England’s National Institute for Health and Care Excellence (NICE) has recommended Santhera Pharmaceuticals’ treatment for Duchenne muscular dystrophy (DMD) Agamree (vamorolone) following a new deal that includes a discount on the price of the drug.
The positive final guidance by England’s cost-effectiveness watchdog opens Agamree’s use to around 1,700 people with DMD on the National Health Service (NHS), as per a 10 December press release. Patients aged four and older with the genetic disorder, that primarily affects boys, will qualify for the treatment.
Shares in Santhera opened 5.2% higher on 10 December compared to a pre-announcement market close. The Switzerland-headquartered pharma has a market cap of $108m.
According to NICE recommendation rules, Santhera expects Agamree to be funded and available on the health system within 90 days. The company added it is also working with the Scottish Medicines Consortium (SMC) on a reimbursement process to secure access for patients in the country.
NICE’s decision marks a contrast to a preliminary one it made in March this year when it decided not to approve Santhera’s drug due to concerns around cost-effectiveness compared to currently available corticosteroids. The agency also stated a lack of evidence to conclude Agamree was a cost-effective treatment option.
NICE stated that after an improved discount on the price of the drug, along with “new analysis” presented by Santhera, it could approve Agamree as a cost-effective use of NHS resources.
Santhera oversaw the drug’s approval in the UK in January this year, following similar nods by the US Food and Drug Administration (FDA) and European Commission (EC) in October 2023 and December 2023, respectively. While it is approved in the same indication in Europe, patients aged two years and older in the US have access to the treatment.
DMD is present from birth, with symptoms usually appearing by three years of age. The disease leads to a gradual decline in muscle function with children often needing to use a wheelchair by early adolescence, even eventually requiring artificial ventilation to breathe.
Agamree became the first drug available in the UK approved to treat patients with the neuromuscular disease regardless of the type of mutation they may have. PTC Therapeutics’ Translarna (Ataluren), approved on the NHS in January 2023, can be prescribed only for DMD patients with the nonsense mutation.
Agamree was created as an alternative to existing corticosteroids, also called glucocorticoids, which are routinely used in the treatment of DMD. While these drugs can reduce muscle inflammation and maintain muscle strength, they have several side effects. Agamree’s ability to keep efficacy whilst showing fewer side effects was a key feature appraised by NICE.
NICE’s director of medicines evaluation Helen Knight said: “The committee felt Agamree could offer important benefits to patients and their families because of its potential to reduce adverse events associated with current steroid treatments.”
Duchenne UK’s chief executive Emily Reuben added: "We are very proud that a treatment that we have supported for so long will now be available in the NHS. The approval of Agamree is the culmination of a global effort of scientists, clinicians and patient advocacy groups investing in and supporting the development of Agamree."
