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- 2026-04-04 03:20:00
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- ‘Pluvicto, a new standard therapy for prostate cancer?'
- by Whang, byung-woo Dec 10, 2024 05:52am
- Prostate cancer is one disease area where the treatment environment has been improving with the increasing number of treatment options listed for health reimbursement benefits. Recently, Pluvicto (vipivotide tetraxetan), a radioligand therapy with a new mechanism of action, has emerged and is attracting attention as a viable new standard of care. Although the prescription is still in its early stages in Korea, it is already expanding its influence in the global market. At a meeting with Dailypharm, SeungJu Lee, Professor of Urology; Byung-Yong Sim, Professor of Oncology; and Hyuk-Jin Yoon, Professor of Nuclear Medicine at Catholic University St.Vincent’s Hospital, expressed their views on how the new treatment option Pluvicto is expected to provide a new option for patients who are not responding to existing treatments. (From the left) Byung-Yong Sim, Professor of Oncology; Seung Ju Lee, Professor of Urology; and Hyuk-Jin Yoon, Professor of Nuclear Medicine at Catholic University St.Vincent’s Hospital Prostate cancer has been ranked as the leading cancer among men in recent years, and its diagnosis rate has been increasing due to the aging population and westernization, as well as active PSA (Prostate Specific Antigen) screening. Metastatic castration-resistant prostate cancer (mCRPC) is estimated to account for 20-30% of all prostate cancer patients. Its prognosis is favorable if detected early, as its 5-year survival rate exceeds 90%, but if the disease has already metastasized by the time of diagnosis and become mCRPC, the prognosis differs significantly from that of early-stage prostate cancer, as it cannot be cured with drugs. “Ten years ago, there was no way to cure mCRPC other than androgen deprivation therapy, but the introduction of antiandrogens had improved the condition and response of the patients,” said Professor Seung-Ju Lee. ”However, as even these treatments can lead to resistance, so we are looking forward to the development of further therapies.” Professor Byung-Yong Sim said, “In the treatment of mCRPC patients, patients are forced to move on to the next line of treatment due to resistance and eventually are left to receive chemotherapy. However, since most prostate cancer patients are elderly, there is also an issue that they cannot receive chemotherapy properly.” Radioligand therapy emerges for mCRPC...multidisciplinary care is key One such option is Pluvicto, a radioligand therapy (RLT). The drug was approved by the Ministry of Food and Drug Safety Korea in May for the treatment of adult patients with PSMA-positive mCRPC who had previously received ARPI therapy and taxane-based chemotherapy. In other words, the drug is expected to become a new treatment option for patients who have had a poor prognosis despite receiving existing therapies. “Previously, anticancer drugs were administered systemically through pills or injections, which then reached the cancer cells, but radioligand therapy’s mechanism of action allows radioactive substances to find and enter the target,” said Professor Lee. ”It is an effective treatment option that aligns with the characteristics of prostate cancer that can be administered with less impact on other cells.” It is also expected to be effective in terms of resistance, which is one of the challenges that exist with mCRPC treatment. “Continued treatment can lead to resistance, but Pluvicto selectively binds to prostate-specific membrane antigen (PSMA), which increases during the process of developing resistance to existing treatments,” said Professor Hyuk-Jin Yoon. ”Before treatment, patients can be tested with a radiopharmaceutical called PSMA PET-CT to determine their suitability in advance, allowing for more effective treatment.” However, due to the complex mechanism of RLT, multidisciplinary care is essential during the patient screening process. For this reason, only a few hospitals in Korea, including St. Vincent's Hospital, are currently able to prescribe Pluvicto. “Collaboration for RLT is only possible in hospitals that are large enough and are equipped with the facilities to handle nuclide materials,” said Professor Sim. ”We are forging multidisciplinary care, supported by a nuclear medicine department equipped with the scale to use radiotherapy materials related to bone metastases, and other departments screening the right patients.” “Pluvicto is administered in 6 cycles at 6-week intervals, and when you come to the hospital, you visit all 3 departments together,” he said. ”The whole body blood test is done by the oncology department, and the prostate is also related to urinary problems, so the urology department checks each of these problems and provides total care.” So how does Pluvicto actually work? While it's still early days for the drug and is hard to definitely say, the evaluation on-site is that has been shown to be effective in relieving bone pain. “A decrease in PSA levels by more than 50% is an important indicator of response, which can take as early as 2 weeks or as long as 4 weeks if the response is slow, so it's still too early to tell,” said Professor Yoon. ”However, one of the patients we recently treated was satisfied with the elimination of bone pain after the first treatment and is continuing onto the second treatment.” Pluvicto shows promise, but its high cost remains a challenge Aside from its therapeutic benefits, its high price tag is likely to be a hurdle like in many other new drugs. Currently, 6 cycles of Pluvicto are estimated to cost around KRW 200 million. Without reimbursement constraints, it is estimated that around 10% of men with castration-resistant prostate cancer (mCRPC), which accounts for 20-30% of all metastatic prostate cancer, would be eligible for Pluvicto. The number may be further reduced when screening eligible patients with PSMA PET-CT. “A lot of people could be helped if they didn't consider the price. Patients are highly interested in the drug, and we have visitors who received information about PSMA PET-CT and the list of hospitals that can treat it in patient advocacy groups, cafes, etc.” “There are some prostate cancer patients who are in good physical condition even though they have undergone all chemotherapy and hormonal treatments and have seen no effect. In such cases, Pluvicto would be a good option despite its high cost.” In the long run, however, the challenge will be its reimbursement. Professor Lee predicts that the reimbursement discussions will be made with the accumulation of prescription experience. “Reimbursement discussions have not been very active at academic conferences because Pluvicto is still new. Once we have a year or two of patient prescription experience, demand will also rise, and once there is demand, reimbursement discussions will be made one way or another.” “In urology, I think the introduction of Pluvicto will serve as momentum to the evolution of prostate cancer treatment, along with robotic surgery and antihormonal drugs,” added Professor Lee. ”I think the basis of the change is PSMA PET-CT, which will be a game-changer in diagnosis, and I think the related treatments will become an important issue that will attract the attention of academics for the next 2-3 years.”
