Swiss company Santhera Pharmaceuticals has announced its Marketing Grant application (MAA) for vamorolone (DMD) for Duchenne muscular Dystrophy (EMA). In the United States, the company expects to submit a vamorolone new drug application (NDA) for treating DMD to the U. S. Food and Drug Administration (FDA) in the fourth quarter of 2022.
vamorolone is a pioneering (first-in-class), a "dissociation" steroid (dissociative steroidal) with a new mode of action, an orphan drug qualification for DMD (ODD) by the FDA for fast track (FTD) and Rare Paediatric Diseases (RPDD), and "Potential Innovation (PIM)" by the UK Medicines and Health Products Agency (MHRA).
Duchenne muscular dystrophy (DMD) is a rare, inherited X-chromosome-linked disease that almost exclusively affects men. DMD is characterized by an inflammatory response at birth or shortly after birth, leading to muscle fibrosis, which clinically manifested as progressive muscle degeneration and weakness. Key milestones in disease progression in DMD include loss of ability to walk, loss of self-feeding, initiation of assisted ventilation, and development of cardiomyopathy. Life expectancy in DMD patients is usually less than 40 years old due to respiratory and / or heart failure.
vamorolone was developed by ReveraGen BioPharma, USA. On September 2,2020, the Santhera exercised its option to gain the global right of the vamorolone to treat DMD and all other indications.
vamorolone is a first of all (first-in-class) drug that is the same receptor bound to corticosteroids but changes downstream activity of the receptor, and therefore is therefore considered a "dissociation" anti-inflammatory agent. This mechanism shows the potential to "disentangle" efficacy from steroid safety issues. Thus, vamorolone has the potential to serve as a valuable alternative to the currently available corticosteroids. Currently, corticosteroids are the standard of care therapy for the treatment of children and adolescents with DMD. However, the medical needs of the high-dose corticosteroid population are far from being met due to the severe systemic side effects, which can reduce the quality of life of the patients.
Central to the MAA submission is positive data from the critical Phase 2b VISION-DMD study consisting of: (1) 24 weeks to evaluate the efficacy and safety of vamorolone (2 and 6 mg / kg / day) versus prednisone (0.75 mg / kg / day) and placebo, and then (2) 24 weeks to assess the maintenance of efficacy and to collect more long-term safety and tolerability data. In addition, this MAA includes data from three open-label studies in which vamorolone doses of 2 to 6mg / kg / day for a total treatment period of up to 30 months.
In the critical VISION-DMD study, vamorolone reached the primary endpoint —— standing time (TTSTAND) speed (p=0.002) when compared to placebo, and demonstrated good safety and tolerability. In that study, the most common adverse effects of vamorolone compared to placebo were Cushing-like features, vomiting, and vitamin D deficiency. Adverse events were generally mild to moderate.
