VAS-101 (Vasceptor) Receives Orphan Drug Designation for Sickle Cell Disease

On 12 June 2025, Vascarta Inc. announced its receipt of the Orphan Drug Designation from the US Food and Drug Administration (FDA) for its lead drug candidate, VAS-101 (Vasceptor), to treat sickle cell disease (SCD). This designation will provide VAS-101 with 7 years of market exclusivity on approval and exemption from some FDA fees, among other benefits.1

VAS-101 is a patented topical curcumin formulation delivered through Vascarta’s patented transdermal technology. The company intends for the treatment to fill the niche made by limited bioavailability and effectiveness of oral curcumin dosing. Previous animal studies have suggested that VAS-101 may reduce chronic pain, stabilize red blood cells, and lower inflammation in patients with SCD.1

“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 Richard Prince, PhD, president and chief executive officer of Vascarta. “We plan to work closely with the United States Food & Drug Administration to bring Vasceptor to market as rapidly as possible.”1

The phase 1 clinical trial is currently ongoing at the Foundation for Sickle Cell Disease Research in Hollywood, Florida, and includes 10 patients who will be treated with VAS-101 twice a week for 4 weeks. Patients will be split into 2 groups of 5 individuals, one treated topically and one treated sublingually.2

Primary endpoints for this study include assessing the safety and tolerability of VAS-101 as well as its effects on impaired blood flow dynamics such as adhesion molecule expression and erythrocyte fragility parameters. Secondary objectives include mean change in red blood cell sickling kinetics and oxygen dissociation curves, as well as the effect of VAS-101 on inflammatory markers related to the activation of sterile inflammation over the 29-day study duration.2

“I’m proud that we are taking part in Vascarta’s pilot study, which explores a new transdermal approach to pain management for individuals living with Sickle Cell Disease (SCD),” said Lanetta Bronte-Hall, MD, MPH, president of FSCDR. “Pain remains one of the most challenging and life-disrupting aspects of this disease, and new approaches are urgently needed. While early in development, this research represents an important step toward finding new options that could one day help ease that burden.”2

VAS-101 is also currently being tested to treat osteoarthritis in a 60-patient population in a randomized, double-blind, placebo-controlled phase 1 study in Perth, Australia. A total of 19 patients received therapy as of March 2025; pending positive results, a phase 2 study is planned for early 2026.3

References

1. PR Newswire. Vascarta Receives FDA Orphan Drug Designation for Vasceptor in the Treatment of Sickle Cell Disease. June 12, 2025. Accessed June 12, 2025. https://prnmedia.prnewswire.com/news-releases/vascarta-receives-fda-orphan-drug-designation-for-vasceptor-in-the-treatment-of-sickle-cell-disease-302480299.html

2. PR Newswire. Vascarta commences Proof of Concept Phase 1 clinical study of VAS-101 in sickle cell disease in the United States. April 15, 2025. Accessed June 12, 2025. https://www.prnewswire.com/news-releases/vascarta-commences-proof-of-concept-phase-1-clinical-study-of-vas-101-in-sickle-cell-disease-in-the-united-states-302428782.html?tc=eml_cleartime

3. PR Newswire. Vascarta commences a Phase I clinical study of VAS101 in osteoarthritis. March 18, 2025. Accessed June 12, 2025. https://www.prnewswire.com/news-releases/vascarta-commences-a-phase-i-clinical-study-of-vas101-in-osteoarthritis-302403061.html