The U.S. Food and Drug Administration (FDA) granted accelerated approval to pemigatinib, the first treatment approved for adults with certain types of previously treated, advanced cholangiocarcinoma.

“This approval demonstrates that while we continue to focus our efforts on addressing the COVID-19 pandemic, the FDA remains committed to the important work of reviewing treatments for patients with cancer and other serious conditions,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research. “With pemigatinib, we considered the observed efficacy results to be clinically meaningful and the overall risk to benefit assessment for patients with tumours harbouring FGFR2 gene fusions and other rearrangements to be favourable, particularly when we considered that these patients have no other good options following first line treatment with chemotherapy.”

Cholangiocarcinoma is a rare form of cancer that forms in bile ducts, which are slender tubes that carry the digestive fluid bile from the liver to gallbladder and small intestine.

Today’s approval is for patients with cholangiocarcinoma that is locally advanced (when cancer has grown outside the organ it started in, but has not yet spread to distant parts of the body) or metastatic (when cancer cells spread to other parts of the body) and who have tumours that have a fusion or other rearrangement of a gene called fibroblast growth factor receptor 2 (FGFR2).

At diagnosis, a majority of patients with cholangiocarcinoma have advanced disease, meaning that the disease is no longer treatable with surgery.

For these patients, until today, there have been no FDA-approved therapies; a combination of chemotherapy drugs has been the standard initial treatment.

FGFR2 fusions have been found in the tumours of approximately 9% to 14% of patients with cholangiocarcinoma.

Pemigatinib is a tablet that works by blocking FGFR2 in tumour cells to prevent them from growing and spreading.

Pemigatinib’s approval was based on the results of a clinical trial that enrolled 107 patients with locally advanced or metastatic cholangiocarcinoma with an FGFR2 fusion or rearrangement who had received prior treatment.

During the clinical trial, patients received pemigatinib once a day for 14 consecutive days, followed by 7 days off, in 21-day cycles until the disease progressed or the patient experienced an unreasonable level of side effects.

To assess how well pemigatinib was working during the trial, patients were scanned every eight weeks.

The trial used established criteria to measure how many patients experienced a complete or partial shrinkage of their tumours during treatment (overall response rate).

The overall response rate was 36%, with 2.8% of patients having a complete response and 33% having a partial response.

Among the 38 patients who had a response, 24 patients (63%) had a response lasting 6 months or longer and 7 patients (18%) had a response lasting 12 months or longer.

The most common adverse reactions occurring in 20% or more of patients who received pemigatinib are hyperphosphatemia and hypophosphatemia (electrolyte disorders), alopecia (spot baldness), diarrhoea, nail toxicity, fatigue, dysgeusia (taste distortion), nausea, constipation, stomatitis (sore or inflammation inside the mouth), dry eye, dry mouth, decreased appetite, vomiting, joint pain, abdominal pain, back pain and dry skin. Ocular (eye) toxicity is also a risk of pemigatinib.

Source: The Food and Drug Administration (FDA)