Hope for Friedreich's ataxia patients

• Friedreich's ataxia is a genetic, debilitating and life-shortening neuromuscular disease
• Biogen is expanding its drug portfolio for rare neuromuscular diseases for patients who have so far been left without any treatment.

Friedreich's Ataxia (FA) is a rare genetic neuromuscular disease that shortens life and devastates the patient's body. It is the most common form of hereditary ataxia. Early symptoms of FA, such as progressive loss of coordination, muscle weakness and fatigue, usually appear in childhood and may initially suggest other diseases. Most people with FA will need to start using a wheelchair within 10-20 years of the first symptoms. The average reported age of death for patients with FA is only 37, although with appropriate and targeted care, these individuals can live many years using a wheelchair.

Biogen announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended marketing authorization for the drug omaweloxolone for the treatment of Friedreich's ataxia (FA) for use in people 16 years of age and older. If approved by the European Commission (EC), omaweloxolone will be the first drug approved in the European Union for this rare, genetic, progressive neuromuscular disease.

"The CHMP's recommendation of omaweloxolone is a significant milestone in our goal of making a drug that slows the progression of Friedreich's ataxia available to patients," - said Dr. Priya Singhal, Chief Development Officer at Biogen. "We are ready to commit all of Biogen's expertise and capabilities in rare diseases to ensure that once the drug omaweloxolone is approved, patients with Friedreich's ataxia will have access to this breakthrough therapy in the European Union."

The CHMP's positive opinion for omaweloxolone is based on efficacy and safety data from the placebo-controlled portion of the MOXIe study. At the end of the 48-week portion of the MOXIe study, patients receiving omaweloxolone showed less physical disability, as measured by the modified Friedreich`s Ataxia Rating Scale (mFARS), compared to patients receiving placebo.
Patients treated with omeloxolone, compared to placebo, also showed improvements in subscales of the mFARS, including upright stability, improved lower limb coordination, swallowing ability and upper limb coordination. Additional data supporting these observations were collected from a post hoc analysis using Propensity Score Matching (PSM), in which patients treated with omeloxolone in MOXIe (Parts 1 and 2) achieved a lower mFARS score (less physical disability) after 3 years, compared to an adjusted control group with a natural history of the disease.

The CHMP recommendation for omaweloxolone will now be evaluated by the European Commission for marketing authorization in the European Union, with a final decision expected in the first quarter of 2024. The U.S. Food and Drug Administration (FDA) approved omaweloxolone, for the treatment of Friedreich's ataxia in adults and adolescents aged 16 and older, in February 2023.

Omaveloxolone is an oral, once-daily medication indicated for the treatment of Friedreich's ataxia (FA), for use in adults and adolescents 16 years of age and older in the United States. Omaveloxolone has been granted orphan drug status by the U.S. Food and Drug Administration (FDA) under the fast track approval program as a therapeutic option for the treatment of the rare disease. Omaveloxolone has been granted orphan drug status in Europe for the treatment of FA by the European Commission.

Source: Press Release