Friedreich’s Ataxia Research Alliance (FARA) is encouraging Reata Pharmaceuticals to submit a New Drug Application (NDA) for Omaveloxolone, which recently completed a Phase III clinical trial as a treatment for Friedreich’s Ataxia (FA). FARA is also urging the Food and Drug Administration (FDA) to consider approving the NDA. FARA has prepared a letter that will be submitted to Reata Pharmaceuticals and the FDA; they are looking for supporters to sign the letter. NAF submitted a letter of support (links to PDF). To date, FARA has received more than 40,000 signatures from patients, their family and friends, and rare disease advocates. We want you to sign on too!
Why is FARA Sending this Letter?
There are currently no approved treatments for FA. The MOXIe study included 2 placebo-controlled clinical trials and pre-clinical studies in FA. Data from the MOXIe trials showed improvement in neurological function in people with FA.
At this point in the drug approval process, sometimes the FDA will require an additional clinical trial. However, federal law allows for some exceptions, particularly when the treatment is for a rare disease. The 21st Century Cares Act requires the FDA to consider the patient perspective in making approval decisions for new drugs. Given the positive results in the well-controlled clinical trials and lack of treatment options, it is imperative that Reata Pharmaceuticals and the FDA move as quickly as possible to make this medication available to FA patients.
It is important for drug and biologic sponsors and the FDA to hear the voices of rare disease communities and to know that these patients and their family members do engage in understanding the data from clinical trials in making decisions about safety and efficacy.
Why is NAF in Support?
NAF has had the opportunity to review the results of the MOXIe studies, and as many of our stakeholders are individuals living with FA, we, like FARA, believe it is important to facilitate opportunities for direct patient engagement and incorporate the patient experience in the interpretation of results and decision making. It is based on these understandings and beliefs that NAF strongly supports the efforts of FARA and the FA Community Call to Action requesting Reata to submit an NDA on an urgent basis and FDA to exercise the flexibility granted by law and contained in FDA guidance in considering approval of an NDA for Omaveloxolone in FA based on the existing evidence from clinical trials.
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