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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.

Monday, February 8, 2016

The U.S. Food and Drug Administration has granted Orphan Drug designation to tazemetostat (also known as EPZ-6438) for the treatment of patients with malignant rhabdoid tumors (MRTs). Tazemetostat is a first-in-class inhibitor of EZH2, a histone methyltransferase that is increasingly understood to play a potentially oncogenic role in a number of cancers.

The Orphan Drug designation—to encourage development of drugs in the diagnosis, prevention, or treatment of a medical condition affecting fewer than 200,000 people in the U.S.—grants a product market exclusivity for a seven-year period if the sponsor complies with certain FDA specifications, as well as tax credits and prescription drug user fee waivers. The designation does not, though, shorten the duration of the regulatory review and approval process.

Tazemetostat is being investigated in an ongoing Phase II study in adults and a Phase I study in children with genetically-defined solid tumors, both of which began late last year and include patients with MRTs (tumors defined by loss of INI1 protein as measured by immunohistochemistry). The studies also include patients with INI1-negative tumors and synovial sarcoma. Interim data from the trial in adult patients with genetically defined solid tumors, including MRT, other INI1-negative tumors, and synovial sarcoma, are expected to be presented at a medical meeting sometime this year, according to a news release from the drug company developing the drug, Epizyme, Inc.


Monday, February 8, 2016

The U.S. Food and Drug Administration has granted Fast Track designation to the multiple-epitope Folate Receptor Alpha Peptide Vaccine (TPIV 200) with GM-CSF adjuvant for maintenance therapy in patients with platinum-sensitive advanced ovarian cancer whose disease is stable or who have had a partial response following completion of standard of care chemotherapy.

The Fast Track designation, established under the FDA Modernization Act of 1997, is designed to facilitate frequent interactions with the FDA review team to expedite clinical development and submission of a New Drug Application for medicines with the potential to treat serious or life-threatening conditions and address unmet medical needs. The designation permits the drug developer the opportunity to submit sections of an NDA on a rolling basis as data become available, allowing the FDA to review those materials on a rolling basis as well.

The FDA had granted orphan drug designation to TPIV 200 for the same indication late last year.

A Phase II trial for TPIV 200 is being planned, according to a news release from the drug company developing the agent, TapImmune Inc.


Wednesday, February 3, 2016

The U.S. Food and Drug Administration has granted a new breakthrough therapy designation to venetoclax for use in combination with hypomethylating agents (HMAs) for the treatment of patients with untreated acute myeloid leukemia who are ineligible to receive standard induction therapy, high-dose chemotherapy. Venetoclax is an inhibitor of the B-cell lymphoma-2 (BCL-2) protein.

 

The breakthrough therapy designation, enacted as part of the FDA’s 2012 Safety and Innovation Act, was created to expedite the development and review time of a potential new drug for serious or life-threatening disease where early clinical evidence suggests the drug may demonstrate substantial improvement compared with existing therapies.

 

Venetoclax has previously received breakthrough therapy designation for the treatment of patients with relapsed or refractory chronic lymphocytic leukemia (CLL) with the 17p deletion genetic mutation (OT 6/25/15 issue); and earlier this year the drug received another breakthrough therapy designation for the treatment of patients with relapsed or refractory CLL with the 17p deletion mutation for use in combination with rituximab.

 

This breakthrough therapy designation for venetoclax is based on data from a Phase II clinical trial that were presented at the American Society of Hematology Annual Meeting in 2014.

 

Venetoclax is being developed in collaboration with Genentech and Roche.


Tuesday, February 2, 2016

The U.S. Food and Drug Administration has approved Halaven (eribulin mesylate) Injection for the treatment of patients with unresectable or metastatic liposarcoma who have received a prior anthracycline-containing chemotherapy regimen. Halaven is a microtubule dynamics inhibitor with a distinct binding profile that exerts its effect via a tubulin-based antimitotic mechanism, ultimately leading to apoptotic cell death after prolonged and irreversible mitotic blockage.

"Halaven is the first drug approved for patients with liposarcoma that has demonstrated an improvement in survival time," Richard Pazdur, MD, Director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research, said in a statement. "The clinical trial data the FDA reviewed indicates that Halaven increased overall survival by approximately seven months, offering patients a clinically meaningful drug."

Halaven is also currently approved for the treatment of patients with metastatic breast cancer who have received at least two prior chemotherapy regimens for late-stage disease (OT 12/10/10). And the drug had also received priority review designation for this indication last year (OT 10/25/15 issue); and was granted orphan drug designation for soft tissue sarcoma in 2012.

Safety and Efficacy
Efficacy and safety for Halaven were evaluated in a Phase III, clinical trial of 143 patients with advanced liposarcoma that was unresectable, locally advanced, or metastatic, all of whom had been treated with chemotherapy. Patients received either Halaven or dacarbazine until their disease had spread or until they were no longer able to tolerate the side effects of treatment. The median overall survival for patients with liposarcoma receiving Halaven was 15.6 months compared to 8.4 months for patients who received dacarbazine.

Side Effects
The most common side effects for the patients who received Halaven were fatigue, nausea, alopecia, constipation, peripheral neuropathy, abdominal pain, and pyrexia. Halaven may also cause neutropenia or decreased levels of potassium or calcium, according to a news release from the FDA. The most common serious adverse reactions for patients receiving Halaven were neutropenia and pyrexia.

Serious side effects from treatment with Halaven may also include a decrease in white blood cell count, which can increase the risk of serious infections that could lead to death; numbness or neuropathy; harm to a developing fetus; and changes in heartbeat that may also lead to death, according to the FDA.


Wednesday, January 27, 2016

The U.S. Food and Drug Administration has approved Kyprolis (carfilzomib) for Injection in combination with dexamethasone or with lenalidomide (Revlimid) plus dexamethasone for the treatment of patients with relapsed or refractory multiple myeloma who have received one to three lines of therapy. And the FDA also approved Kyprolis as a single agent for the treatment of patients with relapsed or refractory multiple myeloma who have received one or more lines of therapy. This FDA decision converts to full approval the initial accelerated approval Kyprolis received in July 2012 as a single agent.

The FDA had previously approved Kyprolis to treat patients with multiple myeloma who have received at least two prior therapies, including treatment with Velcade (bortezomib) and an immunomodulatory therapy (OT 8/25/12 issue). And the FDA had also approved another expanded indication for Kyprolis in combination with lenalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received one to three prior lines of therapy in 2015.

Trial Data
Approval for the new indication is based on data from the Phase III head-to-head ENDEAVOR study of 929 patients with multiple myeloma whose disease had relapsed after at least one, but not more than three, prior therapeutic regimens. The data (which were presented at the American Society of Clinical Oncology Annual Meeting earlier this year) showed that progression-free survival was nearly twice as long (18.7 months) for patients with relapsed multiple myeloma who were treated with Kyprolis and low-dose dexamethasone compared with patients treated with bortezomib and low-dose dexamethasone (9.4 months) (OT 7/25/15 issue).

Adverse Reactions
The most common adverse reactions among the patients receiving Kyprolis were anemia, diarrhea, dyspnea, fatigue, insomnia, pyrexia, and thrombocytopenia. Serious adverse reactions among the patients receiving Kyprolis were hypertension, dyspnea, cardiac failure, acute renal failure, ischemic heart disease, and pulmonary hypertension.

Kyprolis is marketed by Onyx Pharmaceuticals, Inc.

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