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FDA Commissioner Marty Makary intends to resign on Tuesday, according to several sources. This report follows a tumultuous 13-month tenure in which Makary oversaw the controversial rejections of several rare disease drugs and “predictable volatility” within the agency.
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New guidelines from two leading medical associations suggest that efforts to reduce bad cholesterol should focus on maintaining low levels of two key lipoproteins. Big pharma is all in, looking to improve on the standard statins to help vanquish America’s number one killer: heart disease.
The FDA’s decision last year to make complete response letters public provides new insight into why therapies sometimes fail to get the regulatory greenlight. Analysts say the information could help sponsors refine their regulatory strategies.
The Department of Health and Human Services is spinning its wheels, unable to establish steady leadership at three major divisions—the CDC and the FDA’s two primary review units.
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Robert F. Kennedy Jr.’s health department has consistently touted radical transparency as being key to its mission. Recent instances—the FDA’s decision not to disclose the recipients of three Commissioner’s National Priority Vouchers and FDA and CDC choices not to publish vaccine-related papers—call this intent into question.
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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.