NICE Recommends Santhera's AGAMREE® for Duchenne Muscular Dystrophy

Santhera Pharmaceuticals has announced that the National Institute for Health and Care Excellence (NICE) has given a positive recommendation for AGAMREE® (vamorolone) to be used within the National Health Service (NHS) in England, Wales, and Northern Ireland for treating Duchenne muscular dystrophy (DMD) in patients aged four years and above. This development is set to enhance treatment options for those affected by this severe form of muscular dystrophy, which predominantly affects males and is characterized by muscle degeneration and weakness, leading to significant health challenges and reduced life expectancy.

Dario Eklund, CEO of Santhera, expressed the company's commitment to ensuring prompt access to AGAMREE for patients, highlighting the urgent need for improved treatments for this devastating condition. Similarly, Emily Reuben, Chief Executive of Duchenne UK, recognized the significance of this approval, attributing it to the collaborative efforts of scientists, clinicians, and advocacy groups dedicated to supporting DMD patients.

AGAMREE is notable for its innovative mechanism, acting on the same receptor as glucocorticoids but modifying its downstream activity, distinguishing it from traditional corticosteroids. This mechanism potentially separates the drug's efficacy from the safety concerns associated with steroids, such as effects on bone metabolism. Notably, AGAMREE has received full approval in the EU, US, and UK, making it a groundbreaking treatment option for DMD. Following the Medicines and Healthcare products Regulatory Agency’s (MHRA) approval earlier in the year, the recommendation from NICE is expected to facilitate funding and availability within 90 days in the regions mentioned.

Professor Michela Guglieri, a Consultant Neurologist, emphasized the importance of this recommendation, noting AGAMREE's potential to provide an alternative treatment that slows disease progression while minimizing side effects commonly associated with corticosteroids. The drug's approval represents a significant advancement in the care of DMD patients across the UK.

Santhera is also pursuing reimbursement through the Scottish Medicines Consortium (SMC) to ensure patients in Scotland can access AGAMREE. This step is crucial for making the treatment widely available across the UK, providing a new hope for families affected by DMD.

The efficacy of AGAMREE was demonstrated in the pivotal VISION-DMD study, where it successfully met the primary endpoint, showing improved Time to Stand (TTSTAND) velocity compared to placebo. The study also reported a favorable safety and tolerability profile, with side effects such as cushingoid features, vomiting, weight increase, and irritability being generally mild to moderate. Importantly, AGAMREE does not restrict growth or negatively impact bone metabolism, unlike traditional corticosteroids, as evidenced by normal bone formation and resorption markers.

Santhera Pharmaceuticals, a Swiss company specializing in innovative treatments for rare neuromuscular diseases, holds an exclusive license for vamorolone from ReveraGen worldwide. The company has out-licensed rights to AGAMREE for North America to Catalyst Pharmaceuticals and for China to Sperogenix Therapeutics. This strategic positioning underscores Santhera's commitment to addressing unmet medical needs in the field of neuromuscular diseases, offering new treatment possibilities for those affected by DMD.