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Biotechnol Healthc. 2010 Fall; 7(3): 14–15.
PMCID: PMC2957734

Clinical Trials, Research, and FDA Actions

Amy Krajacic, Senior Editor

Late-stage successes

According to a study published in the August 19 issue of the New England Journal of Medicine, Bristol-Myers Squibb’s ipilimumab was found to improve overall survival in patients with metastatic melanoma. The phase 3 trial of the targeted T-cell antibody was conducted with 676 patients who had previously received treatment for their disease. Patients given ipilimumab in addition to a peptide vaccine had a median survival of 10 months, compared with 6.4 months for patients who received the ipilimumab vaccine alone (P<.001), as well as a near doubling of survival rates at 12 months (46 percent vs. 25 percent) and 24 months (24 percent vs. 14 percent).

Positive results from an open--label, single-arm, phase 2b study of Onyx’s carfilzomib have added to the agent’s potential as a treatment for relapsed and refractory multiple myeloma. The proteasome inhibitor achieved an overall response rate of 24 percent and a median duration of response of 7.4 months in 266 patients who had received a median of five previous therapies and whose disease was refractory to their last regimen. The drug was well tolerated with no new toxicities reported. Based on these results, Onyx will file a new drug application by year end.

Trial disappointments

Just a few days after positive late-stage data, Pfizer’s experimental pain relief drug tanezumab ran into several disappointments. Patients who received the therapy as treatment for osteoarthritis saw their condition worsen to the degree that they required joint replacement surgery, and the trial was suspended. Reports of adverse events then caused the U.S. Food and Drug Administration to halt any additional investigations into other indications.

AstraZeneca’s cediranib (Recentin), a potential first-line therapy for colon cancer, was shown to be no more effective than currently available chemotherapy drugs at improving overall patient survival. Although the drug met its coprimary endpoint of progression-free survival, AstraZeneca plans to shift focus and investigate other indications for the agent.

Other research

A team of researchers at Genentech have found that heavy smokers who go on to develop lung cancer may have as many as 50,000 genetic mutations. The findings, which were published in Nature, compared all of the genetic changes found in a single male patient’s lung tumor with that of healthy tissue from the same patient. Researchers suggested that for every three cigarettes the man smoked, one genetic mutation occurred. The man had smoked an average of 25 cigarettes a day for 15 years before his tumor was removed.

According to study results published recently in The Lancet, Roche/Genentech’s trastuzumab (Herceptin) helped patients with gastric cancer live an average of three months longer than those patients treated with chemotherapy alone. The research found that patients with HER2-positive advanced gastric cancer who received the drug lived an average of 13.8 months, while those given chemotherapy alone lived for approximately 11.1 months. Researchers, however, did question the cost-effectiveness of the treatment for the additional months of life gained.

Did you hear?

In the first sign that the FDA plans to crack down on companies that claim to predict inheritable diseases through the use of DNA samples, five genetic test makers have been told they must obtain federal approval before marketing their products for consumer use. 23andMe, deCODE Genetics, Illumina, Navigenics, and Knome were recipients of an online letter from the FDA that stated their products are considered medical devices and, thus, must be federally approved as safe and effective and requested that each company make arrangements to begin the review process.

In a 12–1 vote, an FDA advisory panel called for rescinding approval of Roche/Genentech’s bevacizumab’s (Avastin) as a breast cancer treatment. The panel said that newer studies have indicated that the drug’s benefit in treating advanced breast cancer does not outweigh its risks. These studies have shown more side effects as well as a smaller impact on progression-free survival among women being treated with the agent. If the FDA concurs with the panel’s decision and removes the indication, the action will not affect colon, lung, and other cancers for which bevacizumab has been approved. It is anticipated that the FDA will make its decision by Sept. 17.

SELECTED FDA BIOLOGIC DRUG APPROVALS, MAY 2010–AUGUST 15, 2010
Action dateManufacturerDrug (trade name)IndicationAdministration
Biologics license approval
May 24Genzymealglucosidase alfa (Lumizyme)Patients 8 years of age and older with noninfantile (late-onset) Pompe disease who do not have evidence of cardiac hypertrophyIntravenous injection
June 1Amgendenosumab (Prolia)Treatment of postmenopausal women with osteoporosis at high risk for fractureSubcutaneous injection
SELECTED FDA ACTIONS, JUNE 2010–AUGUST 2010
ManufacturerDrug (trade name)Type of drugIndication soughtNotes
Biologics license application
GlaxoSmith-Kline and Human Genome Sciencesbelimumab (Benlysta)Human monoclonal antibodyTreatment of systemic lupus erythematosusTwo phase 3 trials showed positive data on the drug’s ability to reduce disease activity as well as lower the frequency of flare-ups
Genentech/Rochetrastuzumab-DM1Conjugated monoclonal antibodyTreatment-resistant HER2 positive breast cancerIn a phase 2 trial, the drug shrank tumors in 33 percent of women who had received an average of 7 prior medicines for their disease
Fast-track designation
Acceleron PharmaACE-031Protein therapeuticTreatment of Duchenne Muscular DystrophyA phase 1 trial in healthy postmenopausal women found rapid and sustained dose-dependent increases in lean mass and muscle volume
Genzymealemtuzumab (Campath)Monoclonal antibodyTreatment of relapsing forms of multiple sclerosisThe drug is currently being evaluated in two multicenter, multinational trials
Orphan drug designation
BioSante PharmaceuticalsGVAX CML VaccineVaccineTreatment of chronic myeloid leukemiaGranted June 7
Tarix PharmaceuticalsTXA127Naturally occurring small peptideTreatment to enhance engraftment in patients receiving a stem cell transplantGranted June 28
Cyclacel PharmaceuticalsSapacitabineOrally available nucleoside analogueTreatment of acute myeloid leukemia and myelodysplastic syndromesGranted July 1
Synageva BioPharmaSBC-102Enzyme replacement therapyTreatment of lysosomal acid lipase deficiency, also known as Wolman disease and cholesteryl ester storage diseaseGranted July 7

Sources: Manufacturers’ news releases, FierceBiotech, BIO SmartBrief, wire reports, and weblogs


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