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Volasertib Granted ‘Orphan Drug’ Designation for Acute Myeloid Leukemia

Late-stage trial evaluates Polo-like kinase inhibitor

The FDA has granted an “orphan drug” designation to volasertib (Boehringer Ingelheim) for the treatment of acute myeloid leukemia (AML). The drug is currently being evaluated in a phase III clinical trial for the treatment of patients 65 years of age or older with previously untreated AML who are ineligible for intensive remission-induction therapy.

Volasertib has not been approved by the FDA, and its safety and efficacy have not been established.

AML is an aggressive cancer of the bone marrow and blood. It accounts for approximately one-third of all adult leukemias in the Western world and has one of the lowest survival rates of all leukemias. AML is primarily a disease of later adulthood; the average age of an AML patient is 65 to 70 years. The recommended standard of care is intensive chemotherapy, but many patients, because of age and comorbidities, cannot tolerate this therapeutic approach. For them, treatment options are limited, and their prognosis is typically poor. Volasertib is currently being investigated in this specific patient population.

The FDA’s “orphan drug” designation is a status given to investigational compounds intended to treat a rare disease or condition that has limited treatment options. To qualify for the designation, a medication must, among other requirements, address a disease that affects fewer than 200,000 people in the U.S.

Volasertib is an investigational compound that inhibits Polo-like kinase (Plk) enzymes. Plk1 is the best characterized kinase of the Plk family. Plk1 regulates mitosis. Its inhibition can result in prolonged cell-cycle arrest, ultimately leading to apoptosis.

Volasertib is currently being evaluated in clinical studies for the treatment of various solid tumors and hematologic cancers.

Initiated in January 2013, the POLO-AML-2 trial is an ongoing global phase III study designed to assess the efficacy and safety of volasertib in combination with low-dose cytarabine compared with placebo and low-dose cytarabine in patients 65 years of age or older with previously untreated AML who are ineligible for intensive remission-induction therapy. The trial is currently enrolling eligible patients.

Source: Boehringer Ingelheim; April 17, 2014.

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