- Policy
- Cancellation policy for innovative pharmas certification
- by Kang, Shin-Kook Dec 09, 2024 05:47am
- Choi Sang-Mok, deputy prime minister and Minister of Economy and Finance It has been reported that the cancellation standards of 'Innovative Pharmaceutical Companies Certification' will be relaxed for pharmaceutical companies fined for rebates. Some argue that certification requirements for illegal rebates are strict. Further criticism is anticipated as these measures deviate from the government’s longstanding anti-rebate policies. On December 4, the government finalized the "Innovate Economic Regulatory Plan for Enhancing Corporate Dynamism and Promoting New Industries" at the Emergency Ministerial Meeting on Economic Affairs. This plan was prepared by the Ministry of Economy and Finance and the Office for Government Policy Coordination of South Korea, actively·promptly reflecting suggestions from economic organizations and companies. ◆Improvement of certification cancellation standards for 'Innovative Pharmaceutical Companies' = The government has determined that the strict certification requirements related to illegal rebates in the 'Innovative Pharmaceutical Companies Certification' system could discourage pharmaceutical companies from developing new drugs. In detail, the government plans to revise the standards stipulating that certification can be canceled if ▲A company receives two or more penalties for violating the Pharmaceutical Affairs Act due to illegal rebates within three years or ▲The total amount of rebates exceeds KRW 5 million. After consulting with experts from the industry and academics, the government plans to devise a draft for the revision to the cancellation standards for the 'Innovative Pharmaceutical Companies Certification' (according to the Ministry of Health and Welfare notice) next year. Meanwhile, 'Innovative Pharmaceutical Companies Certification' offers several benefits, including: ▲Incentives when participating in government R&D projects related to new drug development ▲Corporate tax deductions for research personnel development costs ▲Eased location regulations for constructing research facilities ▲Special loan arrangements for policy funds. ◆'Advanced regenerative clinical research' results to be used for drug approvals = Different standards from clinical trials under the Pharmaceutical Affairs Act have been applied to Advanced regenerative clinical research, making it difficult to use as a basis for approval in developing advanced biopharmaceuticals. Guidelines will be established this month to allow advanced regenerative clinical research results to be used as approval review data for advanced biopharmaceuticals. Guidelines will be established this month to allow advanced regenerative clinical research results to be used as approval review data for advanced biopharmaceuticals. A draft guideline, tentatively titled "Guidelines relating to the Advanced Regenerative Medicine Clinical Research and Marketing Authorization," will be developed to utilize research results that meet specific standards as part of the review data for marketing authorization. ◆To introduce simultaneous analysis methods for vitamin B products in functional foods = Currently, the "Standards and Specifications for Functional Foods" do not include a test method for simultaneously analyzing the Vitamin B group (B1, B2, B6, niacin, and folic acid), which has delayed the product development. In response, the government plans to develop·implement a test method capable of analyzing properties across various formulations for functional foods through a research project starting in January next year. ◆To develop a pilot service of a health management system for young children·adolescents = A health management service utilizing AI and digital technology will be implemented to minimize long wait times for treatment and reduce public inconvenience. In particular, with declining birth rates exacerbating the reduction of pediatric medical demand and resources, the government aims to develop a health management system for young children to address reduced access to pediatric care. The system will be designed to include AI-based support for diagnosis and treatment guidance, assistance with medical record documentation, health consultations for managing pediatric diseases and providing information to caregivers on pediatric rare diseases. The government will allocate a total of KRW 32 billion over four years for this project. Additionally, the government suggested other innovative measures, ▲Establishment·operation of a regulatory support center for digital medical products ▲Regulatory improvements for consumer direct-to-consumer (DTC) genetic testing for minors ▲Enactment of legislation to promote alternatives to animal testing ▲Support for precision oncology technology development using next-generation sequencing (NGS) panel data ▲Simplification of required documentation for filing innovative medical technology applications.
