dm+d

Unassigned

New Medicines

Friedreich's ataxia

Information

New molecular entity
Reata
Reata

Development and Regulatory status

Phase II Clinical Trials
Phase II Clinical Trials
Pre-registration (Filed)
Yes
Yes
Aug 22In response to a complete response letter from the FDA in February Reata has submitted an updated analysis of MOXIe extension study using data up to March. As this constitutes a major amendment to the application the PDUFA date has been pushed back three months to Feb 29, 2023 [15].
May 22FDA grants Priority review and has assigned PDUFA target action date of 30 November 2022 [14]
Mar 22Reata completes the rolling submission of an NDA to the FDA. It is supported by efficacy and safety data from the MOXIe Part 1, Part 2, and MOXIe Extension studies [12].
Feb 22Reata continues the regulatory procedures and submissions required for filing an MAA with the EMA for approval of omaveloxolone for the treatment of patients with FA [13].
Jan 22Reata Pharmaceuticals initiate rolling submission of NDA to US FDA for omaveloxolone for the treatment of Friedreich’s Ataxia [11].
Nov 21Granted Fast Track status in US [10].
Nov 21In its latest annual report, Reata states its intention to also seek approval for omavexolone in Europe. Preliminary discussions with the EMA indicated that additional nonclinical or clinical studies may be required [9].
Sep 21Reata completes its pre-New Drug Application (NDA) meeting with the FDA and reaffirms its plan to file in the US in Q1 2022 seeking standard approval. It is not planning to conduct a second pre-approval clinical study prior to the submission [8].
Aug 20Reata announces that if the FDA accepts their proposed approach, it expects to complete the crossover study in Q4 20 and file to the FDA in Q1 21. If the FDA rejects the proposal or if the data are not supportive, Reata will evaluate whether it is feasible to conduct a second pivotal study in FA patients as suggested by the FDA. Regardless of the interaction with the FDA, the company plans to pursue marketing approval outside of the US [7].
Aug 20Reata reports completion of type C meeting with the FDA. The FDA did not express any concern over primary endpoint data from the part 2 of the PII MOXIe trial, however, deemed the data to be insufficient to support a single study approval without additional evidence. The company in response enlisted complications for conducting additional clinical trial, including COVID-2019 pandemic. The Friedreich ’s Ataxia Research Alliance (FARA) then proposed a crossover study to address the US FDA requirement. The study will provide additional evidence on of efficacy of omaveloxolone by measuring effect of treatment on mFARS in patients who were previously randomised to placebo in the part 2 of the MOXIe study and are being treated in the open-label extension study. The US FDA agreed conceded that the proposed trial may provide additional information and requested the company to submit the trial design [5].
Jul 18Granted orphan drug designation in the EU for the treatment of Friedreich’s ataxia.[2]
Jun 17Granted orphan drug designation for the treatment of Friedreich’s ataxia inteh USA.[2]

Category

Activator of Nrf2 (antioxidant inflammation modulator) - taken once daily to increase ATP production and suppress oxidative stress and inflammation and promote restoration of mitochondrial function.
Friedreich’s ataxia is a rare genetic neuromuscular disorder that causes progressive loss of coordination, muscle weakness, and fatigue. prevalence is about 1.8 per 100,000 in the UK [2,3].
Friedreich's ataxia
Oral

Trial or other data

Oct 20Reata announces that results from the pivotal part 2 portion of the MOXIe trial were published in the journal Annals of Neurology [6].
Oct 19In the 2nd part of the PII MOXIe trial, pts treated with 150mg of omaveloxolone saw a placebo-corrected 2.40 point improvement in mFARS at 48-weeks. Omaveloxolone led to a mean improvement in mFARS of -1.55 points from baseline vs. a mean worsening in mFARS of +0.85 points from baseline with placebo.[3,4]
Nov 18Part 2 of PII MOXIe study fully enrolled with 103 pts.[2]
Mar 18Positive proof-of-concept data released from Part 1 of the trial. Omaveloxolone showed improvement in mFARS scores of 3.8 points (p=0.0001) at the optimal dose level vs. baseline and a placebo-corrected improvement in mFARS scores of 2.3 points (p= 0.06).[2]]
Jun 17Results from part 1 of PII MOXIe trial demonstrated that omaveloxolone induced Nrf2 and was associated with improvements in mitochondrial and neurological function.[2]
Nov 14The Global randomised, double-blind, dose-ranging (2.5mg orally once daily or 10mg, 20mg, 40mg, 80mg, 160mg, 300mg and 150mg), 2 part, placebo-controlled PII MOXIe trial (NCT02255435, RTA 408-C-1402) of omaveloxolone in 172 pts with Friedreich´s ataxia started. The primary efficacy endpoint is change from baseline in the modified Friedreich´s ataxia rating scale (mFARS) at 48 weeks. [2-4]