Karyopharm drug wins 'orphan' designation
Natick pharmaceutical company Karyopharm Therapeutics said its leading drug candidate has received federal designation as an “orphan drug” to help treat acute myeloid leukemia (AML).
Karyopharm said the designation for the drug Selinexor is designed to encourage the development of drugs that may help patients who suffer from rare diseases.
The U.S. Food and Drug Administration (FDA), which approves drugs in development, provides orphan status to drugs that are used to treat, diagnose or prevent rare diseases and disorders that affect at most 200,000 Americans, or that affect more than 200,000 but for which companies are not expected to recover the costs of developing and marketing the drug.
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, tax credits on U.S. clinical trials, eligibility for orphan drug grants and a waiver of certain administrative fees, according to a statement from Karyopharm.
The FDA action is a "significant milestone in the Selinexor development program,” Karyopharm CEO Michael Kauffman said in a statement. "We look forward to sharing updated clinical data on Selinexor at upcoming medical meetings."
AML is the most common form of acute leukemia in adults, accounting for more than 80 percent of such cases. According to the American Cancer Society, an estimated 18,860 people will be diagnosed with AML in the United States in 2014, with 10,460 patients expected to die from the disease, Karyopharm said.
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