Sarepta Therapeutics is meeting with the FDA in June to discuss expanding the use of its Duchenne muscular dystrophy gene ...

The government 'did not act contrary to the law' by requiring drugmakers to seek approval before implementing proposed new 340B rebate models, a federal judge ruled.

Rocket Pharmaceuticals reports early success in gene therapy trial for heart condition PKP2-ACM, with 3 patients showing ...

The FDA is prepping to renegotiate its multibillion-dollar, quinquennial user fee agreements with the drug industry later ...

The US Supreme Court should decline to review a generic drug labeling fight between Amarin and Hikma Pharmaceuticals because ...

American Society of Gene & Cell Therapy meeting sees 10% drop in attendance. Notable absences include FDA officials, David ...

The Trump administration is partnering with Mark Cuban’s compounding business and four other entities to improve the ...

BioMarin plans to buy Boston-based Inozyme Pharma and its Phase 3 enzyme replacement therapy for about $270 million, the ...

Leo Pharma is doing an extensive revamp of its leadership team as it splits product strategy and international operations ...

RFK Jr. advisor Calley Means challenges drugmakers considering pulling their investment in the US over Trump's most favored ...

The comments come just a couple months after the company slashed its 2027 sales expectations for the Biogen-partnered ...

Vinay Prasad may have just started as the new director of the FDA's biologics center, but he's 'planning to unleash a massive ...