Omaveloxolone update

Update on availability of Skyclarys for adults with Friedreich’s ataxia (FA) in the UK 

Ataxia UK has received a communication from Biogen informing us that they now have a timeline for the UK regulatory process for Skyclarys (omaveloxolone).

There are 2 stages: 

1. the Medicines and Healthcare products Regulatory Agency (MHRA) decide whether to approve the drug for use in the UK  

and then  

2. the National Institute for Health and Care Excellence (NICE) assesses the cost effectiveness of the drug and will decide whether if approved, will be made available on the NHS in England (Wales and Northern Ireland can also adopt NICE recommendations).  

In Scotland the Scottish Medicine Consortium (SMC) will decide whether it should be made available on the NHS in Scotland using a similar process to that of NICE.

These are the required significant steps to making the drug available to adults living with FA in the UK.  

Details of the timeline

MHRA – Biogen intends to submit Skyclarys for regulatory approval to the MHRA in Q4 2024 using the International Recognition Procedure (IPR). This is the fast-track process introduced in the UK in January 2024, and it means the recent approval from the European Medicines Agency (EMA) will be the reference for the UK submission and the review will be in a shorter timeline than normal. In line with these IRP timelines, we anticipate we will know the outcome of this decision within Q2 2025 (ie: April-June 2025).  

NICE  – NICE have confirmed they intend to evaluate omaveloxolone for treating Friedreich’s ataxia. Once the dossier has been prepared for NICE Biogen will adapt it and also submit it to the Scottish Medicines Consortium.  

The first step in the NICE process is called the ‘scoping phase’ and we are expecting this to take place in July 2024. It involves scoping the population (defining the patient group, care pathway, and comparators) and determining the means of evaluating the medicine. Ataxia UK will be invited to contribute and we intend to do so.   

Here is the link to the NICE website. NICE update the appraisal webpage at each stage of the appraisal. 

After the scoping phase, Biogen and other relevant stakeholders (such as Ataxia UK) will be formally invited to participate in the omaveloxolone appraisal. We understand the anticipated start date for the appraisal will be around October 2024 (although dates may be subject to change). This appraisal process usually takes between 35 and 45 weeks.  

The submission date for Biogen’s evidence dossier and that of other invited stakeholders including patient groups such as Ataxia UK, is likely to be in January 2025. This date will be confirmed by NICE at the start of the appraisal process. On this basis a decision from NICE on whether to recommend omaveloxolone would likely happen between July and September 2025. 

Next steps – Biogen is now working on their evidence submission to NICE. As the biggest patient group in the UK, we are also very involved in this process, and have already started to engage with NICE.  

Whilst the news that the process has started is welcome, unfortunately the approval process is a long one, involving a number of steps, and we acknowledge that people impacted by FA in the UK will be disappointed about the length of time before Skyclarys could be available on the NHS. We understand and continue to do everything we can to ensure access to Skyclarys is provided as soon as possible.

To that end, Ataxia UK is doing our utmost to ensure that the voices of the FA community are heard by the regulators, and are already engaging in these processes at every opportunity. We have started work on making as big an impact as we can through a strong submission to NICE, engaging with all parties and hiring expert consultants.

Having a strong voice 
In addition, at the end of May, Ataxia UK wrote to the Secretary of State for Health and Social Care and the Labour Shadow Secretary of State highlighting the urgent need for a treatment for FA, and seeking opportunities for exploring earlier access to this drug than on the timeline outlined above.

In some countries in Europe there were compassionate use schemes offered whilst Biogen’s submission was being considered by the European regulator. We are also looking at other actions we can take as an ataxia community. As soon as we have more information, we will let you know the next steps and how you can get involved.      

How about Skyclarys for children with FA? 
In order for the drug to be licensed for use in children, there will need to be further trials and Biogen plan to start a paediatric trial in the US this summer, as a first step. This will hopefully be followed by trials in other countries, and we are encouraging Biogen to have a paediatric trial in the UK too. 

