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- 2025-12-22 14:57:54
- Company
- Bispecific multiple myeloma drug 'Talvey' can be prescribed
- by Eo, Yun-Ho Mar 21, 2025 06:00am
- Product photo of Talvey New bispecific multiple myeloma drug 'Talvey' can now be prescribed at general hospitals. According to industry sources, Janssen Korea's Talvey (talquetamab) has passed the drug committees (DC) of tertiary general hospitals, including Samsung Medical Center and Seoul National Univeristy Hospital, and medical institutes, including Pusan National University Hospital and Chung-Ang University Hospital. Talvey was approved by the Ministry of Food and Drug Safety (MFDS) as a monotherapy for adult patients with relapsed or refractory multiple myeloma who have received over three prior lines of treatments, including proteasome inhibitors, immune modulators, and anti-CD38 monoclonal antibody. Multiple myeloma is a type of cancer with the number of patients increased by over 60% in the past 10 years. Many patients with multiple myeloma experience relapse. According to a cohort study, multiple myeloma patients who had received more than three types of treatments, including anti-CD38 monoclonal antibody, demonstrated an overall response rate (ORR) of below 30%, with short life expectancy and poor prognosis. Consequently, patients needed a treatment option with a new mechanism. Talvey is a first-in-class bi-specific antibody drug approved in South Korea, targeting CD3 receptor and G protein-coupled receptor class C group 5 member D (GPRC5D). It can be administered to patients who had previously used proteasome inhibitors, immune modulators, or anti-CD38 monoclonal antibodies. The efficacy of Talvey was demonstrated through the global clinical trial, 'MonumenTAL-1 study.' The study results showed that 143 patients who do not have prior treatment experience with T-cell redirecting therapy were treated with Talvey 0.4 mg/kg every week. The patients treated with Tarvey had an ORR of 74.1% and a stringent complete response (sCR) of 24%. Patients demonstrated 10% complete response (CR) and 26% very good partial response (VGPR). Meanwhile, bispecific antibody drugs are immune cell therapy consisting of two monoclonal antibodies that recognize targeted antigens and T cells of multiple myeloma. For instance, bispecific IgG2 kappa antibody, consisting of two monoclonal antibodies that recognize B-cell maturation antigen (BCMA) and CD3 antigens, is commonly used. It is a new therapy that directly delivers cytotoxicity-triggering T-cells into multiple myeloma cells expressing BCMA. Bispecific antibodies approved in South Korea include Pfizer's 'Elrexfio (elranatamab)' and Janssen's 'Tecvayli (teclistamab),' and Talvey. These drugs remain as non-reimbursed drugs.
- Company
- Prevnar 20 added to the National Immunization Program
- by Whang, byung-woo Mar 21, 2025 05:59am
- Pfizer is signaling full-fledged competition with the addition of its new pneumococcal vaccine, Prevnar 20, to the National Immunization Program (NIP) for children. 프리베나20 제품사진According to industry sources on the 21st, the Korea Disease Control and Prevention Agency recently reviewed the introduction of PCV20 NIP for children during the 1st Korea Expert Committee on Immunization Practices meeting. As a result, the committee decided that PCV20 would be introduced under the NIP, for the same target population and standard immunization schedule as for PCV13 and PCV15, which were already covered through the NIP. Initially, it was widely believed that the entry of Prevnar 20 into the NIP would be delayed to next year because the Public Procurement Service announced a plan to execute a tender for the purchase of Prevnar 13, but discussions progressed with Pfizer compromising its price with the government. At the meeting, the committee allowed the cross-vaccination of Prevnar 20 as an exception for the additional immunization after completing basic immunization with Prevnar 13. Currently, the vaccine price and immunization practice for pneumococcal vaccines are fully covered by the government for children under the age of 12. Both Prevnar 13 and Prevnar 20 are administered once to adults aged 18 or older, but infants and those who have not received the basic vaccination are required to receive multiple doses at regular intervals depending on the number of months. For example, Prevnar 13 is administered 3 times at 0.5 mL per dose at 2, 4, and 6 months of age in infants up to 6 months of age, so those who started this vaccination with Prevnar 13 may receive subsequent vaccination with Prevnar 20. In fact, Prevnar 