A novel treatment regimen involving sapropterin dihydrochloride
AssigneeDipharma S.p.A.
InventorsGilles PAIN, Fabio CANNATA, Bareket GELBHART
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Abstract
The invention relates to an improved tetrahydrobiopterin formulation for treating patients affected by hyperphenylalaninemia. More specifically, the formulation of the invention permits a prolonged residence of tetrahydrobiopterin in the plasma, and/or a higher concentration of sapropterin in the plasma, enabling notably the treatment of HPA patients that would usually not be classified as responders to tetrahydrobiopterin loading test, and therefore left without any pharmacological treatment opportunity, but only with a strictly controlled protein diet.
Claims19
-13. (canceled)
A method of treating hyperphenylalaninemia, comprising administering daily:
- a) once only, a first fast-release formulation containing from 5 mg/kg to 20 mg/kg of sapropterin and a stabilizing agent, the stabilizing agent being present in an amount of up to about 5% w/w of the sapropterin;
- and
- b) a second formulation containing from 2 mg/kg to 20 mg/kg of an antioxidant;
- to a patient in need thereof.
Dependencies are inferred from the claim text.