Vtesse Rakes in Breakthrough Therapy Designation for VTS-270

The FDA has granted breakthrough therapy designation to Vtesse’s VTS-270, thereby offering the company a chance for more guidance and speedier development and review processes.

The cyclodextrin treatment, intended for patients with the rare genetic Niemann-Pick Type C1 Disease, is being tested in a Phase 2b/3 clinical study.

Previously given orphan drug status in both the U.S. and Europe — and with earlier research sponsored by the NIH — VTS-270 is being assessed for its ability to control neurologic symptoms of the fatal disease.