20 is indicated for “Infants, children, and adolescents who have received at least 1 dose of Prevnar/Prevnar 13 may switch to Prevnar 20.” Prevnar 20 is a new pneumococcal vaccine that Pfizer has introduced in 14 years, and it is a vaccine that adds 7 serotypes (serotypes 8, 10A, 11A, 12F, 15B, 22F, and 33F) to the previously supplied Prevenar13. The industry believes that although Prevnar 13 is already on the market, the weight will naturally shift to using Prevnar 20. Pfizer is expected to accelerate preparations for the launch as the pediatric NIP, which had been a concern for the launch, has been resolved. The industry expects the launch of Prevnar 20 to take place in April, and the sales of Prevnar 20 for infants and toddlers will be maintained by Korea Vaccine, which was previously in charge of sales of Prevnar 13. The company is likely to employ a sales strategy that naturally absorbs the market share of its predecessor, Prevnar 13. In the case of Vaxneuvance, the company’s strategy is important as its competitor entered the market just one year after its launch. It is predicted its company will seek opportunities in the timing when people switch from Prevenar 13 to another vaccine. During the media seminar that was held last year for Vaxneuvance, Jaeyoung Cho, Executive Business Unit Director of Vaccines at MSD, said, “The number of infants and toddlers receiving their initial dose of the pneumococcal vaccine, which is covered through NIP, as well as those switching from the existing 13-valent vaccine to Vaxneuvance have been increasing.” “Like Vaxneuvance, which was also applied to the NIP upon its release, we expect Prevnar 20 to quickly expand its influence in the market,” said a vaccine industry official. ”Since both Prevnar 13 and 20 are Pfizer vaccines, the switching hurdle is relatively low, so MSD would likely be considering countermeasures.”
- Policy
- Enhertu’s reimb may be extended to gastric cancer
- by Lee, Tak-Sun Mar 21, 2025 05:59am
- The number of patients who can use the new antibody-drug conjugate (ADC) anticancer drug ‘Enhertu Inj (trastuzumab deruxtecan, Daiichi Sankyo Korea), which has been reimbursed since April last year, is expected to be expanded to cover eligible gastric cancer patients. This is an expansion in the scope of existing patients eligible for reimbursement. The Health Insurance Review and Assessment Service's Cancer Disease Deliberation Committee voted on the 19th to expand the reimbursement coverage of Enhertu. Enhertu is a next-generation ADC that combines a monoclonal antibody with the same structure as trastuzumab, which binds to a specific target receptor overexpressed on the surface of cancer cells, and a topoisomerase I inhibitor payload with a tumor-selective cleavable linker, which is a novel and highly potent mechanism of action. The drug is expected to have a higher therapeutic effect than existing anticancer drugs. The drug was added for reimbursement in April last year by applying a flexible incremental cost-effectiveness ratio (ICER) threshold, an indicator of economic efficiency. As a result, the patient's burden from annual medication costs was reduced from KRW 80 million to KRW 4 million. At the time, the reimbursement standard for gastric cancer patients was set as locally advanced or metastatic gastric adenocarcinoma or adenocarcinoma of the gastroesophageal junction that satisfies all three conditions. The conditions were: patients who ①failed two or more prior therapies, including trastuzumab (Herceptin) +(fluorouracil or capecitabine)+cisplatin; ②have HER2 overexpression (IHC 3+ or 'IHC 2+ and FISH+ or SISH+’) metastatic gastric adenocarcinoma or gastroesophageal junction adenocarcinoma; and ③have an ECOG performance status (PS) of 0 or 1. This expansion of the reimbursement standard this time will extend the scope of the drugs limited to cisplatin in the first condition to other platinum-based anticancer drugs. As a result, it is expected that not only cisplatin but also patients with gastric cancer who have failed to receive oxaliplatin (original Eloxatin) will be able to receive Enhertu with reimbursement. “Enhertu’s reimbursement expansion agenda extends the conditions limited to cisplatin among platinum-based anticancer drugs in the existing reimbursement standards to other platinum-based anticancer drugs,” said a HIRA official. “This is expected to further expand patient access.” The applications for which the reimbursement standards have been established at this meeting will be reviewed by the Drug Reimbursement Evaluation Committee to determine their reimbursement adequacy.
