Tolero's alvocidib receives EMA orphan drug designation for treatment of AML patients

Tolero Pharmaceuticals, Inc., a clinical-stage company developing treatments for serious hematological diseases, today announced that the European Medicines Agency (EMA) has granted orphan drug designation for alvocidib for the treatment of patients with acute myeloid leukemia (AML). Alvocidib is in clinical development for patients with relapsed/refractory and frontline intermediate or high-risk AML.

"The granting of orphan drug designation by the EMA for alvocidib underscores the great unmet medical need for patients with AML, many of whom experience limited effectiveness with current therapies," said David J. Bearss, Ph.D., Tolero's Chief Executive Officer. "Alvocidib has demonstrated high response rates to date in patients with AML, and we look forward to initiating Phase 3 clinical trials this year."

Alvocidib has been evaluated in multiple Phase 2 clinical trials involving approximately 400 patients. These Phase 2 clinical trials included patients with relapsed/refractory AML as well as those with frontline intermediate or high-risk AML. Intermediate and high-risk AML patients are determined by a profile of clinical, cytogenetic and molecular features, and typically these patients have a poor prognosis due largely to the limited effectiveness of current therapeutic options.

The EMA orphan designation is a status assigned to a medicine intended for use against a rare condition (prevalence of the condition in the European Union must not be more than 5 in 10,000) and allows a pharmaceutical company to benefit from incentives offered by the EU to develop a medicine for the treatment, prevention or diagnosis of a disease that is life-threatening or a chronically debilitating rare disease. In addition to a 10-year period of marketing exclusivity in the EU after product approval, orphan drug designation provides incentives for companies seeking protocol assistance from the EMA during the product development phase and direct access to centralized marketing authorization.