Lymphatic Malformation Alliance meets FDA to seek new treatments

SAINT PETERSBURG, Fla., Sept. 22, 2026 /CourierPR/ -- The Lymphatic Malformation Alliance (LMA) has met with senior leaders at the U.S. Food and Drug Administration (FDA) to raise awareness of lymphatic malformations (LMs) and the urgent need for safe and effective treatments. The LMA, a patient advocacy organization dedicated to improving the lives of individuals and families affected by LMs, brought together patient and physician perspectives to bring greater attention to these rare and progressive diseases.

The meeting, which included LMA Executive Director Michael Kelly, MD, PhD, and senior FDA representatives such as Michael Davis, MD, PhD, Director of the FDA’s Center for Drug Evaluation and Research (CDER), Lisa Yanoff, MD, Deputy Director of CDER’s Office of Cardiology, Hematology, Endocrinology, and Nephrology, and Amy Comstock Rick, JD, Associate Director for Rare Disease Strategy and Director of Strategic Coalitions for the FDA’s Rare Disease Innovation Hub, highlighted the significant unmet need for FDA-approved treatments for LMs.

A patient living with LMs participated in the meeting, sharing directly with FDA leaders the impact of living with the condition. The patient described the challenges of managing the disease, which can cause chronic lymphatic leakage, recurrent infections, pain, functional impairment, disfigurement, and substantial psychosocial burden. The patient emphasized the importance of having the opportunity to share their experience directly with FDA leadership, as they wanted to ensure their voice was heard.

Dr. Kelly, the LMA’s Executive Director, shared the experiences and perspectives of the LM community, including the challenges faced by patients and families, and the absence of an FDA-approved treatment specifically for microcystic LMs. The absence of such a treatment underscores the need for continued investment in research and development and treatment approaches that can provide patients with safer and more durable options.

The LMA supports the full and appropriate use of FDA’s existing regulatory framework, including the consideration of natural history and patient experience data, to help advance safe and effective treatments. The patient who participated in the meeting also highlighted the need for regulatory pathways and administrative tools that can expedite development and review for rare and ultra-rare diseases, and how those tools may support treatment development for conditions with very small patient populations.

The LMA will continue to work with patients, families, physicians, researchers, industry partners, and the FDA to increase recognition of LMs, support research and drug development, and advocate for treatments that address the needs of patients and families affected by the disease.

Lymphatic malformations (LMs) are rare vascular anomalies caused by abnormal development of the lymphatic system, often due to mutations in the PIK3CA gene. They can occur anywhere in the body and may involve large balloon-like cysts (macrocysts) or clusters of small, fluid-filled spaces (microcysts) involving the skin and/or deeper soft tissues, organs, and bones. While LMs are often diagnosed in childhood, they can also be identified later in life. Current treatment remains largely focused on symptom management through the improvised use of surgery, sclerotherapy, laser therapy, and off-label or compounded medications. Despite a tremendous need, there are currently no FDA-approved therapies specifically indicated for LMs.

The Lymphatic Malformation Alliance (LMA) serves as a trusted hub for patients, families, clinicians, and researchers affected by isolated lymphatic malformations and complex lymphatic anomalies. Their mission is to accelerate understanding, expand access to care, and advance meaningful therapeutic breakthroughs for a community that today has no FDA-approved therapies. With the patient voice at the center of its work, LMA provides education and resources, fosters collaboration across the lymphatic malformation community, and works to ensure that no one has to navigate these rare conditions alone. The LMA is committed to building a connected global community and creating meaningful pathways toward greater knowledge, improved care, and new treatment options.