- Company
- "Leclaza combo improves survival than Tagrisso monotherapy"
- by Son, Hyung Min Mar 21, 2025 05:58am
- It has been reported that the Leclaza plus Rybrevant combination therapy demonstrated improved overall survival (OS), in addition to progression-free survival (PFS), than Tagrisso. According to industry sources on March 20, the European Society for Medical Oncology made abstracts available for the 'European Lung Cancer Congress 2025(ELCC 2025),' which is scheduled to be held in Paris, France, for four days from March 26. One of the most anticipated study results at the ELCC 2025 is the Phase 3 'MARIPOSA' trial, evaluating the efficacy of Leclaza plus Rybrevant combination therapy. Leclaza, developed by Yuhan, is a new drug for EGFR-positive non-small cell lung cancer (NSCLC). It is a third-generation tyrosine kinase inhibitor (TKI) targeting exon 19 and exon 21 (L858R). Johnson & Jonhson acquired global sales rights of Leclaza and is conducting the MARIPOSA clinical trial, evaluating the efficacy of the drug in combination with Rybrevant, targted treatment option for mutations in exon 20 and MET. Previously, Leclaza plus Rybrevant combination therapy demonstrated efficacy regarding the primary endpoint of PFS. However, only a favorable trend of the combination therapy was shown in terms of the secondary endpoint of OS. The final OS data of Leclaza plus Rybrevant combination therapy were presented at this conference. PFS indicates how long a new treatment can delay the cancer progression, while OS shows data on how long it can extend overall survival. YuhanThe clinical trial compared the efficacy and safety of Leclaza plus Rybrevant combination therapy to Tagrisso monotherapy in 1074 patients with NSCLC harboring exon 19 and L858R mutations who do not have prior treatment experience. Leclaza monotherapy was included to evaluate the contribution component. Patients were randomly assigned at a 2:2:1 ratio to Leclaza plus Rybrevant combination therapy group (429 individuals), Tagrisso group (429 individuals), and Leclaza group (216 individuals). During the median value of 37.9 months, Leclaza plus Rybrevant combination therapy group had a statistically significant improvement in survival rate compared to Tagrisso group. In detail, the median OS of Leclaza plus Rybrevant combination therapy group could not be estimated (42.9-NE), whereas Tagrisso group had an OS of 36.7 months. Considering the survival rate index distribution of the two groups, Leclaza plus Rybrevant combination therapy group is expected to extend an OS over at least 12 months. Furthermore, at 36 months follow-up, Leclaza plus Rybrevant combination therapy group had a 60% survival rate, whereas Tagrisso group had a 51%. "The Leclaza plus Rybrevant combination therapy is the first therapy in patients with progressive NSCLC harboring EGFR mutation that significantly lowered the death risk compared to Tagrisso. The latest clinical trial showed the potential of the drug as the new standard therapy. The study results indicate that the combination therapy can improve survival rate by at least 12 months compared to Tagrisso," the research team remarked. The study results of the MARIPOSA clinical trial will be orally presented by Professor James Chih-Hsin Yang, Department of Oncology at the National Taiwan University, on March 26.
