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- Company
- Reimb progress for Balversa gains attention in Korea
- by Eo, Yun-Ho May 9, 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 9, 2025 06:01am
- 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&8211;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&8211;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 9, 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&8211;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&8212;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.
- Policy
- Ofev reimbursed from May... inflow of generic versions
- by Lee, Hye-Kyung May 9, 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.
- Company
- Lilly retries to make RET-targeted therapy accessible
- by Eo, Yun-Ho May 8, 2025 06:09am
- Patients are hopeful again for RET-targeted cancer therapy, a treatment option once deemed 'pie in the sky.' According to industry sources, Lilly Korea has recently submitted an application for insurance reimbursement listing for its RET (REarranged during Transfection) inhibitor 'Retevmo (selpercatinib),' indicated for the treatment of non-small cell lung cancer (NSCLC). Retevmo, which was approved in South Korea in March 2022, had not passed the Cancer Disease Review Committee (CDRC) of the Health Insurance Review and Assessment Service (HIRA) in May of the same year. The drug passed the CDRC in November and the Drug Reimbursement Evaluation Committee (DREC) in May 2023. Upon clearing the DREC review, Retevmo was anticipated to be reimbursed as the company entered a pricing negotiation with the National Health Insurance Service (NHIS) in June. However, an agreement had not been met. It was the sole unsuccessful drug pricing negotiation of that year. Consequently, it was expected that RET cancer therapy would not be readily accessible in South Korea. Two RET cancer therapies are domestically approved. In addition to Retevmo, 'Gavreto (pralsetinib),' introduced by Roche Korea from Blueprint Medicines Corp, exists. However, Gavreto's inclusion in the reimbursement list became difficult due to Roche withdrawing the sales rights. Lilly's Korean subsidiary has recently made progress. However, it remains uncertain whether the company will finalize the reimbursement listing process. Retevmo was conditionally approved for agreement to meet the Phase 3 trial requirement. However, the company's application for listing process lacked a Phase 3 study and was asked to submit a document equivalent to the requirement. For this reason, criticism arose regarding the evaluation criteria for the drug that had been conditionally approved to expedite its introduction. This was because the company had quickly applied for listing under the approval&8211;evaluation linkage system, underwent discussions on reimbursement for about a year and a half but ultimately failed. There is still room for a turnaround. Proper Phase 3 trial results are now in hand. What now matters is Lilly's and the government's commitment to introducing a RET-targeted cancer therapy. At the 2023 European Society for Medical Oncology (ESMO 2023) annual meeting, the results of Retevmo's Phase 3 trials, LIBRETTO-431 and LIBRETTO-531, were each disclosed. Those results were published in the New England Journal of Medicine (NEJM). The LIBRETTO-431 study presented at the conference compared Retevmo with platinum-based chemotherapy±pembrolizumab in the first-line treatment of patients with progressive or metastatic RET fusion&8211;positive NSCLC. The key result was that, in the ITT-pembrolizumab cohort, the median progression-free survival (PFS) assessed by the blinded independent central review (BICR) was 24.8 months in the Retevmo-treated group versus 11.2 months in the control group, with a hazard ratio of 0.465. The overall response rate (ORR) by BICR was also significantly higher in the Retevmo group at 83.7% compared to 65.1% in the control group. Meanwhile, Retevmo was granted accelerated approval, priority review, and Breakthrough Therapy & Orphan Drug Designation in the United States in 2020. Then, it was approved as the first treatment option for the treatment of cancer patients with RET gene mutations.
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