First Targeted Therapy for Myeloid/ Lymphoid Neoplasms with FGFR1 Approved by FDA

The US Food and Drug Administration (FDA) granted approval to the first targeted treatment indicated for adults with relapsed or refractory myeloid/lymphnoid neoplasms (MLNs) with fibroblast growth factor receptor 2 (FGFR2) inhibitor.

According to Incyte's announcement of the decision, pemigatinib (Pemazyre) will now be the only treatment for this extremely rare and aggressive blood cancer.

“In patients with relapsed or refractory MLNs with FGFR1 rearrangement treated with Pemazyre in FIGHT-203, the high rate of complete response and complete cytogenetic response in patients with chronic phase disease and the high rate of complete cytogenetic response in patients with blast phase disease is clinically meaningful, especially in light of the lack of these specific responses with existing first-line treatments,” Srdan Verstovsek, MD, PhD, Professor, Department of Leukemia, Division of Cancer Medicine, University of Texas MD Anderson Cancer Center, said in a statement.

Verstovsek served as the principal investigator for the FIGHT-203 study which provided the data that supported the FDA's decision. The multicenter open-label, single-arm clinical trial determined the safety and efficacy of pemigatinib to be significant after assessing 28 patients with the condition.

Participating patients that were included could have experienced a relapse following allogeneic hematopoietic stem cell transplantation (allo-HSCT), a disease modifying therapy, or were not a candidate for either therapy. Those with chronic phase in the marrow demonstrated a complete response rate of 78%.

The complete cytogenetic response rate for all participating patients, including 3 without evidence of morphologic disease, was 79%.

Incyte has worked for over a decade to establish itself as a leader in rare blood cancers by developing the first JAK inhibitor approved by the FDA for treating certain patients with myelofibrosis and polycythemia vera.

“The approval of Pemazyre represents an important treatment advancement for people living with MLNs with FGFR1 rearrangement who currently have limited treatment options,” Hervé Hoppenot, Chief Executive Officer, Incyte stated. “These are complex hematologic malignancies with a range of presentations, and this approval highlights Incyte’s continued leadership and commitment to advancing care for patients with rare blood cancers.”