Reata Pharmaceuticals Announces FDA Filing Acceptance and Priority Review Designation for the NDA for Omaveloxolone for the Treatment of Patients with Friedreich’s Ataxia

Rhea-AI Summary

Reata Pharmaceuticals announced that the FDA has accepted its New Drug Application (NDA) for omaveloxolone, a treatment for Friedreich's ataxia, and granted it Priority Review. The FDA's decision indicates no review issues at this time, with a Prescription Drug User Fee Act (PDUFA) target action date set for November 30, 2022. If approved, omaveloxolone would be the first therapy for this rare disease affecting approximately 5,000 patients in the U.S. The application is supported by data from the MOXIe clinical trials.

Positive

• FDA accepted NDA for omaveloxolone and granted Priority Review.
• No potential review issues identified by the FDA.
• PDUFA target action date set for November 30, 2022.
• If approved, omaveloxolone will be the first therapy for Friedreich's ataxia.

Negative

• None.

Friedreich’s Ataxia is a Rare, Progressive, Life-Shortening, Neuromuscular Disease that Affects Approximately 5,000 Patients in the United States

Application Assigned a PDUFA Date of November 30, 2022

If Approved, Omaveloxolone Would Become the First Approved Therapy for Friedreich’s Ataxia in the United States

PLANO, Texas--(BUSINESS WIRE)-- Reata Pharmaceuticals, Inc. (Nasdaq: RETA) (“Reata,” the “Company,” “our,” “us,” or “we”), a clinical-stage biopharmaceutical company, today announced that the U.S. Food and Drug Administration (“FDA”) has accepted for filing and granted Priority Review of its New Drug Application (“NDA”) for omaveloxolone for the treatment of patients with Friedreich’s ataxia. The FDA indicated that at this time it has not identified any potential review issues. The NDA is supported by the efficacy and safety data from the MOXIe Part 2 trial and additional supporting data from the MOXIe Part 1 and MOXIe Extension trials. Omaveloxolone received Fast Track Designation in November 2021 and Rare Pediatric Disease Designation in May 2022.

The FDA grants Priority Review to medicines that may offer significant improvements in the treatment, diagnosis, or prevention of a serious condition. This Designation shortens the FDA’s review of the NDA to eight months from the time of submission, versus a standard review timeline of 12 months. The FDA has assigned a Prescription Drug User Fee Act (“PDUFA”) target action date of November 30, 2022. The FDA indicated it is currently planning to hold an advisory committee meeting to discuss the application.

“We are pleased with the FDA’s decision to grant Priority Review to our NDA for omaveloxolone for the treatment of patients with Friedreich’s ataxia in the United States,” said Warren Huff, Reata’s Chief Executive Officer. “With the FDA’s acceptance of our NDA for filing, omaveloxolone is now one step closer to potentially providing a treatment option for patients with Friedreich’s ataxia, a rare, genetic, debilitating, and degenerative neuromuscular disorder with no approved therapies. We look forward to working with the FDA during the review process, and if approved, we are looking forward to a commercial launch in early 2023.”

About Friedreich's Ataxia

Friedreich’s ataxia is a rare, genetic, life-shortening, debilitating, and degenerative neuromuscular disorder typically caused by a trinucleotide repeat expansion in the first intron of the frataxin gene, which encodes the mitochondrial protein frataxin. Pathogenic repeat expansions can lead to 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. Patients with Friedreich’s ataxia typically experience symptoms in childhood, including progressive loss of coordination, muscle weakness, and fatigue that commonly results in motor incapacitation with patients requiring a wheelchair in their 20s. Patients with Friedreich’s ataxia may also experience visual impairment, hearing loss, diabetes, and cardiomyopathy. On average, patients with Friedreich’s ataxia die in their mid 30s. Based on literature and proprietary research, we believe 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 Friedreich’s ataxia in the United States. Currently, there are no approved therapies for the treatment of Friedreich’s ataxia.

About Omaveloxolone

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 Orphan Drug, Fast Track, and Rare Pediatric Disease Designations to omaveloxolone for the treatment of Friedreich’s ataxia. The European Commission has granted Orphan Drug Designation in Europe to omaveloxolone for the treatment of Friedreich’s ataxia.

About Reata

Reata is a clinical-stage biopharmaceutical company that develops novel therapeutics for patients with serious or life-threatening diseases by targeting molecular pathways involved in the regulation of cellular metabolism and inflammation. Reata’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.

Forward-Looking Statements

This press release includes certain disclosures that contain “forward-looking statements,” including, without limitation, statements regarding the success, cost and timing of our product development activities and clinical trials, our plans to research, develop, and commercialize our product candidates, our plans to submit regulatory filings, and our ability to obtain and retain regulatory approval of our product candidates. You can identify forward-looking statements because they contain words such as “believes,” “will,” “may,” “aims,” “plans,” “model,” and “expects.” Forward-looking statements are based on Reata’s current expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks, and changes in circumstances that may differ materially from those contemplated by the forward-looking statements, which are neither statements of historical fact nor guarantees or assurances of future performance. Important factors that could cause actual results to differ materially from those in the forward-looking statements include, but are not limited to, (i) the timing, costs, conduct, and outcome of our clinical trials and future preclinical studies and clinical trials, including the timing of the initiation and availability of data from such trials; (ii) the timing and likelihood of regulatory filings and approvals for our product candidates; (iii) whether regulatory authorities determine that additional trials or data are necessary in order to obtain approval; (iv) the potential market size and the size of the patient populations for our product candidates, if approved for commercial use, and the market opportunities for our product candidates; and (v) other factors set forth in Reata’s filings with the U.S. Securities and Exchange Commission, including its Annual Report on Form 10-K for the fiscal year ended December 31, 2021, under the caption “Risk Factors.” The forward-looking statements speak only as of the date made and, other than as required by law, we undertake no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events, or otherwise.

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Reata Pharmaceuticals, Inc.

(972) 865-2219

https://www.reatapharma.com/

Investor Relations & Media Relations:

John Hunter ir@reatapharma.com

Wendy Segal media@reatapharma.com

https://www.reatapharma.com/contact-us/

Source: Reata Pharmaceuticals, Inc.

FAQ

What is the significance of the FDA granting Priority Review to Reata Pharmaceuticals' NDA for omaveloxolone?

The Priority Review indicates the FDA recognizes omaveloxolone as a potential significant improvement in treatment for Friedreich's ataxia, which may expedite the approval process.

What is the PDUFA target action date for omaveloxolone?

The PDUFA target action date for omaveloxolone is set for November 30, 2022.

How many patients are affected by Friedreich's ataxia in the United States?

Approximately 5,000 patients are affected by Friedreich's ataxia in the United States.

What indicates that the FDA has not identified review issues for omaveloxolone?

The FDA's statement of no potential review issues suggests a smoother path towards potential approval.