MedPharm reported that it is supporting Palvella Therapeutics following Palvella’s announcement of positive topline results from the Phase 3 SELVA study of QTORIN 3.9% rapamycin anhydrous gel in microcystic lymphatic malformations (microcystic LMs). In the SELVA trial, QTORIN rapamycin met its primary and key secondary endpoints, demonstrating clinically meaningful improvements in disease burden for patients with microcystic LMs, a rare condition for which there are currently no U.S. FDA‑approved treatments.
According to MedPharm, the company was selected by Palvella to contribute to the formulation and development of QTORIN rapamycin, from early concept through clinical development and manufacturing readiness. MedPharm’s scientists collaborated with Palvella to address rapamycin’s challenging characteristics, including its large molecular weight, poor solubility, chemical instability, and complex physicochemical properties, and helped design a dermal‑targeted, room‑temperature stable anhydrous gel with high drug loading to locally modulate the mTOR pathway in affected skin while aiming to limit systemic exposure. MedPharm also conducted a multi‑year formulation optimization program using in vitro skin permeation testing and in vitro release testing to refine the formulation for targeted delivery to the site of action, incorporated patient‑informed insights into product design, and supported scale‑up activities, including production of clinical trial material and preparations for potential commercial manufacturing.
Palvella’s QTORIN rapamycin has received Breakthrough Therapy, Orphan Drug, and Fast Track designations from the U.S. FDA for microcystic LMs and is also being studied in other indications such as cutaneous venous malformations and clinically significant angiokeratomas. MedPharm is further collaborating with Palvella on QTORIN pitavastatin for porokeratosis, another rare genetic skin disease with no FDA‑approved therapies, which could become the first targeted treatment option for this condition if approved.