Clinical stage pharmaceutical company Lipella Pharmaceuticals has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for its drug candidate, LP-310, which is intended to treat oral Graft-versus-Host Disease (GvHD).
A clinical stage pipeline asset for the company, LP-310 is intended to be indicated for inflammatory diseases of the oral cavity, including oral lichen planus and oral GvHD.
Earlier, LP-10 received ODD from the FDA, thereby making LP-310 the second of the company’s leading product candidates to receive this designation.
LP-310 liposomal tacrolimus is an oral rinse formulation of the company’s lead drug candidate, LP-10.
Recently, the company received FDA approval for a Phase IIa clinical trial to analyse LP-310’s safety and efficacy in patients suffering with symptomatic oral lichen planus, which presently does not have any FDA approved treatment and can severely affect patient’s quality of life.
Lipella CEO Dr. Jonathan Kaufman said, “We are very pleased to have received Orphan Drug Designation for LP-310 in oral GvHD. This designation is an example of the way we build value, pursuing all available resources that can de-risk and accelerate our clinical research programs. Our collaborative practice with the FDA is critical to our ability to increase the value of all of our clinical assets. We look forward to advancing this drug as a potential treatment for this painful complication of chronic GvHD.”
Lipella chief medical officer Dr. Michael Chancellor said, “GvHD occurs when donor immune cells attack the recipient’s body tissues after an allogeneic tissue or bone marrow transplant. 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.”
GvHD occurs when donor bone marrow or stem cells attack the recipient after a treatment for leukemia, lymphoma, and other hematological cancers. The condition of oral lichen planus can happen as a single manifestation of chronic GvHD.
The FDA grants ODD status to drugs and biologics that are meant for the treatment, prevention or diagnosis of a rare disease or condition that impacts lesser than 200,000 people in the country.