Tag Archive for: regulatory approval

Following an initial rejection in 2023, Ascendis Pharma on Tuesday said it faces another regulatory bump in the road for TransCon PTH with a three-month delay in the ongoing FDA review.

The company announced that the FDA declined to approve the expanded use of its hepatitis B vaccine in patients undergoing hemodialysis, citing insufficient safety and effectiveness data.

FDA Commissioner Robert Califf has said that advisory committee meetings should focus more on discussion and be “less about the outcome,” but other agency officials recently advocated for retaining the vote on whether to approve a product under review.

The FDA’s authorization of therapies that fail their efficacy endpoints casts doubt on the regulator’s accelerated approval pathway, according to an article from David Rind, chief medical officer of the drug pricing non-profit Institute for Clinical and Economic Review, published in the Journal of the American Medical Association. In his viewpoint piece, Rind specifically called out Sarepta Therapeutics’ gene therapy Elevidys, which won the FDA’s accelerated approval in June 2023—despite falling short of its primary efficacy endpoint in two studies.

Yesterday Boehringer Ingelheim won the FDA’s approval for a high-concentration and citrate-free version of its Humira biosimilar Cyltezo, targeting AbbVie’s blockbuster drug.

Neurocrine Biosciences won the FDA’s approval for a sprinkle capsule formulation of Ingrezza, providing a more convenient route of treatment for Huntington’s disease patients with trouble swallowing.

Based on strong overall and progression-free survival data in its Phase III confirmatory study, Pfizer and Genmab’s antibody-drug conjugate Tivdak on Monday secured the FDA’s full approval for recurrent or metastatic cervical cancer that had progressed on or after chemotherapy.

X4’s mavorixafor, to be sold under the brand name Xolremdi, is the first therapy to get U.S. approval specifically for the treatment of WHIM syndrome.

Yesterday that FDA approved Pivya (pivmecillinam) tablets for the treatment of female adults with uncomplicated urinary tract infections (UTIs) caused by susceptible isolates of Escherichia coli, Proteus mirabilis and Staphylococcus saprophyticus. 

The agency’s green light is a shot in the arm for ImmunityBio, which had reiterated doubts in a regulatory filing last month about its ability to remain in business.