Both the time it takes to get diagnosed and the cost of treatment may limit the number of infants with spinal muscular atrophy who benefit from Novartis's gene therapy for the condition, according to the Guardian.
The US approved Novartis's Zolgensma (onasemnogene abeparvovec-xioi) in 2019 for children under the age of two who are shown by genetic testing to have a certain type of spinal muscular atrophy. The UK announced in early 2021 that Zolgensma would be available through the National Health Service in England later that year.
But the Guardian writes that diagnosing the disease early enough for the treatment to have the greatest effect can be hard. Because it is so rare — 70 infants a year in the UK have SMA — many may confuse the condition with more common ones like muscular dystrophy or cerebral palsy.
Cost can also be a factor, as the Guardian notes that when Zolgensma was approved in the US, it was the most expensive drug in the world, with a list price of $2.1 million. The Guardian notes that while the UK has negotiated a discount for the drug, families elsewhere must pay full price or enter a lottery to receive free treatment from Novartis.