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- 2025-12-22 08:17:47
- Company
- 'Retevmo' also seeks thyroid cancer reimbursement
- by Eo, Yun-Ho May 12, 2025 05:59am
- Product photo of Retevmo The RET-targeted cancer therapy 'Retevmo' seeks insurance reimbursement listing of its medullary thyroid cancer indication. According to sources, Lilly Korea recently submitted reimbursement applications simultaneously for the non–small cell lung cancer indication and medullary thyroid cancer indication of the RET (REarranged during Transfection) inhibitor Retevmo (selpercatinib). Having failed in its previous two attempts to secure reimbursement listing for lung cancer, the company is now showing its determination to gain listing by adding a new indication. The prevalence of medullary thyroid cancer is only 1–2% of all thyroid cancers worldwide and just 0.6% in South Korea. However, 60% of cases harbor RET gene mutations. In other words, RET-targeted therapies can be used to treat medullary thyroid cancer. First-line treatments for medullary thyroid cancer currently consist of multitargeted kinase inhibitors such as vandetanib and cabozantinib, which inhibit multiple kinases non-selectively and can therefore be associated with higher rates of adverse events. In contrast, Retevmo is a highly selective RET inhibitor. Retevmo demonstrated efficacy in medullary thyroid cancer in the Phase 3 LIBRETTO-531 study. The LIBRETTO-531 study is a Phase 3 open-label trial comparing Retevmo against the approved first-line multikinase inhibitors (cabozantinib or vandetanib) in patients with progressive or metastatic RET-mutant medullary thyroid cancer. A total of 291 medullary thyroid cancer patients with no prior multikinase inhibitor therapy were randomized (193 Retevmo treatment group, 98 control group). At the investigators' discretion, patients in the control group received cabozantinib in 73 cases and vandetanib in 25 cases. The progression-free survival (PFS) comparison yielded positive data in the interim analysis. At a median follow-up of approximately 12 months, the Retevmo treatment group had not yet reached median PFS by blinded independent central review (BICR), while the control group had a median PFS of 16.8 months. The hazard ratio was 0.280. In preplanned subgroup analyses, BICR-assessed and investigator-assessed PFS were longer with Retevmo than with the control. Meanwhile, although Retevmo was approved in South Korea in March 2022, it failed to pass the National Health Insurance Service (NHIS) Cancer Disease Review Committee (CDRC) in May of the same year. Still, it cleared the CDRC in November and passed the Drug Reimbursement Evaluation Committee (DREC) in May 2023. After passing the reimbursement committee, negotiations with the NHIS on pricing began in June, raising hopes for listing, but ultimately, no agreement was reached. The case was the only failed price negotiation of that year. A Lilly representative said, "The company has been continuously preparing for the reapplication process and has submitted all required documents to the NHIS. The government is currently reviewing the application. We will do our best to ensure that more patients can benefit as soon as possible."
- Policy
- Reimb expansion for Benlysta in negotiations with NHIS
- by Lee, Tak-Sun May 12, 2025 05:59am
- Negotiations are underway to expand reimbursement for Benlysta (belimumab, GSK), a treatment for active systemic lupus erythematosus in adults. The drug was added to the reimbursement list in 2021 through the risk-sharing agreement (RSA), 8 years after its approval in Korea in 2013. According to industry sources on the 9th, the National Health Insurance Service added Benlysta to the list of drugs subject to price negotiations in May that was disclosed on its website. The proposal to expand Benlysta's scope of use was approved by the Drug Reimbursement Evaluation Committee of the Health Insurance Review and Assessment Service in March. Benlysta is currently reimbursed for the treatment of adults patients with active, antibody-positive systemic lupus erythematosus who are being treated for 3 or more months with the standard of care (corticosteroids, antimalarial drugs, immunosuppressants alone or in combination) who satisfy all the following conditions: ▲ has a disease activity SELENA-SLEDAI score ≥ 10, ▲ anti‐dsDNA–positive, and ▲hypocomplementemic (C3/C4 level). The drug is currently covered at a coinsurance rate of 10% through the special calculation exemption system for rare diseases. However, the drug’s indication is broader. In addition to adult patients, it is also indicated for the treatment of patients aged 5 years or older with active systemic lupus erythematosus and adult patients with active lupus nephritis undergoing standard therapy. The proposed reimbursement expansion application sought to expand the scope of its covered use in adult patients. Lupus is an autoimmune disease that causes an inflammatory response throughout the body. In particular, when it affects major organs such as the heart, lungs, kidneys, and brain, it can cause irreversible damage and lead to death. Most patients are women of childbearing age, and 19% of pregnant women may experience fetal death, intrauterine growth restriction, low birth weight, or premature birth. Benlysta 's 2023 IQVIA sales are estimated at KRW 1.9 billion. Currently, three risk-sharing agreements have been signed for this drug, including a refund-type agreement, an initial treatment refund-type agreement, and an expenditure cap agreement. If the negotiations proceed smoothly, the drug is expected to be listed for reimbursement in the second half of this year.
