FDA Approves Pemigatinib for Myeloid/Lymphoid Neoplasms With FGFR1 Rearrangement

FDA

The FDA has approved pemigatinib (Pemazyre) for the treatment of adults with relapsed or refractory myeloid/lymphoid neoplasms (MLNs) and FGFR1 rearrangement.

“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 of Incyte, stated in a press release. “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.”

The regulatory decision was supported by findings from the phase 2 FIGHT-203 trial (NCT03011372), which examined the safety and efficacy of pemigatinib in a total of 28 patients with relapsed or refractory MLNs with FGFR1 rearrangement. Patients could have relapsed following allogeneic hematopoietic stem cell transplantation (allo-HSCT) or after a disease-modifying therapy or were not eligible to undergo allo-HSCT or other disease-modifying treatments.

Pemigatinib elicited a complete response (CR) rate of 78% (95% CI, 52%-94%) in patients with chronic phase in the marrow with or without extramedullary disease (EMD; n = 18). In these patients, the median time to CR was 104 days (range, 44-435), and the median duration of CR was not yet reached (range, 1+ to 988+ days).

Of those with blast phase in the marrow with or without EMD (n = 4), 2 patients achieved a CR with pemigatinib; the duration of response ranged from 1+ to 94 days. In those with EMD only (n = 3), 1 patient achieved a CR (duration: 64+ days).

For all patients (n = 28, including 3 patients without evidence of morphologic disease) the complete cytogenetic response rate achieved with pemigatinib was 79% (95% CI, 59%-92%).

Regarding safety, the most common adverse effects, reported in 20% or more of patients, included hyperphosphatemia (74%), nail toxicity (62%), alopecia (59%), stomatitis (53%), diarrhea (50%), dry eye (50%), fatigue (44%), rash (35%), abdominal pain (35%), anemia (35%), constipation (32%), dry mouth (32%), epistaxis (29%), retinal pigment epithelial detachment (26%), extremity pain (26%), decreased appetite (24%), dry skin (24%), dyspepsia (24%), back pain (24%), nausea (21%), blurred vision (21%), peripheral edema (21%) and dizziness (21%).