- Biotech startup Vtesse on Wednesday announced the FDA has granted a breakthrough therapy designation to its lead drug candidate VTS-270 for treatment of Niemann-Pick Type C1 (NPC) disease.
- NPC is a genetic, and usually fatal, disease caused by the accumulation of lipids in the brain, liver, and spleen. It affects one in 100,000 to 150,000 children and most patients die before they reach adulthood.
- Launched almost exactly a year ago, Vtesse was spun out of the Cambridge, MA-based orphan drug accelerator Cydan Development with $25 million in Series A funding.
VTS-270 is currently in a Phase 2b/3 trial, after a Phase 1 study conducted by researchers at the National Institutes of Health returned positive results. This current trial will hopefully enroll around 51 patients located in the US and the EU as well as in other countries.
The drug already has been granted orphan drug status by the FDA and the European Medicines Agency.
"It is both rewarding and validating to receive the FDA’s Breakthrough Therapy designation for VTS-270, which we believe may provide the first effective treatment for slowing the progress or stabilizing the devastating impacts of NPC in children and adolescents,” said Ben Machielse, President and CEO, in a statement.
The Phase 2b/3 trial is enrolling patients aged 4 to 21 for a 12 month double-blind, sham control study.