FDA Grants Orphan Designation to Drug Candidate for Oral GVHD

The FDA has granted Orphan Drug Designation to the drug candidate LP-310, designed for the treatment of oral graft-versus-host disease (GVHD).

LP-310 liposomal tacrolimus is a proprietary oral rinse formulation of LP-10, which was previously granted Orphan Drug Designation by the FDA, according to Lipella Pharmaceuticals, the manufacturer of both LP-310 and LP-10.

The FDA recently cleared a phase IIa clinical trial to evaluate the safety and efficacy of LP-310 in patients with symptomatic oral lichen planus, which currently has no FDA-approved treatment.

“GVHD occurs when donor immune cells attack the recipient’s body tissues after an allogeneic tissue or bone marrow transplant,” Michael Chancellor, MD, Chief Medical Officer at Lipella, said. “GVHD affects approximately 30,000 Americans, and oral GVHD contributes significantly to morbidity in cancer survivors. Morbidity of oral GVHD encompasses significant oral pain and discomfort, making it difficult for patients to eat, drink, and speak. In addition, the risk of oral cavity infection, fibrosis, and even oral cancer increases. Oral GVHD affects patients’ quality of life and is a great unmet need in cancer survivors.”