Following our request for regular updates, Roche is delighted to inform us that today the European Commission (EC) approved Evrysdi™ (risdiplam), the first and only at home treatment for SMA, with proven efficacy in adults, children and babies two months and older.
Risdiplam was reviewed under the European Medicine Agency’s (EMA) accelerated assessment programme, intended for medicines that are of major interest to public health and therapeutic innovation.
Today’s approval means that risdiplam is now authorised in Europe for the treatment of 5q SMA (the most common form of the condition, representing about 95% of all SMA cases 2) in patients 2 months and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies. The decision applies across all 27 European Union member states, as well as Iceland, Norway and Liechtenstein.
Less than five years have passed since the first person enrolled in the risdiplam clinical development programme, which is led by Roche in partnership with the SMA Foundation and PTC Therapeutics. SMA organisations have played a crucial role in this with The SMA Foundation co-leading pre-clinical development and patient organisations in Europe identifying patients for enrolment in clinical trials. Thanks to this determination and resolve from the SMA community, Roche has been able to accelerate progress toward today’s announcement. Meanwhile, more than 3,000 people are being treated with risdiplam in clinical trials, real-world settings and through the compassionate use programme, which is open in more than 60 countries.
Dr. Nicole Gusset, President of SMA Europe says:
"We are delighted with today’s announcement by the European Commission. We are proud to have contributed to the development of Evrysdi and of our partnership with Roche. This has given us the opportunity to ensure that they have a clear understanding and focus on the community’s expectations and unmet needs. Indeed, approval is only one step to access to treatment. The path to access is very different across Europe. A recent survey (EUPESMA) we conducted in our communities, showed that a large proportion of people with SMA in the EU were not receiving an approved treatment, leaving them feeling helpless and frustrated. It is vital that we work together with health authorities, regulators and industry to ensure we can get medicines to all patients who need it, as soon as possible”
This EC approval follows a positive opinion issued in February by the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) and is based on data from two pivotal studies; FIREFISH (in symptomatic infants with Type 1 SMA aged 2 to 7 months) and SUNFISH (in people with Types 2 or 3 SMA aged 2 to 25 years). Both studies have demonstrated the clinically meaningful efficacy and favourable safety profile of risdiplam.
Whilst today marks a significant milestone for the SMA community, Roche is keenly aware of the persisting unmet need that remains in access to treatment options. Roche is working closely with reimbursement and assessment bodies in European countries to enable broad and rapid access for those in need. Our collaborative efforts and the urgent need to treat SMA have resulted in early access pathways to risdiplam, aligned to the European label, in Germany in the coming days and in France from early April through the cohort Temporary Authorisation for Use (ATU). To minimise any delay between EC authorisation and access to risdiplam, Roche has already submitted reimbursement dossiers in many European countries in anticipation of today’s approval.
At the same time, Roche continues to work hard to enable broad and rapid access for patients and families worldwide. In less than eight months, risdiplam has received approval in 38 countries across the world (United States, Chile, Brazil, Ukraine, South Korea, Georgia, Russia, United Arab Emirates, the 27 EU member states and Iceland, Norway and Liechtenstein) and they are pursuing marketing authorisation in a further 33 countries (Argentina, Australia, Botswana, Bolivia, Canada, China, Colombia, Costa Rica, Ecuador, Great Britain, Guyana, Indonesia, Israel, Japan, Kuwait, Malaysia, Mauritius, Moldova, Namibia, New Zealand, North Macedonia, Oman, Pakistan, Paraguay, Peru, Qatar, Saudi Arabia, South Africa, Singapore, Switzerland, Taiwan, Thailand and India – where the first step towards approval was granted). Additional filing in several international countries in the coming weeks and months is also planned.