- Opinion
- [Reporter’s View] New Drug Trends and Choline Alfoscerate
- by Lee, Tak-Sun Mar 21, 2025 05:57am
- The Health Insurance Review and Assessment Service's Drug Reimbursement Evaluation Committee, which holds meetings every month, is the biggest hurdle to reimbursement in Korea and helps identify the latest new drug trends. For several years now, the keywords that have led the trend were “anticancer drugs,” “rare drugs,” “biological drugs,” and “high-priced drugs.” Contrary to how the trend leaned toward chronic disease treatments that owned a large patient population in the past, biologic targeted treatments for a small number of patients have become the trend these days. The characteristic of these drugs is that they are expensive. In line with the trend, the Health Insurance Review and Assessment Service and the National Health Insurance Service are in the process of establishing a system to manage such high-priced anticancer and rare drugs. Global pharmaceutical companies are now investing in anticancer and rare drugs with fewer patients and less competition, rather than developing synthetic drugs for chronic diseases, which become flooded with generics upon patent expiry. The companies’ investments have now been making results at the Drug Reimbursement Evaluation Committee level. However, the names of domestic pharmaceutical companies are rarely mentioned at DREC due to their lack of new drug development. Occasionally, the release of a new homegrown drug arises as a hot topic, but usually, the main players deliberated by DREC are multinational pharmaceutical companies. Domestic pharmaceutical companies are increasingly drifting apart from the new drug trend. Traditional pharmaceutical companies have missed the timing when biosimilar development began in the mid-to-late 2000s. When a bio-venture called Celltrion announced its challenge into the biosimilar field, the other companies ignored it, saying things like “there is no real profit” and “there is little chance of success.” When Samsung followed into the biosimilar market and achieved economies of scale, it was already too late. Currently, the top 2 pharmaceutical and bio companies in Korea are Celltrion and Samsung Biologics. I wonder what would have happened if domestic pharmaceutical companies had invested in biosimilars with the capital they had at the time, with a view to the future. Biosimilars are just one trend. The pharmaceutical market has been changing rapidly for the past decade. New diabetes drugs have been introduced one after another, and the cancer drug market has evolved from treatments targeting resistant mutations to immuno-oncology drugs such as Keytruda. The pace of pharmaceutical trends is too fast for Korean pharmaceutical companies to keep up with them, with the big pharma companies aggressively buying rare disease treatments through mergers and acquisitions. Nevertheless, Korean pharmaceutical companies are still stuck in the past. Many of the top-selling products of Korean pharmaceutical companies are the brain function enhancer, choline alfoscerate, which is already nearly 30 years old. In 2020, the government’s re-evaluation reduced the indications for the drug, and even though clinical trials are still underway due to doubts about their efficacy, sales of choline alfoscerate drugs have grown. It is a reliable cash cow for pharmaceutical companies. On the 13th, the Supreme Court ruled in favor of the government in a lawsuit filed by 26 pharmaceutical companies to cancel the notice of selective reimbursement of choline alfoscerate. With this ruling, it is likely that their selective reimbursement, which has been deferred with the lawsuit, will soon become a reality. If reimbursement for the drug is only applied to dementia diseases and excludes mild cognitive impairment, choline alfoscerate will no longer be able to serve as a cash cow for domestic pharmaceutical companies. While domestic pharmaceutical companies have managed to maintain their sales by defending the reimbursement reduction for their choline alfoscerate products for 5 years, this may have rather pushed companies to fall further behind in the market trends of anticancer drugs and orphan drugs. Of course, there are also pharmaceutical companies, such as Boryung and Handok that are increasing investment in the anticancer and orphan drug markets. However, there is concern whether most pharmaceutical companies will lose their investment timing as they struggle to maintain domestic sales, as was the case during the biosimilar boom. The global market's number one immuno-oncology drug, Keytruda’s follow-on is a biosimilar, not a generic drug. Celltrion and Samsung Biologics have just begun development efforts, but it is just a hope for domestic pharmaceutical companies.
