Duchenne Community Keeps The Faith After Dual New Product Setback
The DMD community could be forgiven for feeling disheartened by recent setbacks to potential new treatments in the US and EU. Patient power will not be enough to get DMD drugs past regulators but companies and advocacy groups are keeping the faith. Among other things, they hope that better natural history data may help their cause.
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Seven Green Lights From EU’s CHMP, Including Drugs For Hemophilia, Diabetes, Alpha Mannosidosis And Hyperkalemia
The European Medicines Agency’s CHMP has OKd seven new products, including drugs for hemophilia A, diabetes, alpha mannosidosis, hyperkalemia and herpes zoster. But it again rejected Santhera’s filing for Raxone in the new indication of Duchenne muscular dystrophy, and also turned down Repros’ Encyzix for hypogonadism.
Santhera had a tumultuous 2017 and recovery is not going to be easy this year as its lead compound for the rare disease Duchenne muscular dystrophy is about to be rejected by regulators in Europe for a second time.
The US biotech has presented positive data from a mid-stage trial of edasalonexent that had previously missed its primary endpoint and hopes the goals it has set for Phase III will be enough to satisfy regulators.