ClinicaSpace

Inspection access is not “an administrative footnote,” leaders of key FDA divisions wrote in an editorial on Wednesday vowing to expand oversight of foreign clinical trial sites.

Well-known for its oncology efforts and making recent headlines for a rumored mega merger with AstraZeneca, Bristol Myers Squibb is also dedicating considerable resources to neurological and psychiatric diseases, with a “continuum of care” mindset.

Current leaders Eli Lilly and Novo Nordisk have cornered the early market for weight loss therapeutics, but several other players, large and small, are bidding for a piece of the potential $200 billion pie.

Following a tumultuous stretch under ex-Commissioner Marty Makary, current FDA leadership is working to restore reliability, integrity and transparency to the agency—but experts say that doesn’t necessarily mean an easier regulatory road for drugmakers.

Three years after Qalsody was approved by the FDA as the first drug to target a genetic cause of ALS, some patients are reporting not only disease stabilization but also improvement of symptoms.

Despite the recent emergence of a new drug class, schizophrenia remains one of the most challenging neuropsychiatric diseases to treat—and Cobenfy’s first steps into the market indicate there is still room to grow.

While there are several FDA-approved psychiatric medicines on the market, many patients still struggle to achieve lasting symptom relief. Psychedelics could help fill this gap.

Replimune’s immunotherapy for advanced melanoma faced a perilous regulatory road with two previous rejections, but the drug—to be marketed as Tudriqev—prevailed with strong support from oncologists and a 10-3 advisory committee vote in its favor.

The FDA has been trying to mend fences with rare disease drug developers and patient groups since the departure of former Commissioner Marty Makary three months ago. But advisory committee meetings last week for Replimune and Capricor Therapeutics only created more confusion and frustration.

The FDA’s new investigational new drug pilot program—one part of HHS’s broader clinical trial modernization initiatives—has lofty goals to expedite first-in-human trials of novel drugs, but experts say it won’t tip the scales much on its own.