Recently authorized in Europe, Zolgensma® (onasemnogene abeparvovec) is one of the innovative treatments for SMA, along with Spinraza® (nusinersen) and Evrysdi® (risdiplam). All of these therapies are most effective if they are administered as early as possible.
To encourage the rational prescription of Zolgensma®, a group of European neuromuscular disease experts issued eleven recommendations on:
- the patient selection criteria: age at the onset of the disease, duration of the disease, state of motor function, number of copies of the SMN2 gene, weight at the start of treatment, presence or absence of symptoms at birth, no evidence of superiority of a double treatment (Zolgensma® and Spinraza®) over a single treatment…
- the prerequisites for administering Zolgensma®: expertise of centres carrying out gene therapy injections in accordance with international standards, resources to deal with potential side effects, neonatal screening program in order to be able to begin treatment at a presymptomatic stage, ideally within a period of less than 14 days after the diagnosis …
- additional data to be collected: collection of systematic safety and efficacy data for all patients treated, long-term follow-up, establishment of registers, development of a more rigorous monitoring protocol for more patients of 13.5 kg, close collaboration between laboratories, associations, academy to improve the benefit / risk ratio …
