2025 06:11am
- The market position of CAR-T cell therapy in the treatment of relapsed or refractory Diffuse Large B-cell Lymphoma (DLBCL) is rapidly expanding. Notably, Yescarta has secured approval in Korea for both second- and third-line treatment, drawing attention as a treatment option that has proven survival benefits over the conventional standard therapy. On November 20, Gilead Sciences Korea held a press conference at its headquarters in Jung-gu, Seoul, to share the role and clinical value of Yescarta (axicabtagene ciloleucel) in the treatment of DLBCL. Yescarta received approval last August for: ▲adult patients with DLBCL who relapsed or were refractory within 12 months of first-line chemoimmunotherapy ▲patients with relapsed or refractory DLBCL and Primary Mediastinal B-cell Lymphoma (PMBCL) after two or more lines of systemic therapy. Yescarta is a CAR-T cell therapy that involves harvesting a patient's T cells, genetically reprogramming them to target CD19 on cancer cells, and then reinfusing them. It is the first CAR-T therapy approved in Korea for both second and third-line treatment. Since its first approval for follicular lymphoma in 2021, Yescarta has successfully secured market adoption by rapidly expanding its indications to DLBCL, leukemia, and others. Its global sales reached $1.57 billion (approximately KRW 2 trillion) last year, making it the only CAR-T therapy to surpass $1 billion in annual revenue. Sungeun Kim, Medical Lead at Gilead Sciences KoreaThe domestic approval of Yescarta is based on the ZUMA-7 clinical trial, which compared the efficacy and safety of CAR-T against conventional standard therapy. The clinical results showed that the median Event-Free Survival (EFS) in the Yescarta group was 8.3 months, which was more than 4 times longer than in the high-dose chemotherapy and autologous hematopoietic stem cell transplantation (ASCT) group. The percentage of patients surviving without disease progression or additional treatment at two years was also 41% in the Yescarta group, 2.5 times higher than the 16% in the standard therapy group. The efficacy was consistently observed even in high-risk subgroups, including elderly patients, first-line refractory patients, and patients with double-hit or triple-hit lymphoma. Sungeun Kim, Medical Lead at Gilead Sciences Korea, said, "Yescarta has been supplied to over 31,000 patients worldwide and is a treatment with a stable foundation in terms of manufacturing success rate," and added, "We have submitted the application for reimbursement listing and will do our best to ensure supply in the first half of next year." High unmet need in relapsed/refractory DLBCL…will CAR-T be the solution? DLBCL is a disease where B cells, which protect the body, grow or proliferate uncontrollably. It is the most common B-cell lymphoma, accounting for approximately 40% of non-Hodgkin lymphomas, and is characterized by aggressive, rapid progression. The number of DLBCL patients in Korea was 14,183 as of last year, up 36% from 10,428 in 2018. Up to 15% of patients fail treatment after the first-line standard therapy, and even 25% of patients who achieve complete response (CR) experience relapse within 18 months. Patients with relapsed or refractory DLBCL typically see a rapid worsening of prognosis as the number of treatment lines increases. The current first-line treatment for DLBCL is the chemotherapy 'R-CHOP' (rituximab, cyclophosphamide, vincristine, prednisone), which uses a monoclonal antibody combination. While R-CHOP offers curative potential for about half of patients, the remainder are refractory or relapse due to poor prognostic factors. Professor Gi June Min of Seoul St. MarySubsequent treatment options include high-dose chemotherapy or ASCT. However, due to the physical burden, these options are difficult for elderly or frail patients. In reality, only about 30-40% of patients who respond to salvage chemotherapy can even be considered for ASCT, and half of those are known to relapse after the transplant. Although CAR-T therapies like Kymriah and bispecific antibodies are used after second-line treatment, Yescarta is the only treatment in Korea to secure the second-line indication. This has intensified calls from experts to apply CAR-T therapy as early as possible in patients at high risk of relapse or refractoriness. Professor Gi June Min of Seoul St. Mary's Hospital's Division of Hematology said, "CAR-T therapy is a priority recommended treatment for patients who relapse within 12 months," and emphasized, "Yescarta, which has confirmed improvement in OS, is a good drug that should be used earlier in the treatment sequence to maximize its effect."
