FDA Approves First-Ever Treatment for Erdheim-Chester Disease

Earlier this month, the FDA approved Zelboraf (vemurafenib) for Erdheim-Chester disease (ECD) with BRAF V600 mutation. The final results indicated a response rate of 54.5%.
This is the first FDA-approved treatment for ECD!

ECD is a rare, slow-moving, multi-system blood cancer that is characterized by the excessive production and accumulation of a type of white blood cells called histiocytes. Histiocytes are large cells that normally help in responding to infection or injury, but when they are in this excess, they gather in different organs and tissues, resulting in an abnormal increase in bone density and organ failure. To learn more about this rare disease, click here.

Patient Worthy covered Zelboraf’s FDA review earlier this year.
“This FDA decision means people living with Erdheim-Chester disease will now, for the first time, have an FDA-approved treatment option,” said Sandra Horning, chief medical officer and head of Global Product Development at Genentech, the drug maker. “We are committed to finding new ways to bring medicines to patients with high unmet need, and we are pleased that this innovative clinical trial helped identify Zelboraf for treatment of this rare disease.”

This signals just another example of a rare disease community getting an opportunity for treatment, and for hope.

Click here to read the FDA’s full press release.


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