- LOGIN
- MemberShip
- 2026-06-25 13:20:48
- Company
- Amvuttra receives orphan drug designation
- by Eo, Yun-Ho Nov 13, 2023 05:23am
- ‘Amvuttra,’ the first new drug for amyloidosis with polyneuropathy to be introduced since ‘Vyndaqel,’ has been designated an orphan drug in Korea. The Ministry of Food and Drug Safety announced so through a recent notice of orphan drug designation. siRNA therapy Amvuttra (Vutrisiran) is administered subcutaneously at once every three-month intervals. It inhibits the production of wild-type and mutant-type transthyretin (TTR) by targeting and silencing specific messenger RNA. Its efficacy was demonstrated through the Phase III HELIOS-A study that evaluated the efficacy and safety of Amvuttra in 164 patients with hATTR amyloidosis in 22 countries. The patients were randomized 3:1 to receive either 25 mg of vutrisiran via subcutaneous injection once every three months (vutrisiran arm, 122 patients) or 0.3 mg/kg of patisiran via intravenous infusion once every three weeks (patisiran arm, 42 patients). The efficacy of the vutrisiran arm was assessed by comparing the data with the landmark APOLLO Phase III study of patisiran that evaluated patisiran’s efficacy and safety in a comparable patient population. During the 9-month treatment period, the vutrisiran arm experienced fewer severe neurological damage than the placebo group and improved quality of life. Also, results of the timed 10-meter walk test that evaluates the patient’s walking speed and exercise ability, the vutrisiran arm showed little change compared to the placebo. Also, the arm showed an improvement in NT-proBNP, a biomarker that evaluates heart function. hATTR-PN, which occurs in 1 in 100,000, is caused by a genetic mutation in the transthyretin gene and causes systemic polyautonomic neuropathy, including symptoms related to the heart and digestive system, and eye disease. Generally, symptoms such as pain, abnormal sensations, and paralysis begin in the nerves of the lower extremities, where abnormal proteins tend to accumulate, and then affect the upper body, gradually spreading to the heart, kidneys, and eyes and causing complications. Its life expectancy is on average 7 to 12 years from symptom onset.
- Policy
- Looking at the review date for GIFT No. 1 Lunsumio
- by Lee, Hye-Kyung Nov 13, 2023 05:23am
- "The goal of the GIFT program is to shorten the general review period by 25%. The statutory review period is 120 days for chemical drugs and 115 days for biopharmaceuticals, but GIFT items In the case of, the goal is to complete the review within 90 days.” Park Jae-Hyeon, head of the rapid review division of the Ministry of Food and Drug Safety, announced on the 3rd that the actual review period for Roche Korea's Lunsumio Injection, the first GIFT product to receive domestic product approval, took 86 working days. GIFT's goal was to shorten the general review period by at least 25%. However, if you look at the period from the expedited review designation on November 29th of last year to the actual approval, it took 11 months. At first glance, you may question whether the expedited review is appropriate. Manager Park said in a recent interview with Dailypharm, "Lunsumio was reviewed for about 86 working days. As it is GIFT's first product, the licensing manager worked together to process it quickly," adding, "It took 11 months from designation to approval. “When I looked at the reason why it took, it took about 7 months to supplement the new application’s data,” he explained. According to the 'Drug Rapid Review Report' published by the Ministry of Food and Drug Safety this year, the time taken for rapid review during the drug review period, which generally takes 120 days, was 26.9 days for COVID-19 vaccines and treatments, and 81.5 days for other products. However, this statutory review period does not include the data supplementation period. The Ministry of Food and Drug Safety does not include the supplementation period in the approval review period under the current legal system. Therefore, the Ministry of Food and Drug Safety's shortened expedited review period refers to the pure review period excluding the data supplementation period. Manager Park said, “Shortening the review period is not an easy task,” and added, “The key is how well the applicant understands the supplementary matters and prepares the materials.” In particular, this year's expedited review designation has more than tripled compared to last year, and Manager Park said, "The pharmaceutical industry is interested and is applying for expedited review designation." He added, "It officially takes 30 days to review the expedited review designation, but the current employees' “Through our efforts, we are completing the designation without completing 30 days,” he said. These days, it is said that an increasing number of pharmaceutical companies do not immediately submit applications for approval even after receiving expedited review designation. This is because