After receiving Orphan Drug Designation earlier this year, Satellos Bioscience Inc. announced that the FDA has granted Rare Pediatric Disease Designation to SAT-3247 for the potential treatment of Duchenne muscular dystrophy (DMD).
Pepgen Inc. fell slightly short of its phase II dystrophin goal with PGN-EDO51 for patients with Duchenne muscular dystrophy (DMD) whose mutations are amenable to an exon 51-skipping approach, but Wall Street reacted in a big way, sending the Boston-based firm’s stock (NASDAQ:PEPG) down 33%, or $5.55, to close July 31 at $11.43.
The EMA’s Committee for Medicinal Products for Human Use (CHMP) recommended approval of 14 drugs and the extension of the label of 11 others at its July meeting, but, inevitably, it was the decision to turn down the Alzheimer’s disease therapy Leqembi (lecanemab) that stirred the greatest reaction.
Myogenica Inc., a University of Minnesota startup company, has obtained IND approval from the FDA for Myopaxon, an induced pluripotent stem cell (iPSC)-derived muscle stem cell product to regenerate skeletal muscle. A planned study will evaluate intramuscular injections of Myopaxon in non-ambulatory adult patients with Duchenne muscular dystrophy.
Satellos Bioscience Inc. has submitted a clinical research proposal to a Human Research Ethics Committee (HREC) in Australia seeking regulatory authorization under the Therapeutic Goods Administration (TGA)’s clinical trial notification scheme to conduct a first-in-human phase I trial of SAT-3247.
Two drugs were pushed back by the EMA last week, with a recommendation that Ocaliva, currently the only second line standard of care for treating primary biliary cholangitis, be withdrawn from the market, and a refusal to grant conditional approval for masitinib in the treatment of amyotrophic lateral sclerosis.
Sarepta Therapeutics Inc. CEO Douglas Ingram said he expects “ferocious” demand for gene therapy Elevidys (delandistrogene moxeparvovec), granted full approval by the U.S. FDA for Duchenne muscular dystrophy (DMD). Shares of the Cambridge, Mass.-based firm closed June 21 at $16.72, up $37.22, or about 30% on the news.
The good news for Sarepta Therapeutics Inc. is bad news for Pfizer Inc. as the phase III study of its mini-dystrophin gene therapy in Duchenne muscular dystrophy (DMD) has missed its primary endpoint. Now Sarepta’s Elevidys (delandistrogene moxeparvovec), a single-dose, adeno-associated virus-based gene transfer therapy for DMD, is barreling toward a June 21 PDUFA date with the U.S. FDA as the near competition shrinks in the rearview mirror.
The EMA has been sent back to the drawing board to re-evaluate PTC Therapeutics Inc.’s Duchenne muscular dystrophy therapy Translarna (ataluren), after failing to get the usual rubber stamp following its recommendation in January that the drug’s conditional approval be withdrawn.
Since utrophin compensates for lack of dystrophin in mdx mice, which results in a milder phenotype of muscular dystrophy compared to humans, the mdx/utrn-/- mouse has been developed to mimic early onset of muscle dystrophy, severe muscle weakness and premature death.
