Regulatory Filings and Submissions

FDA has approved the first topical gene therapy product for treating wounds in patients with dystrophic epidermolysis bullosa.

FDA has approved AbbVie’s EPKINLY (epcoritamab-bysp), a bispecific antibody for treating relapsed or refractory diffuse large B-cell lymphoma.

Biotech firm Lumen Bioscience has received fast track designation from FDA for its oral biologic drug candidate for treating C. difficile infection.

The two draft guidance documents provide recommendations for the approval of pediatric drugs, biologics, and vaccines under the Pediatric Research Equity Act and the Best Pharmaceuticals for Children Act.

The recent revision is intended to provide guidance on QRM principles and tools that can be used for different aspects of pharmaceutical quality.

FDA has granted fast track designation to Caribou Biosciences for its allogeneic CAR-T cell therapy for relapsed or refractory multiple myeloma.

Will court challenges to FDA’s oversight authority open the door for multiple lawsuits to block agency decisions on all sides?

FDA aims to address mounting criticisms of its accelerated approval pathway.

Takeda received approval in Japan for a subcutaneous injection formulation of Entyvio (vedolizumab) for treating ulcerative colitis.

Even after an FDA advisory panel voted 14–1 in October 2022 to remove the drug, Makena manufacturer Covis Pharma continued to press for some continued access to the therapy.

This warning is the second on top of a previous warning not to purchase or use EzriCare Artificial Tears or Delsam Pharma’s Artificial Tears due to potential bacterial contamination.

Apellis Pharmaceuticals’ Syfovre is a pegcetacoplan injection intended for the treatment of geographic atrophy secondary to age-related macular degeneration.

The intention of the FOA is to address diverse topics related to improving the efficiency of biosimilar product development and advancing the development of interchangeable products.

Clinicians, pediatric patients, regulators, and more are being considered to further strengthen focus on unmet medical needs.

A Congressional probe of FDA’s approval of Alzheimer’s treatment targets the agency’s interactions with drug manufacturers.

In a response letter, FDA stated that it could not grant Eli Lilly and Company accelerated approval due to concerns surrounding insufficient quantity of clinical trial data.

The guidance provides examples of required and recommended information in the Dosage and Administration section.

Bylvay has minimal systemic exposure and acts locally in the small intestine as a potent, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi).

New medications show exciting outcomes for weight loss.

FDA keeps its user fees but fails to gain important reforms.

AstraZeneca’s Imjudo (tremelimumab) was approved by FDA in combination with Imfinzi (durvalumab) for treating unresectable liver cancer.

An advisory committee’s vote to remove Makena from the market illustrates how difficult it is for regulators to ensure that such drugs document safety and effectiveness in a timely manner.

CBER maps modernization plan to handle surge in research and applications.

Legislative leaders are looking to approve a bare-bones measure to fund FDA operations as part of a short-term government funding bill.

A Post Approval Change (PAC) to replace identity (ID) testing of incoming liquid drug substance with ID by visual inspection is considered a low risk provided questioned listed in this example have been answered favorably. In that case the PAC can be downgraded from a prior approval to a notification to health authorities, and managed in the PQS with immediate implementation effect.