Nitisinone
- Generic Name
- Nitisinone
- Brand Names
- Nityr, Orfadin
- Drug Type
- Small Molecule
- Chemical Formula
- C14H10F3NO5
- CAS Number
- 104206-65-7
- Unique Ingredient Identifier
- K5BN214699
- Background
Nitisinone is a synthetic reversible inhibitor of 4-hydroxyphenylpyruvate dioxygenase. It is used in the treatment of hereditary tyrosinemia type 1. It is sold under the brand name Orfadin.
- Indication
Used as an adjunct to dietary restriction of tyrosine and phenylalanine in the treatment of hereditary tyrosinemia type 1.
- Associated Conditions
- Tyrosinemia Type 1
- Associated Therapies
- -
Small-Molecules Are More Cost Effective Than Biologics, Tufts Researchers Find
The Inflation Reduction Act (IRA) gives biologics a 4-year pricing advantage over small-molecule drugs, potentially leading to a shift in drugmaker priorities. However, a study by James D. Chambers and colleagues suggests that small-molecule drugs offer similar health benefits at lower costs, making them more cost-effective. The study analyzed 28 small-molecule drugs and 115 biologics, finding that small-molecule drugs had a median incremental cost of $4,738 compared to $16,020 for biologics, and a median incremental cost-effectiveness ratio (ICER) of $108,314 per QALY versus $228,286 for biologics.
Drug Development Updates
Stay informed with timely notifications on clinical trials, regulatory changes, and research advancements related to this medication.
© Copyright 2025. All Rights Reserved by MedPath