FDA Grants Orphan Designation for Lipella's LP-310 Drug Candidate for Oral Graft-versus-Host Disease

Lipella Pharmaceuticals' LP-310 clinical stage asset is formulated for delivering active agents to the oral cavity for diseases of the mouth including oral lichen planus and oral graft-versus-host disease

PITTSBURGH, Nov. 10, 2023 /PRNewswire/ -- Lipella Pharmaceuticals Inc. (Nasdaq: LIPO), a clinical stage pharmaceutical company addressing serious diseases with significant unmet need based in Pittsburgh, PA, announced today that it has been granted "Orphan Drug Designation" from the U.S. Food and Drug Administration (FDA) for its drug candidate, LP-310, which has been designed for the treatment of oral Graft-versus-Host Disease (GvHD). 

LP-310 is Lipella's clinical stage pipeline asset intended to be indicated for inflammatory diseases of the oral cavity, including oral lichen planus and oral GvHD.

LP-310 liposomal tacrolimus is a proprietary oral rinse formulation of Lipella's lead drug candidate, LP-10. Lipella recently received FDA clearance for a Phase 2a clinical trial to evaluate LP-310's safety and efficacy in patients with symptomatic oral lichen planus, which currently has no FDA approved treatment, and can severely impact a patient's quality of life.

Dr. Jonathan Kaufman, Ph.D., Chief Executive Officer of Lipella, 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."

Dr. Michael Chancellor, Chief Medical Officer at Lipella, 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."

The FDA's "Orphan Drug Designation" program provides orphan status to drugs and biologics that are intended for the treatment, prevention or diagnosis of a rare disease or condition that affects fewer than 200,000 people in the United States. Orphan Drug Designation qualifies sponsors for incentives, including tax credits for qualified clinical trials, exemption from user fees and a potential seven years of market exclusivity after approval.

Lipella's LP-10 was previously granted Orphan Drug Designation by the FDA, making LP-310 the second of Lipella's leading product candidates to receive this designation.

About LP-310

LP-310 liposomal tacrolimus is a proprietary oral rinse formulation of Lipella's lead drug candidate, LP-10.

About Graft-versus-Host Disease

Graft-Versus-Host Disease (GvHD) occurs when  donor bone marrow or stem cells attack the recipient following a treatment for leukemia, lymphoma, and other hematological cancers. Acute GvHD most commonly affects the skin, liver, and the gastrointestinal tract, while chronic GvHD affects the skin, mouth, eyes, lungs, stomach, bowel and liver, as well as other organs. Oral lichen planus can occur as a single manifestation of chronic GvHD.

About Lipella Pharmaceuticals Inc.

Lipella Pharmaceuticals Inc. is a clinical stage pharmaceutical company with a focus on capital efficient therapeutic development opportunities, including the proprietary repositioning of generics for rare, morbid diseases that currently have no adequate treatments.  Additional information is available at www.lipella.com.

Forward-Looking Statements

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Contacts
Dr. Jonathan Kaufman, CEO
Lipella Pharmaceuticals
[email protected] 
1-412-894-1853

Jeff Ramson
PCG Advisory
[email protected]
1-917-912-9130

SOURCE Lipella Pharmaceuticals Inc.