each company has its own circumstances, such as taking the licensing step after understanding the global market. Manager Park said, “After being designated for expedited review, the applicant must submit all documents related to the permit to the permit general manager before they are forwarded to the expedited review department.” “It’s going on,” he said. As of November 6, there were 19 items designated as GIFT brands, but the rapid review department was newly established in 2020 and the number of items designated for rapid review is 44. Once designated as expedited review, the Ministry of Food and Drug Safety shares the overall review schedule and holds product information sessions and supplementary information information sessions. In addition, the review period will be shortened through 'rolling review', which reviews materials prepared through prior review. Manager Park said, "It is meaningful that Runsumio was the first to receive approval under the GIFT name. However, shortening the review period also requires efforts from the industry. When the Ministry of Food and Drug Safety delivered the first review opinion, the parts that were not understood were discussed with the person in charge. “I think it will be helpful if we communicate with each other to understand clearly and prepare materials,” he said. Currently, items subject to GIFT are ▲medicines intended to treat serious or rare diseases such as life-threatening cancer, ▲intended for the prevention or treatment of infectious diseases that are likely to cause serious harm to public health, such as bioterrorism infectious diseases or infectious disease pandemics. It is limited to ▲ new drugs developed by innovative pharmaceutical companies designated and announced by the Ministry of Health and Welfare ▲ combinations of drugs and medical devices subject to rapid review ▲ cases where there is no existing treatment or showing clinically meaningful improvement in effectiveness, etc. compared to existing treatments. Manager Park added, “We are receiving a request to expand the GIFT target as an industry suggestion,” and added, “It is not easy as we are conducting screening work with limited manpower, but we are making efforts.” .
- Company
- NMOSD drug Enspryng likely to be reimbursed in December
- by Eo, Yun-Ho Nov 13, 2023 05:23am
- ‘Enspryng’ a new drug for neuromyelitis optica spectrum disorder (NMOSD), is expected to be listed for reimbursement in Korea within the year. According to industry sources, Roche Korea completed drug pricing negotiations with the National Health Insurance Service for the reimbursement of its NMOSD treatment Ensprying (satralizumab). Therefore, reimbursement may be applied from December at the earliest. The company had applied for the reimbursement of its Ensprying in H2 2022 after receiving approval in H1 2021. However, due to its high price, the company and authorities have found it quite difficult to set a reimbursement standard and financial sharing plan. The company had first adopted the strategy of accepting the weighted average price (WAP) of its alternative, Soliris, but due to a delay in Soliris’s reimbursement listing process for NMOSD, the company turned to the pharmacoeconomic evaluation exemption system for its reimbursement. In particular, even after passing a review by the Drug Reimbursement Evaluation Committee of the Health Insurance Review and Assessment Service in August, the company encountered difficulties in accepting the deliberation results and entering drug pricing negotiations due to the tightly set reimbursement standards. In other words, the negotiation was only possible because Roche accepted the ‘fourth or later lines of therapy’ reimbursement standard set by DREC. This therefore also conversely suggests significant restrictions on actual prescriptions after its reimbursement listing. Currently, the immunosuppressant azathioprine is used as first-line maintenance therapy for NMOSD. If a patient fails treatment with azathioprine, mycophenolate or rituximab is prescribed with reimbursement as second-line therapy. Both mycophenolate and rituximab are off-label drugs that do not have NMOSD indications. In other words, Enspryng can only be used as a third or later-line therapy in patients who fail treatment with rituximab. Therefore, it remains to be seen whether the company will seek to extend reimbursement for Enspryng after listing. Meanwhile, Enspryng’s efficacy was demonstrated through SAkuraStar and SAkuraSky clinical trials that were conducted on adult patients with anti-aquaporin(AQP4) antibody-positive NMOSD. In the SAkuraStar monotherapy study’s AQP4 antibody-positive subgroup, 76.5% of ENSPRYNG-treated patients were relapse-free at 96 weeks, compared to 41.1% with placebo. In the SAkuraSky study, which evaluated Enspryng when used concurrently with standard immunotherapy, 91.1% of Enspryng-treated AQP4 antibody-positive subgroup patients were relapse-free at 96 weeks, compared to 56.8% with placebo.
- Opinion
- [Reporter’s view] Successive large-scale technology exports
- by Kim, Jin-Gu Nov 10, 2023 05:19am
- There is some good news coming from the pharmaceutical and bio industry. Chong Kun Dang and Orum Therapeutics have signed large-scale technology export contracts with global pharmaceutical companies. On the 6th, Chong Kun Dang signed a technology export contract with Novartis worth a total of $1.305 billion (about 1.7 trillion won). This is a transfer of global development and commercialization rights, excluding Korea, for ‘CKD-510’, a new drug candidate in the HDAC inhibitor class. On the same day, Orum Therapeutic, an unlisted bio venture, signed a contract with BMS to transfer the technology of ‘DRM-6151’, a new drug candidate for leukemia. The total contract size is $180 million (approximately 230 billion won). What attracts more attention than the total contract size is the down payment. Chong Kun Dang received $80 million (approximately 100 billion won) as a down payment, and Orum Therapeutic received $100 million (approximately 130 billion won) as a down payment. The down payment is money that does not need to be returned even if the rights to develop new drugs are later returned. The down payment is evaluated as an appropriate tool to objectively reflect the value of the candidate material. The total contract size is filled with an optimistic outlook. If a candidate material does not meet detailed contract conditions such as development or approval, it will not be received. In general, it is considered a good condition if the down payment ratio is more than 5% of the total contract size. Chong Kun Dang received 6.1% of the total contract size, and Orum Therapeutic received 55.6% as a down payment. In terms of down payment, it is the largest in four years since 2019. In February 2019, SK Biopharmaceuticals received $100 million (total contract size of $530 million) and signed a technology export contract for an epilepsy new drug with AVELOS Therapeutics. Since then, several technology export contracts have been signed with a total contract size in the trillions, but it is true that looking at the contract amount alone, it was disappointing. In some quarters, controversy arose over the discrepancy between the total contract size and the down payment. However, a series of contracts were concluded with a down payment of over 100 billion won. In the pharmaceutical industry, expectations are growing whether large-scale technology export contracts concluded one after another will be able to reverse the recent slump. Recently, the pharmaceutical and bio-industry has been facing an investment cliff due to the high-interest rate situation that has continued since the coronavirus pandemic. In particular, bio ventures that focused on R&D without consistent sales were pushed into crisis as external investment plummeted. In this situation, it is ultimately R&D, the core asset of the biopharmaceutical industry, that leads to a change in the atmosphere. It is time for another R&D performance. We succeeded in revitalizing the atmosphere through a series of technology export contracts. In order to completely change the atmosphere, new R&D results must be released in the near future. I look forward to hitting consecutive hits following Chong Kun Dang and Oreum.
- Product
- ‘Cutting budget for essential drugs neglects public health'
- by Kang, Hye-Kyung Nov 10, 2023 05:19am
- The Korean Pharmacists for Democratic Society (CEO Hyeong-geun Shin) criticized the full budget for the stable supply of essential national medicines and urged for additional budget allocation. On the 9th, before the National Assembly’s Health and Welfare Committee presented and deliberated on the next year's budget and proceeded with deliberation, KPDS submitted an opinion after analyzing the 2024 budget plan. Regarding the budget plan, KPDA said, “Regarding the development and support of the pharmaceutical and bio industry, the R&D budget has been expanded significantly, including the existing Research-Focused Hospital Development Project (approximately KRW 60.5 billion), the National New Drug Development Project (approximately KRW 57.9 billion), the Pharmaceutical Industry Development And Support Project (approximately KRW 35.9 billion), Pan-Ministerial Regenerative Medicine Technology Development Project (approximately KRW 35.3 billion), Global Research Cooperation Support Project (approximately KRW 28.7 billion), Electropharmaceutical Technology Development Project (approximately KRW 6.6 billion), Drug Delivery Treatment Technology Development Project (approximately KRW 7.6 billion), Biohealth Investment Infrastructure Linked R&D Project (KRW 2.7 billion), and the new Korean ARPA-H Project (KRW 49.5 billion) and the Joint Learning-Based New Drug Development Acceleration Project (approximately KRW 2.3 billion), etc. The investment is more noteworthy as the R&D budgets of only the Ministry of Health and Welfare, the Korea Disease Control and Prevention Agency, and the Ministry of Food and Drug Safety were increased among the 31 ministries in Korea.” However, KPDA pointed out, “Although it is a small amount compared to the budget plan set for developing new drugs and fostering the pharmaceutical and bio-industry, which costs hundreds of billions of won, we analyzed the budget plan and found some points worth pointing out to improve issues that the public can feel.” The first issue was regarding the budget implementation ▲to resolve concerns about the unstable supply and demand of medicines in the field KPDA said, “A total of 247 drugs were reported to have supply interruptions and supply shortages in 2022 to the MFDS, and 172 in the first half of 2023 alone. However, in MFDS’s 2024 budget, the budget that ensures a stable supply of national essential drugs was completely cut. The KRW 1 billion that was allocated to improve the supply of medicines through consignment manufacturing for 6 items in 2023 was cut.” Therefore, assuming that there are at least 25 drugs to which the government must respond, an additional KRW 7.5 billion should be allocated, KRW 300 million per drug. KPDA said, “One of the difficulties pharmacies complain about out-of-stock drugs is the lack of a way to explain when the out-of-stock issue will be resolved or explain the out-of-stock situation to patients or prescribers. This ultimately depends on the drug supply and demand situation. So we need to improve the transparency of information regarding the drug supply and demand. For this, the Ministry of Health and Welfare announced that it will disclose distribution information through the Korea Pharmaceutical Information Service Center (KPIS), but most medical practitioners are not aware of this, and even if they go to the website, it is difficult to find the information they want. Therefore, the KPIS webpage should be completely rebuilt so that pharmacies, medical institutions, and patients can easily check the status of out-of-stock items and be aware of the expected release date of necessary drugs.” The second was ▲expanding the budget for drug safety management and side-effect damage relief Under the Yoon Suk-Yeol administration, the Ministry of Food and Drug Safety announced a fast-track program for innovative products and announced that it would improve the system to allow faster approval of medical products, but the budget for licensing reviewers in charge of reviewing the medical products had not increased for several years. KPDA said, "In the 2024 budget, the number of personnel for registering clinical trial information and reviewing reports has been reduced from 7 to 3. There is a need to increase the related budget to expand the number of medical product-related review personnel to 500 people by 2024, as in Japan.” Regarding the Side Effect Damage Relief Fund, "Even though the related fund was established 10 years ago, many people are completely unaware of it, and there have been only about 100 cases of damage relief per year for several years. The government should have actively provided guidance and promotion so that people who have suffered damage from the side effects of medicines can seek relief, but its budget is only KRW 80 million. Considering how this year’s advertising budget for drug safety is KRW 3 billion, there is an urgent need to actively execute a promotion budget for the fund.” Regarding the budget for ▲expanding the publicness of pharmacies, KPDA requested, "The government must gradually expand the number of support pharmacies to ensure people's access to medicines, and expand the system so that at least one late-night pharmacy can be operated by each local governments in the future." In addition, KPDA also insisted on ▲securing a minimum budget to guarantee reproductive health. They said, “After the 2019 decision to deem abortion unconstitutional, guaranteeing women’s reproductive health became a major social issue, but all content related to reproductive health was missing from the 2024 budget. We ask for continued research, including the opening of a website for providing related information counseling services at the government level.” In particular, KPDA added, “It is more essential than ever to secure a budget for the out-of-stock drug situation that is still unresolved."
