Omaveloxolone

People with Friedreich's ataxia face debilitating symptoms, too few medical specialists and too many health insurance barriers, physicians explain in new policy paper WASHINGTON, Feb. 5, 2025 /PRNewswire/ -- An FDA-approved medication can now treat Friedreich's ataxia, but health plan barriers and formulary exclusions keep many patients from receiving it. In a white paper by the Alliance for Patient Access and the Friedreich's Ataxia Research Alliance, titled "Friedreich's Ataxia: A Rare Condition Facing Common Access Challenges," physician authors outline the disease's debilitating symptoms as well as the treatment challenges that accompany it. The rare genetic condition often begins in childhood and affects the brain, heart and neurological system. Continue Reading Alliance for Patient Access logo. (PRNewsfoto/Alliance for Patient Access) After years with no dedicated treatment option, the FDA approved omaveloxolone in 2023. Hailed as "an exciting step forward for the FA community," the medication remains out of reach for many patients. Some health plans have excluded the new drug from their formulary. Others cover it but only with onerous prior authorization and step therapy protocols, which delay care for patients already struggling with the disease's symptoms. These barriers compound existing challenges. Finding a specialist or clinician who can diagnose Friedreich's ataxia, given its rarity, remains difficult. Clinician knowledge gaps and transportation challenges often mean delayed diagnosis or misdiagnosis. "Due to the progressive nature of Friedreich's ataxia, people living with the condition cannot afford delays in access to knowledgeable healthcare professionals or treatment," says Kellyn Madden, patient engagement manager for the Friedreich's Ataxia Research Alliance. "The FA community has partnered for many years with researchers to bring treatments forward. This white paper marks a significant step in raising awareness and advocating for policies that facilitate access to this care." The white paper proposes several potential policy solutions, including: Limits on health plan utilization management Expanded coverage for telehealth Support for innovation and more opportunities for children to participate in clinical trials STATEMENT FROM JOSIE COOPER, EXECUTIVE DIRECTOR OF THE ALLIANCE FOR PATIENT ACCESS: "Patients with Friedreich's ataxia deserve access to innovative treatments and patient-centered care. With health plan barriers negatively impacting these patients' physical and mental health, policy change is critical." Learn more in "Friedreich's Ataxia: The Rare Condition Facing Common Access Challenges." About the Alliance for Patient Access The Alliance for Patient Access is a network of policy-minded health care providers who advocate for patient-centered care. About the Friedreich's Ataxia Research Alliance The Friedreich's Ataxia Research Alliance (FARA) is a national, public, 501(c)(3), nonprofit, organization dedicated to the pursuit of scientific research leading to treatments and a cure for Friedreich's ataxia. SOURCE Alliance for Patient Access WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Friedreich’s ataxia is a rare, debilitating and life-shortening neuromuscular disease caused by genetic factors 1 If approved, omaveloxolone would be the only disease-specific treatment option for patients living with this progressive and challenging disease This New Drug Submission reflects Biogen’s commitment to advancing treatments for rare diseases and serving the unmet needs of Canadian patients TORONTO , Sept. 17, 2024 /CNW/ - Biogen Canada Inc. (Biogen) is pleased to announce today that Health Canada has accepted for priority review its New Drug Submission (NDS) for omaveloxolone, an innovative therapy for Friedreich’s ataxia for the treatment of adults and adolescents aged 16 years and older. This once-daily oral medication, if approved, would represent the only therapy specifically targeted for Friedreich’s ataxia, addressing a critical unmet treatment need in this patient population. “Friedreich’s ataxia is a devastating diagnosis that profoundly impacts the lives of patients and their families,” said Eric Tse , General Manager at Biogen Canada. “The omaveloxolone NDS acceptance marks a significant step forward for Canadian patients, offering hope and potential for a therapy that could help manage this debilitating neurodegenerative disorder. We look forward to working with Canadian health authorities to bring forward this promising treatment for those in need.” Friedreich’s ataxia is a rare, inherited condition that affects about 1 in 40,000 people and has a profound impact on their lives, causing a slow, progressive loss of muscle coordination, slurred speech, weakness, and sensory loss. 2 Symptoms typically begin in childhood or adolescence, and while the progression varies, it often leads to significant disability over time 2 with individuals becoming wheelchair-bound 8 to 10 years after disease onset. 1 The NDS for omaveloxolone is supported by efficacy and safety data from the placebo-controlled MOXIe Part 2 trial, which indicated that omaveloxolone significantly improved modified Friedreich Ataxia Rating Scale (mFARS) scores at 48 weeks relative to placebo. Omaveloxolone received Orphan Drug, Fast Track, and Rare Pediatric Disease Designations from the U.S. Food and Drug Administration, as well as Orphan Drug status by the European Commission. 3,4 It has received regulatory approval for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older in both the United States and the European Union, with strong support from the medical and patient communities. 3,4 In Canada , priority review submissions follow an accelerated timeline where the standard review period is shortened to 180 days. 5 A regulatory decision on the New Drug Submission for omaveloxolone is anticipated in early 2025. About Biogen Founded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patient lives and to create value for shareholders and our communities. We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, balanced with return on investment, to deliver long-term growth. Biogen has been proudly serving Canadian patients for more than 25 years. For information about Biogen Canada, please visit . References: Ataxia Canada . Friedreich’s Ataxia . Available at: Accessed September 2024 . AboutKidsHealth. Friedreich ataxia . Jan. 2020 . Available at: . Accessed September 2024 . U.S. Food and Drug Administration. (2023, February 28 ) FDA approves first treatment for Friedreich’s ataxia [press release] Accessed September 2024 . Biogen Inc. (2024, February 12 ) Biogen Received European Commission Approval for SKYCLARYS® (omaveloxolone), the First Therapy to Treat Friedreich’s Ataxia [press release] Accessed September 2024 . Health Canada . Priority Review of Drug Submissions (Therapeutic Products) Available at: Accessed September 2024 . SOURCE Biogen Canada Inc.