Update 17/11/2022
Omav data presented at the International Congress for Ataxia Research (ICAR 2022 – Dallas, Texas)
Dr David Lynch, from the University of Pennsylvania and Children’s Hospital of Philadelphia, is one of the investigators who has been working with Reata Pharmaceuticals for many years to study omaveloxolone (Omav) as a potential treatment for Friedreich’s ataxia (FA). At the International Congress for Ataxia Research (ICAR 2022) earlier this month, Dr Lynch presented further clinical data on omav from the multi-centre trial (including the site in London).
On Friday 4 November, Dr Lynch gave a presentation in the late-breaking research section of the conference. He described the use of natural history data in the Omav clinical trials. Natural history data is clinical information collected from people with the condition (in this case FA) over a long period of time. Analysing natural history data from a large group of people gives researchers and clinicians information on how the condition is likely to progress over time. Dr Lynch described how data from people on the Omav clinical trial was compared to natural history data. This allowed the researchers to show that omav provides persistent benefit over three years when compared to natural history data. Progression of FA was slowed by omav.
Dr Lynch also presented a poster at the conference, describing the latest clinical data for Omav. At the end of the MOXIe clinical trial (in which participants were given either placebo or Omav), participants were offered omav as part of an extension study. The latest data showed that Omav slowed progression for those that took it as part of the extension study. You can read the full paper describing these results here.
This data was provided to the FDA earlier this year, in support of the New Drug Application for Omav. A decision from the FDA on whether to approve Omav for the treatment of FA is expected by 28th February 2023 – we’ll keep you updated.
Reata’s late-cycle meeting with the FDA
In a recent press release, Reata described the outcome of a late-cycle meeting with the FDA.
Late-cycle meetings are designed to discuss any issues that the FDA finds during their review of a new drug, but they are not designed to discuss the final regulatory decision of the FDA. During this meeting, the FDA confirmed that they are continuing to review the Omav data, and they currently have no requests for additional data, although this might be requested at a later date as their review is ongoing.
During this meeting, the FDA said that if Omav is approved, they anticipate requiring some further studies, such as an evaluation of pregnancy whilst on the drug, among others. These further studies are not currently being requested before the FDA makes their regulatory decision but will be requested after approval if approval is granted.
Read the latest press release in full here.
Update 14/10/2022
Reata announced that FDA does not plan to hold an advisory committee meeting in connection with its review of the company’s New Drug Application (NDA) of Omaveloxolone for FA. We’re awaiting further details on what this means for the Omaveloxolone approval process and we’ll keep you updated as soon as we receive further information.
What are Advisory Committee meetings?
Advisory committees provide FDA with independent opinions and recommendations from outside experts on applications to market new drugs, and on FDA policies. The marketing applications include data to show the safety and effectiveness of human drugs. The outside experts receive summary information about the applications and copies of FDA’s review of the application documents. Based on this information, advisory committees may recommend approval or disapproval of a drug’s marketing application. FDA generally follows an advisory committee’s recommendation but is not bound to do so.
These meetings are an optional part of the review process. The decision to involve an advisory committee is usually at the discretion of the director of the review division.
Information taken from the FDA.gov website.
To read Reata’s full press release, please follow this link, to receive our OMAV-only newsletter, please sign-up using this form.
Update 09/08/2022
Reata Pharmaceuticals have announced that they have had a meeting with the US Food Drug and administration (FDA) regarding Omaveloxolone (Omav) for the treatment of Friedreich’s ataxia (FA). The purpose of this meeting was for the FDA to provide Reata with an update on the review of their New Drug Application (NDA), including whether any issues had been identified. The FDA said that it has not identified any new significant issues but have some concerns over the strength of the evidence showing the effectiveness of the treatment. They have not identified any major safety concerns at this stage; however, their safety review is ongoing.
To address the FDA’s concerns, Reata has submitted three sets of additional data to the FDA:
- Updated results from their ‘delayed-start analysis’ study which included later time points and more participants with FA. These results suggested a persistent treatment effect.
