New PKU Gene Therapy Treatment Receives Testing Approval

As most of us are aware, there are a number of exciting new treatments for PKU that have been introduced recently or currently are in development that promise to radically change the PKU lifestyle, allowing for much more flexibility in the diet, or eliminating the need for a special diet altogether. Long term, some studies being done using gene therapy techniques have the potential to at least partially “correct” the inborn error, allow the body to produce the phenylalanine hydroxylase enzyme naturally.

In this vein, BioMarin announced recently that the FDA has granted Investigational New Drug (IND) status approval of a BioMarin investigational gene therapy known as BMN. This means that BioMarin can now begin clinical study of the drug. (A similar approval was also just received in the UK under its Clinical Trial Application (CTA).

This is good news for all of us with an interest in PKU. For more information, please see the Press Release.