Vascarta Inc., a biopharmaceutical company focused on developing therapies for underserved patient populations, announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation to its lead drug candidate, Vasceptor (VAS-101), for the treatment of Sickle Cell Disease. The designation represents a significant regulatory milestone for the company's novel topical curcumin formulation.
Regulatory Benefits and Market Impact
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 substantial 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."
Novel Therapeutic Approach
VAS-101 is a patented topical curcumin formulation delivered using patented transdermal technology, designed to overcome the limited bioavailability and suboptimal effectiveness of oral curcumin dosing. The drug targets multiple pathways involved in Sickle Cell Disease pathophysiology.
Dr. Joel Friedman, Professor in the Department of Microbiology & Immunology at Albert Einstein College of Medicine and Vascarta Scientific Founder & Head of the Scientific Advisory Board, explained the therapeutic rationale: "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."
Clinical Development Status
A study published in PNAS Nexus suggests VAS-101 may reduce chronic pain, stabilize red blood cells, and lower inflammation in Sickle Cell Disease. The drug is currently in a Phase 1 clinical trial at the Foundation for Sickle Cell Disease Research in Hollywood, Florida. Vasceptor is exclusively licensed to Vascarta from the Albert Einstein College of Medicine.
While curcumin is recognized for its safety profile and anti-inflammatory, antioxidant, and anti-sickling properties, its clinical application has been historically limited by poor oral bioavailability. Vascarta's transdermal delivery system aims to address this limitation.
Addressing Significant Unmet Medical Need
Sickle Cell Disease is the most common inherited genetic disorder that affects primarily African American and non-Hispanic Black individuals in the United States. 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. The disease is characterized by severe pain, inflammation, oxidative stress, and organ damage, contributing to poor quality of life and reduced survival. SCD-associated complications include anemia, acute and chronic pain, infections, pneumonia and acute chest syndrome, stroke, kidney, liver, and heart disease.
Recently approved SCD therapies do not mitigate pain, and patients are often prescribed multiple drugs that frequently have undesirable side effects. The unmet medical need for most SCD sufferers remains significant, with demand for safer therapies that improve outcomes, optimize compliance for patients of all ages, minimize the need for blood transfusions, and reduce dependence on potentially harmful chronic pain medications.