- Company
- Pemazyre lands in major hospitals in Korea
- by Eo, Yun-Ho May 12, 2025 05:59am
- Pemazyre, a treatment for intrahepatic bile duct cancer(cholangiocarcinoma), may now be prescribed at tertiary hospitals in Korea. According to industry sources, Handok's Pemazyre (pemigatinib) has passed the drug committee (DC) of the Big 5 general hospitals in Korea, including Samsung Medical Center, Seoul National University Hospital, Asan Medical Center, and Sinchon Severance Hospital. As the drug has been listed for reimbursement starting this May, the stage has now been set for its full-scale prescription in Korea. The drug is indicated for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement. Pemazyre is the first drug in Korea to be approved for this patient population. In March 2022, Handok signed an agreement with the global biopharmaceutical company Incyte Corp for the registration and supply of Pemazyre in Korea. Pemazyre was designated as an orphan drug by the Ministry of Food and Drug Safety in November 2021. A dire need existed in the field of cholangiocarcinoma due to the lack of a standardized second-line therapy for patients who fail first-line therapy. Surgery is the best treatment for bile duct cancer, but typically only 20-30% of patients are eligible for surgery at the time of diagnosis. Even after surgery, bile duct cancer has a high recurrence rate of 60-70% and a reported 5-year survival rate of less than 20%. Specifically, fusions or other rearrangements of the FGFR2 gene, for which Pemazyre is indicated, occur in approximately 10-16% of patients with intrahepatic cholangiocarcinoma. The approval of Pemazyre is based on the Phase II FIGHT-202 study. The open-label, single-arm Phase II FIGHT-202 study enrolled 107 adult patients (mean age 56 years) who had received at least one prior therapy for locally advanced or metastatic cholangiocarcinoma with a fusion or rearrangement of the FGFR2 gene. The results showed that the primary efficacy endpoint, objective response rate (ORR), was 35.5%, and the secondary endpoint, median duration of response (DOR), was 7.5 months in the Pemazyre arm. In addition, the reported median progression-free survival (PFS) was 6.9 months, and the median overall survival (OS) was 21.1 months. The most commonly reported treatment-related adverse event was hyperphosphatemia, which was of the lowest severity (Grade 1 or 2) and manageable.
- Company
- Generic market share 13% after 'Trajenta' patent expiration
- by Kim, Jin-Gu May 12, 2025 05:59am
- Generic products have expanded their market share to 13% just nine months after the patent expiration of Trajenta (linagliptin), a DPP-4 inhibitor for diabetes treatment. Compared to previous cases of diabetes treatments in the same drug class that patents expired before Trajenta, generic products are analyzed to have slower market penetration. Analysis suggests that the entry of generic products was blocked until early this year because they failed to receive priority marketing authorization, leading to passive product launches due to the original manufacturer’s active resistance. Original prescription sales KRW 29.3 billion→KRW 21.6 billion in one year… and generics totaled KRW 3.1 billion According to UBIST, a pharmaceutical market research firm, on the 9th, the outpatient prescription market size for linagliptin-based diabetes treatments in the first quarter was KRW 24.7 billion. It decreased by 16% year-over-year (YoY). Prescription sales of the original products, Trajenta and Trajenta Duo, dropped. Trajenta monotherapy sales dropped from KRW 14.7 billion to KRW 9.7 billion, a 34% decline, while the metformin combination therapy decreased from KRW 14.6 billion to KRW 11.8 billion, a 19% decline. The combined prescription amount shrank from KRW 29.3 billion to KRW 21.6 billion, a 26% decrease. Such an outcome is attributed to the expiration of the compound patent, the entry of generics, and the resulting price reductions. The substance patent for Trajenta expired in June of last year. Since then, generic products have been listed for reimbursement, leading to price cuts for the original products. Trajenta price fell by 30% and Trajenta Duo by 11%. Quarterly prescription sales of Trajenta original drug (dark green) & generics (light green). (unit: KRW 100 million, source: UBIST) The generics that entered the market immediately after patent expiration have gradually increased their market influence. The combined prescription value of Trajenta and Trajenta Duo generics rose from KRW 1.8 billion in Q3 of last year to KRW 2.7 billion in Q4, and KRW 3.1 billion in Q1 of this year. By company, as of Q1 of this year, KyungDong Pharmaceutical had the highest combined generic prescriptions for the single and combination products at KRW 600 million, followed by Daewon Pharmaceutical at KRW 500 million, Kyungbo Pharmaceutical at KRW 400 million, Yuhan and Hanmi Pharmaceutical each at KRW 300 million, and Boryung at KRW 200 million. Except for one company, all generic manufacturers recorded less than KRW 500 million prescriptions in Q1. The average prescription value per generic-launching company was just over KRW 100 million. Penetration rate↓ compared to previous cases of patent expiry… Did the original company's defensive strategy work? Analysis suggests that generic market penetration has been somewhat slow, even with other diabetes drugs of the same class whose patents expired earlier. For example, generics for 'Galvus (vildagliptin),' which lost patent protection before Trajenta, achieved a 39% market share three quarters after expiration. Generics for 'Tenelia (teneligliptin)' reached a 48% market share nine months after patent expiry. Generics for 'Januvia (sitagliptin),' whose patent expired in September 2023, had a 15% share three quarters after launch, two percentage points higher than Trajenta generics at the same period (13%). Trajenta's generics have shown the slowest market penetration among DPP-4 inhibitor diabetes drugs. The pharmaceutical industry suggests several analysis. First, the lock on priority marketing authorization for Trajenta generics was lifted earlier this year. After Trajenta's compound patent expired in May last year, 26 companies obtained priority marketing authorization and launched products. Their priority marketing periods ended in March of this year. Companies without those priority marketing authorization approvals were barred from launching generics for about ten months. Only after those periods expired in March were an additional 12 companies, including Hanmi Pharmaceutical·Genuone Sciences, able to enter the market. In addition, the original company, Boehringer Ingelheim Korea, actively defended its position by enforcing so-called 'unlisted patents' on Trajenta to defend generic launches. Generic manufacturers faced the choice of launching products without entirely circumventing or invalidating those unlisted patents, risking patent infringement findings and damages in future litigation. For these reasons, some companies reportedly held back on generic launches. Only 15 firms listed the Trajenta monotherapy generic for reimbursement, whereas 39 listed the combination therapies, because reformulating the combination can partially avoid unlisted patent risks. Indeed, of the 40 firms that listed a Trajenta Duo generic, 27 secured approval by changing the formulation to an extended-release tablet. Strong brand loyalty to the original product in clinical practice may have also contributed. Trajenta held the number 2 spot in the market for DPP-4 inhibitors for a long time after Januvia before its patent expiry, and despite generic entry, it maintains high loyalty in prescribing.
