Posted by Michael Wonder on 09 Aug 2017
FDA grants priority review and breakthrough therapy designation for Zelboraf (vemurafenib) in Erdheim-Chester disease with BRAF V600 mutation
7 August 2017 - There are currently no FDA-approved treatments for Erdheim-Chester disease, a rare blood disease.
Genentech today announced that the U.S. FDA has accepted the company’s supplemental New Drug Application (sNDA) and granted Priority Review for Zelboraf (vemurafenib) for Erdheim-Chester disease (ECD) with BRAF V600 mutation. ECD is a rare, serious blood disease characterised by the abnormal multiplication of certain white blood cells called histiocytes. Zelboraf was also granted breakthrough therapy designation by the FDA for this indication. The FDA is expected to make a decision on approval by 7 December 2017.
The sNDA includes data from the open-label, Phase II VE-BASKET study. Basket studies use an innovative clinical trial design that helps collect data faster and may accelerate the development of medicines for diseases with high unmet need. Instead of enrolling people based primarily on their disease or its location, basket studies match a disease’s underlying genetic profile to the mechanism of action of the medicine. This allows researchers to evaluate multiple diseases simultaneously. This is particularly important for diseases that are extremely rare, making it difficult to fully enrol a disease- or tumour-specific trial.
