Niall Collins
Ceist:600. Deputy Niall Collins asked the Minister for Health if there are plans to approve skyclarys (omaveloxolone) for Friedreich's ataxia; and if he will make a statement on the matter. [2552/24]
Amharc ar fhreagraTuesday, 23 January 2024
Ceisteanna (600)
Freagraí scríofa
On 14 December 2023, the European Medicine Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Skyclarys 50mg Capsules, intended for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older. The Applicant for this medicinal product is Reata Ireland Limited.
The active substance of Skyclarys is omaveloxolone. Omaveloxolone activates the Nuclear factor (erythroid-derived 2)-like 2 (Nrf2) pathway which is involved in the cellular response to oxidative stress. The precise mechanism by which omaveloxolone exerts its therapeutic effect in patients with Friedreich’s ataxia is unknown, but as Nrf2 activity is reduced in patients with Friedreich's ataxia, Nrf2 activators may be involved.
The benefit of Skyclarys is an improvement in the mFARS (modified FA rating scale) score measuring disease progression, as compared with a placebo control in a randomised clinical trial. The most common side effects are increased ALT and AST, decreased weight and appetite, nausea, vomiting, diarrhoea, headache, fatigue, oropharyngeal and back pain, muscle spasms, and influenza.
Detailed recommendations for the use of this product will be described in the summary of product characteristics (SmPC), which will be published in the European public assessment report (EPAR) and made available on the EMA’s website after the marketing authorisation has been granted by the European Commission. This normally occurs within 2-3 months after a positive CHMP opinion.
It is not known at present if the company has applied for pricing and reimbursement in Ireland or if / when they plan to launch this medicine in Ireland.