The oral spinal muscular atrophy (SMA) drug Risdiplam has been approved by the FDA (U.S. Food and Drug Administration).
The treatment, which could be the third for the condition, alongside Spinraza and Zolgensma, will be available in the USA for children and adults with SMA Type 1, 2 and 3 over two months old.
It follows positive results in two clinical trials – FIREFISH in symptomatic infants aged two to seven months and SUNFISH in children and adults aged two to 25 years old.
What is Risdiplam?
Risdiplam – or Evrysdi as it is known in the USA – is an oral drug, which is intended to treat children and adults with SMA. It has been developed through a partnership between Roche, PTC Therapeutics and the SMA Foundation and is marketed by Roche in Europe. It is the only medicine for SMA to date that can be taken at home.
Clinical trials showed Risdiplam helped infants survive without permanent ventilation. Some were also able to sit without support – a key milestone that many children with SMA may not reach.
Risdiplam is the third potential treatment for SMA. Spinraza was approved for use in the NHS last year and is available in many countries across the world, while Zolgensma – a gene therapy – has been given marketing authorisation by the European Commission. It is due to be assessed by NICE for use in England and the SMC for Scotland.
What do the FDA and Roche say?
Roche said the treatment will offer “meaningful benefits” in a press release published on its website yesterday. You can read this here.
Levi Garraway, Roche’s Chief Medical Officer and Head of Global Product Development, said:
The strength and resolve of the SMA community has continually inspired us as we developed this first-of-its-kind medicine for SMA, so today we celebrate our collective accomplishment together with them.
The FDA has said Evrysdi will be available in the USA within two weeks. It had granted the application fast track designation and priority review.
When will the treatment be available in the UK?
The treatment will first need to be considered by the European Medicines Agency (EMA). Its decision will be applicable to all 27 European Union member states, as well as Iceland, Norway, Liechtenstein and the United Kingdom. Roche says an application is imminent.
In the UK, treatments need to be further approved before being available in the NHS. NICE assesses treatments for use in England, while the Scottish Medicines Consortium does so in Scotland. Wales and Northern Ireland typically follow NICE’s guidance. There are no timelines for this as of yet.
What do we think?
This is an important step forward, and it’s encouraging to hear an application will be made to the EMA imminently. If Risdiplam does become available in the NHS in the future, it would – for the first time – give families and individuals living with the condition a choice of treatments.
We will continue to work closely with other SMA charities, Roche, and clinicians, and provide all the information and support the SMA community needs.