Santhera Pharmaceuticals has achieved significant milestones for its drug Agamree (vamorolone), securing a positive recommendation from the National Institute for Health and Care Excellence (NICE) in the UK and approval from China's National Medical Products Administration (NMPA). These decisions pave the way for the treatment of Duchenne muscular dystrophy (DMD) in patients aged four years and older.
NICE Recommends Agamree for NHS Use
NICE has recommended Agamree for use within the National Health Service (NHS) in England, Wales, and Northern Ireland. This decision is expected to make the drug available to approximately 1,700 individuals with DMD. The recommendation follows a revised agreement that includes a price discount, addressing previous concerns about cost-effectiveness compared to existing corticosteroid treatments. Emily Reuben, Chief Executive of Duchenne UK, expressed pride in the availability of a treatment that they have long supported within the NHS.
Professor Michela Guglieri, Consultant Neurologist at Newcastle Hospitals NHS Foundation Trust, noted that Agamree represents a significant advancement in DMD treatment, offering an alternative to slow disease progression while reducing side effects associated with traditional corticosteroids.
China NMPA Approves Agamree
In addition to the NICE recommendation, Agamree has received approval from China's NMPA for treating DMD in patients aged four and older. This marks the first approved treatment for DMD in China, where over 70,000 families are affected by the condition. The NMPA's decision highlights the drug's clinically significant safety benefits, including improved bone metabolism, density, and growth compared to standard corticosteroids.
The approval was granted through the priority review program, reflecting the government's focus on rare disease drug development. Santhera has partnered with Sperogenix Therapeutics for the development and commercialization of Agamree in China. Yan Zhiyu, chairman and CEO of Sperogenix, emphasized the company's commitment to addressing the urgent unmet needs of rare disease patients in China.
Clinical Evidence and Safety Profile
Agamree's efficacy and safety are supported by data from the pivotal VISION-DMD study. The study met its primary endpoint, demonstrating a statistically significant improvement in Time to Stand (TTSTAND) velocity compared to placebo at 24 weeks (p=0.002). The drug also exhibited a favorable safety and tolerability profile, with commonly reported side effects being generally mild to moderate in severity. Notably, available data suggest that Agamree, unlike traditional corticosteroids, does not restrict growth or negatively impact bone metabolism.
About Agamree (Vamorolone)
Agamree is a novel dissociative steroid with a unique mode of action. It binds to the same receptor as glucocorticoids but modifies its downstream activity. Unlike corticosteroids, Agamree is not a substrate for 11β-HSD enzymes, potentially reducing local drug amplification and associated toxicity. This mechanism allows Agamree to dissociate efficacy from typical steroid safety concerns, positioning it as an alternative to existing corticosteroids for children and adolescents with DMD.
Duchenne Muscular Dystrophy (DMD)
DMD is a rare, inherited X-chromosome-linked disease primarily affecting males. It is characterized by chronic inflammation leading to 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 or cardiac failure.
