The Food and Drug Administration has granted Priority Review to two drugs for multiple myeloma:
- Empliciti, as a combination therapy for the treatment of patients who have received at least one prior therapy; and
- Ixazomib for the treatment of patients with relapsed and/or refractory disease.
Empliciti (elotuzumab), co-developed by Bristol-Myers Squibb and AbbVie, is an investigational monoclonal antibody targeted against Signaling Lymphocyte Activation Molecule (SLAMF7, also called CS1), a glycoprotein expressed on myeloma and natural killer cells, but not detectable in normal tissue.
The FDA's priority review designation shortens the time to complete a drug's review and aims to deliver a decision on marketing approval designation for drugs that may offer major advances in treatment or provide a treatment where no adequate therapy exists within six months under the Prescription Drug User Fee Act (PDUFA). The FDA action date for Empliciti for this indication is February 29. The drug previously received Breakthrough Therapy status (OT 6/25/15 issue).
Empliciti has been evaluated in the randomized, open-label, Phase III ELOQUENT-2 trial of 646 patients with relapsed or refractory multiple myeloma (OT 6/10/15 issue). Patients were randomized to receive a standard lenalidomide-dexamethasone or a lenalidomide-dexamethasone-Empliciti combination. The lenalidomide-dexamethasone-Empliciti combination increased progression-free survival compared with lenalidomide-dexamethasone at one year—68 versus 57 percent; and two-year progression-free survival rates were 41 versus 27 percent, respectively.
Additionally, the drug was evaluated in the randomized, open-label, Phase II CA204-009 study, which compared treatment with Empliciti with bortezomib and dexamethasone versus bortezomib and dexamethasone alone in 152 patients with relapsed or refractory multiple myeloma. For the patients receiving the combination with Empliciti, 39 percent survived one year without progression of their cancers, compared with 33 percent of the patients receiving the combination therapy without Empliciti.
Median progression-free survival for the Empliciti combination therapy was 9.7 versus 6.9 months for the combination therapy without Empliciti during the one-year follow-up. The name Empliciti was recently proposed by Bristol-Myers Squibb, and will be the drug's brand name if approved.
Ixazomib, marketed by Takeda Oncology, is an investigational oral proteasome inhibitor, and its FDA action date for this indication is March 10.
The drug is also being studied in patients with systemic light-chain amyloidosis and has previously received Breakthrough Therapy status for the indication (OT 12/25/14 issue).
Approval for the multiple myeloma indication will be based on results from the randomized, double-blind, Phase III TOURMALINE-MM1 trial, which is evaluating ixazomib plus lenalidomide and dexamethasone versus placebo plus lenalidomide and dexamethasone in adult patients with relapsed or refractory myeloma.