- Analysis which compared the progression of mFARS (clinical rating scale for FA) in people with FA who were treated with Omav compared to untreated people with FA who were part of the Clinical Outcome Measures in Friedreich’s ataxia (FA-COMS) study. The FA-COMS study is the largest natural history study of FA and looks at how the condition progresses over time. Reata’s analysis found that progression in mFARS was 55% slower in people with FA treated with Omav compared with untreated people with FA in the FA-COMS study.
- Information which shows the relevance of Nrf2, which is the target of Omav, to FA.
The submission of this new data has extended the review process, but this additional evidence should add further support for the approval of Omav. We now hope to hear a response regarding the FDA’s decision on whether to approve Omav in the US by 28 February 2023.
Read the full press releases here and here.
Update 26/05/2022
Reata Pharmaceuticals have announced that the FDA have granted their Omaveloxolone application ‘Priority Review’ designation. The FDA grants Priority Review to medicines that may offer significant improvements in the treatment, diagnosis, or prevention of a serious condition. This means the FDA will review their application for regulatory approval in the US within 8 months rather than the usual 12 months.
Read the full press release here.
Update 12/05/2022
Reata Pharmaceuticals have released a press release regarding their New Drug Application (NDA) to the US FDA for Omaveloxolone for the treatment of Friedreich’s ataxia. They state that they are looking forward to working with the US FDA on its review of their NDA throughout this year. If Omaveloxolone is approved by the FDA, they are preparing to launch the treatment in early 2023.
In this press release, they also gave an update on the status of their application with the European Medicines Agency (EMA). They are continuing to complete the regulatory procedures and submissions required prior to filing a Marketing Authorization Application (“MAA”) in Europe for approval of omaveloxolone. They plan to submit an MAA to the EMA in the fourth quarter of 2022.
Read the full press release here.
Update 06/04/2022
Ataxia UK is very pleased to see that Reata Pharmaceuticals have completed the submission of a New Drug Application (NDA) to the US FDA, for the potential Friedreich’s ataxia treatment Omaveloxolone. This NDA includes data from the MOXIe Part 1, Part 2 and MOXIe Extension studies. The next step in this process is for the FDA to use this NDA to decide whether to approve the treatment.
Read the full press release here.
Update 01/03/2022
In February, we reported that Reata Pharmaceuticals had initiated a rolling submission of a New Drug Application to the US FDA for omaveloxolone to treat Friedreich’s ataxia (FA). We are now encouraged to see from yesterday’s press release that Reata are “actively preparing for the commercial launch of the drug early next year”. Omaveloxolone still needs to be approved by the regulatory agencies before it can be prescribed, but we are very pleased to see that Reata Pharmaceuticals are looking ahead to the next stages required for Omaveloxolone to reach people with FA.
Read the press release here.
Update 01/02/2022
Reata Pharmaceuticals has announced they have initiated a rolling submission of a New Drug Application to the US FDA for omaveloxolone to treat Friedreich’s ataxia. A rolling submission allows Reata to submit portions of the application to the FDA for review on an ongoing basis. Reata expects to complete the submission of the NDA by end of the first quarter of 2022.
Read the press release here.
Update: 23/11/2021
Reata Pharmaceuticals have announced that the US Food and Drug Administration (FDA) has granted Fast Track Designation for omaveloxolone for the treatment of Friedreich’s ataxia. This will enable Reata to have more frequent communication with the FDA and if relevant criteria are met, they will be eligible for FDA programs such as priority review. Reata plan to submit a New Drug Application to the FDA for approval of omaveloxolone during the first quarter of 2022.
Read the press release here.
Update: 30/09/2021
Reata Pharmaceuticals announced that it has completed its pre-New Drug Application (pre-NDA) meeting with the United States Food and Drug Administration (FDA) for omaveloxolone for the treatment of patients with Friedreich’s ataxia (FA) and reaffirmed its plan to submit an NDA in the first quarter of 2022. The purpose of the pre-NDA meeting was to discuss the content of Reata’s planned NDA submission. They plan to submit the NDA seeking standard approval for omaveloxolone for the treatment of FA. They are not planning to conduct a second pre-approval clinical study prior to the submission. For more information, read the press release here.
Update: 10/08/2021
In a recent update on their clinical programmes, Reata Pharmaceuticals announced that they have scheduled a pre-New Drug Application (pre-NDA) meeting with the US Food and Drug administration (FDA) during the third quarter of this year. This is typically the last formal interaction with the FDA prior to a New Drug Application (NDA). They plan to submit the NDA for omaveloxolone to the FDA during the first quarter of 2022.
