Priced at $125,000 per injection and $750,000 for the first year of treatment, Biogen Inc.’s Spinraza (nusinersen) is the the first US FDA-approved treatment for rare spinal muscular atrophy (SMA), and now the company is looking to bring the high-priced drug to China.
A Long March After Approval: What You Need To Launch Orphan Drugs In China
Without automatic reimbursement in place, makers of orphan and rare disease drugs must look at innovative alternative ways to make their high-priced new therapies accessible to patients in China.

More from China
AstraZeneca remains committed to investing in R&D and alliances in China, where Susan Galbraith, the UK major’s head of oncology R&D, sees innovation eventually reaching parity with the US and Europe.
After a more than three-year hiatus, China's Hengrui has signalled its return to multiregional Phase III trials as it looks to globalize its innovative pipeline. Meanwhile, a number of other Chinese players have announced plans to kick off Phase III trials this year and beyond.
RemeGen is planning to complete enrolment in the global Phase III RemeMG study with telitacicept in generalized myasthenia gravis by the end of 2025 or early 2026. The Chinese firm has already sidelined two other global Phase III trials with the molecule to prioritize the indication.
UK pharma will invest $2.5bn in Beijing R&D hub, build a vaccine manufacturing site with BioKangtai, and partner in chronic disease with Syneron and in oncology/immunology with Harbour BioMed.
More from Focus On Asia
Senior executives from AstraZeneca, BMS, Novo Nordisk, Takeda and Regeneron outline how big pharma's global capability centers (GCCs) in India are evolving beyond cost efficiency, focusing on innovation, “agile experimentation” and new technology including GenAI, virtual & augmented reality, with some positioned as COEs. Will Indian multinationals use the GCC approach?
Plus deals involving GV20/Mitsubishi Tanabe, Kaken/Alumis, AstraZeneca/Alteogen and deal terminations involving Clover/Gavi Alliance and Rhythm/RareStone.
RemeGen is planning to complete enrolment in the global Phase III RemeMG study with telitacicept in generalized myasthenia gravis by the end of 2025 or early 2026. The Chinese firm has already sidelined two other global Phase III trials with the molecule to prioritize the indication.