Drugs

Takeda/Protagonist NDA Gets Priority Review for Polycythemia Vera

FDA has accepted for priority review a Takeda and Protagonist Therapeutics NDA for rusfertide, positioning the drug as a potential first-in-class treatment for adults with polycythemia vera (PV). The agency has set a user fee review action target date for the third quarter.

Rusfertide is described as a subcutaneous, once-weekly hepcidin mimetic peptide designed to regulate iron metabolism and suppress excess red blood cell production — the underlying driver of PV. The rare blood cancer is characterized by overproduction of red blood cells, which increases blood viscosity and raises the risk of life-threatening thrombotic events such as stroke and pulmonary embolism, the companies say. Standard management focuses on maintaining hematocrit levels below 45% to reduce those risks, often through repeated phlebotomy and cytoreductive therapies.

The submission is based on data from the Phase 3 VERIFY trial (NCT05210790), along with long-term efficacy and safety results from the Phase 2 REVIVE study and its extension trial, THRIVE. According to the companies, rusfertide met the primary endpoint and all four key secondary endpoints. Patients receiving rusfertide plus standard of care more than doubled clinical response rates compared with standard therapy alone, they say. Benefits included improved hematocrit control, reduced need for phlebotomy and improvements in patient-reported fatigue and symptom burden.

Across 52 weeks of treatment in Phase 3, rusfertide was generally well tolerated. The most common adverse events were injection-site reactions (47.4%), anemia (25.6%) and fatigue (19.6%), most of which were grade 1 or 2. Serious adverse events were reported in 8.1% of treated patients.

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