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.