Santhera Pharmaceuticals' Agamree (vamorolone) has received a positive recommendation from the Scottish Medicines Consortium (SMC) for use within NHS Scotland, and positive final guidance from the National Institute for Health and Care Excellence (NICE) in England, Wales and Northern Ireland. This decision allows for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older. Agamree is now the first and only fully approved treatment for DMD in the EU, US, and UK, marking a significant milestone for patients with this rare genetic disorder.
The approval from the Medicines and Healthcare products Regulatory Agency (MHRA) and the subsequent recommendations are based on clinical trials, including the pivotal VISION-DMD study. These trials demonstrated Agamree's efficacy and a favorable safety profile compared to standard corticosteroids, which are the current standard of care for DMD. The VISION-DMD study met its primary endpoint, Time to Stand (TTSTAND) velocity, versus placebo (p=0.002) at 24 weeks of treatment.
Clinical Benefits of Agamree
Agamree is a novel dissociative steroid that binds to the same receptor as glucocorticoids but modifies its downstream activity. Unlike traditional corticosteroids, Agamree is not a substrate for the 11-β-hydroxysteroid dehydrogenase (11β-HSD) enzymes, potentially reducing corticosteroid-associated toxicities in local tissues. This mechanism allows for the dissociation of efficacy from steroid safety concerns, positioning Agamree as an alternative to existing corticosteroids.
Data indicates that Agamree does not restrict growth or negatively affect bone metabolism, unlike corticosteroids. This is demonstrated by normal bone formation and bone resorption serum markers. Common side effects reported in trials were generally mild to moderate, including cushingoid features, vomiting, weight increase, and irritability.
Duchenne Muscular Dystrophy (DMD)
DMD is a rare, inherited X-chromosome-linked disease primarily affecting males. It is characterized by inflammation leading to muscle fibrosis, progressive muscle degeneration, and weakness. Major milestones in the disease include loss of ambulation, self-feeding difficulties, the need for assisted ventilation, and the development of cardiomyopathy. DMD typically reduces life expectancy to before the fourth decade due to respiratory and/or cardiac failure.
Santhera's Commitment
Dario Eklund, CEO of Santhera, expressed his delight at securing the approvals, stating, "I am delighted that the team has secured this latest approval, which will ensure Scottish patients can benefit from this important treatment for DMD. We will work closely with NHS Scotland, along with all the key regulatory and health authorities in all our approved markets, to ensure patients have access to AGAMREE."
Santhera Pharmaceuticals is a Swiss specialty pharmaceutical company focused on developing and commercializing innovative medicines for rare neuromuscular diseases with high unmet medical needs. They hold an exclusive license from ReveraGen for Agamree (vamorolone) for all indications worldwide.
