Breaking New Ground: FDA Approves Pemazyre, the First Targeted Therapy for Cholangiocarcinoma

Apr 22, 2020 | News

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In the midst of such a challenging time, I wanted to take a moment to share some very positive and exciting news with you.

Late last week, the FDA granted approval to the first targeted therapy in cholangiocarcinoma: Pemazyre (pemigatinib) , developed by Incyte .

There are many reading this email who, like my family, have been directly impacted by cholangiocarcinoma. For all of us, and for so many facing other rare cancers, the message was the same- no approved or effective treatments are available. With this approval, that message finally begins to change.

TCF has had a close partnership with Incyte for several years as they developed Pemazyre, and we want to thank them for their commitment to the cholangiocarcinoma community. In addition, we are grateful to all of the doctors and researchers whose tireless work contributed to the development and approval of this new therapy.

We also want to extend our congratulations to our friends at Foundation Medicine , as their test was also approved by the FDA as the companion diagnostic for Pemazyre. You can read the press release here, which provides a perspective from TCF Executive Director Jim Palma.

Of course, there is still a very long way to go. This drug will only be available to patients who have a FGFR2 fusion driving their cancer, which is about 15% of cholangiocarcinoma patients. But this represents a major step forward for treating cholangiocarcinoma, and we are hopeful that many more therapies will follow.

Bringing a drug to approval takes many years and requires the collaborative efforts of countless individuals, and we are proud to be a part of that community. Here’s to more good news and many more new effective treatments for rare cancers to come.