Just in time for Rare Disease Day, patients with a rare neuromuscular disorder have been given a life-changing gift: the FDA has approved the first-ever medicine specifically designed to treat their condition. Omaveloxolone, developed by Reata Pharmaceuticals, is a major breakthrough in the fight against rare diseases and brings hope to those living with the disorder.
After two weeks of anticipation and volatile market activity, Reata Pharmaceuticals emerged the victor as the FDA granted the approval of omaveloxolone (marketed as Skyclarys) to treat Friedreich’s ataxia in individuals aged 16 and older. This triumphant moment came nearly two hours after the market closed on Tuesday, thus bringing hope to those affected by this rare disorder.
Reata Pharmaceuticals made history with their approval, as they were awarded a Rare Pediatric Disease Priority Review voucher – a first of its kind. This milestone accomplishment means that the company can access expedited review for their future drug applications.
Warren Huff, the CEO of Texas-based company, declared the approval of their milestone as “transformative” in a statement released today. This approval marks a significant step forward for the company and their long-term goals.
Reata Pharmaceuticals is thrilled to announce the launch of Skyclarys, the first FDA-approved treatment for patients who suffer from spinal muscular atrophy (SMA). According to Reata CEO Warren Huff, the company is committed to making Skyclarys available to eligible patients with urgency and efficiency.
Approval of a new treatment option for those suffering from the ultra-rare Friedreich’s ataxia is a major breakthrough, according to Susan Perlman, M.D, clinical professor at UCLA’s David Geffen School of Medicine. This new development marks the first disease-specific treatment for the condition, providing hope and potential relief to those affected.
Named after the German physician Nikolaus Friedreich, who first discovered it in the 1860s, Friedreich’s Ataxia (FA) is a debilitating neurological disorder caused by a trinucleotide repeat expansion in the first intron of the frataxin (FXN) gene. This expansion inhibits the production of the mitochondrial protein frataxin, leading to an overload of iron in the mitochondria and a range of symptoms, including muscle weakness, cardiomyopathy, and neuropathy.
Friedreich’s Ataxia (FA) is an incurable, progressive neurological disorder that affects approximately 5,000 people in the United States. Symptoms of the condition, such as unsteady posture, difficulty walking and scoliosis, typically begin in childhood and can lead to cardiomyopathy, ultimately leading to an early death in most patients by their mid-30s. Despite the grim prognosis, advances in medical care have improved quality of life for those afflicted with this disorder.
Skyclarys jumpstarts the body’s natural healing process with its activation of Nrf2 – a powerful transcription factor that helps to combat inflammation. By restoring mitochondrial function, reducing oxidative stress, and blocking pro-inflammatory signals, Nrf2 helps the body to recover and gain balance.
The anticipation ahead of Tuesday’s decision left Reata shareholders on edge. With bated breath, they awaited the final verdict, hoping for a positive outcome.
Investors were sent into a panic on Monday as news of the sudden departure of FDA neuroscience head Billy Dunn sent the Texas-based biotech’s stock into a tailspin. Speculation arose that the unexpected exit could jeopardize the chances of a much anticipated approval.
In a note released Monday, RBC Capital Markets praised the flexibility of Dr. Dunn when it comes to approving drugs for high unmet need conditions, even with limited or mixed data. Goldman Sachs was even more enthusiastic in their assessment, citing Teresa Buracchio, now acting director of the Office of Neuroscience, as sharing the same viewpoint as Dr. Dunn.
Just a week before, the market swung in the opposite direction, with Reata’s stock jumping 14% on February 17. Investors responded enthusiastically to the news, sending the stock soaring to a new high.
Reata emerged victorious following a Phase II clinical trial of 103 patients, in which Skyclarys treatment resulted in “significant neurological function improvements” compared to the placebo group. The success of the trial was a major milestone for the company.
Patients treated with Skyclarys experienced a remarkable improvement in their motor function, as evidenced by their significantly lower modified Friedreich Ataxia Rating Scale (mFARS) scores compared to placebo. The mFARS scale is the gold standard tool for assessing the progression of Friedreich Ataxia, making the results of this study particularly noteworthy.
A 2021 study published in the Annals of Neurology has revealed that a certain drug is both effective and safe to use. Participants in the study reported that the drug was well-tolerated, with no major adverse effects. These results suggest that the drug could provide a viable option for those looking for a safe and effective treatment.
Up Next
With the first FA approval now in the record books, companies are vying to be next in line. The competition is fierce as each organization vies to secure the coveted approval. It’s a race to the finish, and only the most prepared and ambitious contenders will cross the finish line.
PTC Therapeutics is pushing the boundaries of medical research with their experimental small molecule, Vatiquinone. Inhibiting 15-Lipoxygenase, a primary regulator of the oxidative stress and inflammation response pathways, Vatiquinone is currently undergoing a Phase II/III study with the potential to deliver positive results in the second quarter of 2023. This groundbreaking drug could revolutionize the way we treat inflammation and oxidative stress-related conditions.
Larimar Therapeutics is hot on the heels of the leading treatment options for Friedreich’s Ataxia (FA). The Pennsylvania-based company is developing CTI-1601, a recombinant fusion protein that hopes to deliver human frataxin into the mitochondria of FA patients. With Phase II trials underway, Larimar Therapeutics is aiming for topline data to be available by the second half of 2023.