- Company
- RPE stem cells may address the cause of AMD
- by May 12, 2025 05:58am
- What if damaged human tissues and organs could be “restored”? If severed spinal cords could be reconnected, and eyes that have lost their sight could once again detect light on their own. The technology to revive human tissues that have lost their function has been a long-standing aspiration of humanity. Tissue regeneration using stem cells is considered the key to opening this new frontier in treatment. Stem cells are cells that possess both self-replication and differentiation capabilities. They can not only replicate themselves infinitely but also transform into various types of cells in the body as needed, making them a focal point in regenerative medicine. However, despite the high expectations for stem cells, there is prevalent skepticism both within and outside the academic community. This is due to the limited number of cases where its therapeutic efficacy has been clearly demonstrated, as well as unresolved challenges such as long-term safety and the potential for tumor formation. In Korea, stem cell technology has often been labeled as the “technology of doubt” since the 2005 scandal involving the fabrication of research papers by Woo Suk Hwang. Despite this, some companies continue to believe in the potential of this technology and are actively pursuing its development. One such company is Biotechnology. Luxa is a U.S. joint venture established in 2019 by Y2 Solution and Neural Stem Cell Institute (NSCI), the first independent stem cell research institute in the U.S. Luxa is currently developing “RPESC-RPE-4W,” a candidate drug for the treatment of dry age-related macular degeneration using retinal pigment epithelial stem cell (RPESC). Luxa's Chief Scientific Officer is Dr. Sally Temple, co-founder of Luxa and co-founder of NSCI. Dr. Temple is a world-renowned authority in the field of stem cells, having first identified neural stem cells in the adult central nervous system. Dr. Jeffrey Stern is Dr. Temple's spouse and a retina specialist ophthalmologist who co-founded NSCI with her, and is jointly leading the development of stem cell-based therapies. Dr. Keith Dionne is also a key member of Luxa's executive team. Dr. Dion is a seasoned executive with over 20 years of experience in the biotechnology industry, having served as CEO of four biotechnology companies, including Alantos Pharmaceuticals, Surface Logix, Constellation Pharmaceuticals, and Casma Therapeutics, where he led the growth and mergers and acquisitions (M&A) of these companies. Dr. Dion currently leads the company as co-CEO. Dailypharm met with Dr. Temple, Dr. Stern, and Dr. Dionne at the 2025 annual meeting of the Association for Research in Vision and Ophthalmology held in Salt Lake City. Utah, to hear about the competitiveness of RPESC-RPE-4W, the latest clinical results, and the company’s future development strategies. (from the left) Dr. Keith E. Dionne, Dr. Sally Temple, Dr. Jeffrey Stern - What kind of treatment is Luxa’s RPESC-RPE-4W? Dr. Temple: RPESC-RPE-4W is a cell therapy candidate for dry age-related macular degeneration (AMD) based on adult retinal pigment epithelial stem cells (RPESC). Dry AMD is caused by the degeneration of RPE cells located in the central part of the retina, ultimately leading to central vision loss. This candidate aims to improve the damaged retinal environment and help restore vision by culturing RPE cells externally and transplanting them into the patient's retinal layer. It is currently in Phase 1/2a clinical trials. - I understand that NSCI has begun preclinical research on RPESC-RPE-4W. What prompted the development of a treatment for dry AMD? Dr. Temple: The discovery of RPE cells in 2012 opened up new opportunities for treating retinal diseases. At the time, my husband, Dr. Stern, was treating many patients with macular degeneration, including my mother. It was then that I learned there was no effective treatment for dry AMD. After realizing that RPE cells could potentially address the underlying cause of AMD, we conducted animal experiments and observed significant recovery of vision loss. Dr. Temple: Central nervous system tissue was long considered incapable of regeneration, but this perception is changing as recent research suggests that some regeneration is possible. The discovery of how certain levels of recovery can occur after disease or injury has raised the possibility that stem cells or progenitor cells responsible for regeneration may exist within the CNS. -There is still skepticism within the academic community regarding stem cell therapy. What is your perspective on this? During my PhD, I studied progenitor cells in single-cell culture, which was a highly suitable approach for investigating stem cells in the brain or retina. During the research, Dr. Stern provided significant assistance in developing a time-lapse video system that enabled us to track and observe brain cells in culture. Using this system, we identified a rare but actual population of stem cells within the central