The U.S. Food and Drug Administration has granted Orphan Drug status to Selinexor (KPT-330), an oral selective inhibitor of nuclear export (SINE) compound, for the treatment of patients with diffuse large B-cell lymphoma (DLBCL). Orphan drug status had previously been granted to the drug for the treatment of patients with acute myeloid leukemia (http://bit.ly/1kddAGI).
The Orphan Drug designation—to encourage development of drugs in the diagnosis, prevention, or treatment of a medical condition affecting fewer than 200,000 people in the U.S.—grants a product market exclusivity for a seven-year period if the sponsor complies with certain FDA specifications, as well as tax credits and prescription drug user fee waivers. The designation does not, though, shorten the duration of the regulatory review and approval process.
Approximately 21,000 patients will be diagnosed with DLBCL in the United States in 2014, according to American Cancer Society estimates.
Phase I clinical trials for Selinexor, which is marketed by Karyopharm Therapeutics, are ongoing and continuing to enroll patients.