Complete Response Letters recently issued by the FDA signal heightened scrutiny of trial design and reinforce the agency’s shifting regulatory expectations for sponsors and CROs.

The European Union’s health regulatory agency did not endorse approving Elevidys for ambulatory patients with Duchenne ...

The European Medicines Agency determined testing failed to prove Elevidys’ benefit and issued a negative opinion that could ...

Morning. Today, we discuss how Duchenne patients and their families are responding to the shelving of Sarepta Therapeutics’ ...

Shares of Sarepta Therapeutics Inc. (NASDAQ:SRPT) plunged 13% on Friday after a major regulatory blow in Europe. The European ...

Roche Holding AG’s gene therapy Elevidys failed to get the backing of European regulators, a blow to the Swiss drugmaker and to Sarepta Therapeutics Inc., the embattled US biotech that developed the ...

Separately, Sareptea said last week that it would pause the development of most of its experimental gene therapies for a different type of muscular dystrophy. The stoppage came after one of the ...

CBER is unanimously against Elevdiys’ return to the market without additional evidence, according to media reports citing an ...

Sarepta Therapeutics suffered another major setback on Friday as Europe's drug regulator decided not to recommend the ...

The drug, which was designed to treat Duchenne muscular dystrophy, has been linked to the deaths of two teenage boys.

Since Dr. Vinay Prasad’s appointment as a key political voice within FDA, the tone of U.S. biotech policy has shifted from ...

Dispatch Bio launched with $216 million – backed primarily by Parker Institute for Cancer Immunotherapy, founded by entrepreneur Sean Parker, and ARCH Venture Partners. SAB BIO announced an ...