Sarepta Therapeutics Announces Positive Interim Results from Study of Casimersen to Treat DMD Amenable to Exon 45 Skipping

On Mar. 28, Sarepta Therapeutics announced encouraging interim results from a phase 3 clinical trial that suggest the experimental drug casimersen potentially may be effective as a treatment for Duchenne muscular dystrophy (DMD). Earlier this year, the U.S. Food and Drug Administration (FDA) accepted Sarepta Therapeutics’ New Drug Application (NDA) seeking approval under Priority Review for . . .

Read More

FDA Grants Orphan Drug Designation to Acceleron Pharma’s ACE-083 Muscle Growth Drug for Charcot-Marie-Tooth Disease

On Mar. 5, the U.S. Food and Drug Administration (FDA) awarded Orphan Drug designation to Acceleron Pharma’s ACE-083, a locally acting muscle agent, for treating Charcot-Marie-Tooth disease (CMT). Delivered by intramuscular injection, ACE-083 is based on the naturally occurring protein follistatin and is designed to enhance the body’s own promoters of muscle growth, specifically in . . .

Read More

Sarepta Therapeutics Announces Positive Interim Results in Gene Therapy Trial for LGMD2E and Acquisition of Myonexus Therapeutics

On Feb. 27, Sarepta Therapeutics announced positive interim results of a phase 1/2a clinical trial for MYO-101, a gene therapy candidate developed by Myonexus Therapeutics for patients living with limb-girdle muscular dystrophy type 2E (LGMD2E), also known as beta-sarcoglycanopathy. The first three patients dosed demonstrated significant expression of the protein beta-sarcoglycan in the muscle fiber, . . .

Read More

FDA Grants Breakthrough Therapy Designation to Amicus’ Experimental Therapy, AT-GAA, for Late-Onset Pompe Disease

On Feb. 25, Amicus Therapeutics announced that the U.S. Food and Drug Administration (FDA) granted breakthrough therapy designation (BTD) to AT-GAA, Amicus’ investigational combination drug therapy ATB200/AT2221 for treating late-onset Pompe disease. The company’s combination drug therapy pairs ATB200, a synthetic human acid alpha-glucosidase enzyme meant to replace the missing enzyme in Pompe disease, with pharmacological chaperone AT2221, . . .

Read More

FDA Accepts Sarepta Therapeutics’ New Drug Application (NDA) for Golodirsen to Treat DMD Amenable to Exon 53 Skipping

On Feb. 14, Sarepta Therapeutics announced that the U.S. Food and Drug Administration (FDA) accepted its New Drug Application (NDA) seeking approval under Priority Review for golodirsen (SRP-4053), its drug candidate for treating Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 53. Priority Review status requires the FDA to review the application and . . .

Read More

CMTA and MDA Co-Fund Proof-of-Concept Study Using AAV Vector to Deliver Gene Replacement Therapy in X-linked Charcot-Marie-Tooth Disease

On Mar. 1, the Charcot-Marie-Tooth Association (CMTA) and the Muscular Dystrophy Association (MDA) announced that they have jointly awarded a research grant totaling $276,430 over three years to Kleopas Kleopa, M.D., professor and senior consulting neurologist at the Cyprus Institute of Neurology and Genetics, Cyprus School of Molecular Medicine, in Nicosia, Cyprus. Dr. Kleopa is . . .

Read More

Catalyst Pharmaceuticals Announces Positive Results from Phase 2b Trial of Firdapse in Patients with Muscle-Specific Kinase Antibody Positive Myasthenia Gravis (MuSK-MG)

On Jan. 4, Italian researchers published positive results in the journal SAGE Open Medicine of a Phase 2b clinical trial (MSK-001) evaluating Firdapse (amifampridine phosphate) for treating MuSK antibody positive myasthenia gravis (MuSK-MG). Their results showed the drug to be safe and effective in treating patients with this rare disease. The study was partially funded . . .

Read More

FDA Awards Fast Track Status to RNS60, Revalesio’s Investigational Therapy for ALS

On Jan. 4, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to Tacoma, Wash.-based Revalesio Corporation’s experimental drug for amyotrophic lateral sclerosis (ALS), RNS60.   Fast Track status helps to shorten the time it takes to develop and approve a drug that’s been designed to treat a serious condition and fill an unmet . . .

Read More