12 November 2018 - Palvella Therapeutics today announced that the U.S. FDA has granted fast track designation for its lead product candidate PTX-022 (novel, high-strength rapamycin topical formulation, optimised for dermal targeting) for the treatment of pachyonychia congenita. 

PTX-022 leverages Palvella’s proprietary and patent-pending QTORIN formulation and delivery technology. QTORIN employs a highly specific composition of excipients that enables distribution of mTOR inhibitors into the basal keratinocytes, which harbour the mutant keratin genes that are the primary defect in pachyonychia congenita. QTORIN further enables drug penetration into the reticular dermis where neovascularization and inflammatory components of the pachyonychia congenita pathology manifest.

PTX-022 is supported by multiple issued method of use patents in the U.S. broadly covering the use of mTOR inhibitors in pachyonychia congenita through 2032. PTX-022 has also received U.S. FDA orphan drug designation and EMA orphan drug designation.

Read Palvella Therapeutics press release