In October 2019, Reata Pharmaceuticals announced positive top-line results from their MOXIe trial testing Omaveloxolone (or Omav) in Friedreich’s ataxia (FA). The results of this trial have now been published (read the abstract here).
After showing positive results in a trial such as this, the next step is to apply for regulatory approval to market the drug. Since these results were announced, this is what Reata Pharmaceuticals has been working towards in the USA.
In the MOXIe trial, Reata used the modified Friedreich’s Ataxia Rating Scale (mFARS) to measure the symptoms of those taking Omav vs those taking placebo (a placebo is a substance that has no therapeutic effect, used as a control in testing new drugs). The mFARS results showed that patients taking Omav for 48 weeks had a statistically significant improvement in their symptoms associated with FA than those on the placebo trial.
However, the Food and Drug Administration (FDA), which is the drug regulatory body in the USA, suggested that while it does not have concerns that the mFARS data is reliable, it is not convinced that this data will be enough to support approval of Omav based on this trial alone.
The FDA suggested that Reata consider carrying out another trial to confirm the mFARS results from the MOXIe study. Reata has instead proposed a second ‘crossover study’ to provide additional evidence of the effect of Omav. This crossover study would use data from those involved in the MOXIe trial that are still taking Omav as part of an open-label extension study. They reason that this would be a faster way to obtain more information about the effectiveness of Omav. Reata is now waiting to hear if the FDA will accept this approach.
This is clearly very disappointing news for the FA community, and will undoubtedly lead to a delay in the approval of Omav. If the FDA approve the crossover study that Reata suggested, then Reata still hope to apply for regulatory approval in the USA in the first quarter of 2021. However, if the FDA do not approve the approach – or the results of the crossover study are not positive – Reata will need to evaluate whether it is possible to conduct a second trial.
It is worth noting that regardless of these interactions with the FDA, Reata still plans to seek approval outside of the US and we will continue to recommend to them that they apply to the NHS England ‘Early Access to Medicines Scheme’ (EAMS) to enable Omav to be made available here in advance of US approval.
Read the full press release from Reata Pharmaceuticals here.
Posted on 14/08/2020