Positive top-line data from the pivotal phase III study of AAV5-based etranacogene dezaparvovec by Uniqure NV, shows 54 patients met the primary endpoint in treating severe to moderate hemophilia B.
The FDA has issued Sanofi SA a complete response letter (CRL) regarding its complement pathway inhibitor sutimlimab, citing deficiencies found during a pre-license inspection of a third-party manufacturing facility.
Shares of Bluebird Bio Inc. (NASDAQ:BLUE) sank 16.6%, or $9.72, to close at $48.83 as Wall Street reacted to news that the U.S. regulatory filing for Lentiglobin in sickle cell disease (SCD) will be delayed. Previously expected in the second half of next year, the filing won’t happen until late 2022.
An earlier-than-intended release of abstracts for the American Society of Hematology annual meeting spilled multiple market-moving updates Nov. 4, though with no apparent shocks so far. Shares of Allogene Therapeutics Inc. sunk 8% on Nov. 4 over some metered disappointment around initial data for ALLO-715, a potential medicine for relapsed/refractory (r/r) multiple myeloma. By contrast, shares of Global Blood Therapeutics Inc. climbed nearly 15%, buoyed by new data supporting the long-term use of its sickle cell disease therapy, Oxbryta (voxelotor).
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