News
The FDA plans to hold an advisory committee meeting to discuss Capricor Therapeutics’ application for deramiocel, which the agency rejected last July. The news surprised CEO Linda Marbán, who told BioSpace the FDA has not communicated any issues of concern with the company’s resubmitted application.
FEATURED STORIES
Dual and even triple or quadruple track processes have come roaring back in 2026 thanks to a glut of M&A that has refilled investors’ wallets. Big Pharma is being put on notice that time is critical if they want to acquire.
Policymaking at FDA has been anything but business as usual under the Trump administration, but former regulators cite the agency’s new investigational new drug pilot program as a sign of normalcy.
The FDA’s recently altered outlook on the evidence required for approval of rare disease drugs could have immediate benefits for companies including Skyhawk Therapeutics, Capricor Therapeutics and Biohaven.
FROM OUR EDITORS
Read our takes on the biggest stories happening in the industry.
If cell and gene therapy makers are going to achieve their mission to improve patients’ lives, the industry must come together to share information across stakeholders, from regulators to manufacturers to payers.
THE LATEST
Commissioner Marty Makary said that the FDA will soon start requiring only one pivotal trial, instead of two, for companies seeking approval for new drugs.
Hypersensitivity reactions in a mouse model prompted the agency to suspend Denali’s planned Phase I development for DNL952 for Pompe disease.
Praxis Precision Medicines has also announced a “successful” pre-NDA meeting with the FDA for its essential tremor drug candidate ulixacaltamide, for which an approval application is slated for early 2026.
Days after Johnson & Johnson’s posdinemab failed to slow clinical decline in patients with Alzheimer’s disease, Eisai Chief Clinical Officer Lynn Kramer expressed unwavering conviction in his company’s own anti-tau asset, while others suggest the Alzheimer’s field is heading in a completely different direction.
Investor optimism has waned as final minutes from uniQure’s pre-BLA meeting with the FDA convey that data from the company’s Phase I/II studies of AMT-130 are “unlikely” to provide the primary evidence to support a biologics license application.
Writing in separate editorials in two leading medical journals, former chiefs of federal scientific agencies issued warnings about the changes being proposed to vaccine frameworks by current officials.
The partnership will focus on Crescent’s PD-1/VEGF inhibitor CR-001 and Kelun-Biotech’s SKB105, both of which the companies plan to push into Phase I/II development for solid tumors early next year.
Companies who run their early-stage clinical development outside the U.S. would “experience higher fees” according to an FDA proposal made during the negotiation process for the eighth cycle of the user fee program.
Last week, Center for Biologics Evaluation and Research Director Vinay Prasad claimed in an internal memo—without providing evidence—that COVID-19 vaccines were responsible for the deaths of 10 children between 2021 and 2024.
Høeg is the fifth person to lead the Center for Drug Evaluation and Research this year.