The FDA approves Truseltiq for patients with previously-treated locally advanced or metastatic cholangiocarcinoma with an FGFR2 fusion or rearrangement.
FDA passed Truseltiq.
The U.S. Food and Drug Administration (FDA) approved Truseltiq (infigratinib) for patients with previously-treated locally advanced or metastatic cholangiocarcinoma (CCA) with an FGFR2 fusion or rearrangement. The announcement was made by BridgeBio Pharma through its affiliate QED Therapeutics and Helsinn Group.
BridgeBio and Helsinn Group’s affiliate, Helsinn Therapeutics, will jointly handle commercialization in the U.S. and share U.S. profits and losses equally. Helsinn Group will handle marketing except for in China, Hong Kong, and Macau. BridgeBio will be eligible for tiered royalties as a percentage of adjusted net sales and payments reaching up to $2.45 billion U.S.
More marketing applications are being reviewed in Australia and Canada under Project Orbis. Orbis is a program out of the FDA’s Oncology Center of Excellence that lets companies submit and review cancer drugs concurrently among participating international regulators.
Truseltiq is an oral ATP-competitive, tyrosine kinase inhibitor of FGFR. CCA is a cancer of the liver bile ducts. It affects about 20,000 people in the U.S. and Europe annually and has a median five-year survival rate of 9%. FGFR2 genetic mutations are observed in 15% to 20% of the illness. Advanced, unresectable CCA is rare and aggressive with a poor prognosis.
“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 QED’s chief medical officer.
“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 was built on results from a Phase II trial of 108 patients with at least one previous treatment. They received 125 mg of Truseltiq daily for 21 days of 28-day cycles. Of them, 99% had Stage IV CCA. The primary endpoint demonstrated a confirmed objective response rate (ORR) of 23% and a median duration of response (DR) of 5.0 months.
“Today’s FDA approval of Truseltiq for patients with previously-treated locally advanced or metastatic CCA harboring an FGFR2 fusion or rearrangement provides a new therapy option for patients with a very low rate of survival,” said Paul Rittman, chief executive officer of Helsinn Therapeutics.
“This new therapy has the potential to make a life changing impact on patients with few treatment options, and Helsinn Therapeutics looks forward to working with BridgeBio to make it widely accessible to health care providers and patients in the U.S.”
The Phase II data was presented at the 2021 American Society of Clinical Oncology Gastrointestinal Cancers Symposium by lead investigator Milind Javle, professor of Gastrointestinal Medical Oncology at The University of Texas MD Anderson Cancer Center.
Javle said, “While targeted treatments have extended survival for many types of cancer, people diagnosed with cholangiocarcinoma have previously been presented with extremely limited treatment options coupled with low statistical survival data. In this study, Truseltiq showed promise as a targeted treatment option for patients with FGFR2-fusion-driven cholangiocarcinoma with a well-tolerated safety profile in line with previous observation sin this patient population.”
BridgeBio also announced today that its encaleret for the treatment of autosomal dominant hypocalcemia (ADH1) received Fast Track designation by the FDA. ADH1 is a rare, genetic form of hypoparathyroidism caused by variants in the calcium-sensing receptor gene (CASR).
Approximately 12,000 people in the U.S. carry these variants. Patients with ADH1 have low blood calcium, low or low-normal parathyroid hormone levels, and excess urinary excretion of calcium. It can lead to severe muscle cramps and seizures, impaired kidney function, and kidney stone formation.
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