Updates and more information will follow via our usual communication channels, via our monthly eNews, magazine and social media so, keep your eyes peeled for opportunities to get involved.

Webinar and Q&A about Skyclarys UK approval
Date: Tuesday 23rd July 2024 12 - 1pm

This webinar & Q&A session will give an overview of the timeline for the UK approval process for Skyclarys in adults with FA. There will be an opportunity to ask questions to Ataxia UK and Realise Advocacy, an organisation with expertise in advising charities on the process of drug appraisals in the UK. 

If you would like to send any questions in advance, please email them to our Research team at: research@ataxia.org.uk.

To register for this webinar, please follow this link.

The EMA approves the drug Skyclarys® to treat adults with Friedreich’s ataxia in EU countries   

On 12th February 2024, the European Medicines Agency (EMA) announced that they have approved the drug Skyclarys® (Omaveloxolone) for the treatment of Friedreich’s ataxia in adults in EU countries.  

Andreas Nadke, President of Euro-ataxia, the international non-profit organisation of European ataxia charities, which Ataxia UK is an active member of, shared this statement:  

“On behalf of Euro-ataxia and its members, I am delighted to be able to welcome the EMA approval of Skyclarys®, this first, much needed drug for adults with Friedreich’s ataxia in EU countries. Euro-ataxia member patient groups have worked and waited for this day for many years”.  

Ataxia UK’s Head of Research, Julie Greenfield says: ‘We are making every effort to encourage and support Biogen in seeking UK regulatory approval. In addition, we are discussing ways to cooperate to gather useful information from people with FA in the UK. This information is needed to help people get access to the drug if it is approved’.  

Professor Paola Giunti, lead of the London Ataxia Centre, says ‘We are pleased to have contributed to the basic science that led to the global clinical trials leading to the approval in both the US and the EU. I am committed to helping in the next steps to get approval of the drug in the UK’. 

We will ensure the FA community in the UK is kept up to date with the latest developments as soon as possible while remaining confidential when needed. The interests of families affected by FA, and all other types of ataxias, in the UK, are and will continue to be at the forefront of all our efforts. 

To read the press release from Biogen, the pharmaceutical company which has been developing Skyclarys®, click here.

To read a letter form Biogen to Ataxia UK click here.

You can find out more about Omaveloxolone on this page. 

Positive news regarding Skyclarys® approval in the EU

Ataxia UK is delighted with today’s announcement from the EMA’s Committee for Medicinal Products for Human Use (CHMP), the regulatory body that makes recommendations on drug approvals in the EU.

The CHMP has recommended a market authorization for Skyclarys® for people (16 and older) with Friedreich’s ataxia (FA) in the EU. The final decision will need to be made by the European Commission, in the first quarter of 2024. However, this was a significant hurdle to overcome. See the EMA website here. 

In order for Skyclarys® to be approved in the UK, Biogen will need to submit an application to the UK regulator, the MHRA. We believe an approval in the EU will pave the way for a faster route to approval in the UK. It is worth noting that after drug approval, the drug would need go through the ‘Health technology assessment’ process, where decisions are made on funding of the drug within the NHS. 

Ataxia UK is collaborating with Biogen and doing all we can to ensure access to Skyclarys® for people with FA in the UK. Indeed, a productive meeting was held with five members of the Biogen UK team on the 8th December at the Ataxia UK office. Biogen plans to seek approval from the MHRA. There are a number of regulatory routes available that they are considering. This includes an independent filing or using a new regulatory pathway called the International Recognition Procedure (IRP) which is a ‘fast-track approval process’ that is due to be in place in the UK in January 2024. We will continue to work with them and will keep you updated with progress. 

See Biogen’s press release here and their letter to Ataxia UK about this here. 

