Kate Cardente holds her 3-month-old daughter, Ainsley, while she undergoes gene therapy for Spinal Muscular Atrophy (SMA). SMA is a disease that is the leading genetic cause of death in babies. Ainsley is receiving a single infusion of Zolgensma.
Baltimore Sun | Tribune News Service | Getty Images
LONDON – A drug labeled the “most expensive drug in the world” has been approved by the UK’s National Health Service, a measure that can change the lives of babies and children suffering from a rare genetic disease.
The innovative gene therapy called “Zolgensma” does not come cheap, with a list price of £ 1.79 million ($ 2.48 million) per dose, NHS England said in a statement on Monday.
The drug will be used in babies and young children who suffer from Spinal Muscular Atrophy, a rare and often fatal genetic disorder that causes paralysis, muscle weakness and progressive loss of movement.
Babies born with severe type 1 SMA – the most common form of the disease – have a life expectancy of just two years.
The drug will be available at Britain’s health service, which offers free medical assistance at the point of delivery, “at a fair price for taxpayers after a landmark confidential agreement reached by NHS England,” Chief Executive Simon Stevens announced at the Monday. NHS England did not disclose the price paid.
The NHS is funded primarily by the government through general taxes, so the drugs and treatments it approves and uses must undergo rigorous cost-benefit analysis.
Zolgensma, which is manufactured by Novartis Gene Therapies (part of the American pharmaceutical company Novartis), has shown in studies to help babies reach milestones such as breathing without a fan, sitting on their own and crawling and walking after a single infusion treatment.
The most recent data suggest that Zolgensma can provide rapid and sustained improvement in the motor function of young children with type 1 SMA and prolong their lives.
Up to 80 babies and young children a year could potentially benefit from gene therapy, the NHS said.
Life transformer
Stevens said the deal was “life changing for young people with this cruel illness and for their families”.
“Spinal muscular atrophy is the leading genetic cause of death among babies and young children, which is why the NHS England has moved mountains to make this treatment available, while successfully negotiating behind the scenes to ensure a fair price for taxpayers.”
He said the agreement shows that while the health service remains under pressure due to the Covid-19 pandemic, the NHS is still “caring for millions of other patients, for whom real medical advances are now possible”.
The agreement signed with Novartis Gene Therapies guarantees the medication for NHS patients in England with a substantial and confidential discount and opens the way for the National Institute of Excellence in Health and Care (NICE) – the public agency that issues guidelines on the cost relation -benefit of the drugs and treatments used by the NHS – to publish a draft guideline recommending treatment with Zolgensma.
The terms of the agreement mean that some children who are currently outside the NICE recommendation criteria will also be eligible to be considered for treatment by a national clinical team composed of the country’s leading specialists in the treatment of SMA.
The NHS said it is ready to accelerate the introduction of highly complex and innovative gene therapy and will not wait until NICE publishes the final guidelines to get started. This approach is supported by NICE given the importance of administering single treatment as early as possible, he added.
Drug approval marks the second medical treatment now available for young people with SMA. He added that future treatment possibilities also look promising, with another SMA drug being reviewed by NICE.