- Company
- Padcev seeks reimb as mono and combo therapy in KOR
- by Eo, Yun-Ho Dec 09, 2024 05:47am
- The ADC bladder cancer drug Padcev is seeking reimbursement as both monotherapy and combination therapy in Korea. According to industry sources, Astellas Pharma Korea recently submitted reimbursement applications for Padcev (enfortumab vedotin) as monotherapy and combination therapy with PD-1 inhibitor Keytruda (pembrolizumab) in the treatment of adult patients with locally advanced or metastatic urothelial carcinoma who have received prior treatment with PD-1 or PD-L1 inhibitors and platinum-based chemotherapy agents. The monotherapy option passed the Health Insurance Review and Assessment Service's Cancer Disease Deliberation Committee review in February this year, but the application was rejected after the government and pharmaceutical companies failed to agree on the cost-effectiveness after the company completed the pharmacoeconomic evaluation. In response, Astellas Pharma plans to supplement the monotherapy data and reapply for its reimbursement in addition to the combination therapy. The drug is recommended as Category 1 in the National Comprehensive Cancer Network (NCCN) guidelines. It is a new treatment option for urothelial cancer patients whose cancer has progressed or recurred even after receiving treatment with immunotherapy drugs and platinum-based chemotherapy. The drug was approved in March in Korea for the treatment of adult patients with locally advanced or metastatic urothelial cancer who have received prior treatment with PD-1 or PD-L1 inhibitors and platinum-based chemotherapy, then approved in combination with Keytruda in July. “The Padcev combination is the first first-line therapy in metastatic urothelial cancer in 30 years to change the treatment paradigm and demonstrate unprecedented clinical benefit. We will continue to work closely with the government and stakeholders to ensure that patients with metastatic urothelial cancer in Korea can benefit from the use of our therapy,” said a company official. Padcev’s efficacy as a monotherapy was demonstrated through the EV-301 study, an open-label, Phase III trial that was conducted on 608 patients with locally advanced or metastatic urothelial cancer who have previously been treated with platinum-based chemotherapy and PD-1 or PD-L1 inhibitors. Study results showed that Padcev reduced the risk of death by 30% compared to chemotherapy. The median overall survival (OS) of the Padcev group was 12.9 months, demonstrating a significant improvement in survival compared to chemotherapy's 9.0 months. In addition, Padcev significantly improved progression-free survival (PFS) with a 38% reduction in disease progression or death risk, with the median progression-free survival (PFS) for Padcev being 5.6 months and 3.7 months for the control group. In the case of the Keytruda-Padcev combination, its efficacy was demonstrated through the randomized Phase III EV-302 trial that was presented at the European Society for Medical Oncology Annual Meeting (ESMO 2023) last year. The trial evaluated Padcev+Keytruda versus conventional chemotherapy in 886 patients in 25 countries. In the trial, the combination demonstrated a median progression-free survival (PFS) of 12.5 months over a median follow-up of 17.2 months, with a 55% reduction in the risk of disease progression and death compared to 6.3 months in the placebo arm.
