All the Therapeutic System Drugs
4-Hydroxyphenylpyruvate Dioxygenase Inhibitor. Nitsinone 2, 5, 10 mg. CAPS: 60. Recomm. inititial dose in
ped., adult populat: 1 mg/kg body wt
dly in 2 divid. doses.
Tmt. pts with confirmed diagnos.
hereditary tyrosinemia type 1 (HT-1) in
combinat. with dietary restrict. of tyrosine
C/I: Hypersens., lact.
Monoclonal Antibody. Alirocumab 75 mg/ml, 150 mg/ml. PEN/SYR.: 1,2×75,150 mg/ml
The usual init. dose is 75 mg admin. SC once every 2 wks.
Pts. requir. larger LDL-C reduction (>60%) may be started on 150 mg admin.
SC once every 2 wks.
The dose can be individual. based on pt. characteristics such as baseline
LDL-C level, goal of ther. &response. See lit.
Primary Hypercholesterolaemia (heterozygous familial &non-familial) or mixed dyslipidaemia in adults, as an adjunct to diet:
- in comb .with a statin or statin with other lipid lowering therapies in patients unable to
reach LDL-C goals with the max. tolerated dose of a statin or,
- alone or in comb. with other lipid-low. therapies in pts. who are statin-intoler.
The effