Multiple myeloma (MM) is a hematologic malignancy characterized by the proliferation of clonal plasma cells in the bone marrow. MM mainly affects older adults. In 2014, an estimated 24,050 new cases of MM were diagnosed, and an estimated 11,090 deaths occurred from MM.
The survival rate of patients with MM has improved greatly over the last decade with the introduction of protease inhibitors, such as bortezomib and carfilzomib, and immunomodulatory drugs (IMiDs), such as lenalidomide, pomalidomide, and thalidomide. These treatments do not result in a cure, however, and almost all patients with MM eventually relapse and require additional therapy. Patients whose disease is refractory to protease inhibitors and IMiDs have a poor prognosis, and only about 22% respond to subsequent treatments. Thus, treatments with mechanisms of action different from those of protease inhibitors and IMiDs are needed.
Panobinostat (Farydak®) is an oral agent that is the first histone deacetylase (HDAC) inhibitor approved to treat MM. Two other HDAC inhibitors have been approved for the treatment of some patients with cutaneous T-cell lymphoma.
Panobinostat is approved for use in combination with bortezomib and dexamethasone in patients with MM who have received at least 2 prior regimens, including bortezomib and an IMiD. The approval was based on results from the Phase 3 PANORAMA 1 clinical trial. The results of the PANORAMA 1 study showed significant improvement in progression-free survival (PFS) with the 3-drug regimen.
In a subgroup analysis of PANORAMA 1, outcomes of patients were evaluated based on prior treatments. Of all patients included in the study, 485 had previously received an IMiD, 193 had previously received bortezomib and an IMiD, and 147 had received at least 2 regimens including bortezomib and an IMiD.
The results of the subgroup analysis show that the median PFS in patients who had received at least 2 treatments was 12.5 months for panobinostat compared with 4.7 months for placebo. In patients who had previously received an IMiD, the median PFS was 12.3 months for panobinostat compared with 7.4 months for placebo, and 10.6 months compared with 5.8 months, respectively, in those who had received bortezomib and an IMiD.
In a patient population with limited treatment options and a poorer prognosis, panobinostat plus bortezomib and dexamethasone provided a clear benefit in PFS of 7.8 months for patients who had received at least 2 prior regimens, including bortezomib and an IMiD.
Panobinostat carries a Boxed Warning alerting healthcare professionals and patients to the risks of severe diarrhea and severe and fatal cardiac ischemic events, severe arrhythmias, and changes on electrocardiogram.
The results of this subgroup analysis have been reported in a newly published study in the journal Blood, published online ahead of print on December 2, 2015.
About the Connexion Healthcare Oncology Center of Excellence
Connexion Healthcare is a full-service global provider of strategic medical and scientific communications with decades of experience providing exceptional service to the pharmaceutical industry. We provide services through 2 Centers of Excellence—Oncology and Rare Disease—and possess core expertise in these therapeutic areas and their marketplaces.
Connexion Healthcare’s Oncology Center of Excellence offers executive talent with decades of experience in the pharmaceutical industry. Our medical directors have conducted oncology research at leading institutions and major pharmaceutical companies. Our account leads have years of project execution expertise, and our medical editors are versed in the language of oncology. Our design leads are tops in their fields and ensure that your objectives are met effectively and even surpassed in an industry-eloquent and compliant manner. Our team works with you to conceive a strategy and execute communications through your product’s lifespan – from drug discovery through market launch and beyond. With particular expertise in phase 2 through postlaunch, we will engage with you wherever you are in development and whenever you need us.
For further information regarding the Oncology Center of Excellence at Connexion Healthcare and how we can develop oncology communications to differentiate therapies by their unique attributes, contact:
Larry Lunak, Senior Vice President
Valery Sudakin, PhD
Hematology/Oncology Scientific Services