- Company
- New treatment options for pancreatic cancer arise
- by Son, Hyung Min Dec 09, 2024 05:47am
- New antibody drugs are showing results in pancreatic cancer, a disease area that has been regarded as an incurable disease. Recently, a new bispecific antibody drug from the Dutch company Merus was approved for the treatment of pancreatic cancer in the United States. In clinical trials, the drug has shown therapeutic effects in patients with pancreatic cancer and non-small cell lung cancer. In Korea, efforts to develop new antibody drugs for pancreatic cancer are also underway. Prestige BioPharma, Aptamer Sciences, and ABL Bio have confirmed the potential of antibody drugs in pancreatic cancer. Merus’s HER2·HER3 targeted bispecific antibody secures indications for pancreatic cancer and non-small cell lung cancer According to industry sources on the 7th, the US FDA approved Merus' bispecific antibody Bizengri for the treatment of pancreatic cancer and non-small cell lung cancer. Bizengri is the first bispecific antibody approved for neuregulin 1 gene (NRG1) fusion lung and pancreatic cancer. The drug received accelerated approval and is subject to further confirmatory clinical trials to determine full approval. Bizengri was first under clinical development as a HER2-targeted antibody but was redirected to simultaneously target the NRG1 gene fusion, a variant of the HER2 and HER3 antibodies, to increase the therapeutic effect. Bizengri’s license was based on a Phase I/II eNRGy study. The study included 30 patients with NRG1-positive pancreatic cancer and 64 patients with non-small cell lung cancer who had failed prior therapy. The mean age of the pancreatic cancer patients was 49 years, and 57% were male. The primary endpoints were overall response rate (ORR) and duration of response (DOR) as determined by Blinded Independent Central Review (BICR). Clinical results showed an ORR of 40% in patients with pancreatic cancer. DOR ranged from a median of 3.7 months to up to 16.6 months. Bizengri showed a strong suit in terms of safety. The most common adverse events occurring after treatment with Bizengri were diarrhea, musculoskeletal pain, fatigue, nausea, constipation, vomiting, and abdominal pain. Adverse reactions occurred in approximately 10% of patients and were generally mild. Merus is recruiting patients to participate in a confirmatory clinical trial for full approval of Bizengri. The company expects Bizengri to be effective in all cancer types driven by NRG1 fusions. Monoclonal antibody, ADC, bispecific antibody for pancreatic cancer Korean companies are also trying to develop new drugs for pancreatic cancer through antibody drugs. Prestige Biopharma, LigaChem Biosciences, ABL Bio, and Aptamer Sciences are checking the potential of monoclonal antibodies, ADCs, and bispecific antibodies. Pancreatic cancer has one of the lowest survival rates among cancer diseases. According to the National Cancer Center, the 5-year survival rate for pancreatic cancer from 2017 to 2021 is only 15.9%. Due to its organ location, pancreatic cancer has an early detection rate of less than 10%, making it easy to metastasize to surrounding organs. New drugs from various domestic and foreign pharmaceutical companies have tried their hand in this field, but most of them have failed in the clinical trial stage. As such, antibody drugs are emerging as an alternative. Antibody-drug conjugates (ADCs) and multi-antibodies that target 2 or more biomarkers at the same time are actively being tested to conquer intractable diseases. Prestige Biopharma is developing an antibody drug candidate, PBP1510. PBP1510 neutralizes the pancreatic ductal adenocarcinoma overexpression factor PAUF protein, which is a therapeutic target for pancreatic cancer. PBP1510 is currently in Phase I/IIa clinical trials in Spain, the United States, Singapore, and Australia. Through this study, Prestige Biopharma plans to determine the safety and tolerability of PBP1510 in combination with gemcitabine. Aptamer Sciences recently signed a collaboration agreement with Kolon Pharmaceuticals to jointly develop its ADC candidate AST-203. The two companies plan to conduct clinical studies of AST-203 with the goal of securing an indication for pancreatic cancer. Aptamer Sciences and Kolon Pharmaceuticals will jointly develop its ADC candidate AST-203 for pancreatic cancer AST-203 targets TROP2, a protein mainly expressed in breast, pancreatic, stomach, and lung cancers. The new drug candidate selectively binds to TROP2-positive tumors and penetrates the cell, releasing MMAE, which inhibits cell division, to induce cancer cell death. TROP2 is an intracellular calcium signal transducer involved in cell proliferation and survival. The protein is present in normal cells but tends to be overexpressed in cancer cells and has been linked to drug resistance. The only commercialized drug targeting TROP2 is Gilead's ADC Trodelvy, which is approved for triple-negative breast cancer. In preclinical studies, Aptamer Sciences has shown promise for AST-203 in tumor spheroid (3D aggregates of tumor cells) models. According to the company, AST-203 demonstrated a 6.7-fold higher tumor penetration rate than the existing Trodelvy. ABL Bio is developing bispecific ADCs targeting major solid tumors, including pancreatic, esophageal, colon, and head and neck cancers. Its target antigens are not yet known, but the company says it has shown efficacy compared to single-target ADCs. The company plans to file an IND later next year to test the bispecific ADC in the clinic.
