With the PDUFA date fast approaching for Regenxbio Inc.’s gene therapy RGX-121, the U.S. FDA placed the drug on clinical hold along with another, RGX-111, after preliminary analysis of a single case of neoplasm (specifically, an intraventricular central nervous system tumor) in a participant treated in the phase I/II study with the latter treatment.
With plans in place to launch global pivotal testing of GH-001, its inhaled version of psychedelic mebufotenin (5-MeO-DMT), in treatment-resistant depression in 2026, GH Research plc reported the lifting of a U.S. FDA clinical hold, enabling U.S. subject enrollment. The firm now will seek a meeting with the agency to discuss trial design.
When it won U.S. FDA accelerated approval more than eight years ago, Intercept Pharmaceuticals Inc.’s Ocaliva (obeticholic acid) was viewed as a breakthrough, becoming the first new treatment in 20 years for rare, progressive liver disease primary biliary cholangitis (PBC) and, for several years, the only second-line treatment for PBC patients failing to respond to ursodeoxycholic acid. More recently, however, Ocaliva has faced regulatory and safety stumbles, with Intercept now voluntarily pulling the farnesoid X receptor activator from the U.S. market.
Rocket Pharmaceuticals Inc. CEO Gaurav Shah said his firm is investigating how its gene therapy for Danon disease may have created an “unexpected and paradoxical” effect that led to problems for a phase II patient who ultimately died.
Orum Therapeutics Inc. on April 26 pulled the plug on a U.S.-based phase I study of ORM-5029, its lead oncology degrader antibody conjugate (DAC) asset, a decision that came months after the company first reported a patient death in November 2024.
Amid an overall positive earnings report of $3.2 billion in 2024 revenues, Moderna Inc. disclosed that the U.S. FDA placed its norovirus vaccine on a phase III clinical hold due to a single adverse event of Guillain-Barré syndrome (GBS).
Rapt Therapeutics Inc. has decided to shut down its zelnecirnon (RPT-193) program in asthma and atopic dermatitis, causing the company’s stock (NASDAQ:RAPT) to sharply decline Nov. 11.
The U.S. FDA has lifted the full clinical hold it imposed in June on Biomea Fusion Inc.’ s phase I/II studies of BMF-219 in types 1 and 2 diabetes. A safety review of the phase IIb expansion study was encouraging and none of the elevated lab values confirmed serious liver injury or impairment, said Biomea’s CEO, Thomas Butler.
The U.S. FDA clamped a full clinical hold Biomea Fusion Inc.‘s phase I/II study of BMF-219 for treating type 1 diabetes and type 2 diabetes. The hold sank the stock on June 7 as the company looked to find answers so it could sit down with the agency to discuss next steps.
On the verge of top-line data from its phase IIb trial with oral small-molecule CC chemokine receptor 4 (CCR4) antagonist zelnecirnon in atopic dermatitis (AD), due around the middle of this year, Rapt Therapeutics Inc. said the U.S. FDA has imposed a clinical hold on that study with the otherwise promising drug, also known as RPT-193, in AD as well as the phase IIa trial with the same compound in asthma.