Biogen completes acquisition of Reata Pharmaceuticals 

On 26th September 2023, Biogen announced they have completed their acquisition of Reata Pharmaceuticals. Reata Pharmaceuticals were the original developers of Omaveloxolone, the only FDA-approved treatment for Friedreich's ataxia (FA).  

Biogen is a global technology company. Biogen is now responsible for ensuring Omav is distributed to people with FA.  

In the press release, the President and CEO of Biogen, Christopher Viehbacher, gave an update to say that as of 26 September 2023, ‘over 1,000 patient start forms for SKYCLARYS® have been submitted in the United States’. He also said that they  ’look forward to leveraging Biogen’s rare disease expertise and capabilities to work together with our Reata colleagues as one team to bring SKYCLARYS ® to patients living with this devastating disease’.  

Read the full press release on the Biogen website. 

Reata Pharmaceuticals announce acquisition by Biogen 

On 28th July 2023, Reata Pharmaceuticals announced that they have entered into an agreement under which Biogen will acquire Reata. Reata Pharmaceuticals is the developer of Omaveloxolone (brand name in the US is SKYCLARYS ®), the only FDA-approved treatment for Friedreich’s ataxia (FA). Omaveloxolone is currently under review by the EU regulators (the EMA). In the press release, Christopher Viehbacher, Biogen’s President and CEO says they ‘believe Biogen has the foundation in place to accelerate the delivery of SKYCLARYS ® to patients around the world’. 

The transaction is expected to close in the fourth quarter of 2023. We will be keeping up-to-date with what this means for the FA community and will communicate any updates via our dedicated newsletter. 

Read the full press release at Reata's website. 

Reata Pharmaceuticals announces approval of latest Omaveloxolone submission 

In May 2023, we reported that Reata Pharmaceuticals, the company developing Omaveloxolone (brand name in the US is SKYCLARYS®), had observed a process impurity above the approved specification for SKYCLARYS ®. This meant that they had to submit supplemental information to the FDA. The FDA prioritised the review of this data (with a response expected mid-August), and we are now pleased to hear that they have approved the submission. In their latest press release, Reata Pharmaceuticals state that with this latest approval by the FDA, SKYCLARYS ® is now available to adults with FA in the United States. 

The full press release can be seen here.

Reata Pharmaceutical’s application for Omaveloxolone is under review in Europe by the European Medicines Agency (EMA). We continue to encourage Reata Pharmaceuticals to apply for regulatory approval in the UK, and will continue to update the FA community on this work. 

Reata recently provided an update on their clinical development programmes via a press release and a webcast.

Read on for a summary of these updates. The full press release and webcast can be found on Reata’s website.

Update on Omaveloxolone (brand name in the US SKYCLARYS ®) availability in the US 

In February 2023, SKYCLARYS ® was approved by the FDA (US drug regulators) for people aged 16 and over in the US. Reata have now completed the final stages of drug product manufacturing and packaging. During the process validation they observed a process impurity above the approved specification.  Therefore, they have submitted supplemental information which the FDA is reviewing with an action date of mid-August 2023.  The FDA have stated that the review has been prioritized and the approval should potentially come before mid-August. Subject to approval by the FDA, Reata expect that SKYCLARYS ® will be available through their specialty pharmacy in the US by no later than mid-August.  

Update on application for approval in Europe 

Reata applied to the EU regulators (the EMA) at the end of 2022. Reata have now received a list of questions from the EMA and are on track to provide their responses in the third quarter of 2023. They have said that if the drug is approved in Europe, they are planning for a commercial launch in the second quarter of 2024.  

Please note, if the EMA approves Omaveloxolone, this will not automatically result in approval in the UK. To make Omaveloxolone available in Great Britain, Reata Pharmaceuticals will need to apply to the UK regulators (the MHRA). In Northern Ireland drugs currently go through EMA approval, but under the Windsor Framework (a post-Brexit legal agreement between the EU and the UK,) this will change, and medicines will be regulated by the MHRA. Ataxia UK is doing everything we can to support and encourage Reata Pharmaceuticals to seek approval for Omaveloxolone in the UK. 

