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FDA grants orphan drug designation to IMO-8400 for DLBCL
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The FDA granted orphan drug designation to IMO-8400 for the treatment of diffuse large B-cell lymphoma, according to a press release from the drug’s manufacturer.
IMO-8400 (Idera Pharmaceuticals) — an antagonist of the endosomal Toll-like receptors (TLRs) 7, 8 and 9 — is currently being evaluated in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who harbor a MYD88 L265P oncogenic mutation.
Preclinical study data — presented in 2014 at the American Association for Cancer Research Annual Meeting — suggested that IMO-8400 inhibited the survival and proliferation of human B-cell lymphoma cells harboring the oncogenic MYD88 L265P genetic mutation.
IMO-8400 has been well tolerated in a phase 1 dose-escalating trial composed of 42 healthy subjects who received doses up to 0.6 mg/kg for 4 weeks. The drug has also shown inhibition of immune responses mediated by TLRs 7, 8, and 9 in this population.
"The Orphan Drug designation granted today represents another positive milestone for our B-Cell Lymphoma clinical development program," James J. O'Leary, MD, Idera's interim chief medical officer, said in a press release. "We continue to advance our efforts in DLBCL, as well as our ongoing clinical trial in Waldenström's macroglobulinemia which we expect to complete and have full data available in the fourth quarter of this year."
The FDA’s Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.