FDA
The FDA last October paused Intellia Therapeutics’ late-stage CRISPR studies after detecting life-threatening enzyme elevations in one patient, who died a few days later.
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This week’s Capitol Hill meetings come on the heels of rejections of ultra-rare disease drugs developed by Biohaven and Saol Therapeutics. Physicians and patient groups implored the FDA to expedite these treatments.
Last week, the FDA made its one pivotal trial policy official, sparking myriad questions from industry leaders, including around specific evidence required for the single study and why it hasn’t been implemented across all therapeutic areas before now.
FDA vouchers are normally a coveted prize for biopharma companies, but a surprise rejection for Disc Medicine’s rare disease drug has biopharma reconsidering.
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FDA Commissioner Marty Makary presented a new idea to staff this week: bonus payments for employees that complete regulatory review processes faster than expected.
Hernexeos is the second drug to secure an FDA approval under the agency’s priority voucher scheme, following in the footsteps of USAntibiotics’ Augmentin XR, which was granted the ticket in December 2025.
Aside from creating a toxic work environment, CBER Director Vinay Prasad has also been accused of berating his staff and retaliating against reviewers who questioned his decisions.
A year of significant policy change at the FDA brought momentum and scrutiny into the new year. As 2026 gets underway, biopharma companies are responding to sweeping vaccine changes while concerns surface about the politicization of the agency.
Without naming a specific product, Commissioner Marty Makary referred to an investigational therapy, delivered surgically into the brain, that the FDA was “pressured” to approve even after finding no clinical benefit to patients.
The rejection of Atara Biotherapeutics’ Ebvallo in January was a “complete reversal” of the conesensus FDA reviewers had come to, according to a former agency employee, who said manufacturing problems were the only approvability barrier for the drug.
Regulators overseeing rare disease treatments need better tools to weigh competing risks in real time. Sarepta Therapeutics’ Elevidys is a prime example of why.
In August last year, the Health Department cut around $500 million in mRNA research funding, with Health Secretary Robert F. Kennedy Jr. saying the agency would instead divert the money “toward safer, broader vaccine platforms.”
The framework, first introduced by FDA Commissioner Marty Makary and Center for Biologics Evaluation and Research head Vinay Prasad in November, was criticized for lacking detailed guidance. Agency leaders elucidated on the pathway for personalized medicines on Monday.
Bysanti is based on iloperidone, an active metabolite of a compound that forms the core of Fanapt, another drug by Vanda Pharmaceuticals.