The UK’s National Institute for Health and Care Excellence (NICE) issued a draft guidance recommending the use of risdiplam for spinal muscular atrophy (SMA), a debilitating genetic disorder that leads to progressive loss of muscle function. The recommendation comes as part of a managed access agreement (MAA) between the NHS, NHS Improvement, and Roche Pharmaceuticals, who manufactures risdiplam under the brand name Evrysdi.
SMA in the UK
Currently 1,200 to 2,500 individuals in the UK live with SMA. Spinal Muscular Atrophy UK estimates that 100 children are born with the disease, which has no cure, every year. New treatment options such as risdiplam are therefore always welcome.
According to NICE, the purpose of the MAA is to make risdiplam available to more people while at the same time collecting data on the drug’s therapeutic usage. After the MAA ends, NICE will decide on recommending routine usage of the drug by the NHS.
The NICE committee deemed risdiplam to be “innovative” based on its oral route of administration. Other treatments for SMA such as the leading drug nusinersen are delivered through intrathecal injection, which isn’t an option for many SMA sufferers. Oral administration means the drug can be given at home and that it doesn’t carry the risk of side effects associated with intrathecal injection such as infection.
As for risdiplam’s effectiveness, studies show that the drug can improve muscle function in people with type 1, 2, and 3 SMA. It may even prolong lifespan for people with type 1 SMA. But current research has not shown a long-term benefit to use of the drug.
Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Evaluation at NICE, praised the new managed access agreement.
“We are pleased that there will now be the option of a convenient oral treatment for people with SMA that can be administered at home. This will not only be less burdensome, and therefore have a positive impact on the lives of both people with SMA and their caregivers, but it will also reduce the treatment administration needs to the NHS,” she said.
“In practical terms, the availability of an oral drug should lead to greater adherence to treatment, along with giving access to a treatment to those who aren’t able to have other currently recommended options.”
Written by Natan Rosenfeld