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Drug Development

CanPKU+ recently worked with the University of Toronto and some of their Health Science students for course work in PCL 402.  One team was eager to create some road maps for us regarding Drug Development. The material they provided is excellent at showing the early stages of drug development. To see the PDF click the link below each section.

We wish Eugene Alfonzo II Alteza, Justin Chia, Pei-Yu (Samantha) Chen, and Sa Eun Shin all the best in their future careers.

Global Genes - Allies in Rare Disease has a downloadable document available here. This is a 32 page document regarding the roadmap of drug development. It may not be Canadian Specific. We have captured page 32 and added it here.

Introduction of Generic Drugs

Where some drugs are currently


Kuvan was approved for use but still not covered in some provinces in Canada. Kuvan Fact Sheet in Canada based on Jan 2023

The Generic drug will be introduced into Canada before all provinces are approved for coverage.


CADTH has released their Final Recommendations for use in Canada. (This does not mean the drug is ready to get to the patients. Next steps will still include the price negotiations)

Two years of pegvaliase in Germany: Experiences and best practice recommendations

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