FDA's Approval of Pemazyre a 'Huge Milestone' for Patients with Cholangiocarinoma

The Food and Drug Administration’s recent approval of the first drug designed to treat patients with previously treated, advanced cholangiocarcinoma who have tumors that have a fusion or other rearrangement of FGRF2 is going to help many patients, according to Melinda Bachini.

“I'm a 10-year survivor of cholangiocarcinoma, as a patient this is a huge milestone for us,” Bachini, the advocacy coordinator at the Cholangiocarcinoma Foundation, said in an interview with CURE®. “It’s definitely the first step in the right direction. I know it's not a cure and it won’t help all patients, but it's a first step.”

The approval was based on results from a trial, designed to evaluate Pemazyre (pemigatinib) in 107 patients with locally advanced or metastatic cholangiocarcinoma with FGFR2 fusion or rearrangement who were previously treated.

The overall response rate with Pemazyre was 36%, with 2.8% of patients having a complete response and 33% having a partial response. In the 28 patients with a response, 63% had a response that lasted six months or longer and 18% had a response of 12 months or longer.

Alleviating Some Issues

The approval, according to Bachini, is going to help patients in multiple ways. For instance, cholangiocarcinoma has a high volume of targetable mutations. However, Bachini said, there have been problems in getting patients biomarker testing early to identify what mutations they may have to get them quickly enrolled in clinical trials.

“This approval will help alleviate some of those issues and we hope patients will get t biomarker testing ordered earlier,” Bachini said. “It will give them more options earlier in their treatment pathway. For patients with FGFR, this will make a significant difference, but it will also benefit any patient who is able to get biomarker testing done as soon as possible after their initial diagnosis in regard to treatment options.”

A Good Sign of Things to Come

Bachini stressed that this approval is indicated for patients for whom first-line treatment has failed.

“I don't mean to be negative and keep saying there's more to come, but there is more to come,” she said. “Typically, first-line treatment had to fail for all patients before they could enter into a clinical trial. Which, in this case, is still true.”

Although the drug’s indication is for previously treated patients, Bachini said she hopes that the approval will help speed up the process so that when patients are receiving their first-line chemotherapy treatments that they will subsequently be getting biomarker testing done to prepare for second-line and even third-line treatments.

“There’s no curative option out there, surgery offers only a potential cure, and recurrence rates are high” she said. “Chemotherapy is not going to cure us, and neither is this targeted treatment right now, but it’s a solid step in the right direction and it can give a patient enough time for the next best treatment to come along.”

Next Steps

Currently, there are many patients who have been on a clinical trial and are likely asking questions as to when the therapy is going to roll out for them, Bachini said. However, she also stressed that there are likely patients who have been recently diagnosed and will be wondering how they too can get the therapy.

The goal for the Cholangiocarcinoma Foundation moving forward, according to Bachini, will be to help provide patients, as well as providers, with directions on next steps.

“There’s a lot more work to be done; we have about 50% of our patients who will have a targetable mutation, but that also means we have about 50% of our patients who need to find something to work for their cancer as well,” she said. “There’s a lot of work to be done, but this approval is the first of many additional options that are currently being developed for patients.”

Bachini noted that there are several phase 3 trials currently underway that will likely open more doors down the road in terms of treatment options. However, she wanted to stress something to patients who may not have been addressed with this approval:

“I want to emphasize to our patients who aren't FGFR-positive that you may have a mutation that you may think isn’t important right now, but we don’t know what the future will bring with research,” she said. “It might not be significant right now, but it could be down the road. And even if you don't carry that FGFR mutation, there are other options that are out there that are coming.”