Drug Therapy

Avidity Biosciences Inc. has announced positive data from the phase 1/2 FORTITUDE clinical trial of a proprietary monoclonal antibody that binds to the transferrin receptor 1 conjugated with a siRNA targeting DUX4 mRNA.

Wave Life Sciences has announced positive results from its phase 1b/2a clinical trial of WVE-003, a potential disease-modifying therapeutic for Huntington’s disease.

The European Commission has authorized a subcutaneous injection of Ocrevus to treat relapsing multiple sclerosis as well as primary progressive MS, according to the manufacturer.

The FDA has approved Vyvgart Hytrulo in a once-weekly, 30- to 90-second subcutaneous injection to treat adults with chronic inflammatory demyelinating polyneuropathy, according to the manufacturer.

SAN DIEGO — A novel, oral therapeutic for treatment of migraine was safe and well-tolerated in single ascending doses in a cohort of healthy adults, according to a speaker at the American Headache Society Annual Scientific Meeting.

Newly released data from the open-label extension of the FIREFISH study confirmed sustained safety and efficacy of Evrysdi in children with type 1 spinal muscular atrophy over 5 years.

Annexon Biosciences has announced positive topline results from a phase 3, randomized placebo-controlled clinical trial examining an investigational monoclonal antibody in those with Guillain-Barré syndrome.

Takeda has reported positive results from a phase 2b clinical trial of an investigational, oral orexin receptor agonist for narcolepsy type 1.

The European Commission has granted marketing authorization under exceptional circumstances for Qalsody to treat those with amyotrophic lateral sclerosis associated with a mutation in the superoxide dismutase 1 gene.

The FDA has approved Austedo XR in four new tablet strengths for adults diagnosed with tardive dyskinesia and chorea associated with Huntington’s disease, according to the manufacturer.