According to a story from BioPortfolio, the biopharmaceutical company Incyte Corporation has recently announced that the company’s New Drug Application (NDA) was recently accepted by the US Food and Drug Administration (FDA) for Priority Review. This application is for pemigatinib, an inhibitor of the fibroblast growth factor receptor (FGFR) that is in development as a treatment for cholangiocarcinoma, a rare type of cancer that affects the bile ducts.
Cholangiocarcinoma is a form of cancer that affects the bile ducts. More specifically, the epithelial cells of the bile duct are the ones affected by this disease. The cause is not directly known; many patients who develop the disease also have no known risk factors, but there are some factors that are known to increase risk, such as primary sclerosing cholangitis, chronic liver diseases such as hepatitis, certain genetic disorders such as Caroli’s syndrome, infection with Helicobacter bacteria, and certain liver flukes, a type of parasitic animal that infiltrates the bile ducts and feeds on bile. Symptoms include fever, weight loss, jaundice, itching, and abdominal pain. Treatment may include surgery, chemotherapy, radiation, or liver transplant. Transplant is usually only useful if the disease is detected early; surgery is the best chance for curing cholangiocarcinoma. This disease is rapidly lethal if surgical removal is not possible. To learn more about cholangiocarcinoma, click here.
About Priority Review
Priority Review is reserved for therapies that display the potential to offer a significant improvement in treatment and fulfill an unmet medical need. The standard review period for a medication is twelve months, but Priority Review shortens this period to eight months.
Treating a Rare, Deadly Cancer
The drug is specifically tailored for cases of the disease in which FGFR2 rearrangements or fusions are present. The application follows the results of a clinical study in which pemigatinib was tested in patients with metastatic or locally advanced disease that had been previously treated. This study found that patients with the relevant fusions or rearrangements saw an overall response rate of 36 percent and achieved a median response duration of 7.5 months.
Pemigatinib’s results in the trial were not amazingly impressive, and it only has the potential to help a small percentage of patients (potentially as much as 16 percent) that carry the relevant genetic alterations. However, it is important to keep in mind the very poor prognosis for cholangiocarcinoma, as disease that cannot be treated with surgery or a transplant is almost always fatal.