How a global CRO/CDMO helped develop the world’s first drug for a rare disease affecting children

A U.S.-based company working on rare diseases partnered with global CRO/CDMO Syngene to develop and manufacture a first-in-class treatment for a rare disease affecting children.

The company went on to receive authorization for the sale of the product in the E.U. This was followed by regulatory approvals for sale in the U.S. and U.K. The product has now become the first drug in the world to be approved for children suffering from the rare disease.

Read this case study to learn about the product’s journey from Lab to Commercial.

• How Syngene helped the client define the product’s regulatory starting material
• Successfully developed the manufacturing process to improve the product’s throughput and quality 
• Manufactured and supplied the required quantity of the product for preclinical and clinical supply and, later, for commercial manufacturing
• Provided support for submitting marketing applications for the product in various regions

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