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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, December 17, 2014

The U.S. Food and Drug Administration has approved Somatuline Depot (lanreotide) Injection for the treatment of adult patients with gastroenteropancreatic neuroendocrine tumors (GEP-NETs), which are unresectable, well- or moderately differentiated, locally advanced, or metastatic. The drug is being approved after being granted priority review earlier this year (OT 10/10/14 issue).


The FDA's priority review designation shortens the time to complete a drug's review and aims to deliver a decision on marketing approval designation for drugs that may offer major advances in treatment or provide a treatment where no adequate therapy exists within six months under the Prescription Drug User Fee Act (PDUFA).


Somatuline—which contains lanreotide acetate, a somatostatin analogue that inhibits the secretion of several endocrine, exocrine, and paracrine amines and peptides—will be delivered via a newly approved, ready-to-use, prefilled syringe that includes a retractable needle guard to help avoid needle sticks, and it is manufactured without latex or natural dry rubber, according to a news release from the manufacturer, Ispen Biopharmaceuticals, Inc. The new delivery device does not require reconstitution and is a low volume (0.5 mL) deep subcutaneous injection offering a streamlined process that supports full dose delivery.


The approval is based on data from a Phase III, double-blind study of 204 patients with advanced gastrointestinal and pancreatic neuroendocrine tumors who received treatment with Somatuline or placebo. Median progression-free survival for the patients receiving Somatuline was not reached (and will be greater than 22 months), and 65.1 percent of those patients had no disease progression and had not died at 96 weeks; whereas median progression-free survival for patients treated with the placebo was 16.6 months, and at 96 weeks 33 percent of those patients had no disease progression and had not died.


The most common adverse events reported in the trial for Somatuline were abdominal pain, musculoskeletal pain, vomiting, headache, injection site reaction, hyperglycemia, hypertension, and cholelithiasis. Five percent of patients receiving Somatuline discontinued treatment due to an adverse event, compared with three percent of patients who received the placebo.

Friday, December 12, 2014

The U.S. Food and Drug Administration has expanded the approved use of Cyramza (ramucirumab) for the treatment of patients with metastatic non-small cell lung cancer. Cyramza, an angiogenesis inhibitor that blocks the blood supply to tumors, is intended for patients whose tumor has progressed during or following treatment with platinum-based chemotherapy. Cyramza is to be used in combination with docetaxel.


Cyramza was also approved earlier this year as a single agent to treat patients with advanced stomach cancer or gastroesophageal junction adenocarcinoma (OT 5/25/14 issue). That indication was expanded in November to include the chemotherapy paclitaxel with that use for Cyramza (OT 12/10/14 issue).


“Today’s approval is the third indication that Cyramza has received in 2014,” 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. “The commitment to study Cyramza in a variety of malignancies provides important treatment options to patients.”


Cyramza’s latest approval is based on a clinical study of 1,253 patients with previously treated and progressive lung cancer who were randomly assigned to receive Cyramza plus docetaxel or a placebo plus docetaxel. Median overall survival for patients treated with Cyramza plus docetaxel was 10.5, compared with 9.1 months for the patients who received placebo plus docetaxel. Treatment was stopped for the other patients in the trial due to disease progression or intolerable side effects.


“By adding ramucirumab [Cyramza] to docetaxel, patients were able to live longer than those who were treated with the standard approach," the study’s principal investigator, Edward Garon, MD, a researcher at UCLA's Jonsson Comprehensive Cancer Center, said in a news release from UCLA. "We are pleased to have access to a drug that lengthens survival time in a population of lung cancer patients who often have few treatment options."


The most common side effects associated with Cyramza plus docetaxel from the study were neutropenia, fatigue, and stomatitis. Cyramza can also cause severe bleeding, blood clots, elevation in blood pressure, and may impair wound healing.


The FDA reviewed Cyramza’s application for this new use under the agency’s priority review program, which provides for an expedited review of drugs that are intended to treat a serious disease or condition and, if approved, would offer significant improvement compared to marketed products.


Cyramza is marketed by Eli Lilly.

Wednesday, December 10, 2014

The U.S. Food and Drug Administration has today approved Gardasil 9 (Human Papillomavirus 9-valent Vaccine, Recombinant) for the prevention of certain diseases caused by nine types of Human Papillomavirus—five additional types of HPV than were covered by Gardasil, which was approved by the FDA in 2006 (OT 8/10/06 issue). Gardasil 9 has the potential to prevent approximately 90 percent of cervical, vulvar, vaginal, and anal cancers.


“Vaccination is a critical public health measure for lowering the risk of most cervical, genital and anal cancers caused by HPV,” Karen Midthun, MD, Director of the FDA’s Center for Biologics Evaluation and Research, said in a news release. “The approval of Gardasil 9 provides broader protection against HPV-related cancers.”


