The drug treats Spinal Muscular Atrophy (SMA), a rare and often fatal genetic disease
The latest data suggested that Zolgensma can provide rapid and sustained improvement in motor function for young children with type 1 SMA
The 'most expensive drug in the world' has been approved by the United Kingdom's National Health Service (NHS) to cure a rare genetic disorder. The one-off gene therapy Zolgensma, manufactured by Novartis Gene Therapies, has a reported list price of ₹18 crore (£1.79 million) per dose, NHS England said in a statement Monday.
The drug treats Spinal Muscular Atrophy (SMA), a rare and often fatal genetic disease that causes paralysis, muscle weakness and progressive loss of movement. It will be used for babies and young children suffering from SMA. Babies born with severe type 1 SMA – the most common form of the condition – have a life expectancy of just two years.
Zolgensma, has helped babies to reach milestones such as breathe without a ventilator, sit up on their own and crawl and walk after a single infusion treatment, showed studies.
The treatment is given as a single intravenous infusion and contains a replica of the missing gene SMN1, NHS said. The active ingredient onasemnogene abeparvovec passes into the nerves and restores the gene, which then produces proteins essential for nerve function and controlling muscle movement, it explained.
The latest data suggested that Zolgensma can provide rapid and sustained improvement in motor function for young children with type 1 SMA and prolong their lives.
“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 approval of the drug marked the second medical treatment now available for youngsters with SMA, after Spinraza became available on the NHS to eligible patients in May 2019.The NHS is ready to fast-track the introduction of the highly complex and innovative gene therapy, it said.