- Company
- Auto part company confirms potential of novel antibody drug
- by Son, Hyung Min Dec 09, 2024 05:46am
- Kumho HT's candidate product for cancer immunotherapy demonstrated drug tolerance and effectiveness in human-subject clinical trials. At the European Society for Medical Oncology (ESMO) Asia Congress 2024, held for three days from December 6, the Phase 1 clinical trial result of Kumho HT's DNP-002, a new anticancer drug, has been unveiled. Kumho HT anticipates collaboration with global pharmaceutical companies based on the results from the Phase 1 clinical trial. In 2021, Kumho HT, an automotive electronics company, acquired the antibody drug developer DiNonA, officially making an entry into the biotechnology sector. The company's largest shareholder, S-MAC, aims to secure a new revenue stream through this acquisition, diversifying its business portfolio beyond electronic components and materials. Based on DiNonA's antibody drug development platform, Kumho HT is developing anticancer agents, immune modulators, and COVID-19 therapies. Cancer immunotherapy with a novel mechanism demonstrates results in the Phase 1 trial Kumhu HT According to industry sources on December 9, Kumho HT unveiled the Phase 1 trial result of its 'DNP-002,' a candidate product for cancer immunotherapy targeting CEACAM6, at ESMO ASIA. The current result has been presented in about four years after the approval of the Investigational New Drug Application (IND) in South Korea in 2020. DNP-002 targets 'CEACAM6,' a protein overexpressed in cancer cells and myeloid-derived suppressor cells (MDSC). By targeting both tumor and MDSC, it re-activates the patient's immune system. CEACAM is a novel target protein known to selectively target regulatory T cells expressed within tumors without affecting T cells. Cancer immunotherapy and antibody-drug conjugates (ADCs) targeting CEACAM 1, 5, and 6 are undergoing clinical trials for major solid tumors such as gastric and esophageal cancers. The clinical trial involved 36 patients with solid tumors registered at Asan Medical Center in Seoul and the National Cancer Center. The study aimed to evaluate the efficacy, safety, and tolerability of DNP-002. Participants were 18 years or older, had a prior treatment history, and had an Eastern Cooperative Oncology Group Performance Status (ECOG PS) of 1 or lower. Higher ECOG scores indicate more severe symptoms. The cohort was divided into four groups based on patient characteristics. Clinical results showed that 1 out of 12 patients who could be evaluated for tumor assessment had a partial response (PR) of over 30% reduction in cancer cells. Seven patients were confirmed to have a stable disease (SD) with maintained tumor size. Patients with esophageal cancer registered to another cohort group showed a total of 69% reduction in tumor size compared to the baseline of administration and maintained PR for 30 weeks. Researchers stated, "0.03mg/kg of DNP002 treatment demonstrated a partial response and drug tolerance. We are conducting a study to determine the maximum drug dosage." Automotive electronics company acquires a novel drug developer, making an entry into the biotech industry An automotive electronics company, Kumho HT, began generating results in developing cancer immunotherapy in 2021. At that time, Kumho HT's largest shareholder, S-MAC's Chief Executive Officer & Director Kyung-Suk Cho, acquired DiNonA, a company developing novel antibody drugs. Cho established a corporate governance structure spanning Ohsung Advanced Materials-S-MAC-Kumho HT-DiNonA-Hwail Pharmaceutical through 'East Burgundy,' a management consulting firm wholly owned by Cho. Cho identified biotechnology as a future cash cow, transitioning from a business structure focused on automotive parts and materials. DiNonA, acquired by Kumho HT, specializes in antibody-based drug development and is currently conducting clinical trials for three anticancer drug candidates. One of these is 'DNP-002,' for which the Phase 1 clinical trial results were recently disclosed. Additionally, the company is conducting the clinical development of 'DNP-007,' an immunosuppressant candidate that received IND approval for Phase 1 in 2021. Previously, in 2018, DiNonA also licensed four antibody drug candidates to Aprogen. Kumho HT DNP-007 modulates dendritic cells and targets an antibody that induces acquired immune tolerance. Immune tolerance is the process by which immune cells are prevented from attacking one of our cells. Without proper immune tolerance, autoimmune diseases such as rheumatoid arthritis and multiple sclerosis can occur. DNP-007 is an antibody therapy known as MD-3, a humanized anti-ICAM-1 antibody that employs a novel mechanism to induce immunosuppression in transplanted organs by modulating dendritic cells. The company is conducting joint research with Seoul National University Hospital for DNP-007. According to recently released preclinical study results, monkeys that received continuous administration of DNP-007 maintained normal liver function for over three years, while those treated with conventional immunosuppressants survived less than three months. The company aims to develop 'DNP-007' as an alternative to calcineurin inhibitors, which are known to cause severe side effects such as diabetes, neurotoxicity, renal dysfunction, and alopecia. Based on the antibody drug candidates acquired through DiNonA, Kumho HT is exploring the potential for developing a wide range of antibody-based therapies in addition to the field of oncology. The company has completed Phase 1 clinical trials for its leukemia antibody therapy 'DNP-001.' An ongoing study is also being conducted for the COVID-19 treatment 'DNP-019' and the atopic dermatitis therapy 'KHT-2031' for pets.
