IN THE PIPELINE
for CPX-351 for the treatment of adult patients with therapy-related acute myeloid leukemia ( AML ) or AML with myelodysplasia-related changes . The designation was based on the results from a phase III study of older patients with previously untreated high-risk AML or AML with myelodysplasia-related changes ; patients treated with CPX-351 had a 31 percent reduction in the risk of death , compared with those treated with the standard “ 7 + 3 ” chemotherapy regimen .
Venetoclax : Venetoclax was approved as an orphan drug for the treatment of patients with chronic lymphocytic leukemia ( CLL ) who harbor the 17p deletion ( del17p ) and who have received at least one prior therapy . This is the first U . S . FDA-approved treatment that targets the B-cell lymphoma 2 protein . The approval was based on the results of a phase II , single-arm clinical trial , where venetoclax led to an ORR of 80 percent . Patients receiving venetoclax should not be given live attenuated vaccines .
Iomab-B : Iomab-B , a radioimmunoconjugate composed of BC8 ( a novel urine monoclonal antibody ) and iodine-131 radioisotope , was granted orphan drug designation for the treatment of relapsed / refractory AML prior to HCT in older patients . A multicenter , phase III trial will begin soon to evaluate Iomab-B in patients ≥55 years old with relapsed / refractory AML .
Ibrutinib : Ibrutinib was approved as a first-line treatment for patients with CLL , based on results of the phase III RESONATE-2 trial that compared the use of ibrutinib versus chlorambucil . Ibrutinib improved PFS and reduced the risk of disease progression or death by 84 percent , compared with chlorambucil .
Midostaurin : Midostaurin , an oral , multi-targeted kinase inhibitor , was granted breakthrough therapy designation for adults with newly diagnosed FLT3-mutated AML who are eligible to receive standard induction and consolidation chemotherapy . The decision was based on the results of the phase III RATIFY clinical trial , which found that patients treated with midostaurin and standard induction and consolidation chemotherapy had a significant 23 percent improvement in OS .
Ofatumumab : The anti-CD20 monoclonal antibody ofatumumab was approved as maintenance therapy for patients with CLL who have achieved complete or partial response after receiving at least two lines of therapy for recurrent or progressive disease . The approval was based on results from the phase III PROLONG trial that compared ofatumumab with observation . Ofatumumab resulted in a 50 percent reduction in the risk of disease progression and a longer time to next therapy . and dexamethasone or bortezomib and dexamethasone for the treatment of multiple myeloma ( MM ) in patients who have received at least one prior therapy . Daratumumab was previously granted accelerated approval by the FDA for patients with MM who have failed at least three prior lines of therapy , including a proteasome inhibitor and an immunomodulatory agent . The FDA noted that patients who need a blood transfusion should inform blood banks that they are receiving daratumumab because the drug may interfere with certain tests that they perform .
Nivolumab : Nivolumab was approved as an orphan drug for the treatment of patients with classic Hodgkin lymphoma ( cHL ) who have relapsed or progressed after autologous HCT and post-transplantation brentuximab vedotin . The approval was based on results from two multicenter studies evaluating safety and efficacy , in which nivolumab led to an objective response rate of 65 percent and an estimated duration of response of 8.7 months . Nivolumab carries a boxed warning for complications with allogeneic HCT that occurs following therapy with nivolumab .
Pembrolizumab : Pembrolizumab , a humanized monoclonal anti-PD-1 therapy , received breakthrough designation for the treatment of patients with relapsed / refractory cHL . The designation was based on results of the ongoing phase Ib KEYNOTE-013 and phase II KEYNOTE-087 studies evaluating pembrolizumab as a single-agent therapy for cHL . In both trials , pembrolizumab achieved high objective response rates greater than 64 percent .
Melphalan : The FDA approved a new formulation of melphalan injection for use as a high-dose conditioning treatment prior to HCT in multiple myeloma . The approval was based on results from a multicenter , open-label , phase IIb trial in which 61 patients received 200 mg / m 2 of melphalan followed by transplant . The ORR was 95 percent , and the CR rate was 31 percent . The drug has a boxed warning for severe bone marrow suppression , hypersensitivity , and leukemogenicity .
Obinutuzumab : Under the Priority Review program , the FDA approved obinutuzumab in combination with bendamustine followed by obinutuzumab monotherapy for the treatment of patients with follicular lymphoma ( FL ) who have relapsed after or are refractory to a rituximabcontaining regimen . Approval was based on results from the phase III GADOLIN trial , in which adding obinutuzumab to bendamustine led to longer PFS .
Lymphoma & Myeloma
Daratumumab : The FDA granted breakthrough therapy designation for daratumumab in combination with lenalidomide
4 Focus on Rare Diseases