NHS England has been granted access to the world’s most expensive drug to treat infants with a rare and often fatal disease.
Spinal x-ray
A draft recommendation from the National Institute for Health and Clinical Excellence (NICE) means that the NHS will now be able to access Zolgensma – a £1.79 million gene therapy developed by AveXis and acquired through Novartis to treat spinal muscular atrophy (SMA).
SMA is a rare, inherited, neuromuscular disease that affects around one in every 10,000 births. It’s caused by the lack of a functional SMN1 gene, resulting in the loss of muscle functions, including breathing and basic movement. If untreated, SMA Type 1 can lead to death or the permanent need for ventilation by the age of two.
Zolgensma is a one-time gene therapy for neuromuscular conditions and works by replacing the function of the faulty SMN1 gene. The gene therapy is not designed as a cure however, but rather to halt the progression of the disease. A small-scale clinical trial examining the long-term efficacy of the drug showed that Zolgensma was able to help infants avoid the need for ventilation and be able to walk, sit up, and maintain motor function’s.
Novartis Gene Therapies worked with NHS England and NICE to come to an agreement regarding the cost-effectiveness of Zolgensma. NICE bases its drug approvals of value for money, examining the benefits that a drug offers to the Quality Adjusted Life Years (QALYs).
NICE is currently in the process of finalising guidance for the use of Zolgensma. However, draft recommendations issued by NICE will expedite access for patients during this process. For children with Type 1 SMA who are currently outside of the recommendations, a multidisciplinary team will assess if they can be given the treatment, depending on if they are within the scope of marketing authorisation. If it’s determined that a child is clinically appropriate for Zolgensma, then NHS England will reimburse the costs.
Professor Francesco Muntoni, director of the Dubowitz Neuromuscular Centre at the UCL Great Ormond Street Institute of Child Health and Great Ormond Street Hospital Trust, London said: “This important new advice will enable clinicians in England to have access to this extremely promising novel therapy, which will have a major positive impact for the lives of babies and children diagnosed with this devastating neurodegenerative condition, by addressing its genetic root. Having seen first-hand the impact of severe SMA on infants and their families, this decision offers a glimpse of the potential of one-time gene therapy to replace the burden of years of repeated chronic treatments or palliative care.”
“This ground-breaking gene therapy offers real hope for the future. We truly welcome this decision, so parents in England whose children are covered by this agreement will now be able to discuss a choice of NHS-funded drug treatments with their clinician,” added Liz Ryburn, Support Services manager of community support and advocacy organisation Spinal Muscular Atrophy UK. “We will continue to advocate for this therapy to be fully accessible as a treatment option in the UK for all children for whom it is clinically safe and potentially beneficial.”
Sally-Anne Tsangarides, general manager at Novartis Gene Therapies in the UK, added: “We thank the entire SMA community of families and clinicians for their efforts to help NICE and NHS England appreciate the unmet need in SMA. We are delighted for them that Zolgensma will soon become available to the families in England who need it. Breathing and feeding independently, and developmental milestone achievements like sitting and standing, which have been seen in infants treated with a one-time dose of Zolgensma in clinical studies, are unprecedented in the natural history of the disease.”