Santhera Braced For Another CHMP No In Duchenne
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.
You may also be interested in...
Deal Snapshot: Santhera acquires all rights to vamorolone from Idorsia, making it ReveraGen’s direct partner on the novel Duchenne muscular dystrophy therapy.
Early data for ezutromid in Duchenne muscular dystrophy product give some proof of concept as a broad treatment for the fatal genetic disease – more data are awaited as Summit steps up its pivotal trial plans.
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.