- Company
- Hanmi and Samsung Bioepis join forces to sell Prolia similar
- by Cha, Jihyun Mar 20, 2025 06:01am
- Jae-hyun Park, CEO of Hanmi Pharmaceutical (right), and Kyung-ah Kim, CEO of Samsung Bioepis (left), are taking a commemorative photo after signing a joint sales agreement. (Source: Hanmi Pharmaceutical, Samsung Bioepis) Hanmi Pharmaceutical and Samsung Bioepis announced on the 19th that they had signed a joint sales agreement on the 18th for the domestic launch of the osteoporosis treatment Prolia biosimilar (Project name: SB16, ingredient name: denosumab) in Korea. Under the agreement, Samsung Bioepis is responsible for the production and supply of the product as the developer of the Prolia biosimilar. Afterward, both companies will jointly be in charge of domestic marketing and sales activities. Prolia is an osteoporosis treatment developed by Amgen. As of 2024, its global sales neared approximately KRW 6.5 trillion. According to IQVIA, the size of its domestic market sales in 2024 is expected to be approximately KRW 174.9 billion. Currently, Samsung Bioepis is in the process of obtaining approval for the Prolia biosimilar in Korea. It was approved in the US and Europe, by the respective regulatory authorities in February. Hanmi Pharmaceutical and Samsung Bioepis plan to establish a close cooperative relationship and strive to provide patients with the opportunity to use biopharmaceuticals at more reasonable prices. “Based on our development capabilities and Hanmi Pharmaceutical's sales and marketing expertise, we will work closely together to ensure that Korean patients can experience more benefits from the prescription of our biosimilar,” said Kyung-ah Kim, CEO of Samsung Bioepis. “Hanmi Pharmaceutical is a ready partner that has already established a foothold in the musculoskeletal treatment market,” said Jae-hyun Park, CEO of Hanmi Pharmaceutical. ”Through this partnership, we aim to create innovative results that will enable mutual growth for both companies and will continue to work closely together.”
- Company
- Will COVID-19 vaccines be added to the NIP this year?
- by Whang, byung-woo Mar 20, 2025 06:00am
- As the government began the process of reviewing the inclusion of COVID-19 vaccines in the National Immunization Program (NIP) list, attention has been drawn to whether COVID-19 would be included in the NIP list within this year. It has been reported that the Korea Disease Control and Prevention Agency (KDCA) and major pharmaceutical companies that own COVID-19 vaccines began discussing NIP inclusions.According to industry sources on March 20, the Korea Disease Control and Prevention Agency (KDCA) and major pharmaceutical companies that own COVID-19 vaccines began discussing NIP inclusions. The 2024-2025 seasonal COVID-19 vaccination has been implemented from October 11, 2024, until April 30. The current vaccination is aimed at preventing disease severity and death in high-risk individuals, including immuno-compromised individuals and inpatients‧inmates in facilities that are vulnerable to infections, who are aged 65 and older or 6 months or above. For vaccination, the new vaccine JN.1 COVID-19 vaccine (Pfizer‧Moderna‧Novavax), effective for mutations, has been used. The KDCA recommends vaccinations with both COVID-19 and influenza vaccines for the 2024-2025 season. Both Influenza vaccine and COVID-19 vaccines are provided at no cost, but they differ in terms of NIP coverage. Influenza vaccines are covered with NIP, while COVID-19 vacines are provided through a separate budget outside of the NIP. For the 2025-2026 seasonal vaccination with COVID-19 vaccines, the KDCA plans to change the program as a form of local government business under the NIP. In fact, the KDCA has begun researching and reviewing to evaluate the validithy of including COVID-19 vaccines to the NIP since last year. The research on the 'Evaluation of the validity of newly introducing COVID-19 to the NIP' led by Kangwon University-Industry Cooperation Foundation started on April 29, 2024, and will be completed on April 28. Results are expected to be presented in May, thus the discussion on COVID-19 inclusion in the NIP will be completed in the first half of this year. Considering that NIP vaccines are provided through the Public Procurement Service, several opinions suggested KDCA would decide in April. However, the vaccine industry believes that the decision is not imminent considering the different characteristics of influenza. "Influenza vaccines are produced after the WHO announces the type of virus, so there could be a gap in the time. However, the situation is different for COVID-19, so once the NIP inclusion is determined, procurement will be announced," a vaccine industry personnel said. However, if the type of COVID-19 vaccine for NIP inclusion is limited to one, it could be offered with a private contract. A private contract is established when there is insufficient time for price competition or when meeting the contract's goal through competition is determined challenging. "The number of vaccinations after the NIP inclusion may not differ from the current situation, but we still have to wait for the research results," a vaccine industry personnel said. "Several vaccines will be included in the NIP if there are several types of vaccines. However, if those vaccines do not meet the criteria set by the KDCA, only one vaccine could be included in the NIP."
