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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.

Thursday, January 29, 2015

The U.S. Food and Drug Administration has expanded the approved use of Imbruvica (ibrutinib) for the treatment of patients with Waldenström’s macroglobulinemia. The drug works by blocking the enzyme that allows the abnormal B-cells in Waldenström’s macroglobulinemia to grow and divide.

 

“Today’s approval highlights the importance of development of drugs for supplemental indications,” 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 news release. “Continued research has discovered new uses of Imbruvica.”

 

The drug had previously received a breakthrough therapy designation for the treatment of Waldenström’s macroglobulinemia (OT 3/25/13 issue), and it is also currently approved for the treatment of patients with mantle cell lymphoma who have received one prior therapy (OT 12/10/13 issue) and patients with chronic lymphocytic leukemia who carry a deletion in chromosome 17 (OT 3/10/14 issue and OT 8/25/14 issue).

 

The drug’s most recent approval is based on a clinical study of 63 previously treated patients with Waldenström’s macroglobulinemia, all of whom received a daily 420 mg orally administered dose of Imbruvica until disease progression or side effects became intolerable. The data showed that 62 percent of the patients had their cancer shrink after treatment, and at the time of the study, the duration of response ranged from 2.8 months to approximately 18.8 months.

 

The most common side effects associated with the drug are thrombocytopenia, neutropenia, diarrhea, anemia, fatigue, musculoskeletal pain, bruising, nausea, upper respiratory tract infection, and rash.

 

The product’s new use is being approved more than two months ahead of its prescription drug user fee goal date of April 17, 2015, the date the FDA was scheduled to complete review of the drug application.

 

Imbruvica is co-marketed by Pharmacyclics and Janssen Biotech.


Thursday, January 29, 2015

The U.S. Food and Drug Administration has granted orphan drug designation to tarextumab (anti-Notch2/3, OMP-59R5) for the treatment of both pancreatic and small cell lung cancers. Tarextumab is a fully human monoclonal antibody that targets the Notch2 and Notch3 receptors. Preclinical studies have suggested the drug works by downregulating Notch pathway signaling causing anti-cancer stem cell activity and by affecting pericytes, impacting the stromal and tumor microenvironment.s

 

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.

 

Approximately 46,420 people were diagnosed with pancreatic cancer in 2014 and 31,390 people were diagnosed with small cell lung cancer, according to estimates from the American Cancer Society.

 

The drug company, OncMed, is currently enrolling patients in two trials for the drug: a randomized Phase II clinical trial of tarextumab with gemcitabine plus Abraxane (paclitaxel-bound particles for injectable suspension, albumin bound) in patients with first-line advanced pancreatic cancer; and a randomized Phase II clinical trial of tarextumab in combination with etoposide and platinum-based chemotherapy for patients with first-line extensive stage small cell lung cancer.

 

Phase 1b clinical and biomarker data from a study of tarextumab in combination with standard of care for patients with pancreatic cancer showed that the drug was well tolerated, side effects were manageable, and that 21 of 29 patients had a partial response or whose disease remained stable. A separate Phase 1b study of tarextumab in combination with etoposide and platinum chemotherapy in patients with small cell lung cancer showed that the combination was well tolerated and that 13 patients (out of the 16 who were evaluable for efficacy) had a partial response and for the three other patients disease was stable.


Monday, January 26, 2015

The U.S. Food and Drug Administration has granted orphan drug designation to liposomal encapsulated paclitaxel easy to use (LEP-ETU) for the treatment of ovarian cancer. LEP-ETU is a new formulation of paclitaxel combined with liposomes, designed to maintain or enhance the anti-tumor properties of paclitaxel, while offering the advantages of a shorter infusion time, no required premedication, fewer side effects, and possibly greater effectiveness if higher doses can be delivered without an increase in side effects.

 

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.

 

Approximately 21,980 women will be diagnosed with ovarian cancer in 2014 and 14,270 women will die from the disease, according to estimates from the American Cancer Society.

 

"By entrapping the paclitaxel with liposomes, we believe our LEP-ETU formulation could potentially have reduced toxicity, while maintaining or enhancing efficacy of the cancer-fighting paclitaxel. We are currently evaluating next steps with respect to this drug product candidate," Michael L. Babich, President and Chief Executive Officer of the drug’s manufacturer, Insys Therapeutics, Inc.

 

A Phase I trial for the drug is currently ongoing.


Tuesday, January 20, 2015

The U.S. Food and Drug Administration has granted Fast Track designation to CPX-351 (cytarabine and daunorubicin) for the treatment of elderly patients with secondary acute myeloid leukemia (AML). CPX-351 is a liposomal formulation of a synergistic five-to-one molar ratio of cytarabine and daunorubicin. Preclinical trials, as well as Phase I and II studies suggest that CPX-351 is more effective for treatment of AML than the current standard of care, cytarabine and daunorubicin administered in a “7+3” combination regimen.

 

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.

 

A Phase III study to evaluate CPX-351 versus the current standard of care has completed enrollment of 300 patients who are between 60 and 75 with untreated secondary AML. Overall survival data from that trial is expected in 2016, according to a news release from the drug’s developer, Celator Pharmaceuticals, Inc.


Wednesday, January 07, 2015

The U.S. Food and Drug Administration has granted Fast Track designation to SGX301 (synthetic hypercin) for the first-line treatment of cutaneous T-cell lymphoma (CTCL). SGX301 is a novel first-in-class photodynamic therapy with the active ingredient synthetic hypercin, a potent photosensitizer that has shown anti-proliferative effects on activated normal human lymphoid cells and has inhibited growth of malignant T-cells isolated from patients with CTCL. The drug is topically applied to skin lesions and then activated by fluorescent light 16 to 24 hours later.

 

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.

 

SGX301 had previously received orphan drug designation, which—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. This designation does not, though, shorten the duration of the regulatory review and approval process. 

 

A phase III clinical trial for the drug is expected to begin this year, according to a press release from the drug’s manufacturer, Soligenix, Inc.

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