The Medicines and Healthcare products Regulatory Agency (MHRA) has today, 23 April, approved omaveloxolone (Skyclarys), the first treatment for patients aged 16 and over, in the UK for a rare neurodegenerative movement disorder called Friedreich’s ataxia.
Friedreich’s ataxia is the most common type of hereditary ataxia (caused by genes a person has inherited). It is thought to affect at least 1 in every 50,000 people. Symptoms of Friedreich’s ataxia include problems with balance and movement, which get progressively worse over time.
This medicine is taken as an oral capsule.
Julian Beach, MHRA Interim Executive Director of Healthcare Quality and Access, said:
“Patient safety is our top priority. I am pleased to confirm the first UK approval for the treatment of Friedreich’s ataxia, omaveloxolone.
“We’re assured that the appropriate regulatory standards of safety, quality and effectiveness for the approval of this new treatment have been met.
“As with all products, we will keep its safety under close review.”
People with Friedreich’s ataxia appear to have reduced levels and activity of a protein called Nrf2, which helps cells respond to oxidative stress (a condition that may occur when there are too many free radicals in the body and not enough antioxidants to get rid of them, which can lead to cell and tissue damage). Omaveloxolone activates the Nrf2 pathway.
In a clinical trial, patients treated with omaveloxolone scored better on tests of neurological function than patients who were treated with a placebo (a dummy treatment).
In this clinical trial, involving 103 patients with Friedreich’s ataxia aged between 16 and 40 years, omaveloxolone was more effective than placebo at reducing physical impairment after 48 weeks of treatment.
For the full list of all side effects reported with this medicine, see Section 4 of the Patient Information Leaflet or the Summary of Product Characteristics (SmPC) available on the MHRA’s website.
Anyone who suspects they are having a side effect from this medicine should talk to their doctor, pharmacist or nurse and report it directly to the MHRA Yellow Card scheme, either through the website (https://yellowcard.mhra.gov.uk/) or by searching the Google Play or Apple App stores for MHRA Yellow Card.
Notes to editors
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The new marketing authorisation was granted on 23 April to Biogen Netherlands B.V.
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This product was submitted and approved via a national procedure.
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More information can be found in the Summary of Product Characteristics and Patient Information leaflets which will be published on the MHRA Products website within 7 days of approval.
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The Medicines and Healthcare products Regulatory Agency (MHRA) is responsible for regulating all medicines and medical devices in the UK by ensuring they work and are acceptably safe. All our work is underpinned by robust and fact-based judgements to ensure that the benefits justify any risks.
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The MHRA is an executive agency of the Department of Health and Social Care.
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