Today (February 18), the official website of the Center for Drug Evaluation (CDE) of the NMPA of China announced that the vamorolone oral suspension applied by Sperogenix Therapeutics is scheduled to be included in the priority review, and the indication is Duchenne muscle aged 4 years and above. Public information shows that this is a new rare disease drug introduced by Sperogenix Therapeutics from Santhera Pharmaceuticals for US$124 million. It has been approved to treat DMD in the United States and the European Union.

It is reported that vamorolone has achieved positive results in the key clinical study VISION-DMD and three open-label studies and expansion studies. In the VISION-DMD study, compared with placebo, the vamorolone group reached the primary endpoint after 24 weeks of treatment, and the difference in the time required from lying to standing was statistically significant compared with the placebo group. Additionally, male patients treated with vamorolone maintained similar growth on average compared to the placebo group, whereas those treated with prednisone experienced growth retardation on average. Patients who switched from prednisone to vamorolone after 24 weeks were able to resume growth in average height for the remainder of the study.

Unlike corticosteroids, bone biomarker testing showed that vamorolone did not cause a decrease in bone metabolism after 48 weeks of clinical study treatment, and spinal bone mineral content was not detected by dual-energy X-ray absorptiometry (DXA). Obvious reduction. The European Medicines Agency (EMA) recognized that vamorolone is not only equally effective as current standard hormone therapy, but also has important clinical safety advantages in maintaining normal bone metabolism, bone density and growth. Additionally, patients who switch from standard hormone therapy to vamorolone can restore growth and bone health while maintaining efficacy.
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