- Policy
- Janssen seeks reimb for Balversa in Korea
- by Lee, Tak-Sun Dec 09, 2024 05:46am
- ’Balversa (erdafitinib, Janssen),’ which has been approved as a targeted therapy for bladder cancer, has reportedly applied for reimbursement 2 years after being approved in Korea. It is believed that the company is seeking Balversa’s entry into Korea’s health insurance market based on its proven effectiveness in patients who have used immuno-oncology drugs. According to industry sources on the 6th, Janssen’s Balversa recently applied for a reimbursement decision to Korea’s Health Insurance Review and Assessment Service. Balversa was approved by the Ministry of Food and Drug Safety in January 2022. Specifically, the drug is indicated for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma (mUC) with FGFR2 or FGFR3 genetic alterations whose disease has progressed on or after at least one line of prior systemic therapy, which includes platinum-based chemotherapy, or whose disease has progressed within 12 months of neoadjuvant or adjuvant treatment with platinum-based chemotherapy. The drug gained attention as the first targeted therapy to target a specific gene mutation in bladder cancer. It works by inhibiting a mutation in the fibroblast growth factor receptor (FGFR), which is one of the biological signals involved in cancer cell growth, and 20 to 30% of patients are known to be carrying mutations. However, even after its approval, Balversa was not released in the domestic market for two years. Then, in October, the company held a business meeting and declared that it would officially launched the drug in the domestic market. The delay is explained by the fact that immuno-oncology drugs have settled as a major treatment option for bladder cancer. In response, Balversa has focused on demonstrating efficacy in patients whose disease progressed after immuno-oncology treatment. The THOR study included patients with metastatic urothelial carcinoma with FGFR3/2 mutations whose disease progressed after first-line treatment with an anti-PD-(L)1 immuno-oncology agent. The results showed a median overall survival (OS) of 12.1 months in the Balversa arm compared to 7.8 months in the chemotherapy arm over a median follow-up of 15.9 months, resulting in a 36% reduction in the risk of death. Based on the study, the U.S. FDA formally approved Balversa in January. With the efficacy data, the company seeks to start sales in Korea. It will be interesting to see if Balversa will enter the Korean health insurance market and become a new option in the bladder cancer treatment landscape.
- Policy
- Reimb extension discussions for Kyprolis break down
- by Lee, Tak-Sun Dec 06, 2024 05:57am
- The proposal to extend reimbursement coverage for the multiple myeloma drug Kyprolis (carfilzomib, Amgen) has failed to make it past the negotiating stage with the National Health Insurance Service. The negotiations, which began last October, have broken down. The National Health Insurance Service announced the news of Kyprolis' negotiation failure on the 4th while updating the list of drugs that have completed drug price negotiations on its website. Kyprolis was set reimbursement standards by the Health Insurance Review and Assessment Service's Cancer Disease Deliberation Committee in April as a ‘combination therapy used with daratumumab and dexamethasone (DKd regimen) for the treatment of multiple myeloma patients who have received one or more prior therapies’. However, the coinsurance rate for daratumumab is set at 100%, for the patient to pay in full as out of pocket price. Since then, the agenda has passed the Drug Reimbursment Evaluation Committee review and was in negotiations with the NHIS since October. In February 2018, Kyprolis was successfully listed on the reimbursement list as part of the RSA (risk-sharing agreement, refund type) scheme. The reimbursed therapies at the time were KRd therapy (Kyprolis+lenalidomide+dexamethasone) and Kd therapy (Kyprolis+dexamethasone). With the reimbursement, the out-of-pocket cost for the patients was reduced from KRW 10 million to KRW 500 to 600 thousand. The DKd regimen had shown evidence that it was effective in patients exposed to lenalidomide, which is why it is expected to be beneficial to patients if reimbursed. However, the breakdown in negotiations means it will take longer for the drug to be reimbursed. The drug will have to undergo another review by HIRA DREC before it can be re-negotiated with the NHIS. As a blockbuster drug that recorded sales of KRW 32.2 billion last year (IQVIA), it will be interesting to see how Amgen will respond to its negotiation breakdown.