nervous system. The next question was how these central nervous system stem cells are regulated. Through our research, we discovered that whether stem cells remain in a quiescent state or actively divide to regenerate brain tissue depends on how closely they are in contact with blood vessels. Based on this finding, NSCI was established in 2005. In recent years, we have observed patients with age-related macular degeneration who have shown improvements in vision following RPE stem cell transplantation therapy, which has brought us great joy in seeing that our research is benefiting. - What is the current status and outlook for the dry AMD treatment market? Dr. Dionne: Dry AMD affects approximately 200 million people worldwide, and this number is expected to increase with the aging population. It is one of the leading causes of blindness in adults, and there is currently no fundamental cure. The current best treatment is complement inhibitors, with FDA-approved drugs such as Syfovre and Izervey. However, these treatments require monthly or bimonthly injections into the back of the eye and only slow disease progression without improving vision. Luxa's RPESC-RPE-4W has demonstrated the potential to replace damaged RPE cells and improve vision. Additionally, recent reports indicate that global pharmaceutical giants like Merck in the U.S. are acquiring ophthalmic biotech companies targeting smaller markets than dry AMD for billions of dollars. This demonstrates that the size of the market is not the key factor, but rather how quickly we can respond to it. -Dry AMD treatments are being developed by several companies besides Luxa. Various therapeutic agents with different mechanisms, such as induced pluripotent stem cells (iPSCs), embryonic stem cells, and gene therapies, are under development. What makes RPESC-RPE-4W unique? Dr. Temple: The biggest difference between Luxa's clinical trial and others is the type of RPE cells being transplanted. We use “adult RPE stem cells” that are specialized to develop into RPE cells. In contrast, other therapies first create pluripotent stem cells, such as iPSCs or embryonic stem cells, which can differentiate into various cell types, and then artificially differentiate them into RPE cells for transplantation. This approach involves a complex differentiation process and carries the risk of unwanted cells being mixed in. Luxa eliminates the risk of generating abnormal cells by directly using adult RPE stem cells. Additionally, adult RPE stem cells are highly stable, do not induce tumors, and effectively differentiate into the desired RPE cells, making them more suitable for treatment. -Please tell us about the interim results of the RPESC-RPE-4W Phase 1/2a trial presented at ARVO 2025. Dr. Stern: Luxa is conducting the RPESC-RPE-4W Phase 1/2a clinical trial, and at this conference, we presented data from the low-dose Cohort 1, which involved administering 50,000 cells to six patients. The interim results showed no serious adverse events (SAEs), tumorigenicity, inflammatory responses, or retinal detachment associated with the investigational drug. As a result, the company is now able to proceed to the next phase, which involves increasing the dose to 150,000 and 250,000 cells. ETDRS chart (Source: Luxa) Early signs of vision recovery were also observed in the trial. Luxa measured changes in vision after RPESC-RPE-4W administration using the ETDRS (Early Treatment Diabetic Retinopathy Study) chart. Each line contains five letters. Being able to read 15 more letters means being able to read 3 more lines. Interim results of the Phase 1/2a clinical trial showed that patients with the poorest vision were able to read approximately 21 more letters or 3 more lines on the ETDRS chart. Before treatment, patients could only read the largest letters, but after treatment, they were able to read the fourth line on the ETDRS chart due to an improvement of three lines in visual acuity. Visual acuity improved within approximately one month after administration, and the effect was maintained stably throughout the one-year clinical period. -What is your outlook for the RPESC-RPE-4W clinical trial, and how do you plan to develop it in the future? Dr. Stern: I believe that the current results are most likely to continue. While there is a possibility that the eight patients treated so far are exceptional cases, the probability is very low. To confirm this, we need to analyze all 18 patients who participated in the clinical trial. Since initial safety and tolerability have already been established, we have added two new clinical sites to accelerate the clinical trial. One is the Byers Eye Institute at Stanford University in the United States, and the other is LA Retina, a large private hospital located in Los Angeles, California, United States. The ongoing Phase 1/2a clinical trial is scheduled to be completed by the fourth quarter of this year. Following this, the company plans to seek business partners to conduct large-scale clinical trials and obtain FDA approval for RPESC-RPE-4W.
