News

CDA recommends coverage for Skyclarys, but only at a lower cost

A committee of Canada’s Drug Agency (CDA) has recommended that insurers should cover the cost of Skyclarys (omaveloxolone) for Friedreich’s ataxia (FA), but only under certain conditions, including a substantial reduction in the drug’s cost. “The CDA recommendation represents an important step toward changing that reality by…

Research links genetic ‘spelling errors’ to FA disease severity

Genetic mutations causing Friedreich’s ataxia (FA) and influencing disease severity may be more complex than previously understood, according to research from the University of Oklahoma. Sanjay Bidichandani, PhD, genetics section chief at the university’s medical college, used a new genetic sequencing technique to find “spelling errors” in the FXN gene…

LX2006 designated breakthrough therapy for FA cardiomyopathy

The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to LX2006, a gene therapy for cardiomyopathy in Friedreich’s Ataxia (FA). The status means the Lexeo Therapeutics candidate may be eligible for fast track review and extra FDA guidance during the regulatory process, which could…

MDA, FARA fund grant to study heart fibrosis in FA

A newly awarded $300,000 research grant will fund a study on heart fibrosis, or the accumulation of scar tissue, in people with Friedreich’s ataxia (FA). The Muscular Dystrophy Association (MDA) and Friedreich’s Ataxia Research Alliance (FARA) awarded the grant to researchers at Weill Cornell Medical Center and Indiana…

FDA provides path for accelerated approval of nomlabofusp for FA

The U.S. Food and Drug Administration (FDA) has provided Larimar Therapeutics with clear expectations for an application seeking accelerated approval of nomlabofusp, its potentially disease-modifying therapy for Friedreich’s ataxia (FA), according to the drug developer. Accelerated approval is a type of conditional approval based on early clinical…

Gray matter volume in cerebellum predicts therapy efficacy

The volume of gray matter in the brain region known as the cerebellum helps predict motor and cognitive benefits for Friedreich’s ataxia patients after noninvasive neuromodulation, a therapy that delivers mild electric currents to brain regions, a study found. Gray matter contains a high concentration of nerve cell bodies…

Heart function OK with high dose of Skyclarys in study

A high dose of the Friedreich’s ataxia (FA) treatment Skyclarys (omaveloxolone), far above the approved dose to match blood levels when the therapy is taken with food, did not affect heart function in healthy individuals, a study confirms. The study, “Effect of a Supratherapeutic Dose…

Friedreich’s ataxia mutations disrupted with gene editing

A new gene editing technique disrupted the mutations that cause Friedreich’s ataxia (FA) in a mouse model, a study reports. A type of base editing, the technique swaps one genetic building block, or nucleotide, for another. These edits can interrupt long repetitions of three-letter DNA sequences (trinucleotide repeats, or…