Biologics

Omeros, FDA Agree on Narsoplimab Primary Endpoint

Omeros and FDA have reached agreement on a response-based primary endpoint for its pivotal trial to support a BLA for narsoplimab to treat hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA). Narsoplimab is described by the company as a human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2.

According to Omeros, the response-based primary endpoint “requires a showing of both a beneficial effect on the underlying HSCT-TMA disease process and a meaningful improvement in patients’ clinical status. The endpoint includes laboratory measures and markers of organ function as well as platelet and red blood cell transfusion burden.”

Based on data from an open-label, single-arm, pivotal trial, Omeros says it is confident that the study will meet the primary endpoint. With finalization of the primary endpoint, the company says the next steps are to:

(1) Complete ongoing data collection from medical records of patients already treated with narsoplimab in the clinical trial or under compassionate use. This will enable preparation of complete patient narratives for inclusion in the clinical sections of the BLA.

(2) Complete and submit the BLA. FDA has previously confirmed that Omeros may submit it on a rolling basis, and the company is finalizing for FDA review its proposed schedule of module submissions under that rolling BLA. The nonclinical sections of the BLA have been written and are expected to comprise the first module of the BLA submission.

HSCT-TMA is a significant, costly and often lethal complication of stem cell transplants, according to the company. The condition is a systemic, multifactorial disorder caused by endothelial cell damage induced by infection, graft-versus-host disease, and therapies used as part of stem cell transplantation. In addition to HSCT-TMA, veno-occlusive disease, diffuse alveolar hemorrhage and a range of other disorders are associated with endothelial cell damage.

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