The Saudi Food and Drug Authority (SFDA) has approved the registration of Skyclarys (Omaveloxolone), which was previously designated as an Orphan Drug under the SFDA Orphan Drug Program. The medication is indicated for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older, a rare inherited neurodegenerative disorder that impairs movement and significantly affects patients' quality of life.

Innovative Mechanism of Action

Omaveloxolone functions by activating the Nuclear Factor Erythroid 2-Related Factor 2 (Nrf2), a key protein involved in regulating the cellular response to oxidative stress. In patients with Friedreich’s ataxia, Nrf2 levels and activity are typically suppressed, contributing to disease progression. Activation of Nrf2 enhances antioxidant responses and contributes to neuroprotection by reducing oxidative stress.

 Significant Positive Results

The SFDA confirmed that the approval of Skyclarys followed the evaluation of its efficacy and safety in accordance with applicable regulatory standards. Clinical studies conducted on the drug demonstrated significant positive results in improving patients' conditions. The primary efficacy study showed a reduction in modified Friedreich’s Ataxia Rating Scale (mFARS) scores among patients treated with the medication compared to those who received a placebo after 48 weeks of use, confirming the drug’s effectiveness in slowing disease progression. Long-term data indicate that the positive effects of the treatment persisted for up to three years compared to the expected natural decline of the disease, supporting the ongoing clinical benefits of the medication.

 Side Effects and Interactions

Clinical studies have shown that the medication may cause side effects; the most commonly observed were elevated liver enzyme levels in the blood, headache, back pain, nausea and vomiting, loss of appetite, and weight loss. The studies also revealed interactions with grapefruit juice and with medications that affect the CYP3A4 enzyme.

 SFDA Orphan Drug Program Accelerates Access to Critical Therapies  

The SFDA is committed to facilitating access to effective therapies, particularly for rare and hard-to-treat diseases, which often lack sufficient treatment options. In line with this commitment, the SFDA launched the Orphan Drug Program, which serves as a strategic pillar to promote pharmaceutical innovation and address unmet medical needs, supporting the objectives of Saudi Vision 2030 to enhance the quality of healthcare. An orphan drug is defined as a medication intended to treat a rare disease or condition affecting fewer than 5 individuals per 10,000 people in Saudi Arabia. This program offers several benefits to companies seeking to register such treatments, including pre-submission meetings, priority review for product registration, and scientific consultations provided by the Drug Sector.

 For further information, please refer to the Orphan Drugs Guideline at: (https://www.sfda.gov.sa/ar/regulations/88482) or contact the SFDA via email at Designation.Drug@sfda.gov.sa.