Lymphatic Malformation Alliance (LMA) Meets with FDA Leadership to Elevate Awareness of Lymphatic Malformations and Advance the Urgent Need for Approved Treatments

LMA brings patient and physician perspectives to FDA leadership to elevate awareness of lymphatic malformations

Patient experience highlights the serious, lifelong burden and significant unmet need

LMA calls for greater recognition and continued attention to a community with no FDA-approved treatment

SAINT PETERSBURG, Fla., Sept. 22, 2026 (GLOBE NEWSWIRE) — The Lymphatic Malformation Alliance (LMA), a patient advocacy organization dedicated to improving the lives of individuals and families affected by lymphatic malformations, today announced that it recently met with senior leaders at the U.S. Food and Drug Administration (FDA) to bring greater attention to lymphatic malformations (LMs) and the urgent need for a safe and effective treatment for patients living with these rare and progressive diseases.

The meeting brought together LMA Executive Director Michael Kelly, MD, PhD, and senior FDA representatives, including 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.

A patient living with LMs also participated in the meeting, sharing directly with FDA leaders what it means to live with the condition and how it can affect daily life, health and well-being. Dr. Kelly shared the experiences and perspectives of the LM community while also providing the physician perspective, describing the challenges faced by patients and families and the absence of an FDA-approved treatment specifically for microcystic LMs.

“For too long, patients with LMs have lived with the significant challenges of this disease without an FDA-approved treatment,” said Michael Kelly, MD, PhD, Executive Director of the Lymphatic Malformation Alliance. “LMs are lifelong progressive conditions that can cause chronic lymphatic leakage, recurrent infections, pain, functional impairment, disfigurement, and substantial psychosocial burden. We wanted FDA leadership to hear directly from someone living with this disease and to understand the very real impact it has on our patients and their families. We hope this conversation helps bring greater attention to the severity of LMs and the urgent need to advance safe and effective therapies for this community.”

For patients with LMs, managing the disease can involve procedures such as sclerotherapy, surgery, laser treatment and medications. These approaches may help manage individual manifestations of the disease but do not address its underlying cause and, due to the progressive nature of these diseases, often require repeated interventions. The absence of an FDA-approved 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 patient who participated in the meeting described the importance of having the opportunity to share their experience directly with FDA leadership:

“Living with LMs can be exhausting because it affects so much of everyday life. There are times when I have to change my plans, miss out on things I want to do or spend time dealing with my condition when I would rather be focused on work, friends or just living my life. There are also the appointments, procedures and uncertainty that come with having a condition that doesn’t simply go away. It can be frustrating to have so much of your life shaped by a disease that most people have never even heard of. Having the chance to speak directly with FDA leaders meant a lot to me because I wanted them to understand what it is really like to live with this disease and how urgently patients need better and safer treatments,” said a young adult living with a lymphatic malformation.

The discussion also focused on the regulatory pathways and administrative tools FDA already has in place to 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. LMA supports the full and appropriate use of FDA’s existing regulatory framework, including consideration of natural history and patient experience data, to help advance safe and effective treatments. For a disease with no approved treatment, off-label use may already be embedded in clinical practice or treatment guidelines, raising genuine questions of clinical equipoise. This, along with very small patient populations, can complicate the feasibility of randomized trials. LMA believes these considerations, together with objective clinical endpoints capable of demonstrating treatment effect, deserve meaningful consideration in trial designs for this community.

“LMA’s responsibility as an advocacy organization is to make sure LM patients are heard,” continued Dr. Kelly. “We are grateful for the opportunity to speak directly with FDA leadership and share both the patient experience and the realities faced by clinicians. We want to see meaningful progress toward treatments that can change the lives of patients and families living with LMs. We walked away from our meeting with a sense that FDA leadership shares those goals, and we look forward to working together with both Agency leadership and review personnel to make review of treatments for LM a priority.”

The LMA will continue working 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.

About Lymphatic Malformations

Lymphatic malformations (LMs) are rare vascular anomalies caused by abnormal development of the lymphatic system, often due to mutations in the PIK3CA gene. LMs 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. They can occur in isolation, together (mixed), or as part of associated syndromes. Patients can present with a myriad of symptoms that continue to change as the patient ages as a result of being genetically programmed to grow. Depending on their location and extent, LMs can cause swelling, pain, bleeding, infection, and functional complications like trouble breathing or swallowing. While LMs are often diagnosed in childhood, they can also be identified later in life. LMs are not curable. 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.

About 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. Our 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. LMA is committed to building a connected global community and creating meaningful pathways toward greater knowledge, improved care and new treatment options.

Learn more at lmalliance.org

Contact Information

Michael Kelly, MD, PhD
Executive Director
Lymphatic Malformation Alliance
mkelly@lmalliance.org