Pharma Focus Asia

Palvella Therapeutics Developed First-of-its-kind QTORIN rapamycin™ for Microcystic Lymphatic Malformations

Palvella Therapeutics has developed QTORIN rapamycin™, a revolutionary treatment specifically designed to address Microcystic Lymphatic Malformations (Microcystic LMs).

QTORIN rapamycin, a anhydrous gel formulation containing 3.9 percent rapamycin, is in active development to target Microcystic LMs and other severe skin disorders caused by the overstimulation of the mammalian target of rapamycin (mTOR) pathway. 

This innovative treatment holds promise for alleviating functional impairments associated with these conditions.

Microcystic Lymphatic Malformations (Microcystic LMs) constitute a rare and chronically debilitating genetic disorder resulting from the dysregulation of the PI3K/mTOR pathway. 

This condition is defined by the presence of localised clusters of malformed lymphatic vessels that breach the skin barrier, leading to continuous leakage of lymph fluid (lymphorrhea) and bleeding. Consequently, individuals affected by Microcystic LMs often experience recurrent severe infections and cellulitis, further exacerbating their condition.

The progressive nature of Microcystic Lymphatic Malformations (LMs) and the absence of FDA-approved treatments underscore the critical need for research and development in this area. The lack of specific therapies can significantly impact the quality of life for individuals with these conditions, leading to complications and ongoing morbidity.

QTORIN, a formulation of rapamycin, holds promise in addressing the urgent unmet needs of patients dealing with Microcystic Lymphatic Malformations (LMs), especially those who endure significant morbidity from a very young age due to this lifelong condition.
 

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