- Company
- Korea Pharma applies for approval of its 24hr ADHD drug
- by Lee, Seok-Jun Nov 10, 2023 05:19am
- Korea Pharma announced on the 9th that it had applied for marketing authorization for its 24-hour long-acting ADHD treatment ‘Methydur SR Cap.’ Methydur is a treatment for attention deficit hyperactivity disorder (ADHD) in children and adolescents developed by Orient Pharmaceuticals in Taiwan. The main substance contained in Methydur is methylphenidate hydrochloride, and it is available in three dosages - 22mg, 33mg, and 44mg - depending on symptoms. In Taiwan, the drug underwent 5 Phase I trials and completed a Phase I trial on 113 children and adolescent patients, demonstrating its safety and effectiveness. The drug obtained marketing approval in Taiwan in 2018. Methydur reduced the side effects that commonly accompany CNS drugs and improved the risk of drug abuse by applying the 'ORADUR®' technology. Using the ORADUR technology, the drug’s drug release rate can be controlled while retaining the characteristics of a sustained-release formulation by filling the capsule with a highly viscous gel form liquid. It can reduce discomfort caused by intranasal or intravenous treatments and prevent misuse or abuse. The number of ADHD patients in Korea have been rapidly increasing every year. However, it is an over-monopoly situation, with certain products taking up more than 60% of the market share. Korea Pharma plans to provide a stable environment for drug supply to patients by introducing Methydur, which can be prescribed in various doses and has a proven safety and effectiveness. Eun Hee Park, CEO of Korea Pharma, said, “If we obtain a marketing authorization for Methydur in Korea, we will be able to provide a clinically improved treatment effect to domestic pediatric and adolescent ADHD patients that are increasing every year.”
- Company
- Development active for microbiome-based therapies in Korea
- by Nho, Byung Chul Nov 10, 2023 05:19am
- Microbiome-based therapies have been expanding their therapeutic areas from simple digestives to immunology and oncology, receiving industry attention. Based on research results that showed that imbalances in the human microbiome are highly correlated with various incurable diseases, such as cancer and obesity, and can cause immune and metabolic diseases, research is being conducted in various fields to develop treatments using microorganisms. In particular, Swiss Ferring Pharmaceuticals' Rebyota (prevention of recurrence of Clostridioides difficile (C. difficile) infection) received FDA approval last year, and Seres Therapeutics' Vowst (prevention of recurrence of Clostridioides difficile (C. difficile) infection) was also approved this year. With drugs continuing to be approved one after another, the companies are also speeding up the commercialization of their respective drugs. In line with this global trend, domestic biotech companies are also entering related fields one after another and exploring their possibilities. First, MD Healthcare is developing a new drug with a focus on extracellular vesicle (EV) secreted by microbiome. EV is a lipid bilayer membrane secreted externally by cells that serve as vital mediators of intercellular communication. The company explained that as these particles are much smaller than cells, they have high absorption capacity and thus can provide radical treatment through a mechanism that works from within the cells. MD Healthcare's representative pipeline drug, 'MDH-014', targets central nervous system diseases (CNS) such as autism spectrum disorder, Alzheimer's disease, and Parkinson's disease. The company had submitted an IND for the drug for the autism spectrum disorder indication and is planning to start Phase I trials next year.. Enterobiome is developing treatments for incurable diseases using extreme anaerobic, non-culturable, next-generation probiotic strains. The company has been developing next-generation probiotics akkermansia muciniphila and faecalibacterium prausnitzii strains that show a negative correlation with various immune and metabolic diseases in the body as pharmabiotics. According to domestic and international studies, akkermansia and faecalibacterium significantly reduced the gut microbiota composition of patients with immune diseases such as atopic dermatitis and cancer as well as metabolic diseases such as obesity and non-alcoholic steatohepatitis (NASH) compared to normal people. Also, patients who were administered the two strains saw a treatment effect. Enterobiome has currently completed non-clinical toxicity testing for its akkermansia muciniphila strain EB-AMDK19 for atopic dermatitis and is preparing to apply for an IND as a new atopic dermatitis drug early next year. Akkermansia muciniphila, observed with an electron microscope. Among companies that are developing next-generation microbiomes, the only two companies leading at the akkermansia R&D and commercialization stage are The Akkermansia Company in Belgium and Enterobiome in Korea. Enterobiome owns a source technology for a high-concentration culture that is 1,000 times more concentrated than its competitors. Liveome has been developing a microbiome therapy based on its gene recombination technology. Libiome’s microbiome-based new drug has both the characteristics of a probiotic therapy and a gene therapy and is referred to as a ‘genetically engineered microbiota therapy.’ These treatments have the advantage of being able to increase effectiveness and drug efficacy by designing and manufacturing microorganisms according to the desired mechanism. Libiome is currently conducting Phase I clinical trials in Australia for its LIV001, a candidate in its inflammatory bowel disease pipeline, that was developed using the genetic recombinant eLBP platform. The candidate was selected as a new project by the Korea Drug Development Fund in July of this year and is receiving KDDF support for related R&D costs. In addition, various companies such as KoBioLab, Genome & Company, and CJ Bioscience are developing treatments for incurable diseases using microbiome, therefore whether a next-generation microbiome drug following Vowst’s footsteps will be born in Korea is gaining industry attention.
- Company
- Will Lixiana hit the 100 billion won mark for the NOAC RX?
- by Kim, Jin-Gu Nov 10, 2023 05:19am
- Attention is focused on whether Daiichi Sanko Lixiana will become the first NOAC drug to exceed 100 billion won in annual prescription sales. As of the third quarter, the cumulative prescription amount was 78.2 billion won, and if this trend continues, it is expected that prescriptions will easily exceed 100 billion won by the end of the year. Lixiana's annual prescription volume exceeded KRW 100 billion, making it the only original company to grow According to UBIST, a pharmaceutical market research firm, on the 9th, Lixiana's outpatient prescription volume in the third quarter was 26.8 billion won. Compared to 24.9 billion won in the third quarter of last year, it increased by 8% in one year. Lixiana's cumulative prescription amount in the third quarter was 78.2 billion won. The pharmaceutical industry predicts that if this trend continues, the number of prescriptions will increase to more than 100 billion won by the end of the year. Lixiana recorded prescriptions worth 96.7 billion won last year. In Korea, products that recorded over 100 billion won in prescriptions last year include Viatris 'Lipitor', Hanmi Pharmaceutical 'Rosuzet', HK inno. N 'K-CAB', Daewoong Bio Gliatamin, Handok 'Plavix', Chongkundang Gliatirin, LG Chemical Zemimet, etc. There are only 7. Even though the domestic NOAC market has reached a plateau, Lixiana is the only original drug that continues to see an upward trend in prescription performance. The NOAC market continued to grow rapidly until last year. The NOAC market, which was 147.2 billion won in 2018, reached 225.9 billion won in 2021, reaching the 200 billion won mark. Last year, the market size further expanded to 242.5 billion won. However, on a quarterly basis, it appears to have slowed down since peaking in the second quarter of last year. The NOAC market, which was 61.8 billion won in the second quarter of last year, decreased to 58.3 billion won in the 4th quarter. This year, it stagnated at 59.3 billion won in the first quarter, 60.2 billion won in the second quarter, and 60.9 billion won in the third quarter. BMS Eliquis, the second-largest product in the market, recorded prescription sales of 19.2 billion won in the third quarter. It remained at a similar level to the third quarter of last year. Eliquis has been recording prescription sales of around 19 billion won every quarter since the third quarter of last year. Xarelto's prescription performance decreased significantly due to the release of generics and the subsequent price reduction. Xarelto prescriptions in the third quarter amounted to 7.6 billion won, a 36% decrease from 11.8 billion won in the same period last year. Xarelto had issued quarterly prescriptions worth more than 15 billion won until the third quarter of 2021, but prescriptions have plummeted since the launch of generics. In the fourth quarter of last year, the quarterly prescription amount fell below 10 billion won and has been steadily decreasing since then. Boehringer Ingelheim Pradaxa's slump is prolonged. Pradaxa's prescription volume in the third quarter was 2.5 billion won, a 19% decrease compared to the same period last year. Generics for Xarelto are rapidly expanding their influence. The combined prescription amount for generics for Xarelto in the third quarter was 4.7 billion won. It increased by 72% compared to 2.7 billion won in the third quarter of last year. Its share in the rivaroxaban treatment market increased to 38%. Compared to 18% in the third quarter of last year, the market share has more than doubled in one year. Xarelto generics were released in large numbers in the third quarter of that year following the release of Chong Kun Dang's Riroxia in the second quarter of 2021. Chong Kun Dang's Riroxia, Hanmi Pharmaceutical's Riroxban, and Samjin Pharmaceutical's Rivoxaban are competing. As of the third quarter of this year, Riroxia's cumulative prescriptions were the highest at 3.3 billion won. Riroxban follows this at 2.4 billion won and Rivoxaban at 2.1 billion won. The remaining 30 companies had cumulative prescriptions of less than 1 billion won in the third quarter.
