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. 2020 Jan 1;12(1):e2020010. doi: 10.4084/MJHID.2020.010

Table 2.

Approved Drugs.

Compound Company Structure Mechanism of Action Formulations Indication
Hydroxyurea Numerous Hydroxycarbamide CH4N2O2
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An antineoplastic agent that inhibits DNA synthesis through the inhibition of ribonucleotide diphosphate reductase. Hb F Induction, reduces inflammation and hemolysis Capsules: Hydrea and Droxia; 100, 200, 300, 400, 500 mg
Tablets: Siklos;100 mg, 1000 mg
Solution 100 mg/ml or higher as needed
Sickle cell anemia; Myeloproliferative disorders, certain cancers
L-Glutamine (Endari) Emmaus Medical Inc. 2-Amino-4-carbamoylbutanoic acid
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Not well known. It may improve the NAD redox potential in sickle RBCs through increasing the availability of reduced glutathione. Powder in packets containing 5g each Reduction of the acute complications of sickle cell disease in adult and pediatric patients 5 years of age and older
Crizanlizumab-tmca Novartis Monoclonal antibody Anti-P-selectin Intravenous solution Reduces the frequency of VOCs in adults and pediatric patients aged 16 years and older
Voxelotor (Oxbryta, GBT440) Global Blood Therapeutics Inc Benzaldehyde, 2-hydroxy-6-((2-(1-(1-methylethyl)-1H-pyrazol-5-yl)-3-pyridinyl)methoxy)
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Hb S Polymerization inhibitor Tablets (500 mg) for oral use Treatment of sickle cell disease in adults and pediatric patients 12 years of age and older

NAD = nicotinamide adenine dinucleotid.