UniQure said on Monday the U.S. health regulator has informed the company that data from an early- to mid-stage trial of its ...

United Therapeutics has linked a once-daily drug candidate to a 55% drop in the risk of clinical worsening in a phase 3 trial ...

A Rocket Pharmaceuticals gene therapy has reached the end of its regulatory journey before receiving an FDA decision. The company has withdrawn the submission for its therapeutic candidate for Fanconi ...

The planned filing comes amid heightened scrutiny of the FDA’s rare disease stance and a year after Prime deprioritized the program for economic reasons.

FDA decisions lack majority consensus, experts agree, possibly leading to less nuanced verdicts on new drug applications.

A Regeneron Pharmaceuticals drug that lowers levels of a disease-driving protein has met the goals of a pivotal test in the rare disease generalized myasthenia gravis. Based on these results, ...

Regenerative Medicine Advanced Therapy (RMAT) designation requests update submitted to FDA with recent biomarker data ...

PLAINSBORO, N.J., May 2, 2025 /PRNewswire/ -- Today, Novo Nordisk announced that the U.S. Food and Drug Administration (FDA) accepted its New Drug Application (NDA) submission for an investigational ...

Agency staff "strongly recommended" a sham surgery-controlled trial be conducted before an approval filing, a stance one analyst called a "worst case scenario" for UniQure.

Ocular Therapeutix reported a phase 3 win that it believes can validate its attempts to challenge Regeneron’s entrenched position in wet age-related macular degeneration (AMD) with investigational | ...

The FDA has accepted Moderna’s revised influenza vaccine filing days after controversially refusing to review the original submission, putting the biotech on track to win approval in time for the 2026 ...

MIRA trial data demonstrated a 43% response with 120mg SLK, and a 29 ppt delta vs placebo (p < 0.001) at week 12. VELA-1 met all primary and key secondary endpoints with statistical significance ...