ASH 2021 | The promise of pemigatinib for patients with FGFR1-rearranged MLN
Pemigatinib is a potent, oral, fibroblast growth factor receptor (FGFR) inhibitor approved for the treatment of unresectable, locally advanced, or metastatic cholangiocarcinoma with an FGFR2 fusion. It is currently under investigation for the treatment of patients with myeloid/lymphoid neoplasms (MLNs) with FGFR1 rearrangement. The prognosis of FGFR1-rearranged MLN is poor, reflecting a lack of effective treatment options. Pemigatinib is being studied in the ongoing Phase II FIGHT-203 study (NCT03011372) as a potential novel therapeutic option for these patients. To date, durable and high rates of complete response and complete cytogenetic response have been demonstrated, with no unexpected toxicities. Srdan Verstovsek, MD, PhD, University of Texas MD Anderson Cancer Center, Houston, TX, discusses the findings and the potential of this treatment to represent a new option for patients with MLN. This interview took place at the 63rd ASH Annual Meeting and Exposition congress in Atlanta, GA.
Transcript (edited for clarity)
There is a small group of patients with myeloproliferative neoplasms that suffer from a condition that we officially call myeloid lymphoid neoplasm with eosinophilia, and activation of FGFR. FGFR is fibroblast growth factor receptor. There is activation of this particular gene in these patients through translocation of the chromosome eight. You can see abnormality in these patients when you analyze the chromosomes...
There is a small group of patients with myeloproliferative neoplasms that suffer from a condition that we officially call myeloid lymphoid neoplasm with eosinophilia, and activation of FGFR. FGFR is fibroblast growth factor receptor. There is activation of this particular gene in these patients through translocation of the chromosome eight. You can see abnormality in these patients when you analyze the chromosomes. They carry the 8p11 break point, and that is the marker for that particular disease. Through these chromosome abnormalities, FGFR is activated and drives the disease process. A very small group of people, but very deadly. About a year and a half is average survival. Well, because the FGFR gene makes the protein that we can now target with specific pills, penigatinib is the FGFR inhibitor. The ongoing Phase II study has been presented at ASH and shows phenomenal results. This was one of the best abstracts presented, because we have a very high CR and PR rate, about 80% in total, which you do not see with any other therapy. So, this is really earth-shaking for this group of people. Remember 8p11 MPN patients, they sometimes present with lymphoid markers as well. But this is something that is revolutionizing therapy for these patients, and should be remembered. 8p11, penigatinib follow on, hopefully approved next year.
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