Friedreich's Ataxia (FA) is a rare genetic disorder that affects approximately 1 in 50,000 people worldwide. This progressive condition primarily affects the nervous system, leading to symptoms such as difficulty with balance and coordination, muscle weakness, and speech problems. However, there is hope for those living with this debilitating disease, as the FDA has recently approved a new treatment option for FA, SKYCLARYS, the oral, once-daily medication, also known as omaveloxolone.
“The great news about the approval of omaveloxolone is that patients in need have a first therapy to help improve their quality of life,” said Jonathan S. Stamler, MD, President of the Harrington Discovery Institute at University Hospitals Health System. “It reinforces the importance of the work we do to accelerate discoveries into medicines for unmet needs.”
Omaveloxolone is a novel drug that works by targeting a protein known as Keap1, to downregulate the production of oxidants. In patients with FA, the levels of oxidants are elevated, leading to oxidative stress and damage to nerve cells. By targeting Keap1, omaveloxolone can increase the production of antioxidants, slowing down the progression of the disease.
Clinical trials involving over 100 patients with FA found that treatment with omaveloxolone resulted in significant improvements in neurological function, as well as fewer symptoms and better quality of life compared to those who received a placebo. Based on the positive results of these trials, the FDA granted approval for omaveloxolone as a treatment option for FA on Rare Disease Day 2023 (February 28).
The approval of SKYCLARYS by the FDA is a significant milestone in the treatment of FA. However, the impact of this approval extends beyond the patients who will benefit directly from this medication. It is also a beacon of hope for the field of rare diseases and for nonprofits like ours dedicated to advancing breakthrough discoveries.
Harrington Discovery Institute partnered with The University of Oxford to establish The Oxford-Harrington Rare Disease Centre (OHC). The Oxford-Harrington Rare Disease Centre (OHC) brings together the strengths and capabilities of the University of Oxford in the UK and Harrington Discovery Institute in Cleveland, Ohio, US. We are united in our mission to deliver cures for rare diseases.
With the development and approval of SKYCLARYS, Harrington and its partners can be fueled by the optimism that the fight against FA and other rare diseases can be won.