Dawnzera is the third medicine cleared by U.S. regulators this year for the rare genetic condition, and the second wholly owned medicine Ionis has brought to market.

The deal gives Gilead ownership of a technology that can genetically modify immune cells inside the body, an approach could potentially widen access to CAR-T treatment.

RFK Jr. has his sights set on increasing oversight and enforcement against drugmakers that run afoul of advertising laws.

Anne White, a 30-year company veteran who previously led the company’s oncology unit, will step down as head of Lilly ...

The company’s market value was nearly cut in half following trial results showing higher rates of treatment discontinuations ...

Scientists say HHS’ decision to defund mRNA vaccine research will leave the country less prepared for pandemics, and make it ...

The FDA clearance sets Novo’s medicine up a market battle with Madrigal Pharmaceuticals’ fast-selling Rezdiffra.

The FDA said data for PTC's drug did not prove "substantial evidence of efficacy." Elsewhere, the FDA delayed its decision on ...

The company plans to continue with a lower dose and a change in the pre-treatment regimen after a trial participant died ...

The startup agreed to pay Fosun Pharma as much as $645 million for a drug in testing for multiple lung conditions, including ...

The FDA’s announcement of Papzimeos’ approval contained an unusual quote from newly reinstated CBER director Vinay Prasad. Elsewhere, Eli Lilly and Superluminal Medicines struck a deal.

Miguel Fernández Alcalde, head of Merck KGaA’s U.S. arm, aims to “double down” on innovation. The company’s recent SpringWorks deal is just a start.