NEW YORK – The US Food and Drug Administration on Friday approved Incyte's Pemazyre (pemigatinib) as a treatment for relapsed or refractory myeloid/lymphoid neoplasms (MLNs) with FGFR1 rearrangements.
In MLNs with FGFR1 rearrangements, a piece of the FGFR1 gene is translocated with part of another gene, causing the FGFR1 receptor to remain active. FGFR1-rearranged MLNs occur in 1 in 100,000 people in the US and the biomarker is associated with aggressive blood cancer that becomes resistant to existing first-line treatments.
The FDA approved Pemazyre based on data from the single-arm Phase II FIGHT-203 trial, involving 28 patients with FGFR1-rearranged MLNs who had relapsed on hematopoietic stem cell transplantation or other disease modifying agents or could not receive such treatments. Patients had to have an 8p11 translocation based on conventional cytogenetics or an FGFR1 rearrangement on break-apart fluorescence in situ hybridization testing. Currently, there isn't an FDA-approved test for detecting FGFR1 rearrangements in patients with MLNs.
In FIGHT-203, there was a 79 percent complete cytogenetic response rate in all 28 patients. Among 18 patients with chronic-phase disease in the marrow, with or without extramedullary disease (EMD), 78 percent had a complete response; the median duration of complete responses was not reached at data cutoff. Among four patients with blast-phase disease in the bone marrow, with or without EMD, two had a complete response. In three patients with EMD, one had a complete response.
According to Incyte, Pemazyre is now the first and only targeted treatment available for FGFR1-rearranged relapsed or refractory MLNs. "[T]he 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, a professor in the leukemia department at University of Texas MD Anderson Cancer Center and principal investigator of FIGHT-203, said in a statement.
In the US, Pemazyre is also approved as a treatment for advanced cholangiocarcinoma with a FGFR2 fusion or other rearrangement. In approving this indication in 2020, the FDA also approved Foundation Medicine's FoundationOne CDx as a test for identifying bile duct cancer patients eligible for treatment.
Incyte has a patient access program for Pemazyre, called IncyteCARES, which offers education and financial assistance to those who qualify.