70 Years of PKU Scientific Progress

> Basic Research on
Phenylketonuria (PKU)

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Testing for PAH Mutations

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Key Dates

1934 - Asborn Folling describes PKU

1935 - Lionel Penrose assigns the name PKU

1937 - George Jervis PKU due to enzyme deficiency

1953 - Horst Bickel Suggest dietary treatment

1961 - Robert Guthrie develops simple and inexpensive test for PKU

1966 - Guthrie test becomes standard

1967 - Dick Koch creates National Study on PKU

1992 - Savio Woo identifies gene PAH

1993 - Diet for life recommended McDonald Creates PKU mouse

1996 - Stevens Lab determines PAH 3D Structure

1999 - Kure/Blau propose BH4 treatment

2007 - Kuvan approved

2008 - PEG-PAL enters clinical trials

Total: 5624 Scientific Publications

Sapropterin (KUVAN ^TM) Status

BioMarin recently completed clinical trials conducted in 30 sites in the US, Canada and Europe

Phase 1 trial began November 20, 2003.
Phase 2 trial began December 23, 2004.
Phase 3 trial began April 7, 2005.
FDA drug approval, End of 2007.
8 months of use now on the market
One of the fastest ever FDA clinical trials, thanks in large part to people like Dr. Barbara Burton

RxTrials Institute Drug Pipeline Alert May 28, 2008 | Vol. 6 No. 22

BioMarin Initiates Phase I Clinical Study of PEG-PAL BioMarin Pharmaceutical has started treatment in its Phase I clinical study of PEG-PAL for phenylketonuria (PKU).

The primary objective of the study is to assess the safety and tolerability of single, subcutaneous injections of PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase) in subjects with PKU, the company said. The open-label, multicenter study will be conducted in roughly 35 PKU patients. BioMarin said it expects enrollment to be completed late this year.