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FDA Panel Supports MRD as Endpoint for Accelerated Approval in Multiple Myeloma

• The FDA's Oncologic Drugs Advisory Committee supports using minimal residual disease (MRD) as an endpoint for accelerated approval of multiple myeloma treatments. • This decision addresses the significant unmet need for early endpoints that predict long-term clinical benefits in myeloma patients. • Using MRD could drastically reduce drug development timelines, potentially accelerating access to effective therapies for patients. • The approach offers the opportunity to develop potentially curative therapies, crucial given the lack of curative treatments currently available.

The FDA's Oncologic Drugs Advisory Committee (ODAC) has voted in favor of using minimal residual disease (MRD) as an endpoint for the accelerated approval of new treatments for multiple myeloma. This decision marks a significant advancement in addressing the unmet needs within the myeloma patient population, where curative treatments remain elusive.

The Need for Early Endpoints

Currently, there is a strong demand for early clinical trial endpoints that can reliably predict the long-term clinical benefit of agents in multiple myeloma. According to C. Ola Landgren, MD, PhD, of the University of Miami Miller School of Medicine, this need prompted the development of a meta-analysis designed based on FDA guidance for considering MRD as a clinical endpoint and a potential basis for accelerated approval.

Revolutionizing Drug Development

The adoption of MRD as an endpoint could revolutionize drug development timelines. Instead of the traditional approach of enrolling patients in clinical trials for two years and waiting an additional decade for data maturation, researchers could potentially assess MRD status after just one year. This expedited process could lead to faster drug reviews and approvals, allowing effective treatments to reach patients much sooner.
"Having an early end point that is likely to predict clinical benefit could revolutionize drug development," Dr. Landgren stated. "Instead of enrolling patients in a clinical trial for 2 years and waiting an additional 10 years for data maturation, a large trial could enroll patients for 2 years, check MRD status after 1 year, and obtain results much faster."

Expediting Access to Life-Saving Medications

This streamlined approach could reduce the wait time for certain agents from 12 years to approximately 3 years for newly diagnosed patients. In multiple myeloma, where the clinical unmet need is significant, using MRD as an endpoint presents an opportunity to develop potentially curative therapies. This shift is crucial for providing patients with faster access to potentially life-saving medications.
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Reference News

[1]
Dr Landgren on the Utility of MRD as an End Point in ...
onclive.com · May 29, 2024

C. Ola Landgren highlights the FDA's support for using minimal residual disease (MRD) as an endpoint in clinical trials ...

[2]
Landgren on MRD as an End Point for Multiple Myeloma Trials
targetedonc.com · May 1, 2024
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