- Company
- Pfizer’s Elrexfio fails to pass CDDC's reimb review in KOR
- by Eo, Yun-Ho Mar 20, 2025 06:00am
- New treatment options in the field of multiple myeloma are having difficulty receiving reimbursement listing in Korea. Following the rejection of Janssen Korea’s ‘Tecvayli (teclistamab)' in November last year, Pfizer Korea's ‘Elrexfio (elranatamab)' also failed to pass the Health Insurance Review and Assessment Service's Cancer Disease Deliberation Committee in February. As the Ministry of Food and Drug Safety designated the drug as a Global Innovative Product on Fast Track (GIFT) and approved it through the fast track, the news was received with a great deal of disappointment in the field. However, the pharmaceutical company's will does not seem to be dampened. A Pfizer representative said on the 19th, “We will again attempt reimbursement to increase treatment access for patients. We will do our best to reduce our patients’ burden.” Multiple myeloma occurs in the plasma cells of the bone marrow. As a blood cancer that mainly occurs in the elderly, its continued treatment can prolong life for the patients. Among the various new drugs being developed in this field, monoclonal antibodies and bispecific antibodies are being used in the medical field. In particular, bispecific antibodies are considered a safe and effective treatment option for relapsed or refractory multiple myeloma, where resistance increases with each treatment cycle, resulting in shorter remission periods and fewer available treatment options upon each line of treatment. The approved bispecific antibody treatment options in Korea, include Elrexfio, Tecvayli, and Talvey (talquetamab). However, all of them are not reimbursed in Korea. Amid the failed discussions over coverage of a series of bispecific antibody drugs in the early stages, whether any drug will be granted reimbursement and improve patient access is gaining attention. Meanwhile, Elrexfio was designated by the Ministry of Food and Drug Safety as a GIFT item and was approved as a monotherapy for adult patients who have received more than three lines of treatment, including proteasome inhibitors, immunomodulators, and anti-CD38 monoclonal antibodies, in May last year. The US FDA has also designated it as an innovative drug and granted accelerated approval for the drug. Elrexfio’s efficacy was demonstrated through the Phase II MagnetisMM-3 trial, an open-label, multicenter, non-randomized study that was conducted on 123 who had not received prior BCMA-directed therapy (i.e., BCMA-naïve patients). Results of Cohort A showed that the drug recorded an objective response rate (ORR) of 61.0% and a complete response (CR) of 37.4%. The progression-free survival (PFS) period was 17.2 months, and the overall survival (OS) period was 24.6 months, demonstrating an unprecedented long-term treatment effect. The data proved that long-term survival benefits and slowing disease progression can improve the quality of life of patients who have no other treatment options.
