Drugs

Orphan Status for Imbrium Biliary Cancer Drug

FDA has granted Imbrium Therapeutics an orphan drug designation for its investigational drug etoposide toniribate, described as a novel topoisomerase II inhibitor intended for treating relapsed refractory biliary tract cancer. Also known as cholangiocarcinoma, biliary tract cancer is a rare disease that starts in the bile ducts. It is the second most common primary hepatobiliary cancer, behind hepatocellular cancer, and about 8,000 people in the U.S. diagnosed with it each year.

Etoposide toniribate has shown encouraging data in Phase 2 trials, and these data were key in granting the orphan designation, the company says. “Results of a randomized Phase 2 trial of 23 patients with refractory, metastatic, unresectable biliary tract cancer who had relapsed following treatment with gemcitabine/cisplatin showed a one-year overall survival of 44.4% with etoposide toniribate versus 11.3% with best supportive care (BSC),” it says. “Overall, 55.6% of patients met the primary endpoint of disease control compared with 20% who received BSC. The most common drug-related adverse events were leukopenia, neutropenia, thrombocytopenia, anemia, alopecia, fatigue and abdominal pain.”

Imbrium Therapeutics, in collaboration with Mundipharma EDO, expects to begin a Phase 3 clinical trial of etoposide toniribate in patients with refractory biliary tract cancer in 2020.

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