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