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Orphan drug status for new compound ‘great news’ says Amryt Pharma PLC’s COO

Monday, November 7, 2016 7:29
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Amryt Pharma PLC (LON:AMYT) has been granted orphan drug status in the US for  a compound called AP102 to treat patients with the excess growth condition acromegaly. Chief operating officer Rory Nealon tells Proactive: ”Our focus is to acquire, develop and commercialise products in the orphan drug space … acromegaly is exactly that, it is classified as an orphan condition with an estimated prevalence of 60 patients per million of population”.
”Orphan status is the same in Europe and the USA – it’s designed to encourage companies like ourselves to develop treatments for rare conditions. We get marketing exclusivity and also don’t require the same number of patients for clinical trials”, Nealon added. Amryt has already been awarded orphan drug status for its lead drug EpiSalven, to treat the skin disorder Epidermolysis Bullosa, which is being prepared for a phase III trial early next year.

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