Santhera, ReveraGen’s Duchenne Drug Accepted By EMA While Anticipating FDA Priority Review

Swiss biotech Santhera and its partner ReveraGen have announced that the European Medicines Agency (EMA) has accepted their marketing authorization application (MAA) for vamorolone for the treatment of Duchenne muscular dystrophy (DMD). Vamorolone, positioned as an alternative to current corticosteroids, will now be reviewed by EMA’s Committee for Medicinal Products for Human Use (CHMP). 

Santhera is expecting a positive opinion from the CHMP recommending vamorolone for European Commission (EC) approval in late Q3 2023, according to the company’s press release. 

In the US, Santhera and ReveraGen have completed the new drug application (NDA) filing to the US FDA. The companies are seeking priority review and will know in 60 days if the FDA has granted their application. If granted, vamorolone will be set to become available to patients in the US in the second half of 2023, Santhera said. 

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Primary Endpoint Met 

Vamorolone is a synthetic steroid designed to bind to the same receptor as corticosteroids to exert anti-inflammatory activity and counter the progressive muscle degeneration caused by Duchenne. 

In the pivotal VISION-DMD study assessing vamorolone in two doses for Duchenne, the drug met the primary endpoint of Stand velocity versus placebo at 24 weeks of treatment. In the high-dose arm (6 mg/kg a day) vamorolone-treated patients reduced the time from lying flat to standing from 6 seconds to 4.6 seconds, compared to the placebo (5.4 seconds to 5.5 seconds).

Adverse events ranging from mild to moderate severity included cushingoid features, vomiting, and vitamin D deficiency. 

Delays For The Duchenne Drug

Santhera’s attempts to get a Duchenne treatment into the market have been met with setbacks and delays. The company was forced to restructure and focus on vamorolone after their now-defunct DMD drug idebenone failed an interim analysis in Phase 3 in 2020.

In late June, vamorolone’s rolling submission to the FDA hit a bump after Santhera’s third-party contract manufacturing partner said it could not prepare the drug for FDA inspection, which pushed the regulatory timeline for the drug back by several months.  

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Joy Lin

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