- Company
- ‘Various treatment options emerged for ulcerative colitis'
- by Son, Hyung Min Dec 06, 2024 05:57am
- Tae Oh Kim, Professor of Gastroenterology, Inje University Haeundae Paik Hospital “Various treatment options such as biologics and JAK inhibitors have emerged for ulcerative colitis, but the patients’ options are limited because the government does not allow switching between drugs. Especially as the number of young patients in their 20s and 30s and their life expectancy is both increasing, securing various treatment options for these patients has become the more important.” Tae Oh Kim, Professor of Gastroenterology at Inje University Haeundae Paik Hospital explained so in a recent interview with Dailypharm regarding Korea’s ulcerative colitis treatment landscape. Ulcerative colitis is a chronic inflammatory growth disease of an unknown cause characterized by inflammation localized in the mucosal or submucosal layer of the large intestine. Due to the nature of ulcerative colitis, which is characterized by flare-ups and exacerbations and a varying clinical course, consistent medication is currently regarded as the only treatment option for affected patients. The good news is that there are many different treatment options available for the disease. There are several treatment options available for ulcerative colitis, including anti-inflammatory drugs, corticosteroids, immunomodulators, antibiotics, biologics, Janus kinase (JAK) inhibitors, and S1P receptor modulators. Kim said, “While many of these treatments have proven clinically effective, the patients we see in practice have different comorbidities, ages, etc. Therefore, we can't use the therapies based on clinical data alone,” said Dr. Kim. “We need to try drugs that are not only effective but also safe, but the current prescription standards for ulcerative colitis in Korea do not allow for effective treatment,” he added. Increased patients' life expectancy...“Various treatment options should be secured” According to the Health Insurance Review and Assessment Service, the number of patients with ulcerative colitis and Crohn's disease in Korea increased by 32% from 67,741 in 2017 to 80,289 in 2021. The prevalence of younger patients, especially those in their teens to 20s, has increased significantly due to Westernized eating habits. “As the life expectancy of the patients increased to 80-90 years, the more treatment options we have, the better,” said Kim. “However, all drugs lose their effectiveness over time. In the case of TNF alpha inhibitors, patients develop antibodies in the body, making it difficult to continue the use of the drug.: “We use drugs even if they are only 30% effective, and some patients do not respond to the drug from the beginning. However, due to the limitations of Korea’s insurance reimbursement system, we have to continue administering the ineffective drug to these patients.” In ulcerative colitis, switching between JAK inhibitors is not allowed in Korea. Korea’s insurance reimbursement standards allow patients to use steroids or immunosuppressants first, then switch to other therapies if they don't work. Biologics or JAK inhibitors can only then be used, but if a patient switches from one treatment to another, he or she cannot go back to the previous therapy. JAK inhibitors cannot be cross-administered. This is why the patients’ options are rather limited despite the many treatment options on the market. While developers have supported the use of various medications with efficacy and safety clinical data on switching, overseas cases, and accumulation of real-world data, the regulatory authorities have disallowed switching in ulcerative colitis, citing a lack of clinical evidence. “Some treatments have shown strengths in efficacy, such as Rinvoq, and others are strong in safety, such as Jyseleca,” said Kim. “However, I think it is problematic that we are not able to try new drugs due to Korea’s restrictive treatment selection standards. We should be allowed to switch drugs and then go back if the new option doesn't work for the patient. We may not have known enough about the effectiveness of the previous drug because it was used for a short period of time.” “Oral agents, such as JAK inhibitors and TYK2 inhibitors, have the advantage of not developing antibodies. It is important to increase patient access to a range of oral treatment options in addition to injectables, as antibodies can lead to loss of response. Some patients do not respond to any treatment. There is no set treatment sequence for ulcerative colitis and as patient characteristics also vary, it is important to have multiple treatment options available.”
- Policy
- Expanded clinical use of orphan drug 'Joenja' in children
- by Lee, Hye-Kyung Dec 06, 2024 05:56am
- Product photo of Joenja. The administration·dosage in children for a clinical trial of 'Joenja (leniolisb),' an APDS treatment designated as an orphan drug in South Korea, is expected to be expanded. The meeting record of the Central Pharmaceutical Affairs Advisory Committee (CPAC), released by the Ministry of Food and Drug Safety (MFDS) on December 4, indicates that there has been a consultation regarding the application for approval of a medication on clinical trial intended for therapeutic use, which is currently undergoing clinical trials in a foreign country. It was reported that the medication is Joenja, a treatment for activated phosphoinositide 3-kinase delta syndrome (APDS). In June, Joenja was designated as an orphan drug. APDS is caused by mutations in one of the genes encoding PIK3CD or PIK3R1 that is needed for the development and function of immune cells. The disease occurs in 1 in 1-2 million people. Patients exhibit autoimmunity and inflammatory symptoms. Symptoms include ears infections, paranasal infections, and upper·lower respiratory infections. Lymph nodes or the spleen may enlarge, and patients have increased risk of developing cancer, such as lymphoma. The approval of Joenja was based on the results of multinational, triple-blinded, placebo-controlled, randomized clinical 2/3 trials. The clinical trial involved 31 APDS patients over 12 years old. At the recent CPAC meeting, discussions were held on the age range eligible for approval based on pediatric administration·dosage and predicted through pharmacokinetic (PBPK) modeling data and case studies from a compassionate use program (10 patients). The CPAC concluded that approval could be granted for patients aged four years and older, with further discussions required for those under four. Regarding the PBPK modeling data, one committee member stated, "Metabolism is proportional to body weight and liver volume, with differences of about 20% observed even among adults, making application in children reasonable." "However, since kidney function, liver function, and body composition become similar to adults only after two, predicting results for patients under two years old is challenging." Most committee members state that the submitted modeling document presents no significant issue. One committee member commented, "FDA reports indicate that medication use in ages 4-11 will not pose any problem based on the modeling result. However, the applications below the age of 4 or 2 must need further discussion." Another committee member stated, "The CYP cell maturity is similar after age 2, larger liver size and hepatic clearance. We believe that this has been incorporated in the modeling." Once patients start taking medication, they are likely to continue treatment. Therefore, concerns have arisen that administration should only begin after safety results are reviewed. However, the committee chair noted that data collection could be challenging since the target population comprises children with rare diseases. Regarding this, "The medication has been designated as an orphan drug in both the U.S. and Korea, with four additional approved cases," the MFDS stated. "We need consultation on whether the clinical trial data from U.S. approval, submitted as evidence of safety and efficacy, along with the modeling data and case studies from 10 patients, can substitute for therapeutic confirmatory or exploratory clinical trial results." Consequently, it was concluded that CPAC must give a green light, as the unapproved status in South Korea and CPAC's opposition to access would deny Korean patients treatment opportunities. Meanwhile, since the currently available 70 mg product is a film-coated tablet that children may be unable to swallow, the company plans to provide the medication in 10 mg and 30 mg tablets.
