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.

Money Wave
A Long march towards reimbursement for orphan drugs in China • Source: Shutterstock

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.

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AstraZeneca Expands Emphasis On China, Already A Key Market

 
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RemeGen Fast-Tracks Potential World-First BLys/APRIL Agent For gMG In Global Phase III Trial

 

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.