Pemigatinib

Indication. Pemigatinib is indicated for adult patients with relapsed or refractory myeloid/lymphoid neoplasms (MLNs) with an FGFR1 rearrangement. This indication is not approved for the pediatric population.

Evidence. Twenty-eight patients with relapsed or refractory MLNs with FGFR1 rearrangement were evaluated in the multicenter open-label, single-arm FIGHT-203 trial (NCT03011372). Eligible patients were either not candidates for, or had relapsed disease after, allogeneic hematopoietic stem cell transplant or a disease-modifying therapy (e.g., chemotherapy).[1,2][Level of evidence C3]

Efficacy. For all 28 patients (including three patients without evidence of morphologic disease), the complete cytogenetic response rate was 79% (95% confidence interval [CI], 59%–92%). Among the 18 patients with chronic phase disease in the marrow with or without extramedullary disease (EMD), 14 achieved a complete response (78%; 95% CI, 52%–94%). Among the four patients with blast-phase disease in the marrow with or without EMD, two achieved a complete response (duration, 1+ and 94 days). For the three patients with EMD only, one achieved a complete response (duration, 64+ days) with a median follow-up of 25.3 months.[1,2]

Identification of biomarker. Biomarker identification was performed using local cytogenetics and both local and central fluorescence in situ hybridization results.