Incyte, a Delaware-based biotechnology company, said Friday that its already marketed drug Pemazyre has been approved for a second time, as a treatment for a group of very rare and aggressive blood cancers.
Pemazyre works by blocking FGFRs, or fibroblast growth factor receptors. Typically, these proteins help regulate the development and proliferation of healthy cells; yet, research indicates that they can perform the same function for a variety of cancers. As such, a handful of drug companies, including Amgen, Relay Therapeutics, Eisai and Taiho Oncology, have advanced anti-FGFR drugs into human testing.
In April 2019, the Janssen division of Johnson & Johnson secured the first-ever approval for an FGFR-targeting therapy. Now sold as Balversa, the therapy is used in certain bladder cancer patients. A year later, the Food and Drug Administration authorized Pemazyre as a treatment for cancer that affects the bile ducts.
The approval announced Friday expands Pemazyre’s reach, providing a new treatment option for a category of blood cancers known as myeloid/lymphoid neoplasms. The drug can now be used in adults who have MLNs that either didn’t respond to an earlier therapy or progressed after it, and who have rearrangements in the gene that specifically codes for FGFR1 — one of the FGFR proteins.
“The approval of Pemazyre represents an important treatment advancement for people living with MLNs with FGFR1 rearrangement who currently have limited treatment options,” Incyte’s CEO Hervé Hoppenot said in a statement.
Hoppenot added that these “are complex hematologic malignancies with a range of presentations.” They’re also uncommon. Incyte estimates that MLNs with FGFR1 rearrangement impact less than 1 in 100,000 people in the U.S.
Incyte said the FDA based its decision on an open-label, single-arm study that enrolled 28 patients with MLNs who either were not candidates for stem cell transplants or other disease-modifying therapies, or who were, but their disease still progressed after treatment. The study looked at “complete cytogenetic response rate,” which reflects how much cancer remains after a therapeutic intervention, and found it to be 79% across the group.
In the statement from Incyte, Srdan Verstovsek, the study’s principal investigator, described this rate as “high” and “clinically meaningful, especially in light of the lack of these specific responses with existing first-line treatments.” Verstovsek also serves as a professor in the Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center.
Meanwhile, for Incyte, the second approval may provide a sales lift for Pemazyre. Between April and June, revenue from the drug totaled $19 million, which was 12% lower than what the average Wall Street analyst expected, according to the investment firm SVB Securities.