The Food and Drug Administration has granted Orphan Drug status to three drugs for four indications: E7777 for cutaneous T-cell lymphoma; EPZ-5676 for subtypes of acute leukemias in which the MLL gene is rearranged due to a chromosomal translocation (MLL-r), and MM-111 gastric and esophageal cancers.
E7777, made by Eisai Inc., is currently in a pivotal clinical trial that will be used to support its submission for approval, a news release from the company notes.
EPZ-5676 (Epizyme, Inc.) is a small-molecule inhibitor of the DOT1L histone methyltransferase, and was tested in a Phase I clinical trial last year. The study is currently in a dose-escalation phase with plans to expand in the second half of this year to exclusively enroll patients with MLL-r, the company notes.
The biospecific antibody MM-111 was given two separate Orphan Drug designations-for the treatment of esophageal cancer and for the treatment of gastric and gastroesophageal junction cancers. The drug is designed to inhibit ErbB3 (HER3) receptor signaling in cancers that overexpress ErbB2 (HER2). These cancers are associated with poor prognosis, and research suggests ErbB2 expression to be associated with resistance to current treatments.
MM-111, marketed by Merrimack Pharmaceuticals, Inc., is currently being tested in a Phase II study in advanced gastric, esophageal, and gastroesophageal junction cancers.
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.