NICE Backs Zolgensma for Type 1 SMA Infants, Managed Entry Agreement Set Up for Use in Types 2, 3

LONDON, United Kingdom – The National Institute for Health and Care Excellence has recommended Novartis’ Zolgensma (onasemnogene abeparvovec) as an option for treating 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of type 1 SMA in babies only if they are six months or younger or they are aged seven to 12 months, and their treatment is agreed by the national multidisciplinary team.

In both, permanent ventilation for more than 16 hours per day or tracheostomy must not be needed. For infants aged seven to 12 months, the team must develop auditable criteria to enable treatment with Zolgensma, and the gene therapy must provide them at least a 70% chance of sitting independently.

In addition, Zolgensma is recommended as an option for treating presymptomatic 5q SMA with a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene in babies, only if the conditions of the managed entry agreement (MEA) are followed.

Clinical trial evidence suggests Zolgensma is effective in type 1 SMA babies who are six months or younger, but the studies are too small and do not allow for a comparison with other treatments, including the NHS comparator, Biogen’s Spinraza (nusinersen). Further, there is limited clinical evidence on Zolgensma in type 1 SMA babies who are older than six months at the start of treatment; however, clinical experts advise that some babies aged seven to 12 months would equally benefit from treatment.

Despite cost-effectiveness estimates being uncertain, they are likely to fall within the range of what NICE considers an acceptable use of NHS resources for highly specialized technologies, particularly when used for babies six months and younger and those aged seven to 12 months.

The list price for Zolgensma is £1,795,000 (excluding VAT), but the company has a commercial arrangement in place making the gene therapy available to the NHS at a confidential discount.

As it stands, there is no evidence on Zolgensma in babies with types 2 or 3 SMA with up to three copies of the SMN2 gene—nor is there evidence on its use in type 1 SMA babies who were treated with Spinraza. Clinical trials are ongoing, but NICE cannot make a recommendation. But, since it could provide value for money within the context of a highly specialized service, NICE is recommending an MAE while further data are collected.

The MAE is to aid in describing the uncertainties identified by the NICE Highly Specialized Technology (HST) committee, patient eligibility criteria, and the arrangements and responsibilities for further data collection intended to capture the data that may address these uncertainties. Data collection under the MAE took effect April 21, 2021 and is anticipated to be submitted to NICE in July 2022.