FDA approves pemigatinib for myeloid/lymphoid neoplasms patients with altered FGFR

September 2022 Clinical trials Nalinee Pathak
CHRONIC MYLELOGENOUS LEUKEMIA (also called MYELOGENOUS LEUKEMIA), many MYELOBLASTS (Immature white blood cells or leukocytes), 250X. This is a human blood smear showing numerous Myeloblasts (immature white blood cells or leukocytes). It also shows an Eosinophil with reddish granules and a bilobed nucleus, and a BAND CELL (near the center, has a horseshoe-shaped nucleus) that develops into a NEUTROPHIL (the most common white blood cell). Large numbers of these is considered abnormal. These cells originate in the red bone marrow (MYELOID tissue), hence the name Myelogenous. Leukemia is a cancer of the blood forming tissues, and involves an abnormal proliferation of white blood, cells (leukocytes), many of which are immature, and do not perform normal functions. This great increase in leukocytes can also crowd out red blood cells and platelets. This image also shows numerous, smaller red blood cells (erythrocytes).

The Food and Drug Administration (FDA) has approved pemigatinib for treating patients with relapsed or refractory myeloid/lymphoid neoplasms (MLNs) and rearrangement of fibroblast growth factor receptor 1 (FGFR1).

In 2020, the FDA approved FGFR inhibitor (pemigatinib) for patients with locally advanced or metastatic cholangiocarcinomas they show fusion or other rearrangement of FGFR2. Similarly, pemigatinib can be also used for MLN patients with FGFR1 rearrangements.  The current approval is based on the findings from the FIGHT-203 study.

Study Design

The single arm, multicentre, phase-2 FIGHT-203 study enrolled pateints (n=28) with relapsed or refractory MLNs with FGFR1 rearrangement. The participants had either experienced relapse after, not been candidates for allogeneic hematopoietic stem cell transplantation (HSCT) or other treatments. All the patients were administered pemigatinib (13.5 mg/day) either continuously for 21 days or intermittently (14 days on, 7 days off) until either disease progression, severe toxicity or ability to undergo allogeneic HSCT.

Key Findings

A higher complete response rate of 78% (95% CI, 52-94) was seen in 18 patients with chronic-phase disease in the marrow with or without extra medullary disease. Also, complete response was achieved in a median time of 104 days. Two out of four patients with blast-phase disease in the marrow and one in three with only extramedullary disease reported complete response in 94 and 64 days respectively. Finally, a complete cytogenic response rate of 79% was seen among all the patients. Adverse events were observed in at least 20%.


The high complete response and complete cytogenic response with pemigatinib in MLN patients has led to its accelerated approval by FDA as first-line treatment.