17 Cancer Drugs on Breakthrough Therapy List

The FDA is evaluating proposals for new indications for at least 17 oncology medicines, according to Friends of Cancer Research. The list of therapies includes novel agents and existing drugs whose uses could be expanded if the agency approves the applications.

The breakthrough therapy program is designed to streamline drug development for therapies to treat serious and life-threatening illnesses. The FDA grants the status based on whether a therapy would provide a substantial improvement over existing options based on preliminary clinical evidence.

The Friends advocacy group helped promote the concept of speedier drug development that resulted in the program being signed into law in July 2012. As of late December, the FDA had granted 72 of 252 requests for breakthrough therapy status and awarded 16 approvals to agents that received the designation, according to the Friends group. (The FDA is not permitted to disclose information about the requests; available information comes from the drug companies).

Overall, anticancer agents account for $43.3% of the breakthrough designations, leading all other therapeutic categories, according to the Friends group. These are the cancer drugs and indications with breakthrough therapy status currently under evaluation:

Breast cancer

• Entinostat—In combination with exemestane for postmenopausal women with locally recurrent or metastatic estrogen receptor (ER)—positive breast cancer whose disease has progressed after aromatase inhibitor therapy; a histone deacetylase inhibitor from Syndax Pharmaceuticals
• Palbociclib—First-line treatment for women with advanced or metastatic ER-positive, HER2-negative breast cancer; a CDK4/6 inhibitor from Pfizer

Non—small cell lung cancer (NSCLC)

• Pembrolizumab (Keytruda)—NSCLC in patients who test negative for EGFR mutations and ALK rearrangements after disease progression on or following platinum-based chemotherapy; an anti-PD-1 immunotherapy agent from Merck
• Rociletinib (C0-1686)—NSCLC in patients with the EGFR T790M mutation after progression on anti-EGFR therapy; a selective EGFR tyrosine kinase inhibitor from Clovis Oncology
• AZD9291—Metastatic NSCLC in patients EGFR T790M mutation whose disease progressed during treatment with an EGFR tyrosine kinase inhibitor; EGFR inhibitor from AstraZeneca
• Brigatinib (AP26113)— ALK-positive metastatic NSCLC in patients who are resistant to crizotinib; a dual ALK/EGFR inhibitor from Ariad Pharmaceuticals
• Alectinib—ALK-positive NSCLC for patients who progressed on crizotinib; a receptor tyrosine kinase inhibitor from Roche
• Dabrafenib (Tafinlar)—Metastatic NSCLC in patients who test positive for the BRAF V600E mutation; a kinase inhibitor from GlaxoSmithKline

Hematologic malignancies

• JCAR015—Relapsed or refractory B-cell acute lymphoblastic leukemia, a chimeric antibody receptor (CAR) T-cell therapy from Juno Therapeutics in collaboration with Memorial Sloan Kettering Cancer Center.
• CTL019—Relapsed or refractory ALL in pediatric or adult patients; A CAR therapy from Novartis and the University of Pennsylvania’s Perelman School of Medicine
• Elotuzumab— In combination with lenalidomide and dexamethasone for the treatment of multiple myeloma in patients who have received one or more prior therapies; a humanized IgG1 monoclonal antibody from Bristol-Myers Squibb
• Nivolumab (Opdivo)—In Hodgkin lymphoma after for the treatment of patients with Hodgkin lymphoma after failure of autologous stem cell transplant and brentuximab; an anti-PD-1 inhibitor from Bristol-Myers Squibb
• Volasertib—In acute myeloid leukemia for patients aged 65 or older with previously untreated AML who are ineligible for intensive remission induction therapy; a PLK inhibitor from Boehringer Ingelheim
• Daratumumab—Multiple myeloma in patients who have received at least three prior lines of therapy and are double refractory to a proteasome inhibitor and an immunomodulatory agent; an anti-CD38 monoclonal antibody from Janssen Biotech
• Ibrutinib (Imbruvica)—Waldenström macroglobulinemia; a Bruton tyrosine kinase inhibitor from Pharmacyclics and Janssen Biotech

GI and GU tumors

• CRS-207 plus GVAX Pancreas—Metastatic pancreatic cancer combination immunotherapy treatment from Aduro BioTech. CRS-207 is a Listeria-based vaccine and GVAX Pancreas is a vaccine that expresses granulocyte-macrophage colony-stimulating factor and mesothelin.
• MPDL3280A—Metastatic urothelial bladder cancer; an anti-PD-L1 monoclonal antibody from Genentech