FDA approves Genentech’s Zelboraf for rare blood disease

Officials noted that the method is especially useful for work with rare diseases.
Officials noted that the method is especially useful for work with rare diseases. | File photo

The U.S. Food and Drug Administration recently accepted Genentech’s supplemental New Drug Application (sNDA) for Zelboraf (vemurafenib) — including breakthrough therapy designation — to address a form of Erdheim-Chester disease (ECD), a rare blood disorder.

Additionally, Zelboraf was granted priority review status by the FDA. The specific type of ECD addressed is that with BRAF V600 Mutation. ECD involves irregular replication of histiocytes, a type of white blood cell.

Genentech’s sNDA includes data from its “basket” study, a particular clinical trial design that streamlines data collection by studying a disease’s core genetic profile. Officials noted that the method is especially useful for work with rare diseases.

“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,” Dr. Sandra Horning, Genentech's chief medical officer and head of global product development, said. “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 breakthrough therapy designation granted for this medication is Genentech’s 16th in four years. The FDA is predicted to render an approval decision by Dec. 7.

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