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Ultragenyx将申请加速批准罕见代谢疾病候选药物
2024-06-13 04:40
- Ultragenyx (NASDAQ:RARE) said it would submit a Biologics License Application to the U.S. FDA seeking accelerated approval of UX111, a gene therapy for Sanfilippo syndrome, a rare metabolic disease.
- The company noted that following a recent meeting with the agency, it was agreed that cerebral spinal fluid heparan sulfate could be used as a surrogate endpoint.
- A pre-BLA meeting with the agency is still needed, but Ultragenyx is eying a filing late this year or in early 2025.
- UX111 would be given as a one-time treatment. Sanfilippo syndrome is a fatal lysosomal storage disease that mostly impacts the central nervous system. There are no approved therapies.
- Separately, after Wednesday's closing bell, the company said it has begun a public offering of up to $350M of common stock and pre-funded warrants, sending shares down ~3% in after-hours trading.
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- Ultragenyx Pharmaceutical, Inc. (RARE) Q1 2024 Earnings Call Transcript
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