Ultragenyx Completes Rolling Submission of Biologics License Application (BLA) to U.S. FDA for DTX401 AAV Gene Therapy for Glycogen Storage Disease Type Ia (GSDI…

If approved, DTX401 will be the first therapy approved to treat the underlying cause of GSDIaNOVATO, Calif., Dec. 30, 2025 ...
Ophthalmology Times

FDA update: Outlook Therapeutics receives CRL for resubmitted ONS-5010 BLA

The FDA issued a CRL for ONS-5010, citing the need for additional confirmatory efficacy evidence despite previous trials ...
Seeking Alpha

Denali Therapeutics Announces FDA Review Extension of BLA for Tividenofusp Alfa for the Treatment of MPS II (Hunter Syndrome)

SOUTH SAN FRANCISCO, Calif., Oct. 13, 2025 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (DNLI) today announced that the U.S. Food and Drug Administration (FDA) has extended its review timeline of the ...
The Pharma Letter

Ultragenyx completes rolling submission of BLA for DTX401

US biotech Ultragenyx Pharmaceutical In this week revealed that it has completed the rolling submission of its Biologics ...
TipRanks on MSN

Inovio announces US FDA accepted BLA for INO-3107

INOVIO (INO) announced that the U.S. Food and Drug Administration, FDA, accepted the company’s Biologics License Application, BLA, for INO-3107 ...
Morningstar

Orca Bio Announces FDA Acceptance and Priority Review of the Biologics License Application (BLA) for Orca-T® to Treat Hematological Malignancies

If approved, Orca-T would be the first allogeneic T-cell immunotherapy for the treatment of hematological malignancies including acute myeloid leukemia, acute lymphoblastic leukemia and ...
Benzinga.com

FDA Pushes Back Axogen Avance Nerve Graft Decision By 3 Months

The U.S. Food and Drug Administration (FDA) on Monday extended the Prescription Drug User Fee Act (PDUFA) goal date for Axogen Inc.’s (NASDAQ:AXGN) Biologics License Application (BLA) for Avance Nerve ...

Ultragenyx Pharmaceutical Inc.: Ultragenyx Completes Rolling Submission of Biologics License Application (BLA) to U.S. FDA for DTX401 AAV Gene Therapy for Glycogen Storage ...

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that it has completed the rolling submission of its Biologics License Application (BLA) ...