Patients of all ages with spinal muscular atrophy (SMA) will now be able to be treated with Spinraza (nusinersen) in England, after the National Institute for Health and Care Excellence (NICE) recommended the therapy for funding by the National Health Service (NHS).
NICE’s favorable decision makes Spinraza, marketed by Biogen, the first treatment targeting the underlying cause of SMA to be included in England’s public health program for presymptomatic and symptomatic SMA types 1, 2, and 3.
“This promising treatment has the potential to be life changing for children and their families,” Simon Stevens, NHS England’s chief executive, said in a press release from NICE.
The therapy will be available right away to the youngest and most severely affected patients with SMA type 1, according to NICE.
After the publication of the final guidance document next month, NHS England will begin offering Spinraza to older babies, children, and young adults with less severe symptoms (SMA types 2 and 3).
“We applaud the decision by NICE to recommend funding for SPINRAZA in the United Kingdom. This is a momentous occasion for patients and their families and the result of a strong collaboration between Biogen, NICE, NHS and the SMA community,” Chirfi Guindo, executive vice president of global product strategy and commercialization at Biogen, said in the company’s press release.
NICE is Britain’s independent institution entitled to provide advice and standards for healthcare, including which licensed medicines will be funded by the NHS.
Previously, the agency recommended against Spinraza’s inclusion into the subsidized public health system due to concerns over its long-term effectiveness and high cost. NICE contended that its cost in terms of Quality Adjusted Life Years gained was “too high for it to be considered a cost-effective use of NHS resources.”
But extensive clinical data showing the meaningful benefits of Spinraza for SMA patients of all ages led the agency to reconsider its opinion. Additionally, NHS England and Biogen established an agreement to continue to observe the impact of the NHS-funded treatment for a limited time period, allowing further data to be collected on its effectiveness.
“We are very pleased that we can now recommend nusinersen for people with SMA,” said Meindert Boysen, director of the Centre for Health Technology Evaluation at NICE. “The committee has recognized that nusinersen is a promising treatment that has been shown to improve a range of outcomes important to patients. But it also recognized that there are significant uncertainties, particularly around its long-term benefits.
“Today’s announcement shows that, where companies show appropriate flexibility, it is possible to find a way to provide important treatments to patients in a way that is cost effective for the NHS and taxpayers,” he added.
The latest clinical data showing Spinraza’s long-term efficacy and safety were recently discussed at this year’s American Academy of Neurology (AAN) annual meeting in Philadelphia.
Spinraza not only has shown an ability to improve muscle strength, and motor and respiratory function in adults with SMA, but also results in motor milestone achievements in line with normal motor development in infants in the presymptomatic stage of the disease. In general, treatment with Spinraza has been found to lead to greater improvements in motor function and long-term stability of motor function in patients with SMA.
Spinraza is an antisense oligonucleotide that works by correcting the root cause of 5q SMA, the most common form of the disease, caused by mutations in the SMN1 and SMN2 genes in chromosome region 5q. It instructs cells to increase the production of a full-length survival motor neuron (SMN) protein, which is missing in those with the disease.
The treatment is the only approved therapy for nearly all types of SMA in several countries, including the United States, across the European Union, Australia, Brazil, Japan, Switzerland, South Korea, Canada, and Chile.
Over 7,500 patients with SMA have already been treated with Spinraza in more than 40 countries through expanded access programs, clinical trials, and after it became commercially available.