Swiss firm Santhera Pharmaceuticals and US-based ReveraGen BioPharma, Inc have announced the rolling submission of a New Drug Application (NDA) for vamorolone in Duchenne Muscular Dystrophy (DMD) with the US FDA. The drug, with China rights owned by China-based Sperogenix Therapeutics, is expected to enter the US market during the second half of 2023, pending approval.
Understanding Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a rare X-chromosome recessive genetic disease characterized by progressive muscle degeneration and weakness due to muscle fibrosis. The life expectancy of DMD patients is generally not more than 40 years. The current standard treatment option is glucocorticoid therapy, and there is an urgent need for innovative drugs with better efficacy.
Licensing Deal and Drug Mechanism
In January of this year, Sperogenix Therapeutics struck a USD 124 million licensing deal with Santhera, securing exclusive development and commercialization rights to vamorolone for DMD and other rare disease indications in the Greater China region (including mainland China, Hong Kong, Macau, and Taiwan). Vamorolone is a first-in-class drug that binds to the same receptor as corticosteroids but modifies its downstream activity, acting as a dissociative agonist. The pivotal VISION-DMD study has demonstrated that vamorolone has significant efficacy, along with a good safety and tolerability profile.-Fineline Info & Tech
