subscribe
 

Developing treatment for debilitating rare disease

26th February 2019


MedPharm has announced the expansion of its partnership with Palvella Therapeutics, a Philadelphia-based biopharmaceutical company focused on developing and commercialising therapies for debilitating, rare genetic diseases. To date, MedPharm has employed its specialist formulation expertise to support Palvella’s development of a novel, high-strength rapamycin topical formulation for application to the skin (PTX 022) as a disease-modifying treatment for pachyonychia congenita (PC). Most recently, MedPharm has made arrangements to manufacture the clinical (IMP) batches for use in Palvella’s upcoming Phase 2/3 clinical study.

“MedPharm is very proud to be part of a collaboration with Palvella and the PC community which will potentially benefit PC patients directly. Currently this is a serious unmet medical need that has no approved treatments,” said Professor Marc Brown, MedPharm’s CSO and co-founder. “I have met many PC patients over the years and am excited to be continuing our support of Palvella and PC Project as they move closer to making a real difference to these patients’ lives.”

PC is a rare, chronically debilitating, and lifelong genetic disease in which mutated genes responsible for keratin production lead to extreme cell fragility. This dramatically limits patients’ ability to walk and perform everyday tasks. Most patients suffer from deformed nails and almost all experience some level of pain. No treatments are currently approved for PC in the USA or Europe.





Subscribe

Subscribe



Newsbrief

FREE NEWSBRIEF SUBSCRIPTION

To receive the Scientist Live weekly email NewsBrief please enter your details below

Twitter Icon © Setform Limited
subscribe