- Company
- Reimb progress for Balversa gains attention in Korea
- by Eo, Yun-Ho May 09, 2025 06:01am
- Balversa, a new drug for bladder cancer, has passed the first hurdle toward being listed for insurance reimbursement in Korea. According to industry sources, Janssen Korea’s FGFR targeting urothelial carcinoma (bladder cancer) drug Balversa (erdafitinib) recently passed the Cander Disease Deliberation Committee of the Health Insurance Reimbursement and Assessment Service recently. The covered indication is for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma with FGFR3 gene mutations who have progressed during or after prior systemic therapy, including at least one PD-1 or PD-L1 inhibitor. The drug’s original indication that it had been approved for in 20222 was 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.’ However, the approval of PD-1 and PD-L1-directed immuno-oncology agents in the first- and second-line settings that followed Balversa’s approval led to the need for Balversa to demonstrate efficacy in patients who previously received these agents. The situation was addressed with the publication of Balversa’s Phase III THOR trial study, which demonstrated a prolonged overall survival (OS) benefit with Balversa over chemotherapy in patients with metastatic urothelial carcinoma with FGFR3/2 gene alterations whose disease progressed after first-line treatment with immuno-oncology agents. In the study, Balversa prolonged overall survival (OS) compared with chemotherapy in patients with metastatic urothelial carcinoma. Results showed that over a median follow-up of 15.9 months, the mOS was 12.1 months in the Balversa arm, reducing the risk of death by 36% compared with the 7.8 months in the chemotherapy arm. Based on these findings, the U.S. Food and Drug Administration granted Balversa formal approval in January, but with a more restricted indication than originally approved. The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) also recently recommended expanding Balversa’s indication. Janssen Korea has additionally submitted the results from the THOR study to Korea’s Ministry of Food and Drug Safety. And the added data led to the approval by the Cancer Disease Deliberation Committee. It remains to be seen whether Balversa will be listed for reimbursement within this year and establish itself as a practical treatment option in Korea. Bladder cancer is a representative cancer for which there are no targeted anticancer drugs. Balversa is the first targeted anti-cancer drug for bladder cancer with a novel mechanism of action that inhibits fibroblast growth factor receptor (FGFR). FGFR is a biomarker involved in cancer cell growth that is associated with various cancers. FGFR mutations are particularly common in bladder cancer, with 20 to 30% of patients carrying mutations.
- Policy
- Fitusiran for hemophilia receives orphan drug designation
- by Lee, Hye-Kyung May 09, 2025 06:01am
- Sanofi A long-acting treatment for all types of hemophilia, administered every 2 months, has been designated as an orphan drug in South Korea. According to the “Regulations on the Designation of Orphan Drugs” released by the Ministry of Food and Drug Safety on the 7th, Sanofi's Fitusiran was designated as the 386th orphan drug in Korea. Fitusiran was released under the brand name Qfitlia in the U.S.. In March, the U.S. FDA approved Fitusiran as the first antithrombin inhibitor-based therapy with the convenience of a twice-monthly subcutaneous injection, for the routine prophylaxis to prevent bleeding in patients aged 12 years and older with hemophilia A or B. Fitusiran is an siRNA (small interfering RNA) therapy designed to target antithrombin, a protein involved in blood clotting, for the treatment of patients with hemophilia A and B. It works by lowering antithrombin levels to promote thrombin production, thereby improving hemostasis and preventing bleeding in hemophilia patients. Currently, the hemophilia treatment market is dominated by Hemlibra for hemophilia A, while concizumab by Sanofi and Novo Nordisk is under development as a treatment for both hemophilia A and B. Regarding its designation as an orphan drug, according to the minutes of the Central Pharmaceutical Affairs Council meeting released by the MFDS on the 7th, there was an opinion that Fitusiran is a drug that can meet unmet needs and requires post-marketing surveillance. The committee members believe that Fitusiran is expected to provide patients with a better treatment option by addressing the limitations of current treatments in key areas such as ABR, joint bleeding, patient compliance, and the impact on quality of life. Additionally, while existing medications are currently available, if Fitusiran demonstrates superiority over existing products in terms of ensuring a smooth supply of medications for patients, reducing treatment costs, decreasing administration frequency, alleviating pain, and reducing bleeding, it was deemed to meet the criteria for having an unmet need. In particular, existing treatments have low convenience due to the burden of intravenous injections and frequent administration (2–3 times per week). However, Fitusiran, which is an intravenous injection usable for both hemophilia A and hemophilia B, has the advantage of subcutaneous injection every 4–8 weeks, offering improved convenience. However, while it appears to have improved safety and efficacy compared to alternatives, given its new pharmacological mechanism, careful review is necessary regarding data submission exemptions.. Although the structure of N-acetylgalactosamine and its pharmacologic mechanism when used as a target ligand in oligonucleotide and siRNA therapy are already well known, a detailed review of domestic clinical results would be necessary due to safety concerns identified in previous clinical trials. Therefore, regarding safety, the CPAC decided that the risk level could be judged as acceptable for approval by referencing overseas approval criteria, but appropriate patient monitoring would be necessary. The CPAC members stated that “Hemophilia is a disease with a clear pathophysiology and no significant differences in response between races. The submitted Phase III clinical trial results showed that Asian patients accounted for a relatively high proportion of participants, and submitted the opinion that domestic approval is reasonable. Meanwhile, it is reported that the annual cost of Fitusiran in the United States amounts to approximately USD 642,000 (approximately KRW 944 million).
