Silver Spring, MD (Press Release) – Today the U.S. Food and Drug Admin­istra­tion granted accelerated approval for Darzalex (dara­tu­mu­mab) to treat patients with multiple myeloma who have received at least three prior treat­ments. Darzalex is the first mono­clonal anti­body approved for treating multiple myeloma.

Multiple myeloma is a form of blood cancer that occurs in in­fec­tion-fighting plasma cells (a type of white blood cell) found in the bone marrow. These can­cer­ous cells multiply, produce an ab­nor­mal protein and push out other healthy blood cells from the bone marrow. The disease may result in a weakened immune sys­tem and cause other bone or kidney problems. The National Cancer Institute esti­mates there will be 26,850 new cases of multiple myeloma and 11,240 related deaths in the United States this year.

“Targeting proteins that are found on the surface of cancer cells has led to the devel­op­ment of im­por­tant on­col­ogy treat­ments,” said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research. “Darzalex provides another treat­ment option for patients with multiple myeloma who have be­come resistant to other ther­a­pies.”

Darzalex injection, given as an in­fusion, is a mono­clonal anti­body that works by helping certain cells in the immune sys­tem attack cancer cells.

The safety and efficacy of Darzalex were dem­onstrated in two open-label studies. In one study of 106 par­tic­i­pants receiving Darzalex, 29 per­cent of patients ex­peri­enced a com­plete or partial reduction in their tumor burden, which lasted for an average of 7.4 months. In the second study of 42 par­tic­i­pants receiving Darzalex, 36 per­cent had a com­plete or partial reduction in their tumor burden.

The most common side effects of Darzalex were in­fusion-related reac­tions, fatigue, nausea, back pain, fever and cough. Darzalex may also result in low counts of in­fec­tion-fighting white blood cells (lymphopenia, neu­tro­penia, and leu­ko­penia) or red blood cells (anemia) and low levels of blood platelets (thrombo­cyto­penia).

Blood banks should be informed that patients are receiving Darzalex because the drug may interfere with certain tests that are done by blood banks (such as anti­body screen­ing) for patients who need a blood transfusion. Women who are pregnant should not use Darzalex, and women planning to be­come pregnant should use effective con­tra­­cep­tives during and for at least three months after treat­ment.

The FDA granted breakthrough desig­na­tion for this appli­ca­tion based on pre­lim­i­nary clin­i­cal evi­dence suggesting that if approved, Darzalex may offer a sub­stan­tial im­prove­ment over avail­able ther­a­pies. Darzalex also received priority review and orphan drug desig­na­tions. Priority review status is granted to appli­ca­tions for drugs that, if approved, would be a sig­nif­i­cant im­prove­ment in safety or effectiveness in the treat­ment of a serious con­di­tion. Orphan drug desig­na­tion provides incentives such as tax credits, user fee waivers and eligibility for orphan drug exclusivity to assist and en­cour­age the devel­op­ment of drugs for rare diseases.

Darzalex was approved under the agency’s accelerated approval pro­gram, which allows the approval of a drug to treat a serious or life-threatening disease based on clin­i­cal data showing the drug has an effect on a surrogate end­point reason­ably likely to predict clin­i­cal benefit to patients. This pro­gram provides earlier patient access to promising new drugs while the com­pany conducts con­firmatory clin­i­cal trials.

Darzalex is mar­keted by Janssen Biotech of Horsham, Pennsylvania.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and se­cu­ri­ty of human and veterinary drugs, vaccines and other biological prod­ucts for human use, and medical devices. The agency also is responsible for the safety and se­cu­ri­ty of our nation’s food supply, cosmetics, dietary supple­ments, prod­ucts that give off electronic radi­a­tion, and for regulating tobacco prod­ucts.

Source: The U.S. Food and Drug Admin­istra­tion