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
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/
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