Drugs

Guide on Endpoints for Multiple Myeloma Accelerated Approval

FDA has released a draft guidance outlining how drug developers can use minimal residual disease (MRD) and complete response (CR) as primary endpoints in multiple myeloma trials to support accelerated approval, a move that could further speed access to new treatments in a disease area where outcomes have steadily improved.

Under the guidance, FDA recommends that sponsors may use MRD negativity rates — measured in bone marrow using flow cytometry or sequencing-based assays — in patients who have achieved a complete or stringent complete response as a primary endpoint in trials intended to support accelerated approval. The agency emphasized that the approach is limited to multiple myeloma and does not apply to other disease settings or to the use of MRD for treatment decisions or patient selection.

The guidance also incorporates feedback from a 4/2024 meeting of the Oncologic Drugs Advisory Committee, where members unanimously agreed that MRD can be an acceptable endpoint to support accelerated approval in multiple myeloma. Discussions at that meeting addressed appropriate trial designs, timing of MRD assessments, and disease settings in which the endpoint is most suitable.

The agency cautions, however, that MRD is not yet appropriate as a primary endpoint in all multiple myeloma populations. The draft states that there are currently insufficient data to support its use in maintenance therapy, smoldering multiple myeloma, monoclonal gammopathy of undetermined significance, or extramedullary disease.

Additionally, FDA underscores the importance of careful statistical justification for assumed treatment effects on MRD negativity rates, consideration of toxicity in benefit-risk assessments, and full trial enrollment before analyzing response endpoints to protect trial integrity.

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

Read more

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