Amit Pandya, MD: The Discovery of Oral Tranexamic Acid for Melasma

With the introduction of ruxolitinib cream (Opzelura) as the first drug indicated by the US Food and Drug Administration (FDA) to treat vitiligo last year, it’s natural for dermatologists to consider when more therapeutic advances may come for other pigmentary disorders.

As one leading expert explains, though, these classes of disease are complex and difficult-to-treat. Innovation takes patience—and sometimes a little bit of luck.

In the second segment of an interview with HCPLive during the Maui Derm 2023 NP + PA Summer Conference in Colorado Springs this week, Amit Pandya, MD, director of the Clinic for Pigmentory Disorders at Palo Alto Medical Foundation and adjunct professor at the University of Texas Southwestern Medical Center, discussed the armamentarium status of melasma—a common facial pigmentary disorder most frequently observed in women.

“Melasma has historically been one of the most difficult conditions to treat,” Pandya explained. “That hyperpigmentation of the face is chronic and it affects a large percentage of individuals worldwide. We have been focusing my entire career on topical therapies, to try to reduce the pigmentation.”

But it was by what he called “serendipity” that a game-changing discovery was made for treating melasma. About a decade ago, oral tranexamic acid—indicated for reducing menstrual bleeding—was observed to be associated with improved melasma in treated women.

Further analyses showed a twice-daily regimen of the oral treatment provided significant benefit for melasma pigmentation. When it was studied in laboratory, investigators discovered that tranexamic acid blocks conversion of plasminogen to plasma—explaining its effect on menstrual bleeding—but it also impacts plasmin’s role in stimulating melanocytes to increase pigmentation.

The end result is a secondary indication for a drug that has “revolutionized” melasma management, Pandya said. It’s since become available in oral, topical and intradermal forms—but oral administration appears to be the most effective.

“Having an oral agent is convenient, and the efficacy is very good—and of course we combine it with topical agents and sunscreen, and that combination has worked better than anything I’ve seen so far for melasma,” Pandya said.

Pandya additionally discussed the need for advanced screening, diagnostic and disease-monitoring capability in pigmentary disorders. He suggested a smart phone app or other telemedicine feature help provide a more detailed update on the improvement of lesions, pigmentation, or other aesthetic symptoms from pigmentary disorders like vitiligo or melasma.

The field is currently limited to confirming disease improvements at quartile percentages; being able to tell a patient that their vitiligo has improved 10% in the last month, versus 25% in the last 6 months, may have a major impact on their commitment to necessary long-term treatment regimens.

“In the future, if we had some type of technology that told us there’s this definite improvement of pigmentary disorders, that will motivate patients and physicians to continue that treatment,” Pandya said.