The National Institute for Health and Care Excellence (NICE; UK) has published draft guidelines on the use of onasemnogene abeparvovec (Zolgensma^®) for the treatment of spinal muscular atrophy (SMA) in the NHS in England and Wales. The Scottish Medicines Consortium (Glasgow, Scotland) has also approved the use of the gene therapy, which is made by Novartis Gene Therapies (formerly AveXis).

“SMA is a very serious, debilitating and distressing condition that has very significant effects on every aspect of life of those with SMA, and their families and carers,” explained Meindert Boysen (Centre for Health Technology Evaluation at NICE).

The drug currently recommended by NICE for the treatment of pre-symptomatic SMA and SMA Types 1, 2 and 3 is the antisense oligonucleotide nusinersen, which must be taken on a regular basis.

Zolgensma is administered as a single intravenous infusion and has been approved for use in babies up to 12 months old with Type 1 SMA. The NICE draft guidance also recommends the use of Zolgensma in babies with pre-symptomatic SMA as part of a managed access arrangement. For children aged 7–12 months, their treatment will need to be discussed by a national multidisciplinary clinical team due to limited clinical data available for this age group.

“Zolgensma represents an important development in treating SMA which could not only allow babies to gain important motor milestones such as independent sitting and walking, but for some babies who are diagnosed before they have symptoms, it might come close to being a cure,” commented Boysen.

The gene therapy costs £1.79 million per dose. “As is the case with many new treatments for very rare diseases, limited evidence means there are uncertainties about the long-term benefits of Zolgensma,” continued Boysen. “The collaborative effort of all involved in the evaluation has made it possible to recommend a treatment which, at its list price, is the most expensive drug NICE has ever evaluated, allowing the lives of babies who might otherwise have died before their second birthday to be transformed.”

“We expect the pharmaceutical industry will work on improving its efficiency to deliver such high-cost innovations at lower cost, which is essential for the sustainability of healthcare systems around the world,” concluded Mark MacGregor (Scottish Medicines Consortium).

Link:https://www.regmednet.com/zolgensma-gene-therapy-for-spinal-muscular-atrophy-approved-for-use-in-the-uk/