Bio­Marin heads to FDA with he­mo­phil­ia A gene ther­a­py; Covid-fo­cused biotech signs a small SPAC deal

Bio­Marin’s gene ther­a­py is get­ting a shot at the FDA as the agency ac­cept­ed the BLA, the biotech said Wednes­day night.

With a de­ci­sion date of March 31, 2023, an ap­proval would come two and a half years af­ter the reg­u­la­tor orig­i­nal­ly re­ject­ed the AAV gene ther­a­py, which the biotech net­ted con­di­tion­al ap­proval for in the EU in late Au­gust.

Bio­Marin is at­tempt­ing to be­come the first gene ther­a­py mak­er in the US to se­cure an ap­proval for se­vere he­mo­phil­ia A, which is char­ac­ter­ized by spon­ta­neous episodes of bleed­ing.

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