The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency adopted a positive opinion for Spinraza™, a first step towards market authorisation.
The European Medicines Agency (EMA) Committee on Medicinal Products for Human Use (CHMP) issued a positive opinion on the Marketing Authorisation Application (MAA) in Europe for Spinraza™ (or nusinersen) for all types of SMA. Following this opinion, the European Commission has 2 months to decide whether to validate the marketing authorisation. The opinion of the CHMP is merely advisory but it is rare for the European Commission to decide not to approve a drug if it has received a positive opinion from CHMP.
Once the medicinal product is authorised at European level, steps remain to be taken within each country before it becomes available. The MAA only validates a positive benefit / risk ratio, i.e. the fact that the drug “does more good than harm”. Then, each country must evaluate the value of the drug for its patients, decide to reimburse it and negotiate a price.
In France, two different committees of the French National Authority for Health (HAS), are required to render opinions to evaluate the therapeutic and economic value of the medicinal product. Only then will the Economic Committee for Health Products negotiate a price that will allow standard access to the drug. This whole process will take time. For a medicine such as SpinrazaTM, the delay could be one year, or even longer, after the AMM decision.
The opinion of the CHMP has no influence on early access to Spinraza™ in the form of Temporary Use Authorisation (TUA). Until the MAA, it is possible that a cohort TUA is granted by the National Agency for the Safety of Medicines (ANSM). This type of authorisation would allow access to treatment to a large number of people without going through a case-by-case validation, as is the case for a nominated TUA.
