FDA
The FDA has several big-ticket decisions lined up to close out July, including applications in lymphoma, rare diseases and a hormone deficiency, while GSK dares to DREAMM again in multiple myeloma.
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The overturning of the FDA’s lab-developed tests rule is just the tip of the iceberg. With the loss of Chevron deference, power has shifted from federal agencies to the courts, with potential implications for everything from the FDA shortage list to CMS drug price negotiations.
Arguably the most notable of the FDA’s upcoming decisions is that regarding Gilead’s twice-yearly HIV prophylaxis lenacapavir.
Here’s how companies can ensure they’re in compliance with new requirements that go into effect in August.
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Speaking at BIO2025, rare disease leaders from Ultragenyx, Amylyx and Yale questioned the need for the new regulatory pathway proposed by FDA Commissioner Marty Makary. They acknowledged, however, that creative thinking is required to enable more treatments for patients with ultrarare diseases.
Jacqueline Corrigan-Curay, who stepped into the role as the agency’s top drug regulator in January, is departing in July, according to an email sent to agency staff.
Harliku is the only FDA-approved drug for alkaptonuria, a rare condition characterized by skin discoloration, arthritis and heightened risks of heart failure and stroke.
Exemptions to new export control policies have allowed companies to ship patients’ biological materials to foreign laboratories, including those in China and other adversarial nations, according to the FDA.
The well-respected director of the FDA’s cell and gene therapy office was seen as a stabilizing and trustworthy voice inside the quickly reshaping FDA, especially since the late-March exit of CBER Director Peter Marks.
Lenacapavir, to be marketed as Yeztugo, could “redefine the PReP market,” according to analysts.
Industry watchers responded mostly positively to the commissioner’s new voucher program, but worries remain over staffing cuts at the agency.
Speaking at BIO2025, Makary bemoaned what he called “unnecessary steps” and “avoidable delays” in the U.S. regulatory landscape.
The program will bring together experts from across the FDA for a team-based review, rather than having an application move across numerous offices within the agency before getting a yay or nay.
According to an internal email, the agency may be in for more consolidation in areas including human resources, communications, travel and acquisitions.