KLA Breakthrough Consulting

The FDA drafts Q&A guidance on cell and gene therapy development They said the guidance was developed under its mandate to increase efficiency in the development of CGT products under the Prescription Drug User Fee Act (PDUFA VII) agreement. It includes a total of 36 questions and answers on issues such as regulatory review, chemistry, manufacturing, and controls, pharmacology/toxicology, clinical trials, and clinical pharmacology. The first and one of the most basic questions addressed by the guidance is, what sponsors should know about submitting an investigational new drug (IND) application. FDA noted that sponsors of commercial INDs should submit their applications through the Electronic Submission Gateway (ESG) in electronic common technical document (eCTD) format; eCTD is optional for sponsors of noncommercial INDs. ? “A commercial IND is generally one for which the sponsor (usually a corporate entity) intends to commercialize the product by eventually submitting a marketing application,” FDA clarified. “In comparison, a noncommercial IND is an IND for a product that is not intended for commercial distribution and includes research and investigator-sponsored INDs.” ? “The sponsor of a noncommercial IND may generally be an individual investigator, academic institution, or non-profit entity,” the agency added. “The studies proposed in these INDs are generally for research, and may result in publications in peer-reviewed journals.” ?

FDA Approves First Gene Therapy for Treatment of Aromatic L-amino Acid Decarboxylase Deficiency “AADC deficiency can cause a range of debilitating symptoms, including life-threatening complications,” said Nicole Verdun, M.D., director of the Office of Therapeutic Products in CBER. “Today’s approval represents important progress in the advancement and availability of safe and effective treatments for debilitating genetic disorders.” The application received Priority Review and Orphan Drug designation, and was granted a rare pediatric disease priority review voucher by the FDA. PTC Therapeutics, Inc. ??

On Thursday, the FDA updated the?Breakthrough Device designations data, as well as the number of Breakthrough Devices that have received marketing authorizations. From the launch of the Breakthrough Devices Program through September 30, 2024, the FDA has granted 1,041 Breakthrough Device designations and authorized 128 Breakthrough Devices for marketing. The program is intended to provide patients and health care providers with timely access to certain medical devices by speeding up development, assessment, and review for premarket approval, 510(k) clearance, and De Novo marketing authorization. Through the program, manufacturers can interact with the FDA’s experts to receive feedback on device development, receive help navigating the path to the FDA’s marketing authorization, and obtain prioritized review on regulatory submissions.?

FDA Pilot to Gain Experience Reviewing a Small Number of Original Applications following Split Real Time Application Review (STAR) Paradigm Starting on December 2, 2024, CDER and CBER will each be accepting, on a voluntary basis, a limited number (1-2) of original marketing applications. Details in the comments below.

The FDA’s Center for Drug Evaluation and Research (CDER) released the 2024 Accelerating Rare disease Cures (ARC) Program Annual Report. Since its launch in 2022, CDER’s ARC Program has become a key resource for the rare disease community and a driver of innovation in rare disease drug treatments. Having built a strong foundation, the ARC Program is enthusiastic about the upcoming year as it continues to strengthen collaboration to accomplish even more progress in rare disease drug development. Full report in the comments below

Astellas Pharma has gained approval from the US Food and Drug Administration (FDA) for VYLOY (zolbetuximab-clzb), in conjunction with chemotherapy, for the treatment of advanced gastric and gastroesophageal junction (GEJ) cancer. Astellas senior vice-president and immuno-oncology development head Moitreyee Chatterjee-Kishore, Ph.D., MBA stated: “The approval of VYLOY as the first and only targeted therapy for CLDN18.2-positive patients in the US further delivers on our relentless pursuit of scientific progress for devastating diseases like gastric and GEJ cancers, which are often only discovered at the advanced stage.’’

FDA approves osimertinib for locally advanced, unresectable (stage III) non-small cell lung cancer following chemoradiation therapy AstraZeneca

FDA accepting requests to participate in CMC development and readiness program

FDA Approves Drug with New Mechanism of Action for Treatment of Schizophrenia “Schizophrenia is a leading cause of disability worldwide. It is a severe, chronic mental illness that is often damaging to a person’s quality of life,” said Tiffany Farchione, M.D., director of the Division of Psychiatry, Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research. “This drug takes the first new approach to schizophrenia treatment in decades. This approval offers a new alternative to the antipsychotic medications people with schizophrenia have previously been prescribed.” Bristol Myers Squibb ??

“The progression-free survival benefits seen in the MARIPOSA-2 study are exciting,” -- Andrea Ferris, President and CEO, LUNGevity Foundation. “It is good to see new therapeutic options like the combination of RYBREVANT and chemotherapy helping to address unmet needs impacting individuals with EGFR-mutated lung cancer, with the potential for positive change, which gives hope to more patients and their families.” Janssen Inc.