Currently available disease management options for Duchenne muscular dystrophy (DMD) are mostly symptomatic. Several strategies based on exon-skipping or gene transfer have been proposed to restore dystrophin expression, but can only address specific subsets of DMD patients and/or provide limited clinical benefits. Upregulating utrophin (UTRN), a structural and functional paralogue of dystrophin, has been proposed as an alternative therapeutic approach that may be suitable for all DMD patients, regardless of their genetic defect.
The strife-marked Duchenne muscular dystrophy (DMD) space drew forth another outspoken political figure in the shape of Sen. Ron Johnson (R-Wisc.), who said he was “enraged” by the U.S. FDA’s refusal to consider PTC Therapeutics Inc.’s Translarna (ataluren) for the treatment of nonsense mutation disease.
At the Breakthroughs in Muscular Dystrophy special meeting held in Chicago Nov. 19-20, 2024, and organized by the American Society of Gene & Cell Therapy (ASGCT), multiple interventions at the RNA level were among the approaches that were presented to fight muscular dystrophies.
Since the isolation of the gene that causes Duchenne muscular dystrophy (DMD), scientists have progressed in understanding the mechanisms that lead to muscular diseases that can be evident from the early stages of childhood. This has led to the development of diagnostics and therapeutics, some approved by the FDA.
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.
Beijing- and Shanghai-based Sperogenix Therapeutics Ltd. said that China’s regulatory agency accepted the NDA filing and granted priority review of Agamree (vamorolone) for Duchenne muscular dystrophy on March 26.
Beijing- and Shanghai-based Sperogenix Therapeutics Ltd. said that China’s regulatory agency accepted the NDA filing and granted priority review of Agamree (vamorolone) for Duchenne muscular dystrophy on March 26.
The long-term use of corticosteroids for treating Duchenne muscular dystrophy (DMD) is tied to several undesired effects, compromising the patient’s quality of life; hence, the use of nonsteroidal drugs is highly desirable in the treatment of DMD. Metriopharm AG is investigating the nonsteroidal drug MP-1032 for DMD and recently presented data from studies in a model of DMD.
Failing to meet the primary endpoint in its confirmatory phase III Embark trial, Sarepta Therapeutics Inc.’s gene therapy, Elevidys (delandistrogene moxeparvovec), which received accelerated approval in June and was priced at $3.2 million, has one of three fates in its future, all of which are dependent on how the U.S. FDA perceives the data. Based on secondary endpoints showing statistical significance and a recent positive meeting with the agency, Sarepta could continue to market Elevidys under its current label for 4- and 5-year-old ambulatory Duchenne muscular dystrophy (DMD) patients; Sarepta is filing the postmarketing requirement needed to transition from accelerated to full approval.
Less than two weeks after getting a thumbs-up from the EMA’s Committee for Medicinal Products for Human Use, Santhera Pharmaceutical AG’s vamorolone secured U.S. FDA approval for use in patients, 2 and older, with Duchenne muscular dystrophy (DMD). A first-in-class drug, vamorolone, branded Agamree, is expected to offer a safer alternative to the steroid therapy, which the company has said will remain a foundational treatment of DMD, even with the introduction of gene therapies.