- Company
- U.S. issues orders to produce pharmaceuticals domestically
- by Kim, Jin-Gu May 09, 2025 06:00am
- An executive order issued by U.S. President Donald Trump to promote domestic production of medicines could impose additional burdens on Korean pharmaceutical and biotech companies seeking entry into the U.S. market. Included among the order’s provisions is a requirement to strengthen inspections of manufacturing facilities outside the United States, a move analysts say will lead to more rigorous FDA audits and potentially higher related fees. On the 8th, the Korea Pharmaceutical and Bio-Pharma Manufacturers Association (KPBMA) released an analysis of the impact on Korea’s pharmaceutical and biotech industry of the U.S. administration’s 'Regulatory Relief to Promote Domestic Production of Critical Medicines' executive order. According to the KPBMA, President Trump on the 5th (U.S. time) announced the executive order titled 'Regulatory relief to promote domestic production of critical medicines.' Its purpose is to strengthen the domestic manufacturing base for medicines in the U.S. and reduce reliance on foreign supply. During his first term, the Trump administration had already pushed policies to boost U.S. production of essential medicines and key raw materials. However, the second Trump administration judged that these policies had not been fully implemented under President Biden. Accordingly, the new order aims to relax manufacturing-related regulations and thereby promote expanded U.S. drug production capacity. The order streamlines the domestic review process for pharmaceutical manufacturing and strengthens inspections of foreign manufacturing sites. Under this measure, within 90 days of the order’s enactment, the U.S. FDA must develop improved, risk-based inspection protocols for overseas facilities supplying medicines to the U.S. The order stipulates that the costs of these inspections be covered by increased fees on foreign manufacturers. The KPBMA predicts, “Enforcement of mandatory production–data reporting from overseas facilities and public disclosure of noncompliant manufacturers will intensify the burden on Korean firms eyeing the U.S. market.” And added, “With strengthened FDA audits of foreign sites, possible fee increases, and public reporting of audit outcomes by country and company, Korean manufacturers must bolster quality-management systems and regulatory-response capabilities.” Moreover, “As the U.S. government increasingly focuses on expanding supplies from domestic producers, securing local production and supply chains and meeting U.S. quality-certification standards will be prerequisites,” the association advised, “and companies must devise closely coordinated strategies to respond to changes in U.S. certification, licensing, and procurement processes.” In the medium to long term, converting Korean facilities into U.S.-based manufacturing sites should also be considered—foreign facilities will face tighter regulations while domestic facilities see relief. The FDA will introduce pre-approval inspections for U.S. sites and conduct only essential, efficient reviews. The FDA also provide clearer guidance and procedures for overseas facilities that shift production to the United States.
- Company
- Pharma closely watches tariffs as U.S. sales surge
- by Chon, Seung-Hyun May 09, 2025 06:00am
- South Korea’s pharmaceutical and biotech companies are focusing on potential tariffs on U.S. exports of their medicines. President Donald Trump’s announcement that tariffs would be imposed on imported pharmaceuticals has left major exporters considering devising countermeasures. Celltrion, whose North American sales have topped KRW 1 trillion, has pre-distributed over a year’s inventory. GC Biopharma, which recently entered the U.S. plasma product market, hopes to secure tariff exemptions on U.S.-sourced raw materials. Companies such as Samsung Biologics, SK Biopharmaceuticals, and Daewoong Pharmaceutical have been accelerating their penetration of the U.S. market, making it imperative to monitor the ramifications of any tariffs closely. Celltrion completes transfer of 15 months’ inventory to prepare for tariffs… last year’s North American sales surpassed KRW 1T On the 8th, industry sources reported that Celltrion posted a statement on its website on the 7th titled 'Our Position and Response Strategy Regarding the Trump Administration's Tariff Policy in the U.S.,' announcing that it 'has completed the transfer of approximately 15 months' worth of inventory for products scheduled for sale in the United States in 2025.' Celltrion explained that it has minimized the impact of tariffs on this year's U.S. sales and projected sales in the first half of next year. Celltrion stated, "We have finalized a manufacturing agreement for finished products with a local U.S. CMO, and have already secured the volume that can be produced domestically. We have also completed measures to negotiate additional contracts with manufacturing sites to cover any further volumes that may be affected by tariffs." President Trump's declaration on the 5th that "within the next two weeks, we will announce item-by-item tariffs on pharmaceuticals" prompted Celltrion to unveil these detailed countermeasures. President Trump also signed an executive order directing regulatory agencies to shorten approval times for pharmaceutical plants built in the United States. Since President Trump took office, Celltrion has introduced three separate countermeasures to address potential tariffs on U.S. exports, demonstrating an active approach to contingency planning. On January 30, Celltrion announced measures stating, "We have secured sufficient inventory for our products currently sold in the U.S. to be sourced locally