The FDA has granted speedy approval for an Incyte drug developed to treat cholangiocarcinoma, a rare cancer affecting the bile ducts.
The regulatory nod for the drug, pemigatinib (Pemazyre), comes more than a month earlier than the anticipated May 30 decision date and it makes the pill the first FDA-approved treatment for cholangiocarcinoma. The decision was made on an accelerated pathway used to more quickly evaluate experimental therapies for serious diseases that have few treatment options. Incyte (NASDAQ: INCY) will need to conduct additional studies to retain approval. The Wilmington, DE-based company says those studies are already underway.
Cholangiocarcinoma leads to tumors that block the tubes carrying bile from the liver to the gallbladder and small intestine. Symptoms include jaundice, itching, dark-colored urine, loss of appetite, and weight loss. The rare cancer affects an estimated 2,000 to 3,000 people in the US each year, according to the National Institutes of Health. Treatment includes surgery, chemotherapy, and radiation therapy.
The Incyte drug, a kinase inhibitor, is approved for patients who have previously been treated for cholangiocarcinoma and whose disease has spread or cannot be removed by surgery. Those patients must also have tumors that have a fusion or other rearrangement of a gene called fibroblast growth factor receptor 2 (FGFR2). Separately, the FDA approved a Foundation Medicine companion diagnostic that tests for these genetic abnormalities.
The FDA based its approval of pemigatinib on an open-label study that enrolled 107 patients. Patients received the Incyte pill once a day for 14 days, followed by seven days off. That treatment cycle continued until the disease progressed or side effects led patients to stop. The FDA says the overall response rate, or the percentage of patients who showed a complete disappearance or partial shrinkage of their tumors, was 36 percent. The complete response rate was 2.8 percent; 33 percent of patients showed a partial response. Of the 38 patients who responded to treatment, 24 of them (63 percent) had a response that lasted six months or longer. Seven patients (18 percent) showed a response of one year or longer.
The FDA warns that the Incyte drug can cause dry or inflamed eyes, as well as inflamed corneas. In rare cases, retinal detachment has been reported in patients treated with the drug. The FDA also cautions that the drug may lead to high levels of phosphate in the blood, and it poses a risk of harming a fetus in women who are pregnant.
Pemigatinib will cost $17,000 for each 21-day cycle of treatment of pemigatinib. Barry Flannelly, the company’s executive vice president and general manager for the US, said on a Monday conference call that the median duration of treatment in clinical trials was six months, which translates to eight or nine cycles of treatment.
In a research note Monday, SVB Leerink analyst Andrew Berens wrote that the $17,000 per cycle price is in line with his estimates, but the firm does not expect pemigatinib to boost Incyte’s market value because FGFR drugs have struggled commercially. As an example, he points to Johnson & Johnson (NYSE: JNJ) bladder cancer drug erdafitinib (Balversa), which has not provided “meaningful revenues” despite addressing a larger disease opportunity.
Incyte CEO Hervé Hoppenot said on the conference call that pemigatinib has potential applications treating other types of cancer. The company is currently testing the drug in separate mid-stage clinical trials for bladder cancer, solid tumors, and tumors positive for FGFR mutations or fusions.
Approval of pemigatinib gives Incyte its second FDA-approved drug. In 2011, the FDA OK’d ruxolitinib (Jakafi), a treatment for myelofibrosis. The drug, a JAK inhibitor, blocks proteins associated with inflammation. Incyte announced earlier this month that it is now evaluating ruxolitinib as a potential treatment for COVID-19 patients experiencing an immune response called a cytokine storm. Hoppenot said that blocking JAK proteins may offer a way to stop this immune response. Incyte is conducting the study, mostly in the US and Europe, with Novartis (NYSE: NVS), the company’s partner on that drug.