Santhera preps UK Duchenne drug launch after NICE says yes

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Santhera Pharmaceuticals will launch its new drug for Duchenne muscular dystrophy in the UK within the next few weeks, after getting confirmation it will be covered by the NHS in all four nations.

The announcement follows finalised recommendations this week that backed Agamree (vamorolone) as a treatment for patients aged four and over with DMD from the National Institute for Health and Care Excellence (NICE), covering England, Wales, and Northern Ireland, as well as the Scottish Medicines Consortium (SMC).

The Swiss pharma company said that it expects to make its first sales of Agamree in the UK later this quarter, after launching the drug in its first market, Germany, almost exactly a year ago. It is also available in Austria and in the US through licensee Catalyst Pharma. It has also been approved in the EU, China, and Hong Kong.

The pending launch continues a turnaround in fortunes for Santhera, which was forced to slash staff and restructure its operations after its first DMD candidate, idebenone, failed a phase 3 trial in 2020.

The company licensed in a three-way deal with fellow Swiss firm Idorsia (formerly Actelion) and US biotech ReveraGen BioPharma shortly afterwards, saying at the time that it expected vamorolone to have peak sales potential of around $500 million a year.

Catalyst, meanwhile, licensed US, Canadian, and Mexican rights to the drug last year in a deal worth up to $231 million, including $90 million upfront, whilst also taking an option on some European markets and Japan.

In Europe, Santhera intends to focus its efforts on six key markets – Germany, France, Italy, Spain, the Benelux countries, and the UK – and seek commercial partners elsewhere. Earlier this month, Santhera signed an exclusive agreement with Clinigen to manage supply and distribution in countries where the product is not otherwise commercially available.

Vamorolone is a novel and fully synthetic glucocorticoid that has been shown in trials to slow down muscle degeneration and prolong the ability of DMD patients to walk without causing the weight gain, bone weakness, and mood changes associated with the use of regular corticosteroids like prednisone – a standard therapy for DMD. It binds to the same receptor as steroids, but avoids a downstream pathway associated with toxicity.

In its final guidance, NICE said that Agamree improves muscle function compared with placebo, but it is uncertain whether the drug is similar at improving muscle function outcomes, and how well it works in the long term, compared with currently available corticosteroids.

"Compared with prednisone, the evidence suggests that vamorolone is likely to have fewer side effects, although to what extent is uncertain," continued NICE, noting that the discount on Agamree's list price of £4,585.87 per 100ml of a 40mg/ml oral suspension means that likely cost-effectiveness estimates "are within the range that NICE considers an acceptable use of NHS resources."

NICE has estimated that there are around 1,700 patients in England alone who could be eligible for treatment with the drug.