Orphan Designation

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Bringing to market novel therapies for orphan, rare and ultra-rare conditions is something patients, patient groups and regulatory authorities encourage. We’ll help you align your plans with the appropriate options.


Our experts will help you identify your target population, and apply for incentives and fast-track options designed to bring new drugs to the patients who need them. We’ll work alongside you to develop your orphan drug strategy, prepare and submit your application, and to identify and capitalize on orphan-related market opportunities.


Signposting you to the tools to accelerate your journey


To optimize your success with orphan drug designation, we’ll help identify the available tools aimed at developers of drugs targeted at orphan, rare and ultra-rare diseases, and take advantage of the available benefits – including reduced fees, development support and, in some cases, accelerated approval. It’s something we’ll help you factor into your integrated drug development strategy.


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Insights Hub

Sharing insights, questioning norms, asking the right questions - we'll never lose sight of what you're striving for. Our insight and expertise will help you bring the most innovative therapies within reach of patients around the world.
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If you’re developing something truly ground-breaking, Biopharma Excellence is the nearest resource you’ll find to someone who’s been there before.


    We are now Cencora PharmaLex 

    PharmaLex is now part of Cencora, a leading global healthcare company with a foundation in pharmaceutical distribution. In 2024 and beyond PharmaLex and all the companies in PharmaLex family will begin a journey to becoming Cencora as well. Cencora brings the companies and services together under one new name. The name will change, but the level of attention and service you receive remains the same.  

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