Vascarta Receives FDA Orphan Drug Designation for Vasceptor® in the Treatment of Sickle Cell Disease

SUMMIT, N.J., June 12, 2025 /PRNewswire/ -- Vascarta Inc., a biopharmaceutical company committed to advancing innovative therapies for underserved patient populations, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its lead drug candidate, Vasceptor^® (VAS-101), for the treatment of Sickle Cell Disease (SCD).

Orphan Drug Designation is awarded to therapies intended to treat rare diseases that affect fewer than 200,000 people in the United States. This designation provides significant benefits, including seven years of market exclusivity upon approval, tax credits for qualified clinical trials, and exemption from certain FDA fees.

"The attainment of Orphan Drug Designation represents an important regulatory milestone for Vascarta and underscores the therapeutic potential of Vasceptor^® to provide safe and effective relief to patients living with this debilitating disease," said Dr. Richard Prince, CEO & President of Vascarta. "We plan to work closely with the United States Food & Drug Administration to bring Vasceptor^® to market as rapidly as possible."

Dr. Joel Friedman, Professor, Department of Microbiology & Immunology, Albert Einstein College of Medicine, and Vascarta Scientific Founder & Head, Scientific Advisory Board, stated, "The preclinical results to date showing pain reduction and therapeutic efficacy resulting from our novel approach of targeting red blood cell instability, neuro-inflammation and vascular inflammation bodes well for the development of a widely accessible therapy that prevents and treats many if not most of the clinical consequences of SCD." Vasceptor^® is exclusively licensed to Vascarta from the Albert Einstein College of Medicine (Bronx, New York, USA).

About Vasceptor^® (VAS-101)

VAS-101, developed by Vascarta Inc. (Summit, NJ, USA), is a patented topical curcumin formulation that is delivered using patented transdermal technology and that is designed to overcome the limited bioavailability and suboptimal effectiveness of oral dosing of curcumin. A study in PNAS Nexus suggests VAS-101 may reduce chronic pain, stabilize red blood cells, and lower inflammation in sickle cell disease (SCD). It is currently in a Phase 1 clinical trial at the Foundation for Sickle Cell Disease Research (FSCDR) in Hollywood, Florida. While curcumin is known for its safety and anti-inflammatory, antioxidant, and anti-sickling properties, its clinical use has been limited by poor oral bioavailability.

About Sickle Cell Disease

Sickle Cell Disease is the most common inherited genetic disorder that affects primarily African American and non – Hispanic Black individuals in the United States. SCD associated complications include anemia, acute and chronic pain, infections, pneumonia and acute chest syndrome, stroke, kidney, liver, and heart disease.

Current estimates indicate there are 175,000 cases of SCD in the USA and 45,000 in European Union countries. The estimated life expectancy of those with sickle cell disease in the USA is more than 20 years shorter than the average expected lifespan.

SCD is caused by a single point mutation in the globin gene leading to sickling of red blood cells. It is characterized by severe pain, inflammation, oxidative stress, and organ damage, which contributes to the poor quality of life and reduced survival. Recently approved SCD therapies do not mitigate pain, and patients are often on multiple drugs which often have undesirable side effects. 

The unmet medical need for most SCD sufferers is significant. Better therapies that are safe, improve outcomes, optimize compliance for patients of all ages, minimize the need for blood transfusions, and reduce the need for chronic administration of potentially harmful pain medications are needed.

About Vascarta

Vascarta Inc. is a clinical stage pharmaceutical company developing efficient transdermal and transmucosal delivery of pharmaceuticals to address inflammatory conditions with an initial focus on Sickle Cell Disease and osteoarthritis. To learn more, contact Vascarta Chairman, CEO & President, Dr. Richard Prince, at [email protected]. Media requests should be directed to David Hymson at [email protected].

SOURCE Vascarta Inc.