Price deal opens door to NHS use of Santhera DMD drug

Santhera's Duchenne muscular dystrophy (DMD) therapy Agamree has been cleared for NHS use after a confidential pricing agreement.

The deal means that Agamree (vamorolone) will be available to around 1,700 people with DMD in England as an alternative to corticosteroids that are used to reduce muscle inflammation and maintain muscle strength and function in people with the muscle-wasting disease.

Patients aged four and older with the genetic disorder, which mainly affects boys, will qualify for treatment with the drug.

In trials, Agamree has been shown to slow down muscle degeneration and prolong the ability of DMD patients to walk, without causing the growth stunting and immune suppression associated with the use of corticosteroid drugs – the current standard of care in children and adolescent patients with DMD.

Santhera's drug binds to the same receptor as steroids, but without activating a downstream pathway associated with toxicity.

In final draft guidance – which is due to come into effect in January – health technology assessment (HTA) agency NICE said that Agamree is likely to have fewer side effects than corticosteroids, although, it said the extent of that benefit is "uncertain" and there are also questions about "whether vamorolone is similar at improving muscle function outcomes, and how well it works in the long term."

However, it said that DMD's severity means that Agamree is likely to offer a cost-effective use of NHS resources at the discounted price, according to NICE, which said that a new analysis provided by the company and an improved price offer tipped the balance.

It also claims that Agamree is one of several non-cancer treatments approved using a new severity modifier that has been criticised by the pharma industry.

The HTA asserts that the new variable "allows NICE's independent committees to give greater weight to severe conditions, including conditions such as DMD, that would not have qualified for additional weighting under the previous end-of-life criteria."

According to Santhera, Agamree will now be available to patients in England, Wales, and Northern Ireland, while access negotiations are still ongoing with the Scottish Medicines Consortium (SMC).

"We are very proud that a treatment that we have supported for so long will now be available in the NHS," said Emily Reuben, chief executive of Duchenne UK.

"The approval of vamorolone […] is the culmination of a global effort of scientists, clinicians, and patient advocacy groups investing in and supporting the development of vamorolone," she added.

Agamree was approved by the Medicines and Healthcare products Regulatory Agency (MHRA) in January and has a pre-discount list price of £4,585.87 per 100ml of a 40mg/ml oral suspension. Santhera said it was working with the NHS to ensure patients can access the drug "as quickly as possible."

The drug has also been approved in the EU and US and yesterday got the green light from China's National Medical Products Administration (NMPA).