Janssen Biotech, Inc, announced the opening of a daratumumab expanded access program for eligible patients in the United States. Daratumumab is an investigational human anti-CD38 monoclonal antibody being evaluated in clinical trials as a treatment for patients with multiple myeloma. The multicenter, open-label expanded access program is available to multiple myeloma patients who are refractory to both a proteasome inhibitor and an immunomodulatory drug, or who have received three or more prior lines of therapy, including a proteasome inhibitor and an immunomodulatory drug.
Urgent Need
“We understand that heavily pretreated or double-refractory patients are in immediate need of new treatment options,” said Craig Tendler, MD, Vice President of Late Development and Global Medical Affairs, Oncology at Janssen. “Through a research collaboration with the Multiple Myeloma Research Foundation (MMRF), we have broadened our network of sites and sped site activation. Our goal is to provide timely access to daratumumab for multiple myeloma patients who may benefit while its application is under review with the U.S. Food and Drug Administration (FDA). MMRF’s significant experience in multiple myeloma research and innovation has helped to accelerate our efforts to meet this urgent patient need.”
“While we work urgently each day to find solutions for all who are fighting multiple myeloma, our most critical focus is for those individuals whose multiple myeloma has relapsed and often have few or no active treatment options,” commented Walter M. Capone, Chief Executive Officer and President of the MMRF.
In the United States, expanded access programs are conducted as clinical trials and designed to make investigational medicines available for patients with serious or life-threatening illnesses who are ineligible for ongoing interventional trials and have exhausted currently available treatment options.
Up to 40 medical centers in the United States will enroll patients in the daratumumab expanded access program. The MMRF played a key role in identifying sites geographically distributed across the country and opening sites for enrollment.
Regulatory History
On May 1, 2013, daratumumab received Breakthrough Therapy designation from the FDA for the treatment of patients with multiple myeloma who have received at least three prior lines of therapy including a proteasome inhibitor and an immunomodulatory drug, the current standards of care, or who are double-refractory to a proteasome inhibitor and an immunomodulatory drug.
On June 5, 2015, Janssen announced it had initiated the rolling submission of its biologics license application for daratumumab to the FDA for the treatment of this set of multiple myeloma patients. A rolling submission allows the company to submit portions of the regulatory application to the FDA as they are completed.
The regulatory submission for daratumumab will be primarily supported by data from the phase II MMY2002 (SIRIUS) monotherapy study announced in May 2015 at the 2015 ASCO Annual Meeting, along with additional data from four other studies, including the phase I/II GEN501 monotherapy study.
The expanded access protocol has specific inclusion and exclusion criteria for patients to be considered for enrollment in the program, and patients must not be eligible for another daratumumab study. Interested patients should contact their physician to discuss whether they may be a candidate for daratumumab through the expanded access protocol.
Additional information about the expanded access protocol can be found on the National Institutes of Health website ClinicalTrials.gov (search with identifier NCT02477891). ■
