Summary:
Reata Pharmaceuticals has a PDUFA data for Omaveloxolone for FA 2/28/23.
The previous PDUFA date was extended 3 months after additional data was submitted.
There's concerns from the FDA whether the data provided shows evidence for approval.
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Table of Contents
Omaveloxolone For FA Development Timeline:
Omaveloxolone For Friedreich’s ataxia (FA) has had an interesting path working with the FDA towards approval. They announced MOXle Part 2 results in Q4 2019, submitted their NDA which got accepted under Priority Review, but in their Mid-Cycle Meeting the FDA expressed concerns about the data/submission. The company added additional data to the NDA filing which extended the PDUFA to Feb 2023. The article outlines this previous data and upcoming PDUFA.
(source)
Upcoming PDUFA Decision
Reata Pharmaceuticals (RETA) has an upcoming PDUFA decision for omaveloxolone for patients with Friedreich’s ataxia (FA) on 2/28/23. The NDA was accepted by the FDA in May 2022 under priority review. The company submitted additional data and analysis after a a Late-Cycle Meeting, which the FDA deemed as major amendments to the NDA. The PDUFA target date was extended 3 months to 2/28/23. FDA does not plan to hold an advisory committee meeting.
During this Late-Cycle Meeting with the FDA, the FDA noted that if omaveloxolone is approved, they anticipate requiring a drug-drug interaction trial with CYP3A4 modulators, a thorough QT trial, and an evaluation of pregnancy outcomes. The company noted that "with respect to label review, the original proposed label language did not reflect the data and analyses included in the amendments to the NDA and that we [the company] have updated it in connection with the planned filing of our Marketing Authorization Application (“MAA”) in Europe later this year [2022]." (source)
Late-Cycle Meeting Overview / Concerns (source)
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Friedreich’s Ataxia (FA) Disease:
Caused by epigenetic silencing of frataxin. Impaired mitochondrial function, suppressed Nrf2 expression, which impairs energy production. Mean survival is mid-thirties. It's typically diagnosed in teens with wheelchair-dependence in twenties. Currently no approved therapies that can delay functional deterioration. It's a rare disease, in the U.S. an estimated 4,000 patients are diagnosed out of 5,000 total.
Other companies with clinical stage assets for FA include PTC Therapeutics (PTCT) and Larimar Therapeutics (LRMR). PTC Tx is developing Vatiquinone (PTC743) which has phase 3 data coming Q2 2023, and Larimar Tx is developing CTI-1601 which has phase 2 data in the second half of this year, but is under partial clinical hold.
Upcoming FA Catalysts (source)
Prior Data
Part 2 of MOXIe, an international, multi-center, double-blind, placebo-controlled, randomized registrational Phase 2 trial, enrolled 103 patients with FA worldwide, and is the largest global, interventional study ever conducted in FA. Patients were randomized 1:1 to 150 mg of omaveloxolone or placebo. The primary analysis population included patients without pes cavus (n=82), a musculoskeletal foot deformity that may interfere with the patient’s ability to perform some components of the mFARS exam. Safety analyses were evaluated in the all randomized population (n=103).
The topline data read out 10/14/2019 (source).
The primary endpoint for the study was change in the mFARS score relative to placebo after 48 weeks of treatment. The mFARS is a physician-assessed neurological rating scale used to measure FA disease progression. It includes four sections that measure the patient’s performance of activities such as speaking and swallowing, upper limb coordination, lower limb coordination, and standing and walking. The FDA has indicated that mFARS is an acceptable primary endpoint to evaluate the effect of omaveloxolone for the treatment of patients with FA.
Omaveloxolone treatment met the primary endpoint of the study producing a statistically significant, placebo-corrected 2.40 point improvement (decrease) in mFARS (n=82; p=0.014). Patients treated with omaveloxolone experienced a mean improvement in mFARS of -1.55 points from baseline, while patients treated with placebo experienced a mean worsening in mFARS of +0.85 points from baseline. The observed placebo-corrected improvements in mFARS were time-dependent, increasing over the course of treatment with the largest improvement observed after 48 weeks of treatment (source).
MOXle Part 2 Primary Endpoint. (source)
Omaveloxolone was generally reported to be well tolerated in this study. Four (8%) omaveloxolone patients and two (4%) placebo patients discontinued study drug due to an adverse event (AE). The reported AEs were generally mild to moderate in intensity, and the most common AEs (> 20%) observed more frequently compared to placebo were headache, nausea, increased aminotransferases, fatigue, and abdominal pain. Increases in aminotransferases are a pharmacological effect of omaveloxolone, which increases production of aminotransferases in vitro, and we believe are related to restoration of mitochondrial function. In MOXIe, the aminotransferase increases were associated with improvements (reductions) in total bilirubin and were not associated with liver injury. The overall rate of serious adverse events (SAEs) was low, with three patients in each group reporting SAEs while receiving study drug. Two additional omaveloxolone-treated patients reported SAEs approximately two weeks after receiving their final dose.
AE Profile (source)
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