The Food and Drug Administration (FDA) has granted Fast Track designation to a novel formulation of rapamycin for the treatment of pachyonychia congenita (PC), a rare and lifelong monogenic disease that affects the skin, including the soles of the feet. 

The treatment, PTX-022, is a high-strength topical formulation of rapamycin, an mTOR inhibitor. Through Palvella’s proprietary QTORIN formulation, PTX-022 targets basal keratinocytes, which harbor the mutant keratin genes that cause PC.

“The recognition by the FDA of the potential of PTX-022 to treat pachyonychia congenita builds upon the compelling scientific rationale and the encouraging early human clinical results seen with rapamycin in the treatment of PC,” stated Joyce M. Teng, MD, PhD, clinical professor of dermatology and pediatrics at Stanford University School of Medicine. “I look forward to working closely with the Palvella team as we move expeditiously to advance PTX-022 into a Phase 2/3 clinical study.”  

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PTX-022 was previously granted Orphan Drug status for PC, which according to the US National Library of Medicine, probably affects several thousand people worldwide.

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