REGENXBIO Announces FDA Review Extension of BLA for RGX-121 to Treat Patients with MPS II
RGX-121 would be the first and only potential one-time commercially-available therapy designed to directly address the underlying genetic cause of Hunter syndrome, if approved Commercial launch plans remain on track REGENXBIO plans to present updated pivotal data during the ICIEM meeting...
Related Questions
How will the FDA review extension and upcoming data presentation affect RGNX's short‑term price volatility?
What is the potential market size and pricing power for a one‑time gene therapy for Hunter syndrome compared to existing treatments and pipeline competitors?
What are the risks to the commercial launch timeline and reimbursement landscape if the BLA is not approved on the first submission?