Sunesis Pharma gets European orphan drug status for vosaroxin to treat AML – pharmabiz.com

Sunesis Pharmaceuticals, Inc., a biopharmaceutical company, has received orphan drug designation from European Commission to its lead development candidate, vosaroxin, for the treatment of acute myeloid leukaemia (AML).

The designation provides for 10 years of marketing exclusivity in all EU member countries following product approval. Vosaroxin has previously received orphan drug and fast track designations from the US Food and Drug Administration (FDA).

“The European Commission’s decision reinforces the global potential of vosaroxin and recognizes the desperate need for new treatment options in AML, an indication for which treatment standards have not changed appreciably in the past thirty years,” said Dr Adam Craig, EVP Development & CMO of Sunesis. “European orphan drug designation is the latest in a series of regulatory milestones that have strengthened the commercial opportunity for vosaroxin on both sides of the Atlantic. These include the potential for market exclusivity to 2030 and an expedited review process in the US, as well as new patents issued in the European Union with exclusivity to 2025.”

As established by the European Medicines Agency (EMA), orphan designation is granted to product candidates intended for the treatment of a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union. It also provides for scientific advice and regulatory assistance from the EMA during the product development phase, direct access to centralized marketing authorization, and certain financial incentives for companies developing orphan drug candidates. Orphan drugs are eligible for a reduction of fees associated with pre-authorization inspections, as well as marketing authorization application fees and certain other fees for qualifying companies.

Sunesis is currently enrolling patients in its VALOR trial, a phase III, multinational, randomized, double-blind, placebo-controlled, pivotal clinical trial of vosaroxin in combination with cytarabine in first relapsed or refractory acute myeloid leukaemia.

VALOR is a phase III, randomized, double-blind, placebo-controlled, pivotal trial in patients with first relapsed or refractory AML. The trial is expected to enroll 450 evaluable patients at approximately 110 leading sites in the US, Canada, Europe, Australia and New Zealand. The VALOR trial is currently enrolling patients, who are randomized one to one to receive either vosaroxin on days one and four in combination with cytarabine daily for five days, or placebo in combination with cytarabine. Additionally, the VALOR trial employs an innovative, adaptive trial design that allows for a one-time sample size adjustment by the DSMB at the interim analysis to maintain adequate power across a broader range of survival outcomes. The trial’s primary endpoint is overall survival.

Vosaroxin is a first-in-class anti-cancer quinolone derivative, (AQD), a class of compounds that has not been used previously for the treatment of cancer. Vosaroxin both intercalates DNA and inhibits topoisomerase II, resulting in replication-dependent, site-selective DNA damage, G2 arrest and apoptosis.

Sunesis is a biopharmaceutical company focused on the development and commercialization of new oncology therapeutics for the treatment of solid and hematologic cancers.

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Sunesis Pharma gets European orphan drug status for vosaroxin to treat AML – pharmabiz.com

Sunesis Pharmaceuticals, Inc., a biopharmaceutical company, has received orphan drug designation from European Commission to its lead development candidate, vosaroxin, for the treatment of acute myeloid leukaemia (AML).

The designation provides for 10 years of marketing exclusivity in all EU member countries following product approval. Vosaroxin has previously received orphan drug and fast track designations from the US Food and Drug Administration (FDA).

“The European Commission’s decision reinforces the global potential of vosaroxin and recognizes the desperate need for new treatment options in AML, an indication for which treatment standards have not changed appreciably in the past thirty years,” said Dr Adam Craig, EVP Development & CMO of Sunesis. “European orphan drug designation is the latest in a series of regulatory milestones that have strengthened the commercial opportunity for vosaroxin on both sides of the Atlantic. These include the potential for market exclusivity to 2030 and an expedited review process in the US, as well as new patents issued in the European Union with exclusivity to 2025.”

As established by the European Medicines Agency (EMA), orphan designation is granted to product candidates intended for the treatment of a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union. It also provides for scientific advice and regulatory assistance from the EMA during the product development phase, direct access to centralized marketing authorization, and certain financial incentives for companies developing orphan drug candidates. Orphan drugs are eligible for a reduction of fees associated with pre-authorization inspections, as well as marketing authorization application fees and certain other fees for qualifying companies.

Sunesis is currently enrolling patients in its VALOR trial, a phase III, multinational, randomized, double-blind, placebo-controlled, pivotal clinical trial of vosaroxin in combination with cytarabine in first relapsed or refractory acute myeloid leukaemia.

VALOR is a phase III, randomized, double-blind, placebo-controlled, pivotal trial in patients with first relapsed or refractory AML. The trial is expected to enroll 450 evaluable patients at approximately 110 leading sites in the US, Canada, Europe, Australia and New Zealand. The VALOR trial is currently enrolling patients, who are randomized one to one to receive either vosaroxin on days one and four in combination with cytarabine daily for five days, or placebo in combination with cytarabine. Additionally, the VALOR trial employs an innovative, adaptive trial design that allows for a one-time sample size adjustment by the DSMB at the interim analysis to maintain adequate power across a broader range of survival outcomes. The trial’s primary endpoint is overall survival.

Vosaroxin is a first-in-class anti-cancer quinolone derivative, (AQD), a class of compounds that has not been used previously for the treatment of cancer. Vosaroxin both intercalates DNA and inhibits topoisomerase II, resulting in replication-dependent, site-selective DNA damage, G2 arrest and apoptosis.

