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FDA expands Roche drug indication to approve first-ever treatment for rare blood cancer

Published on 07/11/17 at 09:23am

The FDA has announced the approval of Roche’s Zelboraf (vemurafenib) in the treatment of Erdheim-Chester Disease (ECD), expanding its already existing authorisation in metastatic melanoma to become the first US-approved therapy for the rare blood cancer.

The kinase inhibitor is authorised as a treatment of ECD in patients whose cancer cells feature the genetic mutation BRAF V600. Originating in bone marrow, the cancer causes increased production of white blood cell histiocytes, which in turn can lead to the development of tumours which can manifest in a number of locations such as the heart, lungs and brain.

Affecting just an estimated 600 to 700 patients across the world, ECD is a very rare condition, with BRAF V600-mutated forms of the disease accounting for around 54% of cases.

The FDA’s decision to approve the therapy was based on data taken from 22 patients with BRAF-V600-mutation positive ECD, which demonstrated that the drug produced a partial response in 50% of participants, and a complete response in 4.5%.

Zelboraf can cause a range of severe side-effects which range from hypersensitivity reactions and heart abnormalities to kidney failure and the development of new tumours or growth of existing ones.  

"Today's approval of Zelboraf for patients with ECD demonstrates how we can apply knowledge of the underlying genetic characteristics of certain malignancies to other cancers," said Richard Pazdur, Director of the FDA's Oncology Center of Excellence and acting Director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research. "This product was first approved in 2011 to treat certain patients with melanoma that harbour the BRAF V600E mutation, and we are now bringing the therapy to patients with a rare cancer with no approved therapies."

Matt Fellows

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