The Second Ever FDA Approved Drug for Cholangiocarcinoma (CCA)

As reported in Fierce Pharma; BridgeBio has earned FDA approval for their new targeted cancer treatment, Truseltiq. The approval is the biotech company’s first ever approval for a cancer drug, and their second FDA approved pharmaceutical to date.
The treatment was passed with an accelerated approval to treat patients with the bile cancer cholangiocarcinoma, in patients who have cancer that has already been treated or is locally advanced, and metastatic bile duct cancer with an FGFR2 fusion or rearrangement.
The FDA sped BridgeBio’s Truseltiq along with an accelerated approval, to treat patients that have locally advanced or metastatic bile duct cancer, received at least one previous treatment, with an FGFR2 fusion or rearrangement.

Cholangiocarcinoma

Bile duct cancer or cholangiocarcinoma (CCA)  is a rare liver cancer that affects the bile ducts.  The cancer starts in the cells that lines the tubes carrying bile to the gallbladder and small intestine.  This causes patients to experience abdominal pain, tiredness, itchiness, jaundice, white stool, and weight loss. While there are multiple treatment options including transplants, chemotherapy, surgery, biliary drainage, and now, two drug treatment options, none are very effective. Just 9% of patients with CCA make it five years post-diagnosis.

A Successful Trial

The cancer drug Truseltiq, previously known as infigratinib, emerged from a Phase 2 study that administered the treatment to 108 patients with advanced CCA who had already tried a prior therapy. Each participant received the 125 mg of the drug in one arm every three out of four days over 28 day cycles, until their disease progression got too advanced.
The researchers found 23% of the participants responded to the treatment, lasting for an average of five months. This was enough for the FDA’s accelerated approval process.

The Second Drug Option

Many CCA patients undergo surgery to remove the cancer, but for patients for whom that isn’t an option, there aren’t many effective treatment options.  The gruesome condition mean a diagnosis drastically lowers a person’s life expectancy, and for non resectable cases, the five year survival rate is 0%. Tuseltriq will become the second ever pharmaceutical option, following Incyte’s Pemazyre, which was approved last April to treat bile duct cancers with an FGFR2 fusion or other rearrangement.

The prior option, Pemazyre, was the first-FDA approved medication specifically created for the disease, but it came with a steep price tag— the near daily treatment costing patients about $136,000 to $153,000 in the typical six month duration of treatment. In contrast, patients can now also have access to Bridgebio’s offer of $129,000 for the same six month span, while still a huge expense, saves about 5%.

While switching drugs can be disruptive, and patients tend to stick with healthcare routines, Truseltiq’s lower price tag could give the drug a cost edge over Pemazyre when it becomes accessible.
For patients with this devastating disease, any new options and insights into their condition are well welcomed. A second option in two years is a milestone, and the community is finding progress towards better well being.

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