The US Food and Drug Administration (FDA) has rejected PTC Therapeutics’ marketing application for Duchenne drug ataluren (Translarna).
The FDA’s decision was based on the recommendation of an advisory panel that met on 28 September. Ten of the eleven panel members agreed that the data provided was inconclusive and more work is needed to establish whether Translarna is effective.
This does not affect the availability of Translarna in the UK, as drugs in European countries are regulated by the European Medicines Agency (EMA). Translarna gained its conditional approval from the EMA in 2014. This made it possible for PTC to market Translarna in Europe while it collects more data on its effectiveness. Conditional approvals are renewed on a yearly basis until certain obligations have been fulfilled by the pharmaceutical company, at which point the approval is converted into a normal approval.
In England, Translarna is available through a Managed Access Agreement. This is an agreement between PTC and NHS England, which allows Translarna to be available until 2021 at a negotiated price to the NHS. Under this agreement, PTC is collecting further evidence on the effectiveness of Translarna using the NorthStar database.
PTC first applied to the FDA in 2011 but received a ‘Refuse to File’ letter stating that the application was not sufficiently complete to review. The company appealed the FDA’s decision but had this appeal denied.
PTC applied for a second time in 2016 but were again rejected and denied an appeal. The company then escalated its appeal to the next supervisory levels of the FDA, which led to the application finally being accepted for review.
Nic Bungay, Director of Campaigns, Care and Information at Muscular Dystrophy UK, said:
After Translarna became the first drug approved in Europe to address an underlying genetic cause of Duchenne, the NHS and other relevant UK authorities showed faith in the drug’s potential. They created a special five-year programme to allow children to access it – only the second ever of its kind. That programme is well underway, and will give us a clearer understanding of the drug’s efficacy and the difference it makes to children with Duchenne who receive it over a period of several years.
Our thoughts today are with eligible children in the US and their families, and we urge the FDA to explore similar options that would allow access to ataluren, while further evidence is gathered.
If you have any questions, please get in touch with our Campaigns and Engagement Manager, Clare Lucas on 02078034838 or email firstname.lastname@example.org