Plans for paediatric studies 

Currently, SKYCLARYS ® is only approved for people with FA in the US aged 16 and above. Therefore, Reata are evaluating their strategies for expanding the approval for the SKYCLARYS ® for those under 16. In the US, Reata are planning to request a meeting with the FDA to discuss expanding the approval. They plan to conduct an initial paediatric study in the US in the final quarter of 2023. This will look at the safety of SKYCLARYS ® and how it is processed by the body in those with FA under 16. In the EU, Reata has received a positive opinion from the EMA on their paediatric investigation plans and they have recently requested scientific advice from the EMA on the study protocol design. An additional paediatric study will be finalized once Reata has received feedback from the EMA and FDA.  

Ataxia UK will continue to push for Omaveloxolone approval in the UK as a priority, and to update the FA and ataxia community on our activities towards this important goal.

Please let other people and families affected by FA know about our dedicated newsletter. 

Ataxia UK is doing everything we can to support and encourage Reata to seek approval for Omaveloxolone in the UK.

Read on for the latest planned updates to the approval process in the UK, and what we’ve been doing to drive forward the approval of Omaveloxolone (brand name in the US SKYCLARYS ®) in the UK…

UK Chancellor announces a change to MHRA reviews
As reported in our March newsletter, the UK government announced plans to speed up access to new drugs in the UK. Under the plans, the UK regulators (the MHRA) are forming a new international framework whereby drugs approved in some other countries, including the US, will be fast-tracked for approval by the MHRA. This is good news, although it is not immediate, and the Chancellor’s speech states that this will be implemented from 2024.

Our letters to the MHRA & Secretary of State for Health

To make SKYCLARYS available in Great Britain, Reata will need to apply to the MHRA (UK regulators).

In March 2023, we wrote to the Chief Executive of the MHRA and Steve Barclay, the Secretary of State for Health and Social Care, to request their support in getting Omaveloxolone approved for use in the UK as soon as possible. We stressed that whilst we welcome the commitment to developing the new fast-tracked approval process described above, for those with FA, access to this drug cannot come soon enough. We argued that when Reata submit an application to the MHRA, this should be expedited in advance of the proposed timeline of 2024 for the changes to the approval process.

Our letter to Reata Pharmaceuticals

We have been meeting regularly with Reata over the last few years and will continue to do so. When necessary, we have been writing letters to Reata’s CEO from our Chair of Trustees and Chief Executive.

Back in December 2022, we wrote to the CEO of Reata Pharmaceuticals to highlight the opportunity to apply for approval in the UK under the MHRA ‘light-touch review’ process used for drugs approved by the EMA (the EU regulators). Given the planned changes to the MHRA processes described above and the fantastic news that SKYCLARYS ® was approved by the FDA in February 2023, we wrote to the CEO of Reata Pharmaceuticals again in April 2023.

In this letter, we urged Reata to submit an application to the MHRA as soon as possible. We informed them that although the proposed fast-tracked process is due to start in 2024, we have written to both the CEO of the MHRA and the Secretary of State for Health and Social Care to ask for this system to be expedited in the case of Omaveloxolone, due to the urgent need for the drug to reach people with FA. We reiterated our commitment to do all that we can to facilitate them in getting regulatory approval in the UK.

We will continue to push for Omaveloxolone approval in the UK as a priority, and to update the FA and ataxia community on our activities towards this important goal.

Please let other people and families affected by FA know about our dedicated newsletter. 

Ataxia UK is doing everything we can to support and encourage Reata to seek approval for Omaveloxolone in the UK.

