Turbulence at the FDA & bluebird Bio's sale

Today, we talk about BIO chief John Crowley’s views on rare disease innovation, and the “aggressive” slashing of personnel at federal health agencies. Also, Pfizer changes its DEI approach online.

The need-to-know this morning

• Praxis Precision Medicine said independent data monitors recommended the discontinuation of a Phase 3 study involving its experimental treatment for essential tremors. The futility analysis concluded the Praxis drug was unlikely to show a benefit compared to a placebo. Praxis, however, has decided to complete the study and a companion study also underway.

BIO CEO: FDA reform needs to be underpinned by strategy, not ‘indiscriminate’ cuts

The Trump administration could save the lives of countless patients with rare diseases by modernizing and streamlining the FDA’s approval process, John Crowley, the CEO and president of the trade group BIO, opines this morning for STAT. Specifically, he said that excising redundancies and bureaucracies and focusing on patients would help accelerate cures.

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