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Mild PKU
Tetrahydrobiopterin responsive PKU/HPA
March 31, 2008


Auf Deutsch

History It is important to remember that the synthesis was initially started for the treatment of 10 children in 1980 and now there are only approx. 250 BH4 deficient patients being treated with Schircks Tetrahydrobiopterin tablets world-wide.

Use 

 Because of recent publications many mild PKU patients have been tested and found to be tetrahydrobiopterin  responsive.  Not all tetrahydrobiopterin  responders can be successfully treated with tetrahydrobiopterin. Those loaded with tetrahydrobiopterin, who show phenylalanine levels within the normal range over at least a week on no diet or a relaxed diet, can be tried with tetrahydrobiopterin treatment instead of the PKU diet or tried with a combination of both (relaxed diet with tetrahydrobiopterin supplement). We realize that because the PKU diet is difficult to follow that both parents and children will welcome the new therapy (10-20 mg/kg/day tetrahydrobiopterin  in tablet form). Details of the treatment of patients can be found in the SPC and in the Physicians Guide of Dr. Blau.

No testing has been performed to date on pregnant women so although it would seem a suitable treatment for tetrahydrobiopterin responsive maternal PKU further toxicity testing will be required before this treatment can be considered.
Toxicity Tetrahydrobiopterin is a substance which is found in most tissues of healthy individuals. More information on tetrahydrobiopterin can be found on our external links page. Although no formal clinical studies have been performed on Schircks Tetrahydrobiopterin tablets, patients have been treated with low doses of Schircks Tetrahydrobiopterin tablets up to 10 mg/kg/day for over 25 years without any adverse effects been reported. A few patients have been treated with higher doses recently and no ill effects have been reported to us to date but limited long term data is available and each persons response to tetrahydrobiopterin is dependant on so many factors that it is difficult to compare patients.
Financial strain

 

Patients suffering from mild PKU who are responsive to tetrahydrobiopterin will probably require at least 10 mg/kg/day tetrahydrobiopterin. Schircks Tetrahydrobiopterin tablets are not a registered drug. 

Schircks Tetrahydrobiopterin tablets for the treatment of tetrahydrobiopterin deficiency is usually paid for by insurance companies as there is no alternative treatment for this life threatening disorder. 

Mild PKU patients can alternatively be treated with the PKU diet. Although the diet is also expensive because it has been around for longer, more insurance companies pay for it. It can be difficult to convince insurance companies to pay for Schircks Tetrahydrobiopterin tablets for the treatment of PKU patients. Sometimes treatment is covered by research funds etc. These funds usually expire, after a certain time, leaving the parents and patients in a worrying situation.

It is critical that this financial side of the treatment is examined before starting patients on treatment. Otherwise the relief of the strain of the diet will be replaced with bigger financial strain.

We are doing our utmost to reduce our prices by improving the efficiency of the manufacture. But the paperwork required by regulatory authorities is complicated and time consuming and thus costly.