[April 23, 2014] |
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Tolero Pharmaceuticals Receives FDA Orphan Drug Designation for Alvocidib for the Treatment of Acute Myeloid Leukemia
SALT LAKE CITY --(Business Wire)--
Tolero Pharmaceuticals, Inc., a clinical-stage company developing
treatments for serious hematological diseases, today announced that the
U.S. Food and Drug Administration (FDA) has granted Orphan Drug
designation for Alvocidib for the treatment of patients with acute
myeloid leukemia (AML). Alvocidib is being tested in patients with
intermediate or high-risk AML, which is determined by a profile of
clinical, cytogenetic and molecular features, and typically, individuals
with this profile have a poor prognosis due to limited treatment options.
"The granting of Orphan Drug Designation by the FDA for Alvocidib in
acute myeloid leukemia is a very important milestone in the development
program for Alvocidib, and emphasizes our view that this is an important
disease area of great unmet medical need," said David J. Bearss, Ph.D.,
Tolero's Chief Executive Officer. "Tolero Pharmaceuticals is pleased
with the results that Alvocidib has demonstrated in patients with AML
and we are actively pursuing the development of Alvocidib for
intermediate and high-risk AML patients who desperately need new options
for the treatment of their disease. We look forward to sharing detailed
study results of Alvocidib that support further development of the
compound at an upcoming medical meeting this year."
Alvocidib has een evaluated in multiple Phase 2 clinical trials
involving approximately 400 patients with both relapsed/refractory or
frontline, previously untreated intermediate and high-risk AML. In these
trials, Alvocidib has been evaluated as a single agent as well as in
combination with approved agents including cytarabine and mitozantrone.
Alvocidib is currently being investigated in a Phase 2 study for the
treatment of intermediate and high-risk AML in combination with standard
of care agents.
Orphan designation by the FDA is granted to promote the development of
drugs that target conditions affecting 200,000 or fewer U.S. patients
annually and that are expected to provide significant therapeutic
advantage over existing treatments. Orphan designation qualifies a
company for benefits that apply across all stages of drug development,
including an accelerated approval process, seven years of market
exclusivity following marketing approval, tax credits on U.S. clinical
trials, eligibility for orphan drug grants, and a waiver of certain
administrative fees.
About Alvocidib
Alvocidib is a potent cyclin-dependent kinase (CDK) small molecule
inhibitor in development as a frontline combination therapy for acute
myeloid leukemia and relapsed/refractory chronic lymphocytic leukemia
and other hematologic disorders. CDKs are regulatory proteins and
enzymes that are critical to a cell's ability to replicate as well as
critical regulators of gene expression. Because of the key role CDK
deregulation plays in runaway cell division and growth, CDK inhibitors
remain an attractive target for the treatment of various cancers.
About Acute Myeloid Leukemia
About 18,000 plus new cases of AML are expected to be diagnosed this
year in the United States, according to estimates from the American
Cancer Society. AML is a very rapidly growing cancer of the bone marrow
and the myeloid lineage of blood cells. It is the most common acute
leukemia in adults and accounts for approximately one-third of all adult
leukemias in the United States and Europe. Typically, the prognosis of
AML patients is categorized as low, intermediate or high-risk based on a
profile of clinical, cytogenetic and molecular features which are used
to assign risk of a poor outcome for treatment. Intermediate and
high-risk AML constitute a significant percentage of all cases of adult
AML. For these patients, treatment options are limited and their
prognosis is usually poor. Alvocidib is currently being investigated in
this specific patient population.
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