The Board of AIFA approved on 9 March 2021 the reimbursement of the Zolgensma gene therapy paid by the NHS for all children with SMA1 under 13,5 kg in weight.
The final go-ahead came at the conclusion of a long process that began in May 2020 with the negotiation request presented by the Novartis company.
In November 2020, after the AIFA Technical Scientific Commission (CTS) had evaluated this therapy as innovative, it was decided to admit it to reimbursement for children up to six months of age in light of the important expected benefits. At the same time, rigorous negotiation by the Price and Reimbursement Committee (CPR) took place in order to define a price for the drug that was sustainable for the NHS.
AIFA declares its satisfaction with the possibility of making available in our NHS a gene therapy that offers a radically better life prospect for children with SMA1.
The agreement with the Novartis company also included the company's commitment to make the drug available free of charge in clinical trials for children weighing between 13,5 and 21 kg, in order to acquire additional efficacy and safety data on these patients in a controlled setting.