Reata Pharmaceuticals, Inc. has received approval from the U.S. Food and Drug Administration (FDA) for SKYCLARYS™ (omaveloxolone) to treat Friedreich’s ataxia (FA) in adults and adolescents aged 16 years and older. This development is crucial for the approximately 5,000 diagnosed patients in the United States with this ultra-rare, inherited neuro-degenerative disorder, commonly diagnosed during adolescence. The FDA’s approval of the first disease-specific treatment option for Friedreich’s ataxia is a significant step forward to advance research and achieve treatments for this debilitating disease.
Reata Battles Friedreich’s Ataxia
Friedreich’s ataxia is an inherited neuromuscular disorder that affects around 5,000 diagnosed patients in the United States. The expansion of a DNA triplet repeat within the frataxin gene causes this disease, which results in a deficiency of the frataxin protein. This protein deficiency leads to the progressive loss of coordination, muscle weakness, and fatigue in patients. The symptoms of Friedreich’s ataxia can vary from person to person, but they generally include difficulty with coordination and balance, speech problems, and heart problems. These symptoms can worsen over time, and as the disease progresses, patients may also experience vision and hearing loss, scoliosis, and diabetes. Over time, this condition can worsen to the point where individuals require a wheelchair in their teens or early twenties, and it may ultimately lead to premature death.
Currently, a cure does not exist for Friedreich’s ataxia, and treatment options are limited. However, with continued research and innovative therapies, such as the new treatment option by Reata Pharmaceuticals, there is hope for improving the quality of life for patients with this debilitating disease. SKYCLARYS is a novel medication developed by Reata that fights Friedreich’s ataxia in adults and adolescents aged 16 years old and older. The FDA granted a rare pediatric disease priority review voucher in its approval. The designation highlights the company’s commitment to developing and commercializing novel therapies for patients with severe diseases with few or no approved treatments.
Warren Huff, Reata’s Chief Executive Officer, expressed gratitude to Friedreich’s ataxia patients, investigators, U.S. regulators, and the company’s scientists and employees who made this approval possible. He also highlighted that this is a transformative milestone that underscores the company’s commitment to patients. Huff looks forward to delivering SKYCLARYS to eligible patients as quickly as possible. The approval of SKYCLARYS was welcomed by the entire Friedreich’s ataxia community, including patients, clinicians, scientists, pharmaceutical companies, government agencies, and others who have worked collaboratively for decades to enable therapeutic development for this debilitating disease. Susan Perlman, MD, Clinical Professor, Department of Neurology, David Geffen School of Medicine, UCLA, acknowledged that the approval of SKYCLARYS represents a critical step forward in the treatment of Friedreich’s ataxia, providing physicians with the first disease-specific treatment option approved for patients living with this ultra-rare and progressive disease.
Friedreich’s ataxia is a devastating disease affecting thousands of people in the United States and the world. With continued research and investment in innovative therapies, such as the new treatment by Reata, we can work towards a future where Friedreich’s ataxia is no longer a life-limiting condition. It is essential to support organizations and initiatives that promote awareness and funding for this disease and advocate for policies that ensure access to treatments for patients and their families. Together, we can make a difference and improve the lives of those affected by Friedreich’s ataxia. To learn more about tips for getting active, click here.
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