SANTHERA PHARMACEUTICALS (USA), INC.

China’s NMPA approves Santhera’s Agamree for DMD in patients aged four and above, highlighting its safety benefits over standard corticosteroids. Agamree is already approved in the US, EU, and UK. Sperogenix Therapeutics will commercialize it in China under a $124m licence agreement, paying Santhera royalties and milestone payments.

Agamree (vamorolone) recommended by NICE for NHS use in treating Duchenne muscular dystrophy (DMD) in patients 4 and older. Expected to take effect within 90 days, Agamree aims to preserve muscle function and reduce side effects compared to traditional corticosteroids. Approved in the U.S., EU, U.K., and China, it offers an alternative treatment for DMD.

China's NMPA approves Santhera Pharmaceuticals' AGAMREE for treating Duchenne muscular dystrophy in patients aged four and above, marking the first approved treatment in the country for this indication. The approval follows the NMPA's acceptance of the new drug application in March 2024, highlighting AGAMREE's safety benefits over standard corticosteroids. Sperogenix holds exclusive rights to develop and commercialize AGAMREE in China.

NICE recommends Santhera's Agamree for DMD, available to 1,700 NHS patients aged 4+ after a price discount. Agamree, an alternative to corticosteroids, reduces side effects and maintains efficacy, offering benefits over current treatments.

NICE recommends AGAMREE® (vamorolone) for treating Duchenne muscular dystrophy (DMD) in patients 4 years and older in England, Wales, and Northern Ireland. Santhera works with NHS to ensure rapid patient access and pursues reimbursement in Scotland. AGAMREE is the first DMD treatment approved in the EU, US, and UK, offering an alternative to corticosteroids with reduced side effects.

Non-cystic fibrosis bronchiectasis market driven by rising prevalence, early diagnosis, and drug development. Over 15 companies, including Zambon SpA, Insmed, and AstraZeneca, are developing 15+ pipeline therapies. Key drugs in trials include Colistimethate sodium, Benralizumab, and Brensocatib.

DelveInsight's 'Duchenne Muscular Dystrophy (DMD) Pipeline Insight, 2024' analyzes over 75 pipeline drugs from 75+ companies, focusing on recent FDA, EMA, and PMDA approvals, clinical trials, emerging therapies, and key players like Roche, Santhera, and Sarepta. The report covers various stages of development, routes of administration, and mechanisms of action, offering insights into the evolving DMD therapeutics landscape.

Vamorolone, an FDA-approved therapy for Duchenne muscular dystrophy (DMD), met its primary endpoint in the LIONHEART study, showing a significant increase in the urinary sodium/potassium ratio in healthy adult patients previously challenged with fludrocortisone. The study highlights vamorolone's dual mechanism as both a mineralocorticoid receptor antagonist and glucocorticoid receptor agonist, potentially offering cardioprotective benefits for DMD patients.

Santhera Pharmaceuticals announces Swissmedic's acceptance of the marketing authorization application for AGAMREE® (vamorolone) in Duchenne muscular dystrophy, leveraging existing EU approvals to potentially expedite the Swiss approval process.

Santhera Pharmaceuticals reports H1 2024 financial results, including CHF 14.1 million in revenue and a net loss of CHF 15.3 million. AGAMREE® (vamorolone) launched in Germany, Austria, and the U.S. for Duchenne muscular dystrophy (DMD), with strong market uptake. The drug received approval in the UK and NDA priority review in China. Santhera secured CHF 69 million in financing, expecting cash flow break-even by 2026.