BioMarin's gene therapy pipeline has had setbacks, including a three-year delay in US approval of Roctavian and stalled development of PKU gene therapy BMN 307 as the Food and Drug Administration ...

20% of bioreactor runs at the Genzyme facility failed due to contamination or quality issues over 2.5 years. FDA identified 84 overdue deviation investigations, some exceeding 180 days ...

BMRN reports better-than-expected fourth-quarter results, driven by encouraging uptake for Voxzogo. It reiterates its guidance of generating $4 billion in total sales by 2027.

Orchard Therapeutics has tasked Robin Kenselaar with accelerating growth of Lenmeldy, the world’s most expensive drug, ...

THURSDAY, Jan. 23, 2025 (HealthDay News) -- The U.S. Food and Drug Administration has warned ... for active pharmaceutical ingredients at their Genzyme manufacturing plant in Framingham ...

Sanofi SA (NASDAQ:SNY) and Johnson & Johnson (NYSE:JNJ) have discontinued the E.mbrace phase 3 study evaluating a vaccine ...

Fabry Disease Clinical Trial Pipeline Major pharmaceutical companies are working to advance the pipeline space and unlock the future growth ...

After winning approval in the United States back in September, Sanofi Genzyme’s multiple sclerosis drug, aubagio, has won approval for use in Australia. The Australian Therapeutic Goods ...

The recent FDA approvals of two treatments for molluscum contagiosum may represent a shift in how clinicians view this disease, according to a speaker at Masters of Pediatric Dermatology.In an ...