BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) has announced its decision not to renew the Import Drug License (IDL) for Vimizim (elosulfase alfa) in China when it expires in May 2024. The company is currently exploring feasible ways to ensure a continuous supply to patients currently undergoing treatment.
Challenges in China’s Market Access for Rare Disease Drugs
According to the US firm, China’s complex market access rules have rendered the ongoing supply of the rare disease drug Vimizim unsustainable. Despite efforts over the past few years, Vimizim has not been able to enter the medical insurance reimbursement system, leading to the decision not to renew the import drug registration certificate for the product.
Vimizim’s History and Pricing in China
Vimizim was included on the government’s first list of rare disease drugs in 2018, which outlined drugs clinically urgently needed in China and already approved overseas that would receive fast-track treatment. BioMarin secured the first China approval for Vimizim in May 2019 to treat mucopolysaccharidosis type IVA (MPS IVA), priced at RMB 7,500 (USD 1,047) per shot. Given that the drug is administered via a once per week IV infusion dosed at 2mg per kilogram of patient weight for children over 5, the annual treatment costs for Vimizim can reach up to RMB 2 million (USD 280,000) per 12.5 kg of patient bodyweight.
National Reimbursement Drug List (NRDL) and Partial Coverage
BioMarin applied for Vimizim to be included in the National Reimbursement Drug List (NRDL) in 2021, but price negotiations were unsuccessful. The company declined to participate in the NRDL negotiation process the following year. However, Vimizim has gained partial coverage through city-level supplementary insurance schemes (HuiMinBao) in 13 provinces as of June 1, 2023. In Chengdu, Vimizim users on BioMarin’s patient access program could benefit from around a 40% rate of reimbursement worth around RMB 460,000 per year. A similar scheme was available in Jiangsu. Additionally, BioMarin has offered a program allowing patients paying out-of-pocket to enjoy free drugs after a certain number of purchases. Despite these efforts, only a dozen patients are using the drug in China. Vimizim generated USD 664 million in global sales last year according to BioMarin’s 2022 annual report, up 7% year-on-year, with minimal contribution from the China market.-Fineline Info & Tech
