On September 16, 2023, the official website of the Center for Drug Evaluation (CDE) of the State Drug Administration of the People's Republic of China (SDA) showed that vamorolone, a new drug filed by Shufang Pharmaceuticals in collaboration with Santhera Pharmaceuticals, has been included in the program of "Breakthrough Therapeutic Drugs", and its proposed indication is for patients with Duchenne muscular dystrophy (DMD) who are 2 years of age and older.
On January 9, 2023, Santhera Pharmaceuticals and ReveraGen BioPharma announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for vamorolone for the treatment of Duchenne muscular dystrophy (DMD).The FDA has set October 26, 2023 as the target action date for the Prescription Drug User Fee Act (PDUFA).The PDUFA date is the target date for the FDA to complete its review of the NDA. In addition, the FDA has indicated that it does not intend to convene an advisory committee to discuss the application at this time. If approved, Santhera plans to launch vamorolone in the U.S. in the fourth quarter of 2023. vamorolone's NDA filing is supported by clinical data from the pivotal Phase 2b VISION-DMD study, which met its primary endpoint and was statistically significant compared to placebo.
October 27, 2022 - Santhera Pharmaceuticals and ReveraGen Biopharmaceuticals announced that they have completed the rolling submission of a New Drug Marketing Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking priority review of vamorolone for the treatment of Duchenne muscular dystrophy (DMD).
Vamorolone has received orphan drug designation for the treatment of DMD in the U.S. and Europe, as well as Fast Track and Pediatric Rare Disease designations from the U.S. FDA and Potential Innovative Medicines (PIM) designation from the U.K.'s Medicines and Healthcare Act (MHRA).
Duchenne muscular dystrophy (DMD) is a rare X-chromosome recessive disorder that affects almost exclusively males.Pathogenic variants in the DMD gene cause aberrant expression of anti-myasthenic proteins, which leads to inflammation from the onset of the dystrophies.Inflammation usually occurs at or shortly after birth. The major deteriorating turning points of the disease are loss of ability to walk, loss of ability to feed oneself, initiation of assisted ventilation, and concomitant cardiomyopathy.The life expectancy of patients with DMD is typically no more than 40 years. Glucocorticoids are currently the standard treatment option for DMD.