without additional imports through at least the third quarter of 2025." In February, the company announced the completion of a nine-month inventory transfer. It stated, "We have finalized measures to minimize the impact in 2025," once again presenting the status of its tariff mitigation plans alongside further inventory transfers. Celltrion said, "We have already developed strategies to minimize tariff impacts beyond next year," adding, "Regarding the establishment of local API manufacturing facilities in the U.S. as part of our long-term response, we have completed preliminary reviews and are now conducting a comprehensive, detailed assessment." Celltrion sales by regions (unit: KRW 1 billion, source: Celltrion) Celltrion is one of the largest South Korean pharmaceutical and biotech exporters to the United States. Celltrion has achieved U.S. approval for a total of 11 pharmaceutical products. Celltrion's first U.S. approval came in 2016 with Inflectra, the biosimilar to Remicade. In 2018, its biosimilars to the oncology drugs MabThera and Herceptin received FDA approval. In September 2022, Celltrion obtained FDA marketing authorization for Vegzelma, its Avastin biosimilar, and in 2023, its Humira biosimilar Yuflyma received FDA approval. In August 2023, the FDA approved Celltrion's subcutaneous (SC) formulation of Remsima, marketed as Zymfentra, as a standalone product. Last December, its Stelara biosimilar Steqeyma was approved by the FDA. This year, Celltrion has secured U.S. approval for four biosimilars. In January, the FDA approved Aptozma, its Actemra biosimilar for treating autoimmune diseases. In March, it received FDA approvals for Stobocloo, its biosimilar to bone disease treatments Prolia and Xgeva, and Omlyclo, its Zolair biosimilar, also cleared the U.S. regulatory hurdle. Last year, Celltrion's biopharmaceutical sales in the North American market reached KRW 1.0453 trillion. While its North American sales fell from KRW 709.5 billion in 2022 to KRW 629.2 billion in 2023, they surged by 66.1% last year, surpassing KRW 1 trillion for the first time. GC Biopharma begins U.S. sales of plasma product Alyglo… forecasts that "tariffs on U.S.-sourced materials will have limited impact" GC Biopharma, which recently entered the U.S. plasma product market, has anticipated limited tariff impacts. In December 2023, GC Biopharma entered the U.S. market when the FDA approved its plasma product, Alyglo. Alyglo is a liquid immunoglobulin preparation purified from plasma fractions. It is used to treat primary immunodeficiency disorders such as congenital immunodeficiency and immune thrombocytopenia. In its Q1 earnings release, GC Biopharma stated that, because a tariff exemption for U.S.-sourced raw materials is being considered, it expects the impact of tariffs to be limited. GC Biopharma forecasted that tariffs on U.S. pharmaceuticals will not significantly affect the plasma product Alyglo (source: GC Biopharma) The raw material of blood plasma for Alyglo is produced in the United States. GC Biopharma acquired 100% of ABO Holdings for KRW 138 billion in December last year. ABO Holdings is based in California and operates six blood banks across New Jersey, Utah, and California. GC Biopharma produces Alyglo at its Ochang facility using blood supplied by ABO Holdings, and then sells it in the U.S. GC Biopharma said, "We have secured inventory of Alyglo in the U.S. and are reviewing CMO partnerships for finished product manufacturing," outlining its tariff strategy. In Q1, plasma product sales were KRW 127.2 billion, up 42.3% from KRW 89.4 billion during the same period last year. GC Biopharma began full-scale sales after shipping the initial Alyglo batch in July last year. In Q2 2023, plasma product sales were KRW 90.6 billion, rising 50.8% to KRW 136.6 billion in Q3 and increasing to KRW 161.7 billion in Q4. While plasma product sales fell by 21.3% in Q1 2024 compared to Q4 2023, they remained above KRW 100 billion for three consecutive quarters. Green Cross sells Alyglo in the U.S. through its subsidiary, GC Biopharma USA, which recorded its first sales of KRW 48.6 billion last year. U.S. Sales Growth for Samsung Biologics, SK Biopharm, Daewoong surges… Monitoring Tariff Impacts Closely Korean pharmaceutical and biotech firms must closely watch for tariff impacts as their U.S. sales have recently increased. According to the Korea Customs Service, pharmaceutical exports to the U.S. reached USD 1.35809 billion (approx. KRW 1.61 trillion) last year, up 50% year on year. The U.S. accounted for 18% of total pharmaceutical exports. Exports to the U.S. have risen rapidly over the past two years: from KRW 843.94 billion in 2022 to USD 903.30 million (approx. KRW 1.07 trillion) in 2023, a 7% rise, and last year’s surge accelerated further. In 2022, the U.S. overtook Germany as South Korea’s largest pharmaceutical export market, maintaining that position for three consecutive years. Recently, orders for contract development and manufacturing (CDMO) have expanded, and sales of new drugs and biosimilars in the U.S. have increased, driving a significant rise in export volumes. Of Samsung Biologics’ KRW 45.473 trillion in sales last year, KRW 11.741 trillion (25.8%) came from the U.S. Samsung Biologics’ U.S. revenue share was 28.5% in 2022 and 26.3% in 2023. Samsung Biologics calculates regional sales based on the location of its CDMO clients. Samsung Bioepis, Samsung Biologics’ biosimilar subsidiary, is also expected to be affected by U.S. tariffs. Of its KRW 1.5277 trillion in sales last year, KRW 917.5 billion (60.1%) came from Europe and KRW 610.2 billion (39.9%) from non-European regions. Within those non-European sales, the U.S. represents the largest share, and they grew by 46.6% year on year. Since 