Nitisinone review

Nitisinone, which is also known by the trade name of Orfadin and the moniker of NTBC (an abbreviation of its complete chemical name), is a drug previously intended to be a herbicide but is presently utilized as medication for hereditary tyrosinemia type 1 (HT-1).

It has since toppled liver transplants as the top choice for first-line tyrosinemia treatment ever since the FDA approved of its use as a viable treatment for the rare disease way back in 1991. It's presently being tested to see if it could be helpful in the treatment of the related yet less critical condition of alkaptonuria.

Obviously, people who are suffering from hereditary tyrosinemia—a genetic metabolism disorder that usually affects children—should undergo nitisinone therapy, preferably alongside a dietary restriction regimen of phenylalanine and tyrosine amino acids. The rare disease itself is caused by enzyme deficiency, particularly the type that metabolizes tyrosine.

This drug should be taken orally every twelve hours without food and on an empty stomach (that is, at least an hour before meals) or as directed by your healthcare physician or pharmacist. The dosage is mostly based on the patient's weight, response to therapy, and medical condition, so it has no specific recommended daily dose aside from being taken twice daily.

If ingesting or swallowing nitisinone proves particularly difficult, the capsule may be opened and its contents mixed with a small amount of liquid (i.e., water or applesauce) immediately before administration. Don't bother making a supply of liquid-mixed nitisinone solution in advance; only mix a batch before use.

The most common side effects related to this medicine include increased thirst, diarrhea, and headache. If any of the above symptoms continue to occur or worsen, notify your doctor as quickly as possible. You should also tell him straightaway if any of the following critical yet less typical symptoms appear: yellowing skin or eyes, dark urine, and stomach pain.

The same procedure must be followed for these unlikely yet equally serious side effects: eye-related problems such as light sensitivity, pain, soreness, vision changes, burning, swelling, and redness. Finally, consult your doctor immediately if any of these rare yet fatal side effects occur: seizures, easy bleeding or bruising, persistent fever or sore throat, and rash. If you notice any other symptoms not found in the lists above, report it to your pharmacist or doctor without delay.

It's very important to tell your designated healthcare professional your complete medical history before taking this drug, especially if you ever experienced allergies or any other metabolism difficulties (for example, porphyria).

You must also control your diet when it comes to foods that have high amounts of phenylalanine and tyrosine (for example, high protein foods or those containing aspartame). Consult your doctor for further information on the correct low protein diet needed while undergoing nitisinone therapy.

You must also disclose to your doctor each and every last nonprescription and prescription drug you've recently used or are presently using. On that note, don't halt or begin any sort of therapy without approval from your pharmacist or healthcare specialist. You should avoid sharing this medicine with anybody else.

Medical and laboratory examinations for tyrosine levels, alpha-fetoprotein levels, blood counts, and your eyes may be done to check on your progress or any side effects. Furthermore, you should make sure to fulfill all such regularly scheduled laboratory and medical appointments.

Nitisinone has the following structural formula:

Chemical structure of nitisinone

• Molecular formula of nitisinone is C14H10F3NO5
• Chemical IUPAC Name is 2-[2-nitro-4-(trifluoromethyl)benzoyl]cyclohexane-1,3-dione
• Molecular weight is 329.228 g/mol
Nitisinone available : 2mg capsules, 5mg capsules and 10mg capsules

Brand name(s): Orfadin

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