NICE Widens Its Zolgensma Appraisal Due to European Marketing Authorization

England’s National Institute for Health and Care Excellence (NICE) is expanding its appraisal of Zolgensma, a gene therapy for spinal muscular atrophy (SMA), according to a press release from SMA UK.

NICE had previously planned to conduct evaluations of clinical trial and cost-effectiveness data, with the end goal of determining whether the treatment should be covered by the National Health Service (NHS), the U.K.’s public health system.

Now, the committee has expanded its planned Zolgensma appraisal to be more in line with the therapy’s recent European marketing authorization.

SMA is a genetic disorder caused by mutations in the gene SMN1. Zolgensma (formerly known as AVXS-101), which was developed by Novartis and its subsidiary AveXis, now Novartis Gene Therapies, works by delivering a non-mutated copy of the SMN1 gene to motor nerve cells.

Zolgensma was granted conditional approval by the European Commission (EC) earlier this year — which applies to the U.K., Iceland, Norway, and Liechtenstein in addition to the 27 EU countries.

The EC approval covered infants and young children who weigh up to 21 kilograms (about 46 pounds) who have a clinical diagnosis of type 1 SMA or have up to three copies of the SMN2 gene. SMN2 is similar to SMN1 and can partially compensate for the mutated gene; in general, more SMN2 copies lead to less severe SMA.

NICE will be holding a committee meeting on October 8 to evaluate Zolgensma’s cost-effectiveness and clinical trial data. Now, its appraisal will include people with 5q SMA and up to three copies of the SMN2 gene. 5q SMA refers to SMA in which both copies of SMN1 — one inherited from each biological parent — are mutated, which account for about 95% of SMA cases.

If evidence allows, consideration also may be given to a sub-group of people who have not yet developed SMA symptoms.

According to the SMA UK release, “This [expansion] widens the possibilities and is what patient groups have been advocating.”

Of note, a recently published consensus statement from a panel of European experts states that the current EC approval, being fairly broad, covers patient groups that have not been specifically studied in clinical trials of the gene therapy. Thus far, the trials have focused on patients treated early in infancy.

The consensus statement is expected to be a factor in NICE’s deliberations concerning Zolgensma.

The NICE meeting will be attended by SMA UK and other patient and clinical representatives.

A final decision from NICE is expected on March 25, 2021.