Santhera debuts DMD drug Agamree in Germany

Santhera’s new treatment for Duchenne muscular dystrophy Agamree has been launched in its first market, Germany, following its EU approval last month.

The Swiss biotech said Agamree (vamorolone) is the only approved medication in the EU that has been cleared to treat all patients from four years of age with the muscle-wasting disease, and the first DMD treatment approved across the US, EU, and the UK, where it was given a green light last week.

In trials, the drug 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. It binds to the same receptor as steroids, but avoids a downstream pathway associated with toxicity.

“This significant milestone represents Santhera’s commitment to fill the unmet need in DMD and provide a safe and effective treatment for DMD patients,” said Dario Eklund, Santhera’s chief executive (pictured above).

The first launch of Agamree also marks a turnaround in fortunes for Santhera, whose future was in doubt in 2020 after its earlier DMD candidate idebenone failed a pivotal trials programme and was abandoned.

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

Eklund said the German launch represents a “leap forward” as Santhera enters the commercial stage with its first product. He added that the company is continuing to work towards a “staged commercial roll-out in the large European markets, alongside ongoing discussions with distribution partners for other regions.”

In the US, Agamree was approved last October for DMD patients aged two years and over and is scheduled to be launched in the first quarter of this year by Catalyst Pharmaceuticals.

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

Santhera chose to forego its own revenue stream from the drug in North America in return for a cash injection to reduce its debt and future sales-based milestones and royalties. In Europe, it intends to focus its efforts on six key markets – Germany, France, Italy, Spain, the Benelux countries, and the UK – and seek commercial partners elsewhere, with Catalyst having right of first negotiation.

The US company is viewed as a good partner for the product as it already sells epilepsy therapy Fycompa (perampanel) and Firdapse (amifampridine) for muscular disease Lambert-Eaton myasthenic syndrome (LEMS).

Meanwhile, Santhera is also continuing to develop vamorolone for smaller indications, including Becker muscular dystrophy and other rare paediatric diseases, as an alternative to steroids.