On Jan. 2, the U.S. Food and Drug Administration (FDA) awarded Orphan Drug Designation for MYO-102, an investigational gene therapy for limb-girdle muscular dystrophy type 2D (LGMD2D), also known as alpha-sarcoglycanopathy. Myonexus Therapeutics has licensed the technology from Nationwide Children’s Hospital, which holds the Investigational New Drug (IND) application for MYO-102. Treatment using gene therapy, . . .
We were saddened to end 2018 with the passing of one of the true champions of neuromuscular disease research, Dr. Stephen Katz, the long-time director of the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS). Dr. Katz led efforts throughout the National Institutes of Health (NIH) to implement the Muscular Dystrophy Community Assistance . . .
In January 2018, MDA partnered with Edge Research, an established research firm with extensive expertise working with patient advocacy organizations, to conduct an objective and thorough study among the neuromuscular disease community. This survey, a first-of-its-kind for MDA, was called ONEVoice and comprised 50 questions that sought to identify the needs of individuals and families . . .
On Dec. 10, Ra Pharmaceuticals Inc., based in Cambridge, Mass., announced positive results from a Phase 2 clinical trial designed to evaluate zilucoplan (RA101495) for treating generalized myasthenia gravis (gMG). Patients who received the drug had significant reductions in both measured endpoints, the Quantitative Myasthenia Gravis (QMG) score and the Myasthenia Gravis — Activities of . . .
On Dec. 6, Biogen Inc. announced positive Phase 1 clinical trial results and its decision to license and develop partner Ionis Pharmaceuticals’ BIIB067 (IONIS-SOD1RX). BIIB067 is an investigational therapy for treating amyotrophic lateral sclerosis (ALS) caused by superoxide dismutase 1 (SOD1) mutations, a subtype of familial ALS that makes up 2 percent of all ALS . . .
Today, Wave Life Sciences announced that its Phase 1 trial of WVE-210201, an investigational, exon skipping therapy for boys with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping, showed positive safety and tolerability data. Based on these results, Wave announced that it will initiate a Phase 2/3 clinical trial of WVE-210201 in 2019. Wave . . .
Today, Novartis, the parent company of AveXis Inc., announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) under Priority Review for Zolgensma (formerly known as AVXS-101), a gene therapy for the treatment of spinal muscular atrophy (SMA) type 1. Priority Review status requires the FDA to review . . .
On Nov. 26, Biohaven Pharmaceuticals announced that the Food and Drug Administration (FDA) has accepted its 505(b)(2) New Drug Application (NDA) to review BHV-0223, a sublingual (placed under the tongue) formulation of riluzole for the treatment of patients living with amyotrophic lateral sclerosis (ALS). In submitting its NDA using the 505(b)(2) pathway, Biohaven was able . . .
On Nov. 28, the U.S. Food and Drug Administration (FDA) announced the approval of Firdapse (amifampridine phosphate) for the treatment of adults with Lambert-Eaton myasthenic syndrome (LEMS). Firdapse, developed by Catalyst Pharmaceuticals, is an oral potassium channel inhibitor designed to prolong signals released from nerves and allow greater stimulation of muscles. This is the eighth . . .
Stealth BioTherapeutics is looking for participants with primary mitochondrial myopathy (PMM) to participate in the MMPOWER-3 Phase 3 trial. This trial is designed to evaluate the efficacy and safety of daily subcutaneous injections of elamipretide, an investigational drug that associates with cardiolipin, a key structural component of the inner mitochondrial membrane, and has shown to . . .