The Italian Medicines Agency has sent a letter to the President of the Puglia Region, Michele Emiliano, on the case of the two children with SMA1 for whom the administration of Zolgensma therapy is requested, highlighting AIFA’s continuous commitment to guarantee access in the shortest time to all patients who can benefit from it.

“The exclusion from reimbursement of patients with tracheostomy, or with ventilatory support for a number of hours ≥16 per day for 14 consecutive days, or with total loss (100%) of swallowing capacity, derives from the following considerations: the two conditions represented exclusion criteria from clinical trials conducted for approval and, for included subjects, these were indicators of therapy failure. Even in the absence of direct data in such populations, it is documented that the maximum effectiveness of this gene therapy occurs when administration takes place in the first months of life, while it is reduced in older children and in the event of worsening of the disease”, AIFA underlines.

“Any use of Zolgensma in SMA1 patients with tracheostomy is to be considered compatible with the indication authorised by the EMA”, AIFA writes. However, “taking into account that the expected benefits of the aforementioned gene therapy in SMA1 patients with tracheostomy are considered almost absent, and in the face of increasing risks in subjects with advanced disease, AIFA’s Scientific-technical Committee has considered that there are no conditions for admission to reimbursement by the NHS”.

“The multidisciplinary clinical team remains responsible for establishing - in the individual patient and after a careful assessment of the overall conditions - whether it is appropriate to carry out this treatment”, so AIFA. “Should the specialist team of an Italian reference centre consider the patient eligible for treatment, albeit outside the conditions envisaged for the reimbursement by the NHS, the need is not recognised for this treatment to be carried out abroad”.