The drug is a one-off gene therapy, Zolgensma which is manufactured by Novartis Gene Therapies and it is used to treat Spinal Muscular Atrophy, a rare and often fatal genetic disease that causes paralysis, muscle weakness, and the progressive loss of movement. 

The current world’s most expensive drug will be available in Britain’s health service to be used for babies and young children suffering from SMA; infants born with severe type 1 SMA which is the most common form of the condition have a life expectancy of only 2 years. Britain’s health service provides medical care for free at the point of delivery and will be providing the gene therapy “at a price that is fair to taxpayers after a landmark confidential deal struck by NHS England,” its Chief Executive Simon Stevens announced, however, NHS England did not disclose the price paid.

The gene therapy has helped babies to reach milestones like breathing without the assistance of a ventilator, sitting up on their own, as well as crawling and walking after receiving a single infusion treatment, according to studies. Data suggests that it provides rapid and sustained improvement in motor function as well as prolonging the lives of young children with type 1 SMA. According to the NHS, as many as 80 babies and young children could potentially benefit from this gene therapy annually. 

Treatment involves a single intravenous infusion which contains a replica of the missing gene SMN1, according to the NHS, and the active ingredient, onasemnogene abeparvovec, passes into the nerves to restore the gene which then produces proteins that are essential for nerve function and controlling muscle movement. 

“This deal is a life-changer for youngsters with this cruel disease and for their families,” NHS England chief executive Sir Simon Stevens said.

“Spinal Muscular Atrophy is the leading genetic cause of death among babies and young children, which is why NHS England has moved mountains to make this treatment available, while successfully negotiating hard behind the scenes to ensure a price that is fair to taxpayers,” CEO added.

The deal struck between the NHS and Novartis Gene Therapies secures this treatment at a substantial confidential discount and opens the path for the National Institute for Health and Care Excellence to publish guidance recommending treatment with Zolgensma. Current terms mean that some young children outside of the NICE recommendation criteria may also be eligible for treatment consideration by a national clinical team of leading experts in the treatment of SMA. 

This approval is the second medical treatment available for children with the condition. Previously Spinraza was approved by the NHS for eligible patients in May of 2019. According to the NHS, the agency is ready to fast-track the introduction of the highly complex and innovative gene therapy. Additionally NICE is also reviewing another promising SMA drug.

World’s most expensive drugs, as of March 2021:

*This list includes prescription drugs and drugs only administered by a healthcare professional. Quantity of dose may depend on weight or body surface area of the individual, meaning that prices can vary. Source: GoodRx