FDA Approves Pemigatinib for Myeloid/Lymphoid Neoplasms With FGFR1 Rearrangement

The Food and Drug Administration (FDA) granted approval to pemigatinib, an oral selective fibroblast growth factor receptor (FGFR) inhibitor, to treat adult patients with relapsed or refractory myeloid/lymphoid neoplasms (MLNs) and an FGFR1 rearrangement. This application was granted priority review and breakthrough designation, as well as orphan drug designation. Pemigatinib was previously approved for treatment of patients with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a FGFR2 fusion or other rearrangements.

The current approval was based on the open-label, single-arm, phase 2 FIGHT-203 trial, which included 28 patients with relapsed or refractory MLNs with FGFR1 rearrangement. All patients received pemigatinib once daily until disease progression, unacceptable toxicity, or until patients were able to receive allogeneic hematopoietic stem cell transplantation. The primary outcome was complete response (CR).

Of all patients, the complete cytogenetic response rate was 79% (95% confidence interval [CI], 59 to 92). Of the 18 patients with chronic phase in the marrow with or without extramedullary disease (EMD), there were 14 that achieved CR (95% CI, 52 to 94). The median time-to-CR was 104 days, and the median duration was not reached. There were 4 patients with blast phase in the marrow with or without EMD, of which 2 achieved CR. Of 3 patients with EMD only, 1 achieved CR.

The most common adverse reactions, occurring in ≥20% of patients, were hyperphosphatemia, nail toxicity, alopecia, stomatitis, diarrhea, dry eye, fatigue, rash, abdominal pain, anemia, constipation, dry mouth, epistaxis, serious retinal detachment, extremity pain, decrease appetite, dry skin, dyspepsia, back pain, nausea, blurred vision, peripheral edema, and dizziness. The most common grade 3/4 laboratory abnormalities, occurring in ≥10% of patients, were decreased phosphate, decreased lymphocytes, decreased leukocytes, decreased platelets, increased alanine aminotransferase, and decreased neutrophils.

Source:

FDA approves pemigatinib for relapsed or refractory myeloid/lymphoid neoplasms with FGFR1 rearrangement. United States Food and Drug Administration. August 26, 2022. Accessed August 29, 2022. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-pemigatinib-relapsed-or-refractory-myeloidlymphoid-neoplasms-fgfr1-rearrangement