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FDA Actions & Updates

The latest approvals, designations, and new indications from the U.S. Food and Drug Administration for oncology drugs.

Wednesday, August 24, 2016

The FDA has granted a breakthrough therapy designation to the immunotherapy Darzalex (daratumumab) in combination with lenalidomide (an immunomodulatory agent) and dexamethasone, or bortezomib (a proteasome inhibitor) and dexamethasone, for the treatment of patients with multiple myeloma who have received at least one prior therapy.

This marks the second time daratumumab has received a breakthrough therapy designation, which is intended to expedite the development and review timelines of potential new medicines to treat serious or life-threatening diseases, where preliminary clinical evidence shows the medicine may provide substantial improvement over existing therapies.

Breakthrough therapy designation was granted to daratumumab based on data from two phase III studies:


·         The MMY3004 (CASTOR) clinical trial evaluating daratumumab in combination with bortezomib and dexamethasone, compared to bortezomib and dexamethasone alone, in patients with multiple myeloma who received at least one prior therapy. Overall, the daratumumab combination therapy demonstrated a reduction in the risk of disease progression or death. These results were presented at the 2016 ASCO Annual Meeting (Abstract LBA4).

·         The MMY3003 (POLLUX) clinical trial evaluating daratumumab in combination with lenalidomide and dexamethasone, compared to lenalidomide and dexamethasone alone, in patients with multiple myeloma who received at least one prior therapy. Overall, the addition of daratumumab reduced the risk of disease progression or death in these patients. These results were presented at the Annual Congress of the European Hematology Association in June 2016 (Abstract LB2238).


"Despite tremendous progress in the past 15 years, multiple myeloma remains a highly complex and difficult disease to treat, with most patients relapsing or becoming resistant to therapy," said MMY3003 (POLLUX) lead study author Meletios A. Dimopoulos, MD, Department of Clinical Therapeutics, National and Kapodistrian University of Athens School of Medicine, Alexandra General Hospital, Athens, Greece. "Daratumumab has already shown pronounced activity as a monotherapy in heavily pre-treated patients. This designation underscores the potential of daratumumab in combination with either a proteasome inhibitor or an immunomodulatory agent to provide much-needed benefit to patients with at least one prior therapy."

Wednesday, August 24, 2016

The FDA has approved Keytruda (pembrolizumab), an anti-PD-1 (programmed death receptor-1) therapy, at a fixed dose of 200 mg every 3 weeks, for the treatment of patients with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) with disease progression on or after platinum-containing chemotherapy.            

Under the FDA's accelerated approval regulations, this indication for pembrolizumab is approved based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. For HNSCC patients, PD-L1 testing is not needed prior to use of pembrolizumab.

The approval is based on data from the KEYNOTE-012 study, which included patients with recurrent or metastatic HNSCC who had disease progression on or after platinum-containing chemotherapy or following platinum-containing chemotherapy administered as part of induction, concurrent, or adjuvant therapy and ECOG performance status of zero or one. The data showed an objective response rate (ORR) of 16 percent (95% CI: 11, 22), complete response rate of 5 percent, with responses of 6 months or longer observed in 82 percent (n=23/28) of the responding patients. ORR and duration of response were similar regardless of human papillomavirus status.

KEYNOTE-012 was the first clinical study to investigate the role of a PD-1 inhibitor in patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.

Pembrolizumab is a humanized monoclonal antibody that works by increasing the ability of the body's immune system to help detect and fight tumor cells. It blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes that may affect both tumor cells and healthy cells.​

Tuesday, August 9, 2016

TK216 has received Orphan Drug Designation from the FDA for the treatment of Ewing sarcoma.

It is a first-in-class small molecule inhibiting ets-family transcription factor oncoproteins, which are the main disease drivers in Ewing tumors. A first-in-human phase I trial of TK216 in relapsed or refractory Ewing sarcoma has been initiated.

Ninety percent of Ewing sarcoma cases occur in patients between the ages of 5 and 25 years, and two-thirds are found in those between 10 and 20 years of age. The median patient age is 15 years. Ewing sarcoma is a rare disease, with approximately 600-800 new cases diagnosed annually in the U.S., an incidence rate that has remained fairly constant over the past 30 years at nearly three cases per one million people.

The 5-year survival rate for patients with localized disease is approximately 75 percent, but if the tumor has spread or metastasized at the time of diagnosis, the 5-year survival rate drops to 30 percent.

Tuesday, August 9, 2016

The FDA granted Rare Pediatric Disease Designation for ABT-414, an investigational antibody drug conjugate targeting the epidermal growth factor receptor (EGFR) for the treatment of pediatric patients with EGFR-amplified Diffuse Intrinsic Pontine Glioma (DIPG), known to be highly aggressive and difficult to treat brain tumors found at the base of the brain.

Though brainstem tumors are extremely rare among adults, they comprise approximately 10-15 percent of all pediatric brain tumors. DIPG is the most common subtype of tumor in this anatomical region and the second most common malignant brain tumor of childhood, with an estimated 200-400 children affected each year in the U.S.

The FDA granted the Rare Pediatric Disease Designation based on a proposed pediatric sub-study "nested" within the ongoing phase II study of ABT-414 in adults with recurrent EGFR-amplified glioblastoma, conducted in collaboration with the European Organization for Research and Treatment of Cancer.

Wednesday, July 27, 2016

The FDA granted the premarket approval application (PMA) for the new Photofrin 630 PDT Laser.

Photodynamic therapy (PDT) with Photofrin is a light-based cancer treatment that combines a photosensitizing drug called Photofrin (porfimer sodium) with a specific type of light administered

by a laser to attack cancer cells.

The newly approved laser, which is designed for use with Photofrin to treat esophageal cancer, Barrett's Esophagus, and non-small cell lung cancer, has been re-engineered with technological advancements in laser design. These advancements include new controls and peripheral systems while maintaining the same specifications with minimal changes to the treatment procedures.

Photofrin is also being evaluated as a rare disease product candidate through a phase III clinical trial. The ongoing trial is evaluating the product's safety and efficacy as a potential treatment for cholangiocarcinoma, or bile duct cancer, which is a rare disease affecting approximately 2,000-3,000 patients annually in the U.S.​

About the Author

Sarah DiGiulio