Here’s good news for people with the rare blood disease, Erdheim-Chester disease (ECD): The U.S. Food and Drug Administration (FDA) accepted Genentech’s supplemental New Drug Application (sNDA) and granted Priority Review for its drug, Zelboraf, for the treatment of the disease. This is particularly good news because there are currently no drugs approved by the FDA to treat it.
ECD is a rare, serious blood disease characterized by the abnormal multiplication of certain white blood cells called histiocytes. Zelboraf was also granted Breakthrough Therapy Designation (BTD) by the FDA for this indication. The FDA is expected to make a decision on approval by December 7, 2017.
“This first potential treatment submitted to the FDA for this rare disease was identified by an innovative trial studying Zelboraf across different diseases with the same genetic mutation,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development. “We are committed to bringing new treatment options to people with rare diseases and hope to see Zelboraf approved for Erdheim-Chester disease as soon as possible.”
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 enroll a disease- or tumor-specific trial.
A Priority Review designation is granted to medicines that the FDA has determined to have the potential to provide significant improvements in the treatment, prevention or diagnosis of a disease. Breakthrough Therapy Designation is designed to expedite the development and review of medicines intended to treat serious or life-threatening diseases and to help ensure people have access to them through FDA approval as soon as possible.
Erdheim-Chester disease (ECD) is an extremely rare non-Langerhans cell histiocytosis. The exact prevalence and incidence of ECD are difficult to ascertain given the disease is so rare. Based on available published data, it’s estimated there are fewer than 500 cases of ECD in the United States. More than 50 percent of people with ECD have BRAF V600 mutation-positive disease. No large studies have been performed and there are currently no approved treatments for ECD.