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- 2025-12-21 14:56:01
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- The first RSV vaccine 'Arexvy' to launch in May in Korea
- by Whang, byung-woo Mar 25, 2025 05:54am
- As the launching date of Arexvy, known as the first respiratory syncytial virus vaccine, has been announced, the company aims to challenge a market share. Product photo of ArevyAccording to industry sources on March 22, GSK Korea has confirmed the launching date of the RSV-LRTD vaccine, Arexvy, as May. Arexvy received approval from the Ministry of Food and Drug Safety (MFDS) at the end of December 2024 for the 'Prevention of lower respiratory tract disease (LRTD) caused by RSB in adults over 60 years of age and older.' Approval of Arexvy was based on results from two Phase 3 studies, 'RSV OA=ADJ-006' and 'RSV OA=ADJ-004,' involving adults 60 years of age and older. The study results showed that during the first RSV season, Arexvy significantly lowered the RSV-LRTD risk by 82.6% and severe RSV-LRTD risk by 94.1% in participants 60 years of age and older compared to placebo. Furthermore, the efficacy of the vaccine regarding RSV-A-associated LRTD increases and RSV-B-associated LRTD increases were 84.6% and 80.9%, respectively. Arexvy has quickly secured the rank as the blockbuster product in the global market based on its first-in-class RSV vaccine title. The drug's influence in South Korea is gaining attention. If Arexvy launches in May, vaccination using the drug will likely begin in June, considering hospital landing settings. The remaining issue is how much the health authority considers RSV disease burden in seniors. Health authorities in major countries recommend RSV vaccination. However, the Center For Disease Control (CDC)'s Advisory Committee on Immunization Practices (ACIP) has narrowed the scope of vaccination. Previously, ACIP recommended vaccination in all adults of 60 years and older after consulting doctors. The ACIP has recently reduced the scope to adults 75 years and older or adults 60 to 74 years who have a high possibility of developing severe symptoms. In South Korea, some view that people would be less interested in Arexvy than the antibody injection for young children, Beyfortus (nirsevimab). Professor A from a department of infectious disease at one of the tertiary general hospitals in Seoul said, "Theoretically, RSV may likely affect those who are immune-compromised or older age. However, vaccines may not likely gain attention like those for newborns and young children," adding, "An additional investigation and evaluation may be needed to determine the potential impact since an adequate evaluation on disease burden has not been thoroughly established yet." In other words, even if a RSV vaccine targeting seniors are released, it may not immediately expand market compared to those targeting young children. Yet, potential demand may increase after the introduction of vaccine, as there has been little interest in the RSV vaccine for seniors. Considering these factors, GSK Korea will likely focus on raising awareness of RSV prevention along with Arexvy's launch. Hyunji Kwon, GSK Korea's Vaccine Business Unit Head, said, "RSV infection poses a significant physical and economic burden on high-risk groups, such as seniors. GSK will put efforts into preventing adult infections and relieving the disease burden of Korean patients after the successful launch of Arexvy."
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- 'Xeljanz' reimbursed for juvenile idiopathic arthritis
- by Eo, Yun-Ho Mar 24, 2025 05:52am
- Product photo of Xeljanz'Xeljanz' has become the first JAK inhibitor to be reimbursed for the treatment of juvenile idiopathic arthritis. The Ministry of Health and Welfare (MOHW) has recently announced on the administrative notification board regarding the 'The Criteria and Scope of National Health Insurance (Pharmaceuticals)' that the reimbursement criteria for Pfizer Korea's Xeljanz (tofacitinib) will be expanded from April. Xeljanz can be used to treat children (age 2 to 17) diagnosed with juvenile idiopathic arthritis according to the ILAR criteria (2001 revision), including ▲Polyarticular arthritis that affects five or more joints ▲Extended oligoarthritis ▲Psoriatic arthritis ▲Those who discontinued treatments due to inadequate response to one or more biological agent or side effects. After 6 months of usage, an additional 6-month usage will be approved if an assessment indicates a decrease of over 30% in the number of inflammatory joints compared to the initial administration timepoint. After that, the evaluation will be carried out every 6 months, and when the assessment result in the first 6 months is maintained, consistent administration will be approved. This reimbursement approval is the first among the JAK inhibitors. To date, 'Olumiant (baricitinib)' secured the same indication in September last year but is still non-reimbursed. Similarly, 'Rinvoq (upadacitinib)' has not been domestically approved for treating idiopathic arthritis. Following the patent expiration of Xeljanz, Pfizer has made efforts to increase product competitiveness by changing formulations