2017, Samsung Bioepis has obtained U.S. approval for ten biosimilars. Starting with its Remicade biosimilar in 2017, followed by Herceptin, Enbrel, and Humira biosimilars in 2019. Ontruzant in September 2019, Eticovo, and Hadlima in April and July 2019, respectively; the Lucentis biosimilar Byooviz in September 2021. Since last year, Samsung Bioepis obtained FDA approval for its biosimilars in five areas, including biosimilars of Eylea, Stelara, Soliris, Prolia, and Xgeva. Samsung Bioepis’ biosimilar supply chain involves API production at Fujifilm Bio’s Danish plant and Samsung Biologics, with finished products made by overseas CMO partners and sold worldwide. Its U.S. biosimilar sales are handled by Biogen, Organon, Teva, and other partners. SK Biopharm’s new epilepsy drug Xcopri is also accelerating its U.S. penetration. FDA-approved in November 2019, Xcopri has been sold directly in the U.S. via SK Life Sciences since May 2020. SK Biotech produces the Xcopri API, and a Canadian CMO partner supplies the finished drug. Last year, Xcopri’s U.S. sales reached KRW 438.7 billion, up 62.1% year on year, with cumulative U.S. sales of KRW 969.5 billion. SK Lifesciences sales by year (unit: KRW 100 million, source: FSS) Last year, SK Life Sciences’ sales were KRW 667.8 billion, up 36.0% from 2023. Over two years, its sales grew 72.0%, from KRW 388.3 billion in 2022, more than fourteen-fold over six years since KRW 47.5 billion in 2018. SK Group has established U.S. manufacturing through SK pharmteco, which oversees CMO operations. SK pharmteco runs five companies, including SK Biotech, SK Biotech Ireland, Ampac, Yposkesi, and CBM. Ampac has facilities in California, Texas, and Virginia. Among traditional pharmaceutical companies, Daewoong Pharmaceutical is the most active U.S. exporter, selling its botulinum toxin product Nabota (FDA-approved via partner Evolus in 2019) from its Hyangnam plant. Nabota’s exports were KRW 49.2 billion in 2021, more than doubling to KRW 109.9 billion in 2022, then rising to KRW 117.4 billion and KRW 156.0 billion in 2022 and 2023, respectively, over triple in three years. Q1 2024 exports were KRW 37.3 billion, four times the domestic sales of KRW 8.3 billion. U.S. subsidiaries of Korean pharma firms also saw strong growth. Hugel America, a subsidiary of Hugel, is responsible for the U.S. sales and R&D of the botulinum toxin product “Retivo.” Its revenue increased by 86% year-over-year, from KRW 21.1 billion in 2023 to KRW 39.2 billion last year, and its net loss of KRW 19.2 billion turned into a net profit of KRW 15.5 billion. Huons USA, operated by Huons, markets products such as lidocaine injections. Its revenue rose 29%, from KRW 1.1 billion to KRW 1.4 billion, and its net loss of KRW 1.7 billion in 2023 turned into a net profit of KRW 0.1 billion last year.
- Policy
- Ofev reimbursed from May... inflow of generic versions
- by Lee, Hye-Kyung May 09, 2025 05:59am
- The generic drug market is growing for Boehringer Ingelheim's idiopathic pulmonary fibrosis treatment ‘Ofev Soft Capsules (nintedanib),’ which was granted reimbursement and was listed from this month, 8 years after approval. On the 7th, the Ministry of Food and Drug Safety approved 2 items, 100 mg and 150 mg of Whan In Pharm’s ‘Ofenip Tab (Nintedanib Esylate).’ Ofenip is the fourth generic version approved for Ofev, which was approved in 2016. Generic drug approvals began with Yungjin Pharmaceutical's ‘Nintebro Tab’ in December last year, followed by Daewoong Pharmaceutical's ‘Ofevia Tab and Ildong Pharmaceutical's ‘Cuninta Tab’. The Ofev generics have been granted marketing authorizations upon the original drug's patent expiration on January 25. Ofev, which contains nintedanib, has three indications: ▲treatment of idiopathic pulmonary fibrosis, ▲delaying the decline in lung function in patients with systemic sclerosis-associated interstitial lung disease, and ▲treatment of chronic fibrosing interstitial lung disease with progressive phenotype. However, the generic versions of Ofev do not have the indication for the treatment of idiopathic pulmonary fibrosis and have only been approved for the remaining two indications. The reason for this can be found in the reimbursement process for Ofev. According to the results of the reimbursement adequacy review conducted by the Drug Reimbursement Evaluation Committee of the Health Insurance Review and Assessment Service in January, Ofev was only granted reimbursement for two indications: systemic sclerosis-associated interstitial lung disease and chronic fibrosing interstitial lung disease with progressive phenotype. Although Ofev was initially approved for idiopathic pulmonary fibrosis, the pharmaceutical company did not submit separate cost-effectiveness data for the indication, so no discussion had been made regarding its reimbursement. In particular, in South Korea, Ildong Pharmaceutical’s ‘Pirespa Tab (Pirfenidone) had dominated the reimbursement market for idiopathic pulmonary fibrosis since 2015, so Boehringer Ingelheim, which owns Ofev, had no choice but to pursue a differentiation strategy. This is why latecomers following Ofev that are attempting reimbursement have also entered the market with indications that are being reimbursed for the original drug. Currently, the number of patients eligible for Ofev’s use with reimbursement in Korea is estimated to be about 329. With reimbursement, patients will see a significant reduction in their annual medication costs from KRW 19.14 million to KRW 5.74 million. In addition to the pharmaceutical companies that have already received approval, domestic companies such as Chong Kun Dang, Sama Pharm, and Samoh Pharm are also developing generic versions of Ofev, so more generics are expected to be released in the future.
