Regulatory
The regulatory path for companies developing drugs for rare diseases is often fraught with challenges. KemPharm CEO Travis Mickle discussed just some of these with BioSpace.
Curis announced that the FDA has allowed patient enrollment to resume in the monotherapy phase of its TakeAim Leukemia Phase I/II trial studying emavusertib.
Blueprint Medicines’ Ayvakit is headed to the FDA for a new indication after the drug hit the mark in Part 2 of the PIONEER trial for non-advanced systemic mastocytosis.
The FDA has accepted the sNDA for AstraZeneca and Merck’s Lynparza and the sBLA for Genentech (Roche)'s for Polivy in diffuse large B-cell lymphoma.
BrainStorm Cell Therapeutics said it will submit a Biologics License Application to the FDA for ALS hopeful NurOwn. Additionally, a correction has been made to analyses of the Phase III clinical trial.
Daiichi Sankyo and AstraZeneca report another first-in-class approval for Enhertu, Bayer snags an sNDA in metastatic hormone-sensitive prostate cancer and Merck faces contamination challenges with Januvia.
The FDA will still allow continued distribution of sitagliptin containing NTTP with an acceptable intake limit of 37 ng per day and up to 246.7 ng per day.
The FDA has had a busy week, accepting drug applications, approving clinical trials and granting various special designations for Gamida Cell, Cellectis, Scynexis & more.
Experts from U.S. health regulatory agencies have announced plans to conduct a clinical trial to assess the safety and efficacy of TPOXX (tecovirimat) as an antiviral for monkeypox.
In its Q2 conference call, Sarepta Therapeutics indicated plans to accelerate its timeline for SRP-9001 a gene therapy for Duchenne muscular dystrophy (DMD).
PRESS RELEASES