nitisinone (Orfadin)
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Indications
- adjunct to dietary restriction of tyrosine & phenylalanine in the treatment of hereditary tyrosinemia type 1
Dosage
- 1-2 mg/kg divided into two daily doses
Capsules: 2 mg, 5 mg, 10 mg
Suspension: 4mg/mL[2]
Monitor
- serum alpha-fetoprotein
- monitor liver with hepatic ultrasound, CT or MRI
Adverse effects
- thrombocytopenia
- leukopenia
- acute porphyria
- ocular/visual complaints, transient resolving within 1 week
- hepatic neoplasm
- liver failure
Laboratory
Notes
- blood levels of nitisinone in patients it toxic to Anopheles gambiae mosquitoes[3]
- death within 12 hours
More general terms
References
- ↑ RxList http://www.rxlist.com/orfadin-drug.htm
- ↑ 2.0 2.1 Windle ML FDA Drug Approvals and Changes: May edition. Medscape. May 20, 2016 http://reference.medscape.com/features/slideshow/drug-review/2016/may
- ↑ 3.0 3.1 Nield D Drug For Rare Disease Turns Human Blood Into Mosquito Poison Scienc Alert. March 27, 2025 https://www.sciencealert.com/drug-for-rare-disease-turns-human-blood-into-mosquito-poison
Haines LR, Trett A, Rose C et al Anopheles mosquito survival and pharmacokinetic modeling show the mosquitocidal activity of nitisinone. Sci Transl Med. 2025 Mar 26;17(791):eadr4827. PMID: https://pubmed.ncbi.nlm.nih.gov/40138457