On December 30, 2014 Idera Pharmaceuticals reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for IMO-8400, an antagonist of the endosomal Toll-like receptors (TLRs) 7, 8 and 9, for the treatment of Waldenström’s macroglobulinemia (WM) (Press release Idera Pharmaceuticals, DEC 30, 2014, View Source [SID:1234501255]). Additionally, Idera is providing a progress update on the ongoing Phase 1/2 clinical trial being conducted in WM.

Idera is currently conducting a Phase 1/2 clinical trial of IMO-8400 in patients with WM (ClinicalTrials.gov identifier NCT02092909) who have a history of relapse or failure to respond to one or more prior therapies. In B‐cell lymphomas characterized by the MYD88 L265P oncogenic mutation, including WM, preclinical studies have shown that TLR signaling is overactivated, thereby enabling tumor cell survival and proliferation. About 90 percent of WM patients are reported to harbor the MYD88 L265P oncogenic mutation.

The objectives of the trial are to evaluate the compound’s safety, tolerability and potential clinical activity. The protocol includes three dose-escalation cohorts of IMO-8400 administered subcutaneously. The trial’s independent data review committee has completed its review of four-week safety data from the second dose cohort (1.2 mg/kg/week) and has determined that Idera may open enrollment in the third dose cohort (2.4 mg/kg/week). Final 24-week safety and clinical activity data are anticipated in the second half of 2015.

Orphan drug designation is granted by the FDA Office of Orphan Products Development to drugs intended for the treatment of a rare disease or condition that affects fewer than 200,000 people in the United States. This designation provides certain incentives, including eligibility for federal grants, research and development tax credits, waiver of PDUFA filing fees and a seven-year marketing exclusivity period, once the product is approved and as long as orphan drug designation is maintained.

The approval of an orphan drug designation request does not alter the standard regulatory requirements and processes for obtaining marketing approval of an investigational drug. Sponsors must establish safety and efficacy of a compound in the treatment of a disease through adequate and well-controlled studies.