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Incyte Gets FDA Nod for Cholangiocarcinoma Drug Pemazyre

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Incyte (INCY - Free Report) announced that the FDA has approved pemigatinib, a kinase inhibitor indicated for the treatment of adults with previously-treated, unresectable, locally-advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test.

The candidate has been approved under the brand name, Pemazyre.

The New Drug Application (NDA) for Pemazyre was reviewed under the FDA’s Priority Review program.

The FDA approval was based on positive data from the multi-center, open-label, single-arm study, FIGHT-202, which evaluated the drug as a treatment for adults with cholangiocarcinoma. Data showed that Pemazyre monotherapy resulted in an overall response rate (ORR) of 36% (primary endpoint) and the median duration of response (DOR) of 9.1 months (secondary endpoint) in patients harboring FGFR2 fusions or rearrangements (Cohort A).

Pemazyre is the first and only FDA-approved treatment for this rare cancer that forms in the bile duct. It was approved under accelerated approval based on ORR and DOR. Continued approval may be contingent on verification and description of clinical benefit in a confirmatory trial(s).

Additionally, Incyte’s marketing authorization application (MAA) seeking the approval of pemigatinib for cholangiocarcinoma in the EU has been validated by the European Medicines Agency (EMA) and is currently under review.

Meanwhile, the company has granted Innovent Biologics, Inc. rights to develop and commercialize pemigatinib in hematology and oncology in Mainland China, Hong Kong, Macau and Taiwan.

Pemazyre is also being evaluated in other studies for various other indications — myeloproliferative neoplasms and in previously-treated, locally-advanced/metastatic or surgically unresectable solid tumor malignancies. A phase III study is also ongoing to evaluate the drug as a first-line treatment for patients with cholangiocarcinoma with FGFR2 fusions or rearrangements.  It is also being evaluated in a phase II study in combination with Merck’s (MRK - Free Report) Keytruda and as monotherapy in patients with previously-untreated, metastatic or unresectable bladder cancer harboring FGFR3 mutations or fusions/rearrangements, who are not eligible to receive cisplatin.

Approval of new drugs bodes well for Incyte as it reduces its dependence on lead drug, Jakafi.

Incyte’s shares have gained 14.6% in the year so far compared with the industry’s growth of 1.9%.


Earlier, Incyte initiated RUXCOVID, a global, randomized, double-blind, placebo-controlled phase III study evaluating the efficacy and safety of Jakafi plus standard-of-care (SoC) in patients aged 12 and above with COVID-19-associated cytokine storm. The study is sponsored by Incyte in the United States and Novartis (NVS - Free Report) outside of the country.

Jakafi is a first-in-class JAK1/JAK2 inhibitor approved by the FDA for the treatment of polycythemia vera (PV) in adults who have had an inadequate response to or are intolerant of hydroxyurea, those with intermediate or high-risk myelofibrosis (MF), and for the treatment of steroid-refractory acute graft-versus-host disease (GVHD) in adult and pediatric patients 12 years and older.

Currently, there are no FDA-approved treatments for the severe illness caused by SARS-CoV-2.

Given the alarming levels of spread and severity, some approved drugs are being tested to see if they are effective in treating infected patients.

Earlier, Regeneron (REGN - Free Report) and partner Sanofi announced a program to evaluate their rheumatoid arthritis (RA) drug, Kevzara, to treat patients hospitalized with severe infection due to COVID-19.

Incyte currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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