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Regeneron to challenge Ipsen as FDA clears bone disease drug

Pasatru's approval sets up a market battle in a rare indication where the only available treatment has generated disappointing sales.

Cancer vaccine win restores Wall Street’s faith in Moderna

Moderna gained $45 billion in market value in a single day, a surge that brought the company's stock to levels not seen since 2024 - and saddled it with expectations that may be tough to meet.

Ultragenyx, after setbacks, nabs first gene therapy approval

The clearance of Genglycos for a rare glycogen storage disease gives Ultragenyx a sellable voucher and momentum ahead of an eagerly anticipated study readout.

Trump nominates policy aide Overton to lead FDA

Heidi Overton, a medical doctor and veteran of the America First think tank, currently serves as deputy director of the White House Domestic Policy Council.

Moderna and Merck cancer vaccine returns ‘landmark’ result in melanoma

At an interim data check, investigators of a key trial found the individualized mRNA treatment kept disease from returning and stopped its spread, sparking a surge in Moderna shares.

BioMarin acquires experimental drug for rare bone disorder in $275M deal

The Alesta buyout helps restock BioMarin's pipeline with an asset it says could "reach our largest addressable patient population," joining other muscle and skeletal disease treatments in development.

AstraZeneca scores in lung cancer; Sandoz inks a China deal

A combination of AstraZeneca's Tagrisso and Hutchmed's Orpathys extended survival for some lung cancer patients. Elsewhere, MapLight floated the idea of partnering on its autism drug.

Leo Pharma buys rare skin disease drug in $435M deal

Tanabe Pharma has already asked the FDA to approve the medicine for two conditions that cause severe reactions to sunlight.

Amylyx’s bet on a GLP-1 blocker pays off

A late-stage trial found the drug, which Amylyx bought for cheap in a bankruptcy auction, was effective at regulating blood sugar in patients who'd just undergone bariatric surgery.

Argenx jumps on Phase 3 data for blockbuster Vyvgart

The latest results give investors confidence in the drug's ability to be approved in another indication for which there are no approved treatments.