FDA Approves QED and Helsinn’s Truseltiq to Treat Patients with Cholangiocarcinoma

Rare Daily Staff

The U.S. Food and Drug Administration approved BridgeBio affiliate QED Therapeutics and Helsinn Group’s Truseltiq under the accelerated approval program for the treatment of patients with previously treated, locally advanced or metastatic cholangiocarcinoma harboring an FGFR2 fusion or rearrangement.

Cholangiocarcinoma (CCA), a cancer of the bile ducts of the liver, is a serious and often fatal disease which affects approximately 20,000 people in the United States and European Union each year. FGFR2 genetic aberrations are present in approximately 15 percent to 20 percent of people who have this disease. Currently, the five-year survival rate is only 9 percent. Advanced, unresectable CCA is a rare, aggressive malignancy with a poor prognosis.

Truseltiq (infigratinib) is an orally administered, ATP-competitive, tyrosine kinase inhibitor of FGFR. In the pivotal trial of patients with advanced, unresectable cholangiocarcinoma (CCA), an aggressive malignancy with poor prognosis, Truseltiq led to cases of tumor shrinkage.

“This is an important milestone for patients diagnosed with FGFR2-fusion-driven cholangiocarcinoma who have recurred after first-line therapy and are in need of targeted options for further treatment,” said Susan Moran, chief medical officer for QED. “Based on the efficacy seen to date, our team believes infigratinib possesses promise for a range of FGFR-driven conditions, including other cancers. We will continue to evaluate its safety and efficacy in these areas of unmet need.”

The approval of Truseltiq is based on a phase 2 clinical study in which 108 patients who had undergone at least one prior treatment for advanced CCA received 125 mg of Truseltiq daily for 21 days of 28-day cycles. Of these patients, 107 (99 percent) had Stage IV CCA. All patients had received at least one prior line of systemic therapy. The study’s primary endpoint demonstrated a confirmed objective response rate of 23 percent. The study also showed a median duration of response of 5.0 months.

Common adverse reactions and laboratory abnormalities (greater than 30 percent) were increased creatinine, increased phosphate, decreased phosphate, nail toxicity, stomatitis, increased alkaline phosphatase, decreased hemoglobin, increased alanine aminotransferase, dry eye, fatigue, increased lipase, decreased lymphocytes, increased calcium, decreased sodium, alopecia, increased triglycerides, increased aspartate aminotransferase, decreased platelets, increased urate, palmar-plantar erythrodysesthesia syndrome, arthralgia, and dysgeusia.

“The approval of Truseltiq provides a new and exciting treatment option for patients with CCA harboring an FGFR2 fusion,” said Stacie Lindsey, CEO of the Cholangiocarcinoma Foundation. “We appreciate the fact that there is a robust patient support program, ForgingBridges, to help patients access care and support them throughout their treatment journey.”

Additional marketing applications for infigratinib are currently under review in Australia and Canada under Project Orbis, an initiative of the FDA’s Oncology Center of Excellence that allows for concurrent submission and review of oncology drugs among participating international regulatory agencies.

BridgeBio and Helsinn Group’s affiliate, Helsinn Therapeutics (U.S.), will be jointly responsible for commercialization activities in the United States and will share U.S. profits and losses on an equal basis. Helsinn Group will have exclusive commercialization rights on infigratinib outside the United States, excluding China, Hong Kong and Macau. BridgeBio will be eligible for tiered royalties as a percentage of adjusted net sales, and payments totaling up to approximately $2.45 billion in the aggregate. Helsinn Group will fund the majority of ongoing and future research and development related to infigratinib in oncology. BridgeBio and Helsinn Group entered into a global collaboration and licensing agreement in March 2021. BridgeBio previously entered a strategic collaboration with LianBio for development and commercialization of infigratinib in oncology indications in China, Hong Kong, and Macau.

Accelerated approval was granted based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).

Photo: Susan Moran, chief medical officer for QED