FDA Approves First Targeted Treatment for Myeloid/Lymphoid Neoplasms

Pemazyre is the first approved treatment specifically for patients with FGFR1 rearrangement, a rare blood cancer.

The FDA has approved Incyte’s Pemazyre (pemigatinib) to treat adults with relapsed or refractory myeloid/lymphoid neoplasms (MLNs). Pemazyre is a selective fibroblast growth factor receptor (FGFR) inhibitor that specifically targets FGFR1 rearrangement, an extremely rare and aggressive blood cancer.

“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, CEO of Incyte, said 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 FDA approval was based on data from the phase 2 FIGHT-203 study, which evaluated the safety and efficacy of Pemazyre in 28 patients with relapsed or refractory MLNs with FGFR1 rearrangement. In patients with chronic phase in the marrow with or without extramedullary disease, the complete response rate was 78%. The median time to response of complete response was 104 days.

In patients with blast phase in the marrow with or without extramedullary disease, two patients achieved complete response. In patients with extramedullary disease only, one patient achieved a complete response.

Pemazyre is also approved under an accelerated approval based on overall response rate and duration of response to treat adults with bile duct cancer that has spread or cannot be removed by surgery. The wholesale acquisition cost is $17,510 for a 14-count bottle, according to Incyte.