Top of the morning to you, and a fine one it is. Clear blue skies and cool breezes are wafting over the Pharmalot campus, where the official mascots are snoozing in their respective corners. This means we are free to focus on the matters at hand — rummaging through our to-do list and making cups…
Acting U.S. FDA Commissioner Kyle Diamantas met with rare disease groups on Wednesday, according to groups attending and a government official, as the new chief seeks to repair relations with a sector disappointed by his predecessor. Representatives for rare disease organizations including Friends of Cancer Research and the Foundation for Angelman Syndrome Therapeutics are pushing…
The long-time FDA Oncology Center of Excellence director joins former FDA and NCI officials on the FOCR board of directors. Richard Pazdur, the long-time head of the US Food and Drug Administration’s oncology review divisions, was elected to the board of the Friends of Cancer Research, one of the sector’s key advocates. Pazdur, who left…
More flexibility in early-phase development for cell therapies is needed, a panel of US Food and Drug Administration (FDA) representatives and industry members said at a public meeting hosted by Friends of Cancer Research and the Parker Institute for Cancer Immunotherapy. Vijay Kumar, acting director of the Office of Therapeutic Products (OTP) in the FDA…
Rare disease advocates said chemistry, manufacturing and controls requirements in the FDA’s plausible mechanism framework guidance should not resemble standards for large-scale manufacturing. Key Takeaways CMC requirements in the FDA plausible mechanism guidance could limit the diseases and patient populations that could access the framework, advocates said. The FDA should clarify how manufacturing expectations would…
The plausible mechanism framework draft guidance requirement for “well-characterized natural history” could be too high a bar for many rare diseases and more clarity is needed on the patient populations that would qualify, advocacy groups said. Key Takeaways Patient advocates said the requirement for “well-characterized natural history” is vague and may preclude many rare disease…
The FDA’s biomarker qualification process has drawn criticism for its long timelines. A new incubator initiative is meant strengthen the agency’s data aggregation and evaluation capabilities, starting with a pilot project focused on drug-induced kidney injury. Background on biomarkers Biomarkers are a vital tool for disease diagnosis, patient risk assessment and drug development. The FDA describes biomarkers as…
On May 5, the FDA quietly released a new guidance document with considerations regarding its existing policy flexibilities for chemistry, manufacturing and controls issues in cell and gene therapy products. Here, AgencyIQ breaks down what’s in that document and the discussion of it at a timely May 6 Friends of Cancer Research workshop. Catching up:…
Sponsors working outside the US to accelerate first-in-human studies could miss the many new flexibilities the US FDA offers, the agency’s head of cell and gene therapy application reviews said. Key Takeaways Acting OTP Director Vijay Kumar said that overseas first-in-human studies may not provide a long-term benefit. He said sponsors would forego the advantages…
Listen to Dr. Crystal Mackall on the Future of Medicine podcast — a fascinating conversation about the science, breakthroughs, and challenges shaping the next era of cancer treatment. Dr. Crystal Mackall, a pioneer in cancer immunotherapy, traces her journey from a working-class Ohio upbringing to leading cutting-edge T-cell research at Stanford. Inspired by family members…
