Nitisinone: Hereditary tyrosinemia type 1 (HT-1) medicine

 The medicine Orfadin contains Nitisinone, an FDA-approved drug to treat patients with hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine. Nitisinone is a competitive inhibitor drug of 4-hydroxyphenyl-pyruvate dioxygenase, an enzyme upstream of fumarylacetoacetate hydrolase (FAH) in the tyrosine catabolic pathway. 

Dosage and administration

The drug Nitisinone comes in the standard strengths of 2 mg, 5 mg, 10 mg, and 20 mg capsules to administer orally.

 Following are the administration and dosage instructions:

• The recommended initial dosage of Nitisinone is 0.5 mg/kg orally twice daily.
• One should titrate the dose for individual patients based on biochemical and clinical responses.
• One should adjust the dose of Nitisinone capsules individually.
• Healthcare professionals should monitor plasma and urine succinylacetone concentrations, liver function parameters, and alpha-fetoprotein levels.
• If succinylacetone is still detectable one month after the start of nitisinone treatment, increase the Nitisinone dosage to 0.75 mg/kg twice daily. 
• The patient may need a maximum dosage of 1 mg/kg orally twice daily based on evaluating all biochemical parameters.  
• If the biochemical response is satisfactory, the dosage should be adjusted only according to body weight gain.
• In addition to the tests above, during the initiation of therapy or if there is a deterioration in the patient’s condition, it may be necessary to follow all available biochemical parameters more closely. 

Side-effects of Nitisinone

The common side-effects of Nitisinone 20 mg, 10 mg, 5 mg, and 2 mg are:

•  Elevated tyrosine levels
• Thrombocytopenia (deficiency of platelets)
• Leukopenia (decrease in white blood cells)
• Conjunctivitis (inflammation in the eye)
• Corneal opacity 
• Keratitis (inflammation in the cornea)
• Photophobia 
• Eye pain
• Blepharitis (inflammation of eyelids)
• Cataracts
• Granulocytopenia (less no. of a type of white blood cells)
• Epistaxis (bleeding nose)
• Pruritus (skin allergy)
• Exfoliative dermatitis 
• Dry skin
• Maculopapular rash 
• Alopecia (baldness)

Price of Nitisinone

The price of Nitisinone in India is reasonably reasonable, and one can purchase this medicine from any certified generic medicine supplier. Certified pharmaceutical organizations like Indian Pharma Network help to connect the patients with certified suppliers, wholesalers, and dealers of Nitisinone in India and worldwide. 

FAQ

What are the ingredients in the medicine Orfadin capsules?

The active ingredient in the Orfadin capsules is Nitisinone, with the inactive ingredient pregelatinized starch. 

What are the brand names for the drug Nitisinone?

The innovator brand names for Nitisinone are Orfadin, Tisinone, and Nityr. 

How much does Orfadin cost?

The cost of Tisinone (or Orfadin) is sensibly reasonable, and one can purchase this medication from any licensed generic medicine supplier. For more information regarding the drug Nitisinone, contact us on 18008891064.