Santhera's AGAMREE® Approved in Hong Kong for Duchenne Muscular Dystrophy

Santhera Pharmaceuticals, a Swiss company specializing in rare neuromuscular diseases, has announced that the Department of Health (DoH) in Hong Kong has approved AGAMREE® (vamorolone) for treating Duchenne muscular dystrophy (DMD) in patients aged four and older. This approval marks the fifth independent recognition of AGAMREE by health authorities, following similar endorsements from the U.S. FDA, European Medicines Agency (EMA), UK's Medicines and Healthcare products Regulatory Agency (MHRA), and China's National Medical Products Administration (NMPA).

AGAMREE represents a novel approach to DMD treatment, operating through a mechanism that modifies the activity of glucocorticoid receptors without being a substrate for the 11-β-hydroxysteroid dehydrogenase enzymes. This unique mode of action potentially mitigates the risks associated with corticosteroid toxicity, offering a safer alternative to traditional steroids used as the standard of care for DMD.

In a significant clinical trial, VISION-DMD, AGAMREE demonstrated efficacy by achieving its primary endpoint of improving Time to Stand (TTSTAND) velocity compared to a placebo over a 24-week period. The drug also exhibited a favorable safety profile, with most side effects, including cushingoid features, vomiting, weight gain, and irritability, being mild to moderate in severity. Importantly, AGAMREE does not inhibit growth or adversely affect bone metabolism, as evidenced by normal markers of bone formation and resorption.

Duchenne muscular dystrophy is a rare genetic disorder linked to the X chromosome, primarily affecting males. It is characterized by early-onset inflammation leading to muscle fibrosis and progressive degeneration and weakness. Key challenges for those affected include loss of mobility, self-feeding ability, and cardiomyopathy, often resulting in reduced life expectancy due to respiratory or cardiac failure. Corticosteroids have been the traditional treatment option, though they come with significant safety concerns.

The approval in Hong Kong follows a licensing agreement between Santhera Pharmaceuticals and Sperogenix Therapeutics, initially established in January 2022. Sperogenix has the exclusive rights to develop and commercialize AGAMREE for DMD and other rare conditions in Greater China, including Hong Kong, Macao, and Taiwan. In March 2023, the agreement was extended to cover additional Southeast Asian countries such as Brunei, Cambodia, and Vietnam, among others, with Sperogenix making a payment of USD 4 million to secure these rights. By July 2024, Sperogenix had exercised this option, incorporating these territories into their operational strategy.

Santhera will supply AGAMREE to Sperogenix for the ongoing Early Access Program (EAP) and commercial distribution. In return, Sperogenix will pay Santhera royalties in the double-digit percentage range on net sales, along with milestone payments contingent on commercial performance.

Sperogenix Therapeutics, founded in 2019, is committed to bringing genetic and rare disease treatments to the Chinese market, with a focus on neuromuscular and inherited metabolic disorders. The company aims to innovate a commercial model tailored to rare disease management in China, ensuring access to effective therapies for physicians and patients.

This approval in Hong Kong strengthens the global footprint of AGAMREE, positioning it as a promising therapeutic alternative for those affected by Duchenne muscular dystrophy, offering hope for improved management of this debilitating condition.