First-ever Targeted Treatment for Cancer of Bile Ducts Approved by FDA

Recently, the U.S. Food and Drug Administration has granted approval to the use of Pemazyre (pemigatinib)to Incyte Corporation in the treatment of certain types of cancerous formations in the bile ducts of a person.

More specifically, the drug was tested and seen to be effective for advanced stages of cholangiocarcinoma by scientists. Cholangiocarcinoma is a rare form of cancer of the bile ducts, which are tubes that are primarily responsible for the transfer of bile from the liver to the small intestine and gallbladder.

Previously, the only treatment option available for people with a diagnosis of cholangiocarcinoma was using a combination of general chemotherapy drugs.

However, the survival rate for the cancer was very low and the majority of the patients did not have a long life expectancy. This is also because most of the people who are diagnosed with cholangiocarcinoma have late-stage cancer.

Therefore, surgery is not a viable option in nearly all cases. After today’s approval of Pemazyre, there will be a treatment option in cases of metastatic form, in which cancer has spread to other parts of the body.

In addition, patients of locally advanced cholangiocarcinoma, which is when cancer has started to spread outside the impacted organ but has not reached organs or parts of the body that are far away, can also opt for treatment with Pemazyre.

During the diagnosis of cholangiocarcinoma in people, fusion or another rearrangement of a gene called fibroblast growth factor receptor 2 (FGFR2) is present in nearly nine percent to fourteen percent of all cases of cancer regardless of whether it is locally advanced or metastatic.

People with FGFR2 in their tumors can now take Pemazyre, a kinase inhibitor, in the form of tablets that can effectively control and stop the growth of the tumors after today’s FDA approval of the drug.

The FDA approval was given after analyzing the results of a clinical trial that test Pemazyre on one hundred and seven patients, who had either metastatic cholangiocarcinoma with FGFR2 fusion or rearrangement or locally advanced cholangiocarcinoma with FGFR2 fusion or rearrangement.

Secondly, all of the participants had also received treatment with chemotherapy drugs prior to taking Pemazyre.

In the trial, the patients were given Pemazyre in twenty-one days cycles. In these cycles, the patients had to take the drug once a day for fourteen days constantly followed by a seven days break.

The cycles were continued until the patients reported having a lot of adverse effects due to taking the drug or had progressions in the cancerous tumors. In order to track the results of Pemazyre’s effects on cancer, the patients underwent scans every eight weeks.

With established criteria, the reduction or shrinking of the cancerous tumors with Pemazyre was noted by the researchers in the patients. After the trial, it was discovered that the overall response rate in the participants was thirty-six percent.

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Thirty-three percent of these had partial responses and almost three percent had full responses. In the thirty-eight patients with shrinkage, sixty-three percent of twenty-four experienced the response for a time period of six months or longer and eighteen percent or seven months had it for twelve months and longer.

On the other hand, the adverse effect noted in the participants was hypophosphatemia or electrolyte disorder, dysgeusia, stomatitis, abdominal pain, alopecia, nausea, and vomiting, joint pain, nail toxicity, dry eyes, fatigue, back pain, diarrhea, dry skin, and ocular toxicity.

However, these side effects of using the drug can outweigh its advantages in the treatment of bile duct cancer or cholangiocarcinoma.

The application received the Breakthrough Therapy and Priority Review designation. The drug Pemazyre also received Orphan Drug designation.




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