Read on for the latest planned updates to the approval process in the UK, and what we’ve been doing to drive forward the approval of Omaveloxolone (brand name in the US SKYCLARYS ®) in the UK…

UK Chancellor announces a change to MHRA reviews
As reported in our March newsletter, the UK government announced plans to speed up access to new drugs in the UK. Under the plans, the UK regulators (the MHRA) are forming a new international framework whereby drugs approved in some other countries, including the US, will be fast-tracked for approval by the MHRA. This is good news, although it is not immediate, and the Chancellor’s speech states that this will be implemented from 2024.

Our letters to the MHRA & Secretary of State for Health

To make Omaveloxolone available in Great Britain, Reata will need to apply to the MHRA (UK regulators).

In March 2023, we wrote to the Chief Executive of the MHRA and Steve Barclay, the Secretary of State for Health and Social Care, to request their support in getting Omaveloxolone approved for use in the UK as soon as possible. We stressed that whilst we welcome the commitment to developing the new fast-tracked approval process described above, for those with FA, access to this drug cannot come soon enough. We argued that when Reata submit an application to the MHRA, this should be expedited in advance of the proposed timeline of 2024 for the changes to the approval process.

Our letter to Reata Pharmaceuticals

We have been meeting regularly with Reata over the last few years and will continue to do so. When necessary, we have been writing letters to Reata’s CEO from our Chair of Trustees and Chief Executive.

Back in December 2022, we wrote to the CEO of Reata Pharmaceuticals to highlight the opportunity to apply for approval in the UK under the MHRA ‘light-touch review’ process used for drugs approved by the EMA (the EU regulators). Given the planned changes to the MHRA processes described above and the fantastic news that SKYCLARYS ® was approved by the FDA in February 2023, we wrote to the CEO of Reata Pharmaceuticals again in April 2023.

In this letter, we urged Reata to submit an application to the MHRA as soon as possible. We informed them that although the proposed fast-tracked process is due to start in 2024, we have written to both the CEO of the MHRA and the Secretary of State for Health and Social Care to ask for this system to be expedited in the case of Omaveloxolone, due to the urgent need for the drug to reach people with FA. We reiterated our commitment to do all that we can to facilitate them in getting regulatory approval in the UK.

We will continue to push for Omaveloxolone approval in the UK as a priority, and to update the FA and ataxia community on our activities towards this important goal.

Please let other people and families affected by FA know about our dedicated newsletter. 

Changes in UK drug regulatory system – relevance to UK approval of Omaveloxolone. Ataxia UK welcomes the UK government’s announcement to speed up access of new drugs in the UK.

To make Omaveloxolone (brand name in the US SKYCLARYS ®) available in Great Britain, Reata Pharmaceuticals will need to apply to the UK regulators (the MHRA). We reported previously on the post-Brexit agreement for fast-track approval by the MHRA of drugs that have been approved by the EU regulators (EMA). Under the new plans, the MHRA is forming a new international framework whereby drugs approved in some other countries, including the US, will be fast-tracked for approval by the MHRA. This is good news, although it is not immediate, and the Chancellor’s speech states that this will be implemented from 2024.

The situation in Northern Ireland is currently different as drugs go through the EMA approval rather than the MHRA. Under the new Windsor Framework, which is a proposed post-Brexit legal agreement between the European Union and the United Kingdom announced on 27th February 2023, this would change and medicines would be regulated by the MHRA like the rest of the UK.

For more information please see this gov.uk website.

Ataxia UK sets up a Friedreich’s Ataxia Special Interest Group to drive getting UK approval forward.

Ataxia UK has set up a Friedreich’s Ataxia Special Interest Group, which includes clinical experts, Ataxia UK staff, and parents of children with FA, with a goal to help drive forward the approval process and support Reata Pharmaceuticals in their efforts.

The group is meeting regularly and is developing an action plan. Please make sure you sign up to our dedicated newsletter and keep an eye on your inbox as there may be actions you can take to help support this action plan in the future.

Current Situation
Ahead of the FDA approval on the 28th February 2023, Reata applied for approval to the EU regulators (The EMA) at the end of 2022. This process usually takes around a year.

