FDA Accepts NS Pharma’s NDA for Viltolarsen for the Treatment of DMD Amenable to Exon 53 Skipping

On Feb. 7, NS Pharma Inc. (a wholly owned US subsidiary of Nippon Shinyaku Co. Ltd.), announced that the US Food and Drug Administration (FDA) accepted its New Drug Application (NDA) seeking approval under Priority Review for viltolarsen, an investigational therapy for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 53. Priority . . .

Read More

Clinical Trial Alert: LGMD2I Participants Sought for a Phase 3 Study

Researchers at PTC Therapeutics are looking for individuals with limb-girdle muscular dystrophy type 2I (LGMD2I) to participate in a phase 3 study. The goal of the study is to evaluate the efficacy of deflazacort (Emflaza), which researchers hope may reduce inflammation in muscles and potentially lead to improved muscle strength and motor function. Participants will . . .

Read More

Clinical Trial Alert: Mitsubishi Tanabe Pharma Seeks Participants for a Phase 3 ALS Study

Researchers at Mitsubishi Tanabe Pharma Development America are looking for participants with amyotrophic lateral sclerosis (ALS) to participate in a phase 3 study to evaluate the safety of oral edaravone (Radicava). Intravenous edaravone is an FDA-approved treatment for ALS shown to slow the decline of physical function. Edaravone is thought to act as a free . . .

Read More

Antisense Therapeutics Announces Positive Results from Phase 2 Study of ATL1102 in DMD

Australian company Antisense Therapeutics announced encouraging results from its phase 2, open-label clinical trial of the company’s investigational therapy ATL1102 for treating Duchenne muscular dystrophy (DMD). The study met its primary endpoint, which was to assess the safety and tolerability of ATL1102 in patients with DMD who are non-ambulatory (unable to walk independently). After six . . .

Read More

MDA Awards More Than $1 Million in Venture Philanthropy Funding to AavantiBio to Develop Gene-Targeting Therapy for Friedreich’s Ataxia

AavantiBio, a biotechnology company developing a gene-targeting therapy for Friedreich’s ataxia (FA), was awarded MDA Venture Philanthropy (MVP) funding totaling $1,076,232 to advance AavantiBio’s phase 2 clinical trial of a gene-replacement therapy for the disease. MVP is MDA’s drug development program, which is exclusively focused on funding the discovery and clinical application of treatments and . . .

Read More

FDA Approves ITF Pharma’s Tiglutik via PEG Tube for the Treatment of ALS

On Dec. 13, the US Food and Drug Administration (FDA) approved expanded labeling for ITF Pharma’s Tiglutik to include administration via percutaneous endoscopic gastrostomy (PEG) tube for the treatment of amyotrophic lateral sclerosis (ALS). The expanded approval for use with PEG feeding tubes will enable patients who not only have difficulty swallowing but also require . . .

Read More