in many ways. In 2020, the company launched an extended-release formulation with fewer administrations for various arthritis treatments. In 2023, Pfizer also launched a syrup formulation that is more convenient to administer to pediatric patients. It is to be watched whether Xeljanz, with an opportunity to take the market share, prescription will increase in the idiopathic arthritis area. Meanwhile, the efficacy of Xeljanz was demonstrated through the Phase 3 'JIA-I' trial. The study compared the effectiveness and safety of the drug to a placebo in 225 patients aged 2 years and above and those aged 18 years and below. In the study, Xeljanz tablet or syrup formulation (dosage depending on the weight range; 5 mg was administered less than twice a day) was administered for 18 weeks. Patients (142 individuals) who reached the JIA ACR 30 (symptom improvement over 30%) were divided into Xeljanz and placebo groups. The results at week 44 confirmed that the symptom worsening in the Xeljanz group (29%, 72 individuals) was significantly lower than those in the placebo group (53%, 70 individuals). During the same period, the rate of reaching the JIA ACR30∙50∙70 (30∙50∙70% symptom improvement) was higher in the Xeljanz group. Physical function measured by the Childhood Health Assessment Questionnaire (CHAQ) also confirmed significant improvement in the Xeljanz group (-0.11) compared to the placebo group (0.00).
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- RSV vaccine Beyfortus lands in Big 5 Hospitals in Korea
- by Eo, Yun-Ho Mar 24, 2025 05:52am
- The respiratory syncytial virus (RSV) preventive antibody injection ‘Beyfortus’ has landed in the Big 5 tertiary hospitals in Korea. According to industry sources, Sanofi Korea's Beyfortus (nirsevimab) has passed the drug committees (DCs) of the Big 5 tertiary hospitals in Korea, including Samsung Medical Center, Seoul National University Hospital, Asan Medical Center, and Severance Hospital. In addition, the drug has also landed at medical institutions such as Gangnam Severance Hospital, Korea University Anam Hospital, Korea University Ansan Hospital, Bundang Severance Hospital, and Pusan National University Yangsan Hospital. Also, Beyfortus vaccination has begun at local clinics since February. SK Bioscience is in charge of Beyfortus’s promotional activities for medical institutions at the clinic level. Beyfortus is an RSV preventive antibody injection that received approval from the Ministry of Food and Drug Safety in May last year and can be administered to all newborns and infants who are entering their first RSV season. Also, children up to 24 months of age who remain at risk of severe RSV disease through their second RSV season may receive Beyfortus. Previously, RSV prevention products for infants and toddlers in Korea were only administered to high-risk infants and toddlers, such as premature babies, who are at high risk of severe RSV disease. However, Beyfortus is different in that it can be administered to all infants and toddlers. According to the Phase III MELODY trial, which was the basis for the approval of Beyfortus, RSV lower respiratory tract infections were reduced by 74.5% in the Beyfortus-administered arm. This study evaluated the efficacy of Beyfortus against RSV infection with medical management up to 150 days after administration in 3,012 infants born after 35 weeks of gestation in their first RSV season. In addition, according to the interim results of the national vaccination program being implemented in Galicia, Spain, real-world evidence of Beyfortus, hospitalizations due to RSV in infants under 6 months of age who received Beyfortus were reduced by 82% compared to infants who did not receive Beyfortus. Ki-Wook Yoon, a Professor at Seoul National University Hospital, said, “RSV can infect people of all ages, but 90% of infants under the age of 2 are infected. When infected, it can lead to mild cold symptoms to hospitalization due to lung infection. Infants whose bronchial tubes are not fully mature can have more severe symptoms when they are infected with RSV, which can cause losses not only for family members but also to the society and economy.” He added, “Until now, RSV prevention was limited to personal hygiene practices, showing a clear unmet demand for RSV. However, we expect that active RSV prevention will become possible with the introduction of the preventive antibody injection.”
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- 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.
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- 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.”
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- "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.
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- 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.”
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- 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."
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- 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.
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- 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.
