Conditional application of Zolgensma gene therapy

Question for written answer  E-001026/2021
to the Commission
Rule 138
Raffaele Fitto (ECR)

On 19 May 2020 the Commission granted conditional approval for onasemnogene abeparvovec, better known by its trade name Zolgensma, a gene therapy for the treatment of patients with spinal muscular atrophy (SMA) and clinical diagnosis of SMA type 1 or of patients with SMA who have up to three copies of the SMN2 gene.

The European approval concerns infants and children with SMA weighing up to 21 kg (according to the approved dosage) and applies to all 27 EU Member States.

Many Member States, while respecting the principle of autonomy of countries’ individual regulatory agencies, have implemented the 21 kg weight instruction set out in the conditional approval. In Italy, however, AIFA has - under its approval of 17 November 2020 - reserved the treatment only for patients up to the age of six months. To date, only three children have been able to receive the therapy.

In the light of the above, can the Commission say:

• 1.whether it is aware of the different treatment applications in the Member States;
• 2.whether it intends – while respecting the autonomy of individual regulatory agencies in the Member States – to take any measures to ensure that the treatment is fully effective throughout the EU, by encouraging a more uniform application of the therapy in accordance with the guidelines provided by the EMA and allowing access to the many child patients and their families who, at present, are excluded?