Developed by QED Therapeutics, a subsidiary of BridgeBio Pharma, Truseltiq is an orally administered kinase inhibitor. Administered in capsules, the recommended dose is 125mg once daily for a 3-week period (21 days) followed by 1 week (7 days) of no treatment. In addition to its approval, the FDA also approved the FoundationOne CDx. This device helps to identify and diagnoses patients with FGFR2 gene abnormalities so that they can more quickly receive treatment.

Many patients with FGFR2-driven cholangiocarcinoma have not responded well to prior treatments. Thus, Truseltiq offers the potential to fill an unmet need for patients. Additionally, if effective, Truseltiq could potentially be used to treat other conditions associated with FGFR mutations.

Ultimately, the accelerated approval hinged on clinical data which highlighted the safety, efficacy, and tolerability of Truseltiq. Altogether, 108 patients enrolled in the trial. During the trial, patients received treatment either until they reached a level of unacceptable toxicity or their cholangiocarcinoma progressed. The research showed that:

• Altogether, Truseltiq prompted a 23% response rate. 24 patients partially responded to treatment, while one patient achieved complete response.
• At least 8 patients were treated for 6+ months.
• The median response duration was approximately 5 months. This means that the cancer either responded to treatment or failed to spread for this period.
• Truseltiq was relatively safe and well-tolerated. However, some common side effects included:
  • Increased creatinine and serum phosphate
  • Mouth and lip inflammation
  • Fatigue
  • Dry eyes, skin, and mouth
  • Appetite loss
  • Blurry vision
  • Vomiting
  • Small patches of hair loss
  • Nausea and vomiting
  • Diarrhea or constipation
  • Muscle and joint pain

Cholangiocarcinoma

Cholangiocarcinoma occurs in the bile ducts or biliary tract. Normally, the bile ducts and biliary tract connect the liver to the gallbladder and small intestines and facilitate the movement of bile. In this rare cancer, tumors may form within the liver (intrahepatic), outside of the liver (hilar), or close to the small intestine (distal). Most cholangiocarcinoma diagnoses occur in older individuals. Additional risk factors include Thorotrast exposure, obesity, biliary cysts, diabetes, primary sclerosing cholangitis, and a history of other bile duct or biliary tract issues.

Unfortunately, cholangiocarcinoma does not have a positive prognosis. In part, this is due to the fact that symptoms do not often appear until the cancer has spread. Symptoms include:

• Jaundice (yellowing of the skin and eyes)
• Abdominal pain
• Appetite loss
• Itchy skin (pruritus)
• Fatigue
• Dark-colored urine
• Nausea and vomiting
• Fever and chills
• Pale and greasy stools
• Unintended weight loss

Learn more about cholangiocarcinoma.