Telomir Pharmaceuticals Inc. (NASDAQ:TELO) on Wednesday announced new preclinical data showing that its lead candidate, ...

Senate Republicans released their version of the Trump administration’s “One Big Beautiful Bill Act” on Monday with a few ...

Circle Pharma receives US FDA orphan drug designation for its CID-078 to treat small cell lung cancer: South San Francisco, California Wednesday, June 18, 2025, 18:00 Hrs [IST] So ...

FDA recommends inclusion of patients as young as 6 months - younger than Moleculin had proposed - to be allowed in the ...

Global Phase 3 BRAVE study will evaluate the efficacy and safety of omaveloxolone in children 2 to <16 years old with Friedreich ataxia, a rare neurodegenerative disorder BRAVE study will explore the ...

New intellectual property builds on Palvella’s multi-layered exclusivity strategy Patent term expected through at least 2038 ...

Shares of Cero Therapeutics Holdings drew increased retail investor chatter on Tuesday after the company said that the FDA ...

Eli Lilly and Co. is buying Verve Therapeutics Inc. and its gene-editing program for about $1.3 billion. Two of the one-time treatments are in the clinic. Lead candidate VERVE-102, a gene-editing ...

The designation qualifies CERo and CER-1236 for certain incentives, such as FDA assistance in designing clinical trials, access to the FDA Orphan Drug Grants Program, exemption from the drug approval ...

The biopharma industry has hit a setback in its ambition to leverage President Donald Trump’s “big, beautiful bill” to amend ...

Capricor Therapeutics (NASDAQ:CAPR) received the Orphan Drug designation from the U.S. FDA for its lead cell therapy candidate, Deramiocel, to treat becker muscular dystrophy. With its orphan drug ...

ABOUT ORPHAN DRUG DESIGNATION The FDA grants Orphan Drug Designation to investigational drugs intended for the treatment of rare diseases affecting fewer than 200,000 people in the U.S. Benefits ...