Over the past week, the U.S. Food and Drug Administration (FDA) granted Priority Review to treatments for multiple myeloma and prostate cancer; gave Fast Track designation to a targeted gene therapy for lung cancer; granted Orphan Drug designation to a combination therapy for hepatocellular carcinoma; and issued a 501(k) marketing clearance to a device designed to relieve the symptoms of lymphedema.
Priority Review for Belantamab Mafodotin in Relapsed or Refractory Multiple Myeloma
The FDA granted Priority Review to a biologics license application (BLA) seeking approval of belantamab mafodotin (GSK2857916) for the treatment of patients with relapsed or refractory multiple myeloma whose prior therapy included an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody.
Belantamab mafodotin is an investigational immunoconjugate comprising a humanized anti–B-cell maturation antigen monoclonal antibody conjugated to the cytotoxic agent auristatin F via noncleavable linker. In 2017, belantamab mafodotin was granted Breakthrough Therapy designation by the FDA.
The BLA is based on data from the pivotal DREAMM-2 (DRiving Excellence in Approaches to Multiple Myeloma) study, recently published in The Lancet Oncology, which enrolled heavily pretreated patients who had actively progressing multiple myeloma that had worsened despite current standard of care.
Priority Review for Olaparib in Metastatic Castration-Resistant Prostate Cancer
A supplemental new drug application for olaparib has been accepted and granted Priority Review by the FDA for patients with metastatic castration-resistant prostate cancer and deleterious or suspected deleterious germline or somatic homologous recombination repair gene mutations whose disease has progressed following prior treatment with a new hormonal agent. A Prescription Drug User Fee Act (PDUFA) date is set for the second quarter of 2020.
Olaparib is a first-in-class poly (ADP-ribose) polymerase inhibitor and the first targeted treatment to block DNA damage response in tumor cells harboring a deficiency in homologous recombination repair, such as mutations in BRCA1 and/or BRCA2.
The Priority Review is based on results from the phase III PROfound trial, which were presented during the Presidential Symposium at the European Society for Medical Oncology Congress 2019.
Results of the PROfound trial showed olaparib significantly reduced the risk of disease progression or death by 66% based on a hazard ratio of 0.34 (P < .0001) vs abiraterone or enzalutamide in patients with BRCA1/2 or ATM-mutated metastatic castration-resistant prostate cancer, the primary endpoint of the trial.
The trial also showed that olaparib reduced the risk of disease progression or death by 51% based on a hazard ratio of 0.49 (P < .0001) vs abiraterone or enzalutamide in the overall trial population of patients with homologous recombination repair–mutated metastatic castration-resistant prostate cancer (those with mutations in the genes for BRCA1/2, ATM, CDK12, or 11 other homologous recombination repair–mutant genes, a key secondary endpoint). The safety and tolerability profile of olaparib in the PROfound trial was in line with that observed in previous clinical trials.
Fast Track Designation for Gene Therapy that Targets Lung Cancer
The FDA has granted Fast Track Designation for Oncoprex immunogene therapy in combination with the EGFR inhibitor osimertinib for the treatment of patients with non–small cell lung cancer (NSCLC) and EFGR mutations that has progressed after treatment with osimertinib alone.
Oncoprex is comprised of the TUSC2 (tumor suppressor candidate 2) gene complexed with a lipid nanoparticle. TUSC2 is the active agent in Oncoprex.
Genprex, the manufacturer of Oncoprex, has treated more than 50 patients with lung cancer with Oncoprex in phase I and II clinical trials. The company believes the data from these trials are encouraging as to both safety and efficacy.
Genprex is preparing to initiate a phase I/II clinical trial evaluating Oncoprex in combination with osimertinib, as well as a new phase I clinical trial evaluating Oncoprex in combination with a checkpoint inhibitor.
Durvalumab and Tremelimumab Granted Orphan Drug Designation for Hepatocellular Carcinoma
Durvalumab, a human monoclonal antibody that binds to programmed cell death ligand 1 (PD-L1) and blocks the interaction of PD-L1 with programmed cell death protein 1 and CD80, and tremelimumab, an anti-CTLA4 antibody, have both been granted Orphan Drug designation for the treatment of hepatocellular carcinoma.
The phase III HIMALAYA trial is testing durvalumab and the combination of durvalumab plus tremelimumab vs sorafenib in patients with unresectable, advanced HCC who have not been treated with prior systemic therapy and are not eligible for locoregional therapy. HIMALAYA is the first trial to test dual immune checkpoint blockade in the first-line setting for advanced hepatocellular carcinoma. The randomized, open-label, multicenter, global trial is being conducted in 189 centers across 16 countries including in the United States, Canada, Europe, South America, and Asia. The primary endpoint is overall survival, and key secondary endpoints include objective response rate and progression-free survival.
510(k) Clearance to Market Compression Device for Lymphedema Treatment
The FDA granted 510(k) clearance to market the updated AIROS 8 Sequential Compression Therapy device and garment system.
The AIROS 8 uses pneumatic air compression to inflate accessory garments worn on the area of the body affected by lymphedema. The inflation and deflation sequences, delivered at the pressure and time prescribed by a physician or lymphedema therapist, move the lymphatic fluid in the correct physiologic direction. AIROS Medical has sold the AIROS 8 and its garments since August 2018, but this new FDA 510(k) clearance allows the company to treat patients with breast cancer and lymphedema more effectively.
The AIROS 8 is also available for use with leg garments and arm garments. The leg garments are utilized primarily with patients who have venous complications, including chronic venous insufficiency.
AIROS Medical also manufactures the AIROS 6 device and garment system, which operates in a similar fashion as the AIROS 8. The AIROS 6 model is primarily utilized by patients with Medicare insurance.
