On December 7, 2022, FDA’s Center for Biologics Evaluation and Research (CBER) and the Office of Tissues and Advanced Therapies (OTAT) held a town hall to answer questions related to cell therapy and tissue-engineered products chemistry, manufacturing, and controls (CMC). The purpose of these town …
Hosted by American Conference Institute, the FDA Boot Camp returns for its 40th iteration with the continued intent of providing an essential working knowledge of core FDA concepts, and real-world examples that will help you to excel in your everyday practices. This year’s conference co-chairs include …
On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes. …
On September 30, 2022, President Biden signed into law the Continuing Appropriations and Ukraine Supplemental Appropriations Act, 2023, Division F of which is titled the “FDA User Fee Reauthorization Act of 2022” (“FUFRA”). In addition to reauthorizing for an additional five fiscal years—Fiscal Years 2023-2027—several …
On October 26, 2022, US Pharmacopeia (USP) Biologics Stakeholder Forum held a workshop on “Collaborating to solve CMC challenges and support efficient development of lentiviral-mediated CART cell therapies.” Panelists discussed CMC challenges for CART therapies, including potency assay development, analytical method transfer, and demonstrating comparability. …
FDA recently published the final guidance document “Comparability Protocols for Postapproval Changes to the Chemistry, Manufacturing, and Controls Information in an NDA, ANDA, or BLA.” This final guidance provides recommendations to original applicants and holders of approved applications for human drugs and certain biological products …
During last year’s 2021 Pharmaceutical Quality Symposium, CDER described the development of a regulatory framework called FRAME –Framework for Regulatory Advanced Manufacturing Evaluation, to accommodate innovation expected in the next 5-10 years with a focus on 4 technologies: End to End Manufacturing, Distributed Manufacturing, Point-Of-Care, …
Making use of real-world data has long been of interest to stakeholders as having tremendous potential value. These data are routinely collected from a variety of sources, such as electronic health records, providing information on health and healthcare in actual patients, rather than in the …
As we begin the final quarter of 2022 and the leaves here on the east coast begin to turn and fall, it seems the clock may be running out on FDA and the Center for Drug Evaluation and Research (CDER) to meet its goal of …
On September 29, 2022, FDA’s Center for Biologics Evaluation and Research (CBER) and the Office of Tissues and Advanced Therapies (OTAT) held a town hall to answer questions related to gene therapy chemistry, manufacturing, and controls. Wilson Bryan opened this new forum for direct agency …
On September 29, 2022, FDA approved Amylyx’s NDA for its drug, Relyvrio (sodium phenylbutyrate/taurursodiol), for treatment of patients with amyotrophic lateral sclerosis (ALS) (see FDA announcement here). This approval decision charts a path for the exercise of appropriate flexibility in regulatory decisions for other rare …
On September 14, 2022, FDA/CDER/Office of New Drugs, in collaboration with the Duke-Margolis Center for Health Policy, hosted a virtual meeting on advancing premarket safety analytics, including sessions on new FDA Medical Queries and standardized presentations of safety data. For a number of years, FDA has been including …
FDA recently published a Draft Guidance entitled “Charging for Investigational Drugs under an Investigational New Drug Application: Questions and Answers” (the Draft Guidance). This Draft Guidance, when finalized, will replace the Final Guidance issued just six years ago (the 2016 Guidance). As the new Federal …
Last week, FDA issued a draft guidance entitled “Considerations for Rescinding Breakthrough Therapy Designation” (the Draft Guidance), authored by CDER, CBER, and the Oncology Center of Excellence (it is interesting, though not surprising, to see the OCE as an author on this document, given the …
Hyman, Phelps & McNamara, P.C.’s James Valentine and Ellis Unger will be presenting at the DIA 2022 Global Annual Meeting held this June 19-23 in Chicago, IL. This meeting brings together industry, regulators, academics, and patients to discuss global and local challenges in the life …
