fda-and-biotech News
Catalyst Pharma Touts AGAMREE's Superior Safety in Long-Term DMD Study
Five-year data shows the Duchenne muscular dystrophy drug has comparable effectiveness to corticosteroids but with significantly fewer side effects like growth stunting and bone fractures.
Sarepta Plunges After Key DMD Drug Study Misses Endpoint
The failed confirmatory trial for two of its Duchenne therapies adds pressure as the company navigates safety concerns around its flagship gene therapy, Elevidys.
Caribou Biosciences Jumps on Off-the-Shelf Cancer Therapy Data
Phase 1 results for its lead therapy show efficacy and durability in lymphoma patients comparable to existing personalized treatments, boosting its allogeneic platform.
Terns Pharma Shares Soar on 'Unprecedented' Leukemia Drug Data
Phase 1 trial for TERN-701 shows a 64% response rate in hard-to-treat CML patients, setting the stage for a pivotal presentation at the upcoming ASH annual meeting.
UniQure Stock Plummets 65% on FDA Reversal for Huntington's Drug
The U.S. regulator unexpectedly deemed Phase I/II trial data for gene therapy AMT-130 insufficient for an accelerated approval submission, creating significant delays.
FDA Drug Chief Resigns, Casting Shadow Over Pharma Sector
Departure comes amid allegations of misconduct and a lawsuit from a drugmaker, threatening to slow critical drug approvals and creating deep uncertainty for investors.
Alvotech Shares Fall After FDA Rejects Key Biosimilar Drug
The U.S. regulator cited manufacturing deficiencies for the company's AVT05 biosimilar, prompting a cut in Alvotech's 2025 financial forecast.
Cybin Stock Wavers After $175M Capital Raise Sparks Dilution Fears
The biotech firm secured backing from major healthcare funds for its psychedelic-based therapies, but the deal represents nearly 85% of its public market value.