Pharma Focus Asia

Santhera Launches Early Access Program for AGAMREE® in China through Sperogenix Collaboration

Monday, June 10, 2024

Santhera Pharmaceuticals (SIX: SANN) has announced that Sperogenix Therapeutics, its partner, has started a paid Early Access Program (EAP) in China for AGAMREE® (vamorolone), intended for patients with Duchenne muscular dystrophy (DMD).

In April 2024, the Hainan Medical Products Administration (HMPA) granted approval for the EAP, following local regulations and recognizing the drug's existing approvals in the US, EU, and UK. This approval highlights the critical need for treatment options in China, where there are currently no approved therapies for DMD. The EAP commenced in mid-May at the Bo'ao Lecheng Pilot Zone in Hainan Province, where the first patients have begun receiving AGAMREE.

Additionally, the National Medical Products Administration (NMPA) of China accepted the New Drug Application for AGAMREE in DMD for patients aged 4 years and older in March 2024, categorizing it for Priority Review and Breakthrough Therapy designations. If the regulatory review is successful, approval could be granted by the first quarter of 2025.

Duchenne muscular dystrophy affects approximately 70,000 people in China, where there is currently no approved drug, pointing to a significant unmet need. The increasing rates of diagnoses further emphasize the demand for effective treatment options.

As per their licensing agreement, Sperogenix holds exclusive rights for the development and commercialization of AGAMREE in DMD and other rare diseases within China. Santhera will provide the drug for both the EAP and future commercialization, while Sperogenix will compensate Santhera with royalties on net sales and additional milestones based on sales performance.

AGAMREE® (vamorolone) operates by interacting with the same receptors as glucocorticoids but alters their downstream effects. It avoids the action of 11-β-hydroxysteroid dehydrogenase enzymes, which are associated with corticosteroid toxicity. This unique mechanism aims to provide the therapeutic benefits of steroids while minimizing the associated risks, potentially offering a safer treatment option for inflammation in DMD patients.

In the VISION-DMD clinical trial, AGAMREE showed significant improvement in the primary endpoint of standing-to-stand speed compared to a placebo after 24 weeks of treatment, with a favorable profile for safety and tolerability. Most side effects were mild to moderate, including symptoms like Cushingoid appearance, vomiting, weight gain, and irritability.

AGAMREE has received orphan drug status and approval in the US, EU, and UK. It does not impair growth or negatively affect bone health, as indicated by normal markers for bone formation and resorption.

Duchenne muscular dystrophy is a severe genetic disorder predominantly affecting males, linked to the X chromosome. It is marked by early inflammation, muscle fibrosis, and progressive muscle weakness, leading to the eventual loss of mobility, ability to self-feed, need for ventilation, and heart muscle complications. DMD typically shortens life expectancy due to respiratory and cardiac issues, with corticosteroids currently being the standard treatment.



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