FDA approved Pemigatinib for R/R FGFR1+ Myeloid/Lymphoid Neoplasms

The FDA has granted approval for pemigatinib (Pemazyre) to treat adult patients with relapsed or refractory myeloid/lymphoid neoplasms (MLNs) and FGFR1 rearrangement.

The new approval was based on 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.

The primary endpoint of the trial was the proportion of participants who achieve CR, with secondary endpoints including the proportion of subjects who achieve CR or PR, complete cytogenetic response, duration of CR, duration of response, progression-free survival, overall survival, safety, and tolerability.

Pemigatinib results exhibit a complete response (CR) rate of 78% (95% CI, 52%-94%) in patients with chronic phase in the marrow with or without the extramedullary disease (EMD; n = 18). Moreover, 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).

Concerning safety, data showed that the most common adverse effects to be reported in 20% or more patients were 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%).