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.
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 ...
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, ...
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 ...
Regenerative Medicine Advanced Therapy (RMAT) designation requests update submitted to FDA with recent biomarker data ...
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 The company has withdrawn the submission for its therapeutic candidate for Fanconi ...
OX124 is based on Orexo´s world-class drug delivery platform, amorphOX®, and is designed to reverse the effects of the most powerful synthetic opioids, such as fentanyl Synthetic opioids are behind 91 ...
The sNDA filing acceptance initiates substantive FDA review for Ameluz PDT in sBCC, with a target action date of Sept. 28, 2026 and no identified filing deficiencies. Approval would establish the ...
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 ...
FDA decisions lack majority consensus, experts agree, possibly leading to less nuanced verdicts on new drug applications.
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