- Policy
- Public-private meetings on improving drug pricing system
- by Lee, Tak-Sun Mar 20, 2025 06:00am
- Attention has been gathered to recent public-private meetings regarding improving drug pricing system. This month, a public-private discussion session hosted by the Ministry of Health and Welfare (MOHW) and a meeting to discuss the negotiation system for expanded use of scope were held. On March 26, a regular meeting session was attended by three pharmaceutical organizations, including the National Health Insurance Service (NHIS), the Democratic Party of Korea's Future Economic Growth Strategy Committee, the Korean Research-Based Pharma Industry Association (KRPIA), and the Korea Biomedicine Industry Association (KoBIA), is scheduled. The public-private meeting was temporarily discontinued last year and resumed in February. It has been reported that two sessions have been held this year to discuss the agenda for improving the drug pricing system. The public-private meetings are attended by government agencies, such as the MOHW, NHIS, and Health Insurance Review and Assessment Service (HIRA), and three pharmaceutical organizations. As the agenda for 'incentivizing new drugs with innovative values' has been established through the public-private body in 2023, attention has been gathered to the outcome of the discussion on the drug pricing system through the upcoming meeting. The industry is expected to recommend pricing drugs based on specific indications. The organization for the negotiations system for expanded scope of use announced establishing guidelines for priority negotiations this year. Improving the negotiation system for expanded scope of use is based on the research outcome of the research initiated by the NHIS (Professor Euna Han's research team in the College of Pharmacy at Yonsei University) last year. However, the MOHW and the industry have decided to establish negotiation guidelines at this year's meetings rather than bringing improvements to the system itself. The focus of the regular meeting between NHIS-three pharmaceutical organizations on March 26 will be discussing improvements to the negotiations for expanded scope of use and choices of medicines and adjusted criteria based on the draft for 'incentivizing new drugs with innovative values.' The health authority said, "It is a regular meeting attended by pharmaceutical companies every three months," adding, "We plan to develop an agenda for system improvements through continued meetings." Meanwhile, the public-private meeting initiated by the MOHW to improve the system for surveying pharmaceutical prices in the market completed hearing opinions. It has been reported that the MOHW is contemplating on whether to conduct an additional session or write a draft based on gathered views to date. The industry expects the draft to be announced in the first half of the year. The research contains sensitive data, including companies subjected to the survey, so whether it will be reflected in the draft gathers attention.
- Company
- Will the monthly ₩20M Welireg be reimbursed this time?
- by Moon, sung-ho Mar 20, 2025 05:59am
- MSD Korea is re-applying for reimbursement of the rare anticancer drug ‘Welireg,’ following the re-application for reimbursement of its Keytruda Results of the first hurdle, whether the Cancer Disease Review Committee sets a reimbursement standard, are expected to determine the fate of the drug’s quick reimbursment. # According to industry sources, MSD Korea’s oral hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor Welireg (belzutifan) will be once again presented to the Health Insurance Review and Assessment Service’s Cancer Disease Deliberation Committee on March 19th. Von Hippel-Lindau disease is a hereditary disease associated with tumors in various organs and is caused by genetic abnormalities in the tumor suppressor gene VHL. It is characterized by causing various malignant and benign tumors, including central nervous system hemangioblastoma, retinal hemangioblastoma, lymphatic cysts of the inner ear, cysts of the kidneys and pancreas, pheochromocytoma and paraganglioma of the adrenal gland, and adenoma of the gonads, which occur in the brain and spinal cord. Its incidence rate is about 1 in 36,000 people, and it is estimated that about 200,000 people worldwide and about 10,000 people in the United States are suffering from it. In Korea, the number of adult patients with VHL disease is estimated to be in the low to mid-200s, based on data from the Health Insurance Review and Assessment Service. Based on this, Welireg was designated an orphan drug in Korea for the treatment of Von Hippel-Lindau disease in 2023, then formally approved in May of the same year. The company then submitted a reimbursement review application to the Health Insurance Review and Assessment Service (HIRA) in April last year and embarked on a full-fledged reimbursement journey. However, in August of the same year, the application was submitted to the CDDC, but the committee concluded that the reimbursement criteria should not been set, resulting in the first setback. The company then again submitted an application and the application is likely to be resubmitted at the 2025 3rd CDDC meeting scheduled for the 18th of this month. Meanwhile, the voices of patient groups are growing along with the national petition urging for Welireg’s reimbursement. In fact, the petitioner of the petition for national consent urged the government to reimburse the drug, imploring, “The price of one month (90 tablets) of Welireg, the only treatment for VHL, is KRW 22.61 million, aggravating the economic burden of the patients.” As a HIF-2α inhibitor, Welireg reduces transcription and expression of HIF-2α target genes associated with cellular proliferation, angiogenesis, and tumor growth. The drug’s efficacy was demonstrated in the open-label Study 004 trial, which investigated 61 patients with VHL-associated RCC who were diagnosed with at least one measurable solid tumor localized to the kidney.
