Incyte’s Rare Cancer Drug Snags Second FDA Approval

After receiving accelerated approval for a rare bile duct cancer in 2020, the FDA granted Incyte’s Pemazyre a second approval on Friday to treat adults with relapsed or refractory myeloid/lymphoid neoplasms (MLNs) with FGFR1 rearrangement. The approval marks the first and only targeted treatment for the rare blood cancer type. 

Pemazyre’s Value as an FGFR Inhibitor

Fibroblast growth factor receptors (FGFR) bind to the fibroblast growth factor family of proteins and regulate cell differentiation, proliferation, and apoptosis (cell death). Pemazyre is an FGFR inhibitor designed to decrease cancer cell proliferation and survival in FGFR-driven tumors. 

MLNs with FGFR1 rearrangement is a rare blood cancer affecting approximately 1 in 100,000 Americans annually. This type of cancer is aggressive and challenging to treat due to its varied presentation in patients. 

Pemazyre received FDA approval to treat relapsed or refractory myeloid/lymphoid neoplasms (MLNs) with FGFR1 rearrangement after a priority review, meaning the drug received regular approval for the indication. The FDA based the approval on the FIGHT-203 Phase 2 study, consisting of 47 adult patients with the rare blood cancer. 

Hervé Hoppenot, CEO at Incyte, said, “The approval of Pemazyre represents an important treatment advancement for people living with MLNs with FGFR1 rearrangement who currently have limited treatment options. 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.”

Pemazyre received accelerated approval in April 2020 as a second-line treatment for cholangiocarcinoma, a rare bile duct cancer. The FDA granted the approval based on surrogate endpoints on the condition that Incyte performs postapproval clinical trials to prove the drug’s safety and efficacy further. According to the FDA’s postmarket requirements and commitments database, Incyte has yet to embark on the confirmatory trials two years after receiving accelerated approval. 

Related Article: Mounting Criticisms of FDA’s Accelerated Approval Pathway Could Prompt Changes to the System

FIGHT-203 Phase 2 Supporting Data for Approval

The FDA based the approval on data provided by the Phase 2 FIGHT-203 clinical trial. The study demonstrated a complete cytogenetic response rate of 79% across the 47-participant population. 

The duration of the complete response ranged from 44 days to 435 days. The median complete response clocked in at 104 days across the population that achieved a complete response. 

Like many cancer therapies, Pemazyre’s life-saving potential comes with inherent risks and side effects. In its press release, Incyte noted that about 53% of MLNs with FGFR1 rearrangement patients experienced adverse reactions. Fatal adverse reactions occurred in 9% of patients taking Pemazyre. 

Some of the most common adverse reactions included hyperphosphatemia, nail toxicity, hair loss, stomatitis, diarrhea, dry eye, and fatigue. About 80% of patients required dosage changes after experiencing adverse side effects during treatment. 

Still, with the aggressive nature of MLNs with FGFR1 rearrangement and access to limited options, patients and doctors may be happy to add another therapy to their arsenal to treat the rare blood disease. 

Pemazyre’s most recent approval as a second-line treatment for MLNs with FGFR1 rearrangement is another step forward for Incyte and its ambitions to use the drug’s potential to treat rare cancers. In the future, Incyte may look to leverage Pemazyre in other indications as more clinical trials progress and more data becomes available.

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Author

Reed Slater