The Gardasil 9 vaccine is approved for use in females ages nine through 26 and males ages nine through 15. It is approved for the prevention of cervical, vulvar, vaginal, and anal cancers caused by HPV types 16, 18, 31, 33, 45, 52, and 58, and for the prevention of genital warts caused by HPV types 6 or 11. The new vaccine adds protection against HPV types 31, 33, 45, 52, and 58, which cause approximately 20 percent of cervical cancers and are not covered by previously FDA-approved vaccines.


Gardasil 9 was evaluated in a randomized, controlled, clinical study (conducted in the U.S. and internationally) of approximately 14,000 females 16 through 26, all of whom tested negative for vaccine HPV types at the start of the study. The females received either Gardasil or Gardasil 9. Gardasil 9 was found to be 97 percent effective in preventing cervical, vulvar, and vaginal cancers caused by the five additional HPV types (31, 33, 45, 52, and 58) that Gardasil does not protect against. Additionally, Gardasil 9 was found to be as effective as Gardasil for the prevention of diseases caused by the four shared HPV subtypes (6, 11, 16, and 18) based on similar antibody responses in participants in clinical studies.


Due to the low incidence of anal cancer caused by the five additional HPV types, the prevention of anal cancer is based on Gardasil’s demonstrated effectiveness of 78 percent and additional data on antibodies in males and females who received Gardasil 9.


The effectiveness of Gardasil 9 in females and males ages nine through 15 was determined in studies that measured antibody response to the vaccine in approximately 1,200 males and 2,800 females in this age group. Antibody responses for these individuals were similar to those in females 16 through 26. And based on these results, the vaccine is expected to have similar effectiveness when used in this younger age group, the FDA news release notes.


The safety of Gardasil 9 was evaluated in approximately 13,000 males and females; the most commonly reported adverse reactions were injection site pain, swelling, redness, and headaches.


Gardasil 9 is administered as three separate shots, with the initial dose followed by additional shots given two and six months later. For all of the indications for use approved by the FDA, Gardasil 9’s full potential for benefit is obtained by those who are vaccinated prior to becoming infected with the HPV strains covered by the vaccine.


Gardasil 9 is manufactured by Merck Sharp & Dohme Corp.

Wednesday, December 10, 2014

The U.S. Food and Drug Administration has granted Fast Track designation to DPX-Survivac as maintenance therapy for patients with advanced ovarian, fallopian tube, and peritoneal cancer who have no measurable disease following surgery and front-line platinum/taxane chemotherapy to improve their progression-free survival. DPX-Survivac is a novel cancer immune therapy that stimulates the immune system to produce T cell responses targeting the tumor associated antigen survivin.


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 design of a large randomized Phase II trial in ovarian cancer is being finalized, following reports of positive results from earlier Phase I trials for the drug in patients with ovarian cancer, according to a news release from the manufacturer, Immunovaccine Inc.

Thursday, December 04, 2014

The U.S. Food and Drug Administration has approved a new use for Jakafi (ruxolitinib)—for the treatment of patients with the chronic bone marrow disease polycythemia vera. The new use for Jakafi is intended to treat patients who have inadequate response to or cannot tolerate hydroxyurea, another drug prescribed to treat polycythemia vera.


The drug works by inhibiting the enzymes Janus Associated Kinase (JAK) 1 and 2, which are involved in regulating blood and immunological functioning.


“The approval of Jakafi for polycythemia vera underscores the importance of developing drugs matched to our increasing knowledge of the mechanisms of diseases,” 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. “The trial used to evaluate Jakafi confirmed clinically meaningful reductions in spleen size and the need for phlebotomies to control the disease.”


The drug’s approval to treat polycythemia vera is intended to help decrease the occurrence of splenomegaly and the need for phlebotomy, the FDA’s news release also noted.


Jakafi’s safety and effectiveness for this indication were evaluated in a clinical trial of 222 patients with polycythemia vera who had had the disease for at least 24 weeks and who had an inadequate response to or could not tolerate hydroxyurea, had undergone a phlebotomy procedure, and had exhibited an enlarged spleen. The study was designed to measure the reduced need for phlebotomy and reduction in spleen volume. Patients received either Jakafi or best available therapy, as determined by the investigator.


The data showed that 21 percent of patients treated with Jakafi experienced a reduction in the need for a phlebotomy and a reduction in spleen volume, compared to one percent of patients who received best available therapy.


The most common side effects reported for the patients who received Jakafi were anemia and thrombocytopenia. And the most common non-blood related side effects were dizziness, constipation, and shingles.


The FDA reviewed Jakafi’s use for polycythemia vera under the agency’s priority review program because, at the time the application was submitted, the drug demonstrated the potential to be a significant improvement in safety or effectiveness over available therapy in the treatment of a serious condition. Priority review provides an expedited review of a drug’s application. Jakafi also received orphan product designation because it is intended to treat a rare disease.


Jakafi had previously received FDA approval in 2011 for the treatment of patients with immediate or high-risk myelofibrosis, including primary myelofibrosis, post-polycythemia vera myelofibrosis, and post-essential thrombocythemia myelofibrosis.


Jakafi is marketed by Incyte Corp.

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