The FDA approved a new drug that stimulates the body’s immune system to fight multiple myeloma in individuals who have already received one to three prior medications. The drug, elotuzumab, which was co-developed by AbbVie, Inc. and Bristol-Myers Squibb (BMS), will be marketed under the brand name, Empliciti™, solely by BMS, and will reportedly cost as much as $142,000 for the first year of treatment.
Multiple myeloma is a blood cancer that occurs in plasma cells, which are a type of white blood cells that fight infection and are found in the bone marrow. The cancer cells multiply and accumulate in the bone marrow, where they crowd out other healthy blood cells. The cancer cells produce an abnormal protein that can weaken the immune system and cause kidney and bone problems.
Multiple myeloma is considered to be a “relatively rare” type of cancer. The American Cancer Society estimates that 26,850 new cases of multiple myeloma will be diagnosed in the U.S. and 11,240 deaths will be attributable to the disease in 2015.
How it works
Empliciti is only the second monoclonal antibody that has been approved to treat patients with multiple myeloma. The drug works by activating the body’s immune system to attack multiple myeloma cells. The drug is approved to be used in combination with Revlimid (lenalidomide) and dexamethasone (a corticosteroid), both of which had been previously approved by the FDA to treat the cancer. Earlier in the month, Darzalex (daratumumab) became the first monoclonal antibody approved by the FDA to treat patients with multiple myeloma who have received at least three prior treatments.
Richard Pazdur, M.D., Director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research said, “We are continuing to learn about the ways the immune system interacts with different types of cancer, including multiple myeloma.”
The drug was tested in a randomized, open-label clinical study composed of 646 participants whose multiple myeloma either returned or did not respond to prior treatments. The participants who took Empliciti along with Revlimid and dexamethasone saw a delay of the worsening of their disease by 19.4 months as compared to participants taking only Revlimid and dexamethasone, who saw a delay in the worsening of their disease by 14.9 months. Out of the participants who took Empliciti along with Revlimid and dexamethasone, 78.5 percent saw their tumors either completely or partially shrink as compared to 65.6 percent of those taking only the Revlimid and dexamethasone.
Special FDA designations
The application submitted to the FDA for Empliciti was granted breakthrough therapy designation. The Food and Drug Administration Safety and Innovation Act (FDASIA) (P.L. 112-144) established the breakthrough therapy designation, which is only granted in instances when preliminary clinical evidence indicates that a drug that is intended to treat a serious condition may, “demonstrate substantial improvement over available therapies on one more clinically significant endpoints.” The FDA will expedite the development and review of drugs that receive the breakthrough therapy designation.
The drug also received priority review and orphan drug designations by the agency. The FDA grants priority review status to applications for drugs that it determines would provide a significant improvement in the safety and effectiveness for the treatment of a serious condition as compared to standard applications. The Orphan Drug Act (P.L. 97-414) established the FDA’s orphan drug designation, which provides incentives to drug sponsors to encourage the development of drugs that can be used to treat rare diseases. The incentives can include tax credits, fee waivers, and orphan drug exclusivity.
The innovative therapy will reportedly come at a high price, with a monthly cost of $10,000 for bi-weekly doses, and an average first-year cost reaching $142,000, which includes special induction cycles.