Orphan Drugs
Orphan Drugs Specifications
In Europe rare diseases are those affecting less than 1 in 2,000 people and in the USA, fewer than 200,000 people. These diseases are mostly genetic in origin affecting young children and inevitably require long term, lifelong treatment.
Today around 30 million people live with rare and severe diseases in EU and USA and there are more than 7,000 rare and severe diseases known while only around 400 specific orphan drugs are available on the market to treat rare diseases.
In this context, Orphan drugs have become a public health priority in USA, EU and JP supported by various incentives (accelerated regulatory review, market exclusivity, reduced application fees...)
Today around 30 million people live with rare and severe diseases in EU and USA and there are more than 7,000 rare and severe diseases known while only around 400 specific orphan drugs are available on the market to treat rare diseases.
In this context, Orphan drugs have become a public health priority in USA, EU and JP supported by various incentives (accelerated regulatory review, market exclusivity, reduced application fees...)
Our Expertise
As a very experienced CDMO with fully scalable GMP manufacturing facilities Eurofins CDMO network of companies is ideally suited for the development of Orphan medicinal products:
- Customized development and manufacturing facilities for Orphan or Paediatric products
- Regulatory CMC support and a strong background in Orphan and Paediatric projects
- Clinical drug development through to commercial production (sterile & non-sterile) with dedicated facilities for small batche sizes
- Small Molecules, Peptides and Biologics
- Highly potent products
- Several Orphan medicinal products manufactured for commercial use in EU and JP markets
- More than 25 development and clinical batches produced for rare diseases
