On average, patients with Friedreich’s ataxia die in their mid-thirties.
Based on literature and proprietary research, it is believed that Friedreich’s ataxia affects approximately 5,000 children and adults in the United States and 22,000 individuals globally. There are an estimated 4,000 patients diagnosed with FA in the United States.
Currently, there are no approved therapies for the treatment of Friedreich’s ataxia.
About Friedreich’s Ataxia
Friedreich’s ataxia is a rare, genetic and degenerative neuromuscular disorder caused by a trinucleotide repeat expansion in the first intron of the frataxin gene that encodes the mitochondrial protein frataxin. Pathogenic repeat expansions would cause impaired transcription and reduced frataxin expression, which can result in mitochondrial iron overload and poor cellular iron regulation, increased sensitivity to oxidative stress, and impaired mitochondrial ATP production.
Omaveloxolone is an investigational, oral, once-daily, activator of Nrf2, a transcription factor that induces molecular pathways that promote the resolution of inflammation by restoring mitochondrial function, reducing oxidative stress, and inhibiting pro-inflammatory signaling. The FDA has granted omaveloxolone Orphan Drug and Fast Track Designations for the treatment of Friedreich’s ataxia. Also, the European Commission has granted Orphan Drug Designation to omaveloxolone for the treatment of Friedreich’s ataxia in Europe.
From the CEO of Reata Pharmaceuticals
Warren Huff, Reata’s Chief Executive Officer, Reata Pharmaceuticals, said, “This NDA submission marks an important step toward making a treatment available for patients with Friedreich’s ataxia, a serious degenerative neuromuscular disorder with no approved therapies. I want to thank all those who made this possible, especially our patients with Friedreich’s ataxia, their families, and investigators, as well as our employees, for their tenacity and unwavering belief in the omaveloxolone clinical program. We look forward to the next steps on the path to making omaveloxolone available as a first-in-class therapy for Friedreich’s ataxia, pending approval.”
About Reata Pharmaceuticals
Reata Pharmaceuticals is a clinical-stage biopharmaceutical company developing novel therapeutics for patients with serious or life-threatening diseases by targeting molecular pathways involved in the regulation of cellular metabolism and inflammation.
The firm’s two most advanced clinical candidates, omaveloxolone and bardoxolone methyl (bardoxolone), target the important transcription factor Nrf2 that promotes the resolution of inflammation by restoring mitochondrial function, reducing oxidative stress, and inhibiting pro-inflammatory signaling.
Omaveloxolone and bardoxolone are investigational drugs and their safety and efficacy have not been established by any agency.
Taking in glucose and oxygen, mitochondria produce energy which they capture and package as energy-rich molecules of adenosine triphosphate (ATP).
Learning more about the mitochondria opened the door for researchers to understand the function of mitochondria organelles found in the cells of eukaryote organisms. The researchers are learning how mitochondria might function and how they might cause chronic devastating life-threatening diseases.
Regarding the diseases, the researchers are developing treatments for them. Some of these diseases, which included Friedreich’s ataxia and (ALS), are horrible and cause the inevitable death of the patients.
Products that demonstrate an improvement during clinical trials, we believe, should be granted approval, regardless of the improvement’s size, as long as they are safe. As physicians and researchers follow up on patients who are taking the approved drugs they might amass further knowledge that could lead to the improvment of the treatments, or even to miracle cures.
Click here to read more about Reata Pharmaceuticals.
Reata Pharmaceuticals Completed Rolling NDA Submission of Omaveloxolone for Friedreich’s Ataxia
Reata Pharmaceuticals Completed Rolling NDA Submission for Omaveloxolone for Friedreich’s Ataxia
Reata Pharmaceuticals (RETA) announced the completion of the rolling submission of a New Drug Application (NDA) to the U.S. FDA for omaveloxolone’s treatment of Friedreich’s ataxia (FA). The NDA is supported by the efficacy and safety data from the MOXIe Part 1, Part 2 and MOXIe Extension studies.
The FDA has granted Fast Track Designation and Orphan Drug Designation to omaveloxolone for the treatment of Friedreich’s ataxia.
Why is This Important for Patients with Friedreich’s Ataxia?
Patients with Friedreich’s ataxia experience symptoms from childhood which include progressive loss of coordination, muscle weakness and fatigue resulting in motor incapacitation. The patients require wheelchairs in their teens or early 20s.
Patients with Friedreich’s ataxia may also experience visual impairment, hearing loss, diabetes and cardiomyopathy.