Drugs

Elevar Submits Lirafugratinib NDA for Bile Duct Cancer

Elevar Therapeutics has submitted an NDA for lirafugratinib as a second-line treatment for patients with cholangiocarcinoma whose tumors harbor FGFR2 fusions or rearrangements. Cholangiocarcinoma (bile duct cancer) is a rare malignancy, whose treatment options are limited, particularly after first-line therapy.

The NDA is supported by clinical data from the Phase 1/2 ReFocus trial, which evaluated lirafugratinib in patients with advanced or metastatic cholangiocarcinoma and other solid tumors with FGFR2 alterations. In the cholangiocarcinoma population that had not previously received FGFR inhibitor therapy, the drug demonstrated clinically meaningful anti-tumor activity across multiple efficacy endpoints, including objective response rate, duration of response and progression-free survival, according to the company.

According to data presented earlier this month at the American Society of Clinical Oncology Gastrointestinal Cancers Symposium, the independent review committee-assessed objective response rate was 46.5%, with a median duration of response of 11.8 months, Elevar says. More than three-quarters of responses lasted longer than six months. Median progression-free survival was 11.3 months, while median overall survival was 22.8 months, it adds.

Lirafugratinib’s safety profile was described by the company as manageable, with the most common Grade 3 or higher treatment-related adverse events being palmar-plantar erythrodysesthesia and stomatitis. Dose reductions and interruptions were common, though treatment discontinuation due to adverse events occurred in a small proportion of patients, according to the company.

Lirafugratinib is described as a selective oral inhibitor of fibroblast growth factor receptor 2, a molecular alteration found in a subset of cholangiocarcinomas and other solid tumors.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome