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TMCnet17m
Biogen Initiates Phase 3 Pediatric Study of Omaveloxolone for the Treatment of Friedreich Ataxia
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 ...
Palvella Therapeutics Granted Sixth U.S. Patent Covering 0.1–20% Anhydrous Compositions of Rapamycin and Other mTOR Inhibitors
New intellectual property builds on Palvella’s multi-layered exclusivity strategy Patent term expected through at least 2038 ...
Taiwan News26m
Moleculin Receives Positive FDA Feedback on Pediatric Study Plan for Annamycin in Children with R/R AML
Moleculin is in the process of updating its iPSP to incorporate FDA’s recommendations and intends to submit the revised plan to FDA later this quarter. Moleculin expects to initiate pediatric clinical ...
The Pharma Letter1h
Senate drops orphan drug bill amendment opposed by patient advocates
A controversial proposal to change the Inflation Reduction Act’s pricing framework for rare disease drugs has been dropped ...
pharmaphorum3h
FDA unveils fast-track review path for 'national priorities'
The Commissioner's National Priority Voucher (CNPV) programme will be available to companies aligned with pressing matters ...
Stocktwits on MSN10h
Cero Therapeutics Rockets On FDA Orphan Drug Status For Blood Cancer Drug; Retail Bulls Feel Stock 'Still Has A Few Pumps Left'
Shares of Cero Therapeutics Holdings drew increased retail investor chatter on Tuesday after the company said that the FDA ...
CERo Therapeutics stock soars after FDA grants Orphan Drug Designation
Investing.com -- CERo Therapeutics Holdings Inc (NASDAQ: CERO) stock surged 191.6% after the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) for the company’s lead drug ...
BioWorld17h
In a $1.2b deal, Lilly buys gene editor Verve
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 ...
Fierce Pharma20h
In setback for biopharma, Senate version of Trump's tax bill drops Orphan Cures Act
The biopharma industry has hit a setback in its ambition to leverage President Donald Trump’s “big, beautiful bill” to amend ...
RemeGen's Telitacicept (RC18) Received Orphan Drug Designation from EMA for Myasthenia Gravis
RemeGen Co., Ltd. ("RemeGen", stock symbols: 688331.SH/09995.HK) announced that telitacicept (RC18; brand name: 泰爱®) has received Orphan Drug Designation (ODD) from European Medicines Agency (EMA) for ...
Capricor gets FDA's Orphan Drug status for muscle disorder therapy
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 ...
TMCnet21h
FDA Grants Orphan Drug Designation to Medicovestor's First-in-Class ADC for Pancreatic Cancer
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 ...