- Company
- Will Trodelbi become a second-line standard tx
- by Nov 09, 2023 05:43am
- Gilead Sciences Korea held a press conference at the Plaza Hotel in Seoul on the 7th to commemorate the domestic launch of Trodelbi, a treatment for metastatic triple-negative breast cancer Antibody Drug Conjugate (ADC) anticancer drugs first appeared in the domestic triple-negative breast cancer treatment market. To date, there have been no treatment options targeting triple-negative breast cancer treatment other than immunotherapy drugs and PARP inhibitors. Gilead is aiming to make Trodelbi the standard treatment option in this area. Gilead Sciences Korea held a press conference at the Plaza Hotel in Seoul on the 7th to commemorate the domestic launch of Trodelbi, a treatment for metastatic triple-negative breast cancer. Trodelbi is an ADC that targets Trop-2 protein, which is frequently observed on the surface of breast cancer cells and is used as a treatment for advanced or metastatic triple-negative breast cancer. Last May, Trodelbi was approved in Korea for the treatment of unresectable locally advanced or metastatic triple-negative breast cancer that has previously received two or more systemic treatments, at least one of which was for metastatic disease, and was launched in Korea last month. The phase 3 ASCENT study served as the basis for approval. The study was conducted to compare the effectiveness and safety of Trodelbi and chemotherapy in 529 patients with locally advanced or metastatic triple-negative breast cancer who had previously received chemotherapy twice or more. 12% of all patients had brain metastases. The primary endpoint was PFS in patients without brain metastases compared to baseline. Secondary endpoints included overall patient PFS, OS, and ORR. As for clinical results, the median PFS in patients without brain metastases, which was set as the primary endpoint, was recorded by Trodelbi at 5.6 months. This was a higher figure than the 1.7 months recorded for chemotherapy. The OS, measured regardless of the secondary endpoint of brain metastasis, was 11.8 months for Trodelbi and 6.9 months for chemotherapy in the entire patient group. There was a significant difference in ORR, with Trodelbi being 31% and chemotherapy being 4%. In terms of safety, serious adverse reactions such as neutropenia (7%), diarrhea (4%), and pneumonia (3%) occurred during Trodelbi administration. Treatment discontinuation due to adverse reactions was calculated to be 5% for Trodelbi and chemotherapy. Professor Son Joo-hyuk of the Department of Oncology at Yonsei Cancer Hospital said, “Trodelbi is considered a docile anticancer drug. The basic principle is that cytotoxic anticancer drugs kill cancer cells, and as they bind to antibodies, we confirmed a good safety profile without severe toxicity,” he said. “Trodelbi is an anticancer drug that should be used as a standard treatment for metastatic triple-negative breast cancer.” “I think it should be included not only in ESMO or NCCN but also in our country’s treatment guidelines,” he said. According to the NCCN guidelines, Trodelbi is classified as Category 1 for the second-line or higher treatment of adult patients with metastatic triple-negative breast cancer. Rare cancer, triple-negative breast cancer, need for secondary treatment options after treatment failure. Triple-negative breast cancer is classified as a rare cancer among breast cancers. Triple-negative breast cancer, which is negative for human epidermal growth factor receptor type 2 (HER2), hormone receptor (HR), and estrogen, is experiencing difficulties in developing targeted treatments. In particular, compared to HR- and HER2-positive breast cancer, there is a shortage of treatments and treatment outcomes are not as good. In the case of early triple-negative breast cancer, immunotherapy drugs can be used without biomarker analysis. However, in metastatic triple-negative breast cancer, it can only be used if there is PD-L1 expression. If BRCA mutation