Update: 28/06/2021
Euro-ataxia Conference 2021: Reata update on omaveloxolone study in FA
Ataxia UK was delighted to have Dr Colleen Stoyas, who is a Medical Science Liaison at Reata Pharmaceuticals, present an update on the MOXIe study which evaluates the safety and efficacy of omaveloxolone (Omav) in Friedreich’s ataxia (FA) at the Euro-ataxia conference 2021 on Friday 18th June 2021.
Dr Stoyas began with an overview of the mechanism of Omav in FA. She then explained that to test Omav in FA, Reata used mFARS (Modified Friedrich’s Ataxia Rating Scale), which is a physician-assessed neurological exam that tracks progression of FA. This was followed with a summary, key findings, and safety summary of the last 6 years, where Reata has been sponsoring the MOXIe study which consists of 3 parts: MOXIe Part 1, MOXIe Part 2, and the Open-Label Extension.
An important finding shared was data from the MOXIe extension study was also analysed in the Delayed-Start Analyses. The results of this study indicate that the slowing of progression measured by mFARS is due to Omav treatment and Reata feel are indicative of Omav modifying the progression of FA.
During the Q and A session, a question was asked that if Omav was approved in the UK, whether it would be available for children? Dr Stoyas replied that currently the youngest age in the MOXIe inclusion criteria was 16 years old, and therefore, a pediatric safety study will likely be required.
Please find the presentation slides here.
Update: 25/05/2021
Reata Pharmaceuticals is investigating the benefits of a new drug called omaveloxolone – a potential therapy for Friedreich’s ataxia (FA). In order for omaveloxolone to be passed for use in the USA it must be approved by their Food and Drug Administration (FDA). Approval in the USA may hasten approval in other countries.
Reata Pharmaceuticals have announced that they received a communication from the FDA (United States Food and Drug Administration) stating that, after a preliminary review of briefing materials for an upcoming Type C meeting, a pre-New Drug Application (pre-NDA) meeting is the most appropriate format for a discussion of the development program for omaveloxolone in Friedreich’s ataxia.
Reata now plan to withdraw the current request for a Type C meeting and submit a request for a pre-NDA meeting. A Type C meeting is a general meeting regarding the development and review of a drug, while a pre-NDA is a specific meeting to discuss the content of a New Drug Application. It is typically the last formal interaction with the Food and Drug Administration prior to a New Drug Application (NDA). Therefore, this represents a positive step forward for the potential approval of omaveloxolone by the FDA. Find the press release here.
The news follows the Friedreich’s Ataxia Research Alliance’s (FARA) recent petition, supported by Ataxia UK, which was signed by over 74,000 people, asking Reata to submit an NDA for omaveloxolone and the FDA to consider its approval based on existing evidence.
Ataxia UK regards this as positive news. Keep a look out on our website and social media channels for updates regarding Omaveloxolone.
Date posted: 17/05/2021
Reata Pharmaceuticals is a clinical-stage biopharmaceutical company, and one of their most advanced clinical candidates is omaveloxolone, which is being investigated in clinical trials as a potential treatment for Friedreich’s ataxia (FA). To find out more about omaveloxolone, click here.
In the latest press release they reported that new data from ‘Delayed-Start Analyses’ suggest disease-modifying activity with omaveloxolone. They claim this provides evidence supporting the positive primary endpoint findings in MOXIE Part 2, and provides additional evidence of the effectiveness of omaveloxolone in patients with FA.
Reata requested and were granted a Type C meeting with the FDA (United States Food and Drug Administration) to discuss their data analyses, and overall FA development program. The meeting will occur later this year.
Also, later this year, Reata is planning to initiate a second pivotal study, which will incorporate input from the FDA and European Medicines Agency into the protocol, before enrollment begins.
Please read the full press release here and latest news associated with this topic here.
Ataxia UK’s research team have reported on omaveloxolone previously. Please find below links to our previous articles on this topic:
1. https://www.ataxia.org.uk/research-news/letter-reata-and-fda/
2. https://www.ataxia.org.uk/research-news/omaveloxolone-omav-update/
