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 which the administration of Zolgensma therapy is requested, recalling the continuous commitment of AIFA 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, were indicators of therapy failure. Even in the absence of direct data in these populations, it is documented that the efficacy of this gene therapy is maximum when the administration takes place in the first months of life, while it is reduced in older children and in the event of an aggravation of the disease ", underlines AIFA.
“Any use of Zolgensma in SMA1 patients with tracheostomy is to be considered compatible with the indication authorized by the EMA”, writes AIFA. However, "taking into account that the expected benefits of the aforementioned gene therapy in patients affected by SMA1 with tracheostomy are considered almost absent, and in the face of increasing risks in patients with advanced disease, the Technical Scientific Commission of the Agency 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 - writes AIFA - In the event that the specialist team of a center of Italian reference considered the patient suitable for the treatment, although outside the conditions envisaged for the reimbursement of the SSN, the need for such treatment to be carried out abroad is not recognized in any case ".