Lipella Pharmaceuticals secures FDA approval for expanded access program for LP-310 in oral lichen planus

Lipella Pharmaceuticals Inc., a clinical-stage biotechnology company dedicated to addressing unmet medical needs, has announced that it has received approval from the U.S. Food and Drug Administration (FDA) for an Expanded Access Program (EAP) for its investigational therapy, LP-310. This oral rinse formulation is designed to treat oral lichen planus (OLP), a chronic inflammatory condition affecting the mucous membranes inside the mouth.

The FDA’s approval of this program represents a critical milestone for Lipella Pharmaceuticals Inc., as it not only expands access to LP-310 for patients outside of clinical trials but also underscores the growing recognition of the therapy’s potential to address a significant gap in the treatment landscape for oral lichen planus. With no FDA-approved therapies currently available for OLP, this development brings renewed hope to the approximately six million Americans living with the condition.

What is oral lichen planus, and why is there an urgent need for new treatments?

Oral lichen planus is a chronic, immune-mediated inflammatory condition that primarily affects the mucous membranes of the mouth. It manifests as painful white patches, swollen tissues, burning sensations, and, in severe cases, open sores. The disease can significantly impair a patient’s quality of life, making eating, speaking, and basic oral functions difficult. Despite its prevalence, oral lichen planus remains poorly understood, and the absence of FDA-approved treatments has left patients reliant on off-label therapies that often provide inconsistent results.

Current management strategies for OLP typically involve corticosteroids and immunosuppressive agents, which, while sometimes effective in reducing inflammation, come with risks associated with long-term use, including systemic side effects and potential complications. This underscores the urgent need for targeted therapies like LP-310 that can provide localized relief without the broader systemic impact.

How does LP-310 work, and what makes it different from existing therapies?

LP-310 is an oral rinse formulation derived from tacrolimus, an immunosuppressive drug traditionally used in transplant medicine to prevent organ rejection. However, in the case of oral lichen planus, LP-310 has been specifically designed to deliver the therapeutic benefits of tacrolimus locally within the oral cavity, thereby minimizing systemic absorption and reducing the risks typically associated with long-term steroid or systemic immunosuppressive use.

The therapy targets inflammation at its source by modulating the immune response within the affected mucosal tissues. This localized approach allows for effective symptom relief while reducing potential side effects such as immune suppression, which can leave patients vulnerable to infections and other complications. By focusing on targeted delivery, LP-310 aims to offer a safer, more effective option for OLP patients who have exhausted other treatment avenues.

What does FDA approval of the Expanded Access Program mean for patients?

The FDA’s approval of Lipella Pharmaceuticals Inc.’s Expanded Access Program for LP-310 is a significant development for patients with oral lichen planus who have limited treatment options. Expanded Access Programs, sometimes referred to as “compassionate use” programs, allow patients with serious or life-threatening conditions to access investigational therapies that are still undergoing clinical trials.

For OLP patients, this means that even if they are not eligible to participate in ongoing clinical studies, they can still access LP-310 under the supervision of their healthcare providers. This is particularly important given the debilitating nature of oral lichen planus and the lack of effective, approved therapies currently on the market.

Michael Chancellor, Co-Founder and Chief Medical Officer of Lipella Pharmaceuticals Inc., highlighted the significance of the FDA’s decision, stating that it represents a pivotal moment in the company’s mission to address unmet medical needs. According to Chancellor, making LP-310 available through the Expanded Access Program not only broadens treatment options for patients but also reaffirms Lipella Pharmaceuticals Inc.’s commitment to advancing innovative therapies for challenging conditions like oral lichen planus.

What’s next for LP-310 in clinical development?

While the Expanded Access Program marks an important step, Lipella Pharmaceuticals Inc. continues to focus on generating robust clinical data to support the eventual regulatory approval of LP-310. The company is currently conducting a Phase 2a multicenter, dose-ranging clinical trial designed to evaluate the safety, tolerability, and efficacy of LP-310 in adults with symptomatic oral lichen planus.

This ongoing study is being conducted at seven active sites across the United States, with participants receiving one of three different doses of LP-310 (0.25 mg, 0.5 mg, or 1.0 mg of tacrolimus). The trial’s design aims to identify the optimal dosing regimen while providing valuable insights into how the therapy performs across diverse patient populations.

The data collected from this trial will play a crucial role in Lipella Pharmaceuticals Inc.’s efforts to secure full FDA approval for LP-310. In the meantime, the Expanded Access Program ensures that patients who need immediate relief can benefit from the therapy while the company continues its research.

Why does LP-310’s approval matter in the broader context of OLP treatment?

The approval of LP-310’s Expanded Access Program by the FDA reflects more than just a regulatory milestone for Lipella Pharmaceuticals Inc.—it represents a shift in how oral lichen planus is being approached within the medical community. For years, patients with OLP have had to rely on treatments that were not specifically designed for their condition, often facing limited efficacy and troubling side effects.

With LP-310, there is now a therapy in development that directly targets the underlying inflammatory processes driving OLP, offering a tailored approach to treatment. This not only provides hope for patients currently struggling with the condition but also signals a broader trend toward more personalized, targeted therapies in the field of oral medicine and beyond.

As the medical community awaits further clinical trial results, LP-310’s availability through the Expanded Access Program ensures that patients facing the daily challenges of oral lichen planus can access a promising new option that may significantly improve their quality of life.