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.
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
-
The new marketing authorisation was granted on 23 April to
Biogen Netherlands B.V.
-
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.