- Policy
- Linking 'Prior review-performance evaluation-reevaluation'
- by Lee, Tak-Sun Dec 06, 2024 05:56am
- HIRA The Health Insurance Review and Assessment Service announced that it has been reviewing a measure that links preliminary review, performance evaluation, and reevaluation for newly listed high-priced drugs. In other words, for newly listed high-priced drugs, the authorities will conduct a proper cost-effectiveness evaluation through a preliminary review process. In this way, the new system will build a virtuous cycle with the existing preliminary review system. HIRA's Healthcare Review and Assessment Committee (HCRAC)’s Review Department and Review Division explained so at a press conference with press corp reporters on the 3rd. The Review Department and the Review Division are TF-type departments that were established this year to improve HIRA’s review standards. Min-Sun Kim, Head of the HCRAC Department, said, “The HCRAC Department was newly established earlier this year when a new task of overseeing and improving the general review standards was added to the committee’s preliminary review task.” Kim explained that the HCRAC's key achievements this year were the voluntary efforts it had made to improve the review standards and the establishment of a virtuous cycle of the preliminary review system. “As the need for preliminary review has increased with the increase in the number of high-priced drugs, we have established and operated a monitoring and return system that switches items that have been under review for a long time to post-review and quickly introduce newly listed high-priced drugs through preliminary review,” explained Kim. Of the 12 preliminary review items, 5 items, including a treatment for paroxysmal nocturnal hemoglobinuria, were stably transitioned to items for post-review, and a new treatment for the inherited retinal disease was introduced. In addition, the committee improved and expanded the reimbursement standards for 6 items through analysis of the review status and expert discussions. This year, HIRA switched the preliminary review status of Strensiq, Soliris Inj (paroxysmal nocturnal hemoglobinuria), Ultomiris Inj, ICD (implantable cardioverter-defibrillator), and CRT (cardiac resynchronization therapy) as subject to post review. Kim added, “In the case of preliminary review, we will promote the introduction of high-priced new drugs, and consider ways to utilize data that can be linked from preliminary review to performance evaluation and re-evaluation for a solid reimbursement management system of high-priced drugs." Jung Gu Kang, President of HIRA who also attended the conference, added, “Drugs whose data has not been verified at the time of their introduction should be objectively verified for cost-effectiveness through post-evaluations. Newly introduced drugs should undergo preliminary review to reduce unnecessary spending.” However, he said there are no plans to move the atypical hemolytic-uraemic syndrome treatment, which has been receiving many requests to transition the item to post-review due to its low pre-approval rate. “The reason we switched paroxysmal nocturnal hemoglobinuria treatments to post-review is because they are stable in terms of reimbursement coverage, but atypical hemolytic uremic syndrome treatments are not so,” added Kim, ”The Anti-Corruption & Civil Rights Commission did not recommend abolishment of the drug’s preliminary review status.” In addition to the reorganization of the preliminary review system, the HCRAC’s review office said it has improved the review standards for 114 applications. Kim explained, “At the end of last year, we reviewed a total of 410 cases, including spine surgeries that clinical societies and associations filed appeals and opinions on improving the review criteria and revised and reflected their opinion on 114 cases into review guidelines and notices, and improved the review process. We met and discussed with academic societies and held advisory meetings more than 50 times.” “Of the 410 cases, 58%, or 238 cases, were resolved within the year. For 124 cases where the medical community misunderstood the review and criteria, we provided detailed guidance to clinical societies and associations for better understanding of the medical community, and 114 cases with medical grounds were reflected in the review guidelines and notices.” “For the other 172 cases that have not yet been resolved, some cannot be resolved immediately due to lack of clinical evidence, while others need to be improved, but may take several months to a year or more to resolve due to equity issues between medical departments or financial requirements. We have shared these situations with clinical societies and associations, and we ask for the understanding and cooperation of the medical community and the government for their resolution.”
