We are delighted to share with you that the Committee for Medicinal Products for Human Use (CHMP), of the European Medicines Agency (EMA), adopted a positive opinion recommending the approval of risdiplam for the treatment of 5q SMA, in people 2 months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3, or with one to four SMN2 copies.
This positive opinion will now be referred to the European Commission (EC), who will review the CHMP recommendation and give its final decision within approximately two months.
The CHMP recommendation is based on data from two pivotal studies, which evaluate the efficacy and safety of risdiplam in symptomatic infants with Type 1 SMA aged 2 to 7 months and in people with Types 2 or 3 SMA aged 2 to 25 years. Both studies have demonstrated the clinically meaningful efficacy of risdiplam with a favourable safety profile. A liquid medicine administered once daily, risdiplam, if approved, will be the first and only treatment for SMA that can be taken at home by mouth or feeding tube, if required.
The Roche team was very grateful to everyone involved, for the trust and ongoing partnership, since the very beginning of the clinical development programme and all along risdiplam's lifecycle. They thank SMA Europe for the support and strategic guidance, which has been invaluable to them throughout this journey. Starting from 2019, when preparing for the Scientific Advice Working Party meeting with EMA, throughout the filing process and more recently, to define a response strategy for the hundreds of questions received from EMA. This advice, along with our robust survey data and official statements have supported filing documents, Accelerated Assessment request, Orphan Drug Designation and responses to EMA's objections. And last but not least, the interventions of our regulatory experts during the various EMA meetings, making sure that all parties have a clear understanding and focus on the community's expectations and existing unmet needs.
Our President, Nicole Gusset, said:
"We are proud that we have a long-established partnership with Roche. This gives us the opportunity to ensure that they have a clear understanding and focus on the community’s expectations and unmet needs. We are happy that we could contribute our share and that we, the SMA community, will soon have another promising product for people living with SMA approved in the EU. Nevertheless, we do not forget that approval is still pending in other countries. In addition, approval is only one step to access to treatment. This path to access is very different across Europe and SMA Europe is committed to ensuring that we have access for as many people with SMA as possible, as soon as possible."