Duchenne and Becker muscular dystrophy (DBMD) are allelic disorders caused by mutations in dystrophin. Adults with DBMD develop life-threatening cardiomyopathy.
Akashi Therapeutics, Inc., announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to the company’s most advanced product candidate, HT-100 (delayed-release halofuginone), an orally available, small molecule drug candidate intended to reduce fibrosis and inflammation and promote healthy muscle regeneration in boys with DMD. Fast track designation is granted by the FDA to facilitate the development and expedite the review of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.
Prosensa Holding N.V. (NASDAQ: RNA), the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, today announced the successful completion of enrollment in its prospective Natural History Study designed to increase the understanding of Duchenne muscular dystrophy (DMD).
Antisense therapy with both chemistries of phosphorodiamidate morpholino oligomers (PMOs) and 2′-O-methyl phosphorothioate has demonstrated the capability to induce dystrophin expression in Duchenne muscular dystrophy (DMD) patients in phase II-III clinical trials with benefit in muscle functions.
DART Therapeutics Inc., an innovative, new-model biotechnology firm focused on developing therapies for Duchenne muscular dystrophy (DMD), announced today that it has resumed clinical development for its lead drug candidate, HT-100 (delayed-release halofuginone) an orally available, small molecule drug candidate intended to reduce fibrosis and inflammation and promote healthy muscle regeneration in boys with DMD.
PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that following its request for re-examination, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion regarding the company’s application for a conditional marketing authorization of TranslarnaTM (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged five years and older.
SMT C1100 is an oral small molecule utrophin modulator that has the potential to treat all patients with DMD, regardless of the underlying dystrophin fault causing the disease.
The Phase 1b trial was a dose-escalating, open-label study conducted in pediatric patients with DMD to evaluate safety, tolerability and drug exposure.
Santhera Pharmaceuticals (SIX: SANN) announces today that its Phase III DELOS study of orally administered Catena®/Raxone® (INN: idebenone) in patients with Duchenne Muscular Dystrophy (DMD) met the primary endpoint and achieved its primary objective of delaying the loss of respiratory function compared to placebo.
