Drugs

Breakthrough Status for Hemab’s Bleeding Disorder Therapy

FDA has granted Hemab Therapeutics a breakthrough therapy designation for sutacimig, an experimental therapy being developed to prevent bleeding episodes in patients with Glanzmann thrombasthenia. Hemab said sutacimig could become the first prophylactic therapy specifically designed to prevent bleeding in people with Glanzmann thrombasthenia, a rare inherited platelet disorder that can cause frequent and sometimes life-threatening bleeding. Current treatments generally focus on managing bleeding once it occurs rather than preventing episodes.

The designation was based on clinical data from the completed Phase 2 multiple-ascending-dose portion of Hemab’s ongoing Phase 1/2 trial, which showed consistent and clinically meaningful reductions in bleeding events, according to the company. The therapy also reduced the most severe episodes requiring high-intensity interventions such as recombinant factor VIIa, platelet transfusions, plasma products or medical procedures.

Previously, sutacimig received fast track and orphan drug designations for treating Glanzmann thrombasthenia. The disorder is characterized by impaired platelet aggregation, leading to recurrent bleeding that can significantly affect quality of life. In the international Glanzmann’s 360 natural-history study, 88% of participants reported at least one bleeding episode in the week before being surveyed, while 65% had required a bleed-related hospital visit in the prior six months, the company says.

Sutacimig, formerly known as HMB-001, is a subcutaneous bispecific antibody designed to stabilize endogenous factor VIIa and recruit it to activated platelets to promote clot formation.

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