Complete response letters

Sarepta Therapeutics faces serious FDA action after news broke of a third patient death, the FDA gets a new top drug regulator in George Tidmarsh, a handful of new drugs get turned away from the market and pharma companies continue to commit billions to reshoring manufacturing.

BMO Capital Markets pointed to FDA leadership, and CBER Director Vinay Prasad in particular, as potential factors in the agency’s decision to issue a complete response letter for Replimmune’s viral treatment RP1 for advanced melanomas. Shares of the company tumbled 75% on Tuesday.

Roche and Genentech were unable to sufficiently demonstrate the benefit of using Columvi in an earlier treatment setting for DLBCL in a U.S. population, according to the FDA.

Around 3,500 FDA employees received termination emails; FDA Commissioner Marty Makary suggests lowering industry user fees and tying review times to drug prices; the regulator opens its trove of complete response letters in the name of transparency; and two companies receive rejections for rare disease therapies.

The FDA cited manufacturing issues but did not flag problems with Ultragenyx’s data package for UX111, with the biotech noting that the regulator found its neurodevelopmental findings for the gene therapy to be “robust.”

In its complete response letter, the FDA cited insufficient evidence establish deramiocel’s effectiveness for cardiomyopathy associated with Duchenne muscular dystrophy. The decision comes after CBER Director Vinay Prasad canceled an advisory committee meeting for the therapy.

The trove of more than 200 letters is part of a pledge of transparency from the agency, with the intention to increase public insight into the reasons new drug and biologics applications got rejected.

Atara Therapeutics’ Ebvallo, already marketed in Europe for a transplant-related blood cancer, will not hit the U.S. market just yet, forcing the company to “significantly reduce expenses.”

After a series of strongly worded letters—one of which was addressed directly to Commissioner Robert Califf—the FDA has publicly laid out its reasoning for rejecting Vanda Pharmaceuticals’ gastroparesis drug candidate tradipitant.

Lykos Therapeutics is currently working out ways to fund an additional Phase III study for its MDMA-assisted PTSD therapy following an FDA setback last year.