DIPHARMA SA

Nitisinone Dipharma

NITISINONE DIPHARMA

2 mg, 5 mg, 10 mg and 20 mg hard capsules, currently approved in selected EU countries.

Nitisinone is a competitive inhibitor of 4-hydroxyphenylpyruvate dioxygenase, an enzyme which precedes fumarylacetoacetate hydrolase in the tyrosine catabolic pathway. By inhibiting the normal catabolism of tyrosine in patients with hereditary tyrosinemia type 1 (HT-1), nitisinone prevents the accumulation of the toxic intermediates maleylacetoacetate and fumarylacetoacetate.

Nitisinone treatment leads to normalised porphyrin metabolism with normal erythrocyte porphobilinogen synthase activity and urine 5-aminolevulinate levels, decreased urinary excretion of succinylacetone, increased plasma tyrosine concentration and increased urinary excretion of phenolic acids.

Available data from a clinical study indicate that in more than 90% of the patients, urine succinylacetone was normalized during the first week of treatment.

THERAPEUTIC INDICATIONS