On September 29, 2021 Helsinn Group and BridgeBio Pharma, Inc. (Nasdaq: BBIO), through its affiliate QED Therapeutics, Inc., reported that Health Canada has approved TRUSELTIQ (infigratinib), a small molecule kinase inhibitor that targets fibroblast growth factor receptor (FGFR), under the Notice of Compliance with Conditions (NOC/c) policy, for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma (CCA) with a FGFR2 fusion or other rearrangement (Press release, BridgeBio, SEP 29, 2021, View Source [SID1234590454]).
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An NOC/c is a form of market approval granted to a product on the basis of promising evidence of clinical effectiveness following review of the submission by Health Canada. Products authorized under Health Canada’s NOC/c policy are intended for the treatment, prevention or diagnosis of a serious, life-threatening or severely debilitating illness. They have demonstrated promising benefit, are of high quality and possess an acceptable safety profile based on a benefit/risk assessment. In addition, they either respond to a serious unmet medical need in Canada or have demonstrated a significant improvement in the benefit/risk profile over existing therapies. Health Canada has provided access to this product on the condition that sponsors carry out additional clinical trials to verify the anticipated benefit within an agreed upon time frame.
"This is an important next step in growing TRUSELTIQ’s global reach. We are pleased to have achieved this milestone for patients with previously-treated locally advanced or metastatic cholangiocarcinoma harboring an FGFR2 fusion or other rearrangement," said Riccardo Braglia, Helsinn Group Vice Chairman and CEO. "The conditional approvals from the U.S. FDA and Health Canada mark the beginning of our journey delivering this medicine to patients in need. We look forward to working to enter further markets in the months and years ahead and working closely with BridgeBio as we make strides to reach patients."
"We are grateful for our first international approval and the opportunity to reach patients outside the United States who are searching for options to treat FGFR2-fusion-driven cholangiocarcinoma. Helsinn has an impressive track record of advancing and commercializing oncology therapies around the globe and we partnered with them earlier this year in the hope of reaching as many patients with FGFR-driven cancers as possible," said BridgeBio CEO and Founder Neil Kumar, Ph.D. "We believe infigratinib may be able to treat other FGFR-driven conditions and we will continue to evaluate its safety and efficacy in urothelial carcinoma and other areas of unmet need."
Under Project Orbis, an initiative of the FDA, Oncology Center of Excellence that allows for concurrent submission and review of oncology drugs among participating international regulatory agencies, TRUSELTIQ received accelerated approval from the U.S. Food and Drug Administration (FDA) in May 2021. An additional marketing application for infigratinib is currently under review in Australia.
Helsinn Group has exclusive commercial rights for TRUSELTIQ in Canada with BridgeBio eligible for tiered royalties as a percentage of net sales as part of the global collaboration and license agreement entered into between the two companies in March 2021.
As part of this agreement, BridgeBio and Helsinn Group’s affiliate, Helsinn Therapeutics U.S., Inc., are jointly responsible for commercialization activities for TRUSELTIQ in the U.S. and will share U.S. profits and losses on an equal basis. Helsinn Group will have exclusive commercialization rights on infigratinib outside of the U.S., 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 USD in the aggregate. Helsinn Group will fund the majority of ongoing and future research and development related to infigratinib in oncology. BridgeBio previously entered a strategic collaboration with LianBio for development and commercialization of infigratinib in oncology indications in China, Hong Kong and Macau.
About TRUSELTIQ (infigratinib)
TRUSELTIQ (infigratinib) is a small molecule kinase inhibitor that targets FGFR, which obtained accelerated approval by FDA and was conditionally approved by Health Canada for the treatment of adults with previously treated, unresectable locally advanced or metastatic CCA with a FGFR2 fusion or other rearrangement.
Prior to initiation of TRUSELTIQ therapy, FGFR2 fusion or rearrangement should be established using a validated test.
Clinical effectiveness of TRUSELTIQ is based on overall response rate (ORR) and duration of response (DoR) from a single-arm Phase 2 trial in patients with specific FGFR2 fusion or other rearrangements.
Infigratinib is not FDA- or Health Canada-approved for any other indication in the United States and Canada, and is not approved for use by any other health authority.
About Cholangiocarcinoma (CCA)
CCA represents an aggressive group of malignancies that form in the bile ducts. The incidence of this serious and fatal disease varies considerably worldwide. As the disease is usually asymptomatic at early-stages, CCA typically presents at diagnosis as locally advanced or metastatic disease with a poor prognosis. In this respect, the five-year survival rate for patients affected by metastatic CCA is 2%. Depending on the anatomical site of origin, CCAs are classified into two subtypes: intrahepatic (iCCA – 10% of total) and extrahepatic (eCCA – 90% of total) CCA. Approximately 10% to 16% of iCCA harbor FGFR2 genetic alterations.1, 2, 3