Nitisinone

Hereditary tyrosinemia type 1 (HT-1) Nitisinone is indicated for the treatment of adult and paediatric (in any age range) patients with confirmed diagnosis of hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine.

Alkaptonuria (AKU) Nitisinone is indicated for the treatment of adult patients with alkaptonuria (AKU).

18 Page 3 of 21 MDK-Nitisinone Nitisinone capsules PART I: HEALTH PROFESSIONAL INFORMATION SUMMARY PRODUCT INFORMATION Route of Administration Dosage Form / Strength Clinically Relevant Nonmedicinal Ingredients Oral Capsules 2 mg, 5 mg, 10 mg, 20 mg For a complete listing see DOSAGE FORMS, COMPOSITION AND PACKAGING section.

INDICATIONS AND CLINICAL USE MDK-Nitisinone (nitisinone) is indicated for the treatment of patients with hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine. Treatment with MDK-Nitisinone should be initiated and supervised by a physician experienced in the treatment of HT-1.

Geriatrics (≥ 65 years of age):

Clinical studies of nitisinone did not include any subjects aged 65 and over.

Pediatrics (< 18 years of age):

Clinical trials of nitisinone were conducted in patients with HT-1 ranging in age from birth to 21 years of age [see CLINICAL TRIALS]. CONTRAINDICATIONS  Patients who are hypersensitive to this drug or to any ingredient in the formulation or component of the container.

1 INDICATIONS AND USAGE ORFADIN ® is indicated for the treatment of adult and pediatric patients with hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine. ORFADIN is a hydroxy-phenylpyruvate dioxygenase inhibitor indicated for the treatment of adult and pediatric patients with hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine.

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Hereditary tyrosinemia type 1 (HT-1) Orfadin is indicated for the treatment of adult and paediatric (in any age range) patients with confirmed diagnosis of hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine.

Alkaptonuria (AKU) Orfadin is indicated for the treatment of adult patients with alkaptonuria (AKU).