The following is a message from the Director of the FDA Oncology Center of Excellence, Dr. Richard Pazdur:
On August 26, 2022, the Food and Drug Administration approved pemigatinib (Pemazyre, Incyte Corporation) for adults with relapsed or refractory myeloid/lymphoid neoplasms (MLNs) with fibroblast growth factor receptor 1 (FGFR1) rearrangement.
Efficacy was evaluated in FIGHT-203 (NCT03011372), a multicenter open-label, single-arm trial that included 28 patients with relapsed or refractory MLNs with FGFR1 rearrangement. Eligible patients were either not candidates for or have relapsed after allogeneic hematopoietic stem cell transplantation (allo-HSCT) or after a disease modifying therapy (e.g., chemotherapy). Pemigatinib was administered until disease progression, unacceptable toxicity, or until patients were able to receive allo-HSCT.
Selected demographics and baseline characteristics were: median age of 65 years (range, 39 to 78); 64% female; 68% White, 3.6% Black or African American, 11% Asian, and 3.6% American Indian/Alaska Native; and 88% ECOG performance status of 0 or 1.
Efficacy was established based on complete response (CR) rates per the response criteria relevant to the morphologic disease type. Of the 18 patients with chronic phase in the marrow with or without extramedullary disease (EMD), 14 achieved CR (78%; 95% CI: 52, 94). The median time-to-CR was 104 days (range, 44 to 435). The median duration was not reached (range: 1+ to 988+ days). Of the 4 patients with blast phase in the marrow with or without EMD, 2 achieved CR (duration: 1+ and 94 days). Of 3 patients with EMD only, 1 achieved a CR (duration: 64+ days). For all 28 patients (including 3 patients without evidence of morphologic disease), the complete cytogenetic response rate was 79% (22/28; 95% CI: 59, 92).
The most common (≥20%) adverse reactions occurring in patients were hyperphosphatemia, nail toxicity, alopecia, stomatitis, diarrhea, dry eye, fatigue, rash abdominal pain, anemia, constipation, dry mouth, epistaxis, serous retinal detachment, extremity pain, decreased appetite, dry skin, dyspepsia, back pain, nausea, blurred vision, peripheral edema, and dizziness.
The most common (≥10%) Grade 3 or 4 laboratory abnormalities were decreased phosphate, decreased lymphocytes, decreased leukocytes, decreased platelets, increased alanine aminotransferase, and decreased neutrophils.
The recommended pemigatinib dose is 13.5 mg orally once daily on a continuous basis until disease progression or unacceptable toxicity.
This application was granted priority review and breakthrough designation. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics. The application also was granted orphan drug designation.
Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088.
For information on the COVID-19 pandemic, see the following resources:
- FDA: Coronavirus Disease 2019 (COVID-19)
- NCI: Coronavirus: What People With Cancer Should Know
- CDC: Coronavirus (COVID-19)
Please visit the FDA Hematology/Oncology (Cancer) Approvals & Safety Notifications webpage for a list of current and past approvals.