Press Announcements following UK Trial

Topical patidegib gel has shown early promise in a phase 2 clinical study in the UK in 2016. 


Investigators at two sites in the United Kingdom enrolled 17 patients with the rare genetic disease and randomised them to receive one of two doses of topical Patidegib or placebo. The trial treated and tracked subjects for 26 weeks to assess the effect of twice-daily administrations of the hedgehog inhibitor on the basal cell carcinomas that characterise Gorlin Syndrome. 

PellePharm (pharmaceutical company) quote "PellePharm's trial represents important progress bringing hope to people affected by the disease who currently have extremely limited options for addressing their invasive BCCs throughout their lifetime'.

The company are now looking to communicate with the US Food & Drug Administration (FDA) to move forward regards a phase 3 clinical trial. 

For further information see the following two articles. BusinessWire and FierceBiotech



 Originally produced by Professor P. A. Farndon, Clinical Geneticist, Jim Costello (deceased) and Margaret Costello.  We are reliant on a team of medical advisors for the clinical content of the website. We are grateful for their continuing support. 
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