is confirmed, PARP inhibitors can be used, but if there is no corresponding biomarker mutation, there is no treatment. Professor Kim Ji-hyung of the Department of Oncology at Gangnam Severance Hospital said, “Triple-negative breast cancer has aggressive clinical manifestations and there are almost no targeted therapies available. “The disease-free survival rate is usually only 2 to 3 months, and there is no standard treatment,” he said. “Immunotherapy drugs also require confirmation of the PD-L1 expression rate, so treatment options are limited. “An effective treatment is needed for patients with metastatic triple-negative breast cancer who have failed primary treatment,” he said.
- Policy
- Korean researchers develop 1st new substance for Alzheimer's
- by Lee, Hye-Kyung Nov 09, 2023 05:43am
- A Korean research team has developed a new substance (ALT001) that promotes mitophagy, and the recycling of damaged mitochondria, and presented a new door to treating Alzheimer's type dementia. The Korea Health Industry Development Institute (President: Soon-Do Cho) announced that a joint research team that consists of Jin-ho Yoon (College of Medicine, Dong-A University), Jong-Hyun Cho (Department of Medicinal Biotechnology, College of Health Sciences, Dong-A University), Ji-Hoon Jo (Chonnam National University), and Alt Medical (CEO: Eun-Hee Yoo) has succeeded in developing a new mitophagy promoting substance (ALT001) that can treat Alzheimer's type dementia. Summary of ALT001 ALT001 demonstrated low toxicity and safety that does not interfere with cell growth. Also, animal testing in vivo showed that the substance effectively improved damaged cognitive function, proving its value as a clinically applicable treatment substance for dementia. Previously, most studies for the treatment of Alzheimer's type dementia had focused on amyloid beta and tau proteins, but recent study results showed that mitochondrial dysfunction plays an important role in the development of dementia by interacting with amyloid beta, and promoting the mitophagy process that maintains mitochondrial function has been attracting attention as a new treatment strategy. However, due to the absence of mitophagy-promoting substances with proven clinical applicability, dementia treatment through mitophagy has not been implemented in practice. To improve this situation, the joint research team conducted a chemical library screening using an in-house mitophagy activity analysis system. Through the screening, the research team identified an isoquinolium scaffold for mitophagy induction and generated ALT001 through chemical optimization of the isoquinolium scaffold. ALT001 effectively promotes mitophagy and is safe as it does not interfere with cell growth, demonstrating its clinical applicability. The result of an experiment conducted to evaluate the learning and memory ability when using ALT001 for Alzheimer's type dementia in mouse models showed surprising results, where the learning and memory ability of the dementia mouse model was restored, and this therapeutic effect was also identified in a mouse model widely used in other dementia research. Professor Jin-ho Yoon, who led the research, said, “Thanks to the national support and helping hands, we researchers in Korea were able to develop a dementia treatment substance that can be clinically applied in the highly competitive mitophagy-based treatment development field. We are very grateful for the support. Our research will allow the development of a mitophagy-based dementia treatment that has been difficult to commercialize due to the lack of a drug with defined molecular mechanisms. We will devote ourselves to follow-up research with the goal of commercializing a dementia treatment using the results of this research.” The study was conducted with the support of the Korea Dementia Research Center, and its results were published in Theranostics, a world-renowned academic journal ranked among the top 5.8% in the field of medical research.