If the drug is approved by the EMA, it will apply to the EU and for the time being, Northern Ireland.

To make Omaveloxolone (brand name in the US SKYCLARYS ®) available in Great Britain, Reata Pharmaceuticals will need to apply to the UK regulators (the MHRA). Under a scheme announced post-Brexit, the MHRA currently conducts a ‘light touch review’ of drugs approved by the EMA. This means the same information can be submitted to the EMA and the MHRA, and the MHRA review will be conducted within the shorter timeframe of 67 days.

If the MHRA approves the treatment, different bodies will then decide whether it can be provided by the NHS (e.g., NICE for England and Wales, and the Scottish Medicines Consortium for Scotland). We will keep you updated on the progress of this important issue and hope that Omaveloxolone will be available for FA patients in the UK as soon as possible.

Thank you for being a part of this journey.

We were delighted to report the news yesterday that the regulators in the US (the FDA) have approved Omaveloxolone for adults over 16 in the US with Friedreich's ataxia (FA).

This is the first approved treatment for FA, and a very exciting moment for the ataxia community. Following the approval yesterday, some further details have now been announced. We will continue to use this Omav newsletter to send updates as soon as we receive them.

Reata Pharmaceuticals held a webinar on 28 February where they provided some further information about the approved treatment (which now has teh brand name SKYCLARYS®), and answered questions. You can view the full webinar at this link. 

Some key points from the webinar:

• Reata Pharmaceuticals expect Omaveloxolone to be available to adults over 16 in the US in May or June 2023.
• They are speaking with regulators about how the treatment can be approved for those under 16, and are discussing plans for a paediatric trial in Europe. When we know more, such as the location of the trial sites, we will update the community.

You can read the full press release from Reata Pharmaceuticals by following this link. 

This SKYCLARYS website contains additional information, mainly for people with FA in the US. 

Reata applied for approval to the EU regulators (The EMA) at the end of 2022. If approved by the EMA, this approval will apply to the EU and Northern Ireland. For Omaveloxolone to be available in Great Britain, Reata Pharmaceuticals will need to apply to the UK regulators (the MHRA). 

We are delighted that the FDA has approved Omaveloxolone for the treatment of adults with Friedreich’s ataxia (FA) in the US.

As the first drug approved to treat FA, this is a huge step for the entire community, and we are so pleased that people living with FA in the US will soon have access to this treatment. See the press release below with more information on the announcement from Reata.

However, we are obviously keen for people with FA to potentially get access to the drug in the UK too. Ataxia UK is doing everything it can to support and encourage Reata Pharmaceuticals to seek approval for Omaveloxolone in the UK. We were very pleased to hear that Reata submitted an application to the regulatory agency in Europe (the EMA) at the end of 2022. However, if the EMA approves Omaveloxolone, this will not automatically result in approval in the whole of the UK, but only in Northern Ireland. For approval in Great Britain, Reata Pharmaceuticals will have to apply to the UK regulators (the MHRA).

Under a scheme announced post-Brexit, the MHRA currently conducts a ‘light touch review’ of drugs approved by the EMA. This means the same information can be submitted to the EMA and the MHRA, and the MHRA review will be conducted within the shorter timeframe of 67 days. Earlier this year, Ataxia UK’s Chair of Trustees wrote to the CEO of Reata Pharmaceuticals, encouraging them to make use of this scheme, which is currently due to expire in December 2023. Submitting an application to the MHRA under this scheme will result in faster approval in the UK, bringing this treatment to people with FA in the UK sooner.

We will ensure the FA community in the UK is kept up to date with the latest developments as soon as possible while remaining confidential when needed. The interests of families affected by FA, and all other types of ataxia, in the UK, are and will continue to be at the forefront of all our efforts.

Read the latest press release in full here.

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.

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.

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. 

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: 

1. 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.
2. 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.
3. 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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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/