Sunesis is a biopharmaceutical company focused on the development and commercialization of new oncology therapeutics for the treatment of solid and hematologic cancers.

View the original article here

FDA Approves Voraxaze to Treat Patients with Toxic Methotrexate Levels

FDA Approves Voraxaze to Treat Patients with Toxic Methotrexate Levels

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January 17, 2012 — The U.S. Food and Drug Administration today approved Voraxaze (glucarpidase) to treat patients with toxic levels of methotrexate in their blood due to kidney failure.

Methotrexate is a commonly used cancer chemotherapy drug normally eliminated from the body by the kidneys. However, patients receiving high doses of methotrexate may develop kidney failure.

Voraxaze is an enzyme that rapidly reduces methotrexate levels by breaking the chemotherapy drug down to a form that can be eliminated from the body. Voraxaze is administered directly into a patient’s vein (intravenously).

“Prolonged exposure to high levels of methotrexate can result in kidney and liver damage, severe mouth sores, damage to the lining of the intestine, skin rashes, and death due to low blood counts,” said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “Voraxaze is an important new treatment option for cancer patients aimed at preventing these toxicities associated with sustained high levels of methotrexate.”

Voraxaze has an orphan drug designation, given to therapies indicated for rare or specific disease populations.

A single clinical study of 22 patients evaluated the effectiveness of Voraxaze. All patients received Voraxaze treatment. The study considered treatment a success if the methotrexate level fell below a critical level within 15 minutes and stayed below the critical level for eight days. Ten of the 22 patients achieved this standard. Although not all patients experienced this result, Voraxaze eliminated 95 percent of the methotrexate in all patients.

A separate clinical study evaluated the safety of Voraxaze in 290 patients experiencing problems clearing methotrexate from their blood.

The most common side effects observed in greater than one percent of patients in the clinical study were low blood pressure (hypotension), headache, nausea, vomiting, flushing, and abnormal sensation (paraesthesia).

Voraxaze is marketed by BTG International Inc., West Conshohocken, Pa.

Source: FDA

Posted: January 2012

Voraxaze (glucarpidase) FDA Approval History

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Stem-Cell Trial Failed to Treat Heart Failure

SATURDAY, March 24 (HealthDay News) — An innovative approach using patients’ own bone marrow cells to treat chronic heart failure came up short in terms of effectiveness, researchers report.

Use of stem cell therapy to repair the slow, steady damage done to heart muscle and improve heart function is safe, but has not been shown to improve most measures of heart function, the study authors said.

“For the measures we paid most attention to, we saw no effect, there is no question about that,” said researcher Dr. Lemuel Moye, a professor of biostatistics at the University of Texas School of Public Health in Houston.

“Ultimately, this is going to pay off handsomely for individuals and for public health in general, but it’s going to take years of work,” Moye said. “We are the vanguard looking for new promising lines of research.”

While the hoped-for results didn’t materialize, there appeared to be a small improvement in some patients, he said. “When we looked at another commonly used measure of heart function called ejection fraction, or the strength of the heart’s pumping, that’s where all the action was,” Moye noted.

It’s hard to know which measures of heart function to look at, Moye explained. “We have had some difficulty with that,” he said.

Future research will look at other measures of heart function, pay more attention to the characteristics of the cells that are injected and determine which cells are best, he added.

Cardiac cells and other types of specially prepared cells are available now that were not accessible when this study started in 2009, Moye pointed out.

The results of the trial, which was sponsored by the U.S. National Heart, Lung, and Blood Institute, were to be presented Saturday at the American College of Cardiology’s annual meeting in Chicago. The report was also published online March 24 in the Journal of the American Medical Association.

For the study, Moye and colleagues worked with 92 patients, average age 63 and mostly male, who had heart failure with and without chest pain. They were randomly assigned to receive either an injection of 100 million bone marrow cells from their own bone marrow, or an inactive placebo. Patients in both groups also received aggressive medical therapy.

During the trial, the researchers looked for improvements in blood volume in the heart, oxygen use by the heart and blood flow through the heart.

After six months, the researchers said they saw no difference between the groups in these measures. Nor was any difference seen in the extent of heart damage, heart movement during contractions or overall condition.

Moye’s team did find a slight improvement in the heart’s ability to pump blood among patients 62 and younger. The improvement was small, only 3.1 percent, but patients in the placebo group declined 1.6 percent in this measure, they said.

It’s possible that cells of younger people are more potent, and that’s borne out by improvement in heart function seen in younger patients who did not get bone marrow cells, Moye said.

“We have demonstrated that the characteristics of the cells are correlated with heart function, so that the better the cells, the better the response — so even in patients who did not get stem cells, those younger patients did better,” he said.

Commenting on the study, Dr. Gregg Fonarow, director of the Ahmanson-University of California, Los Angeles, Cardiomyopathy Center, said there has been “tremendous interest in cell-based therapies” to treat acute and chronic heart disease and chronic heart failure.

Most studies trying delivery of different types of cells have been small and not adequately powered to demonstrate improvement in cardiac function or clinical outcomes, and results have been mixed, Fonarow said.

“While this study failed to meet any of its primary or secondary endpoints, the insights provided will be helpful in designing future trials,” Fonarow added. “However, whether cell-based therapies will be of therapeutic value to patients with heart disease and heart failure remains to be seen.”

More information

For more information on heart disease, visit the American Heart Association.

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