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First drug receives FDA approval for ultra rare neurodegenerative disorder

6th March 2023

Omaveloxolone is the first drug to receive FDA approval for the progressive genetic neurodegenerative disorder, Friedreich’s ataxia

The manufacturer Reata has announced that its drug omaveloxolone (brand name SKYCLARYS) has become the first treatment to be approved for the treatment of Friedreich’s ataxia, an ultra-rare, genetic, progressive neurological disorder.

Friedreich’s ataxia represents an inherited autosomal recessive disease, that affects the nervous system, producing a progressive ataxia, weakness and sensory deficits. The disease has a median onset age of 10 to 15 years and affects approximately 1 in 50,000 people worldwide. Friedreich’s ataxia is due to a lack of function in a gene responsible for the production of frataxin, a protein involved in mitochondrial iron homeostasis.

During conditions of cellular oxidative stress, there is activation of the transcription factor, NF-E2-related factor and which triggers a cellular antioxidant response, through induction of antioxidant response element driven genes. However, it appears that a lack of frataxin impairs NF-E2 signalling and this is though a contributory factor for the neuro-degeneration seen in Friedreich’s ataxia. Omaveloxolone has been shown to induce NF-E2 and thus protect cells from oxidative stress.

Omaveloxolone clinical efficacy

An initial dosing ranging study identified that a dose of 160 mg/day appeared to improve neurological function. Following on from this, a phase 2 randomised, placebo-controlled trial with 103 patients, demonstrated that omaveloxolone 150 mg daily, significantly improved the modified Friedreich’s Ataxia Rating Scale (mFARS) score after 48 weeks compared to placebo (p = 0.014). In an open label extension trial, after 72 weeks, the response to omaveloxolone compared to placebo was maintained.

Following the FDA approval, the company has created the Reata Education, Access, and Care Helpline (REACH), an integrated specialty pharmacy and patient services program, designed to help eligible patients access prescribed Reata medicines. 

While yet to be approved, omaveloxolone has been granted Orphan Drug designation in Europe for the treatment of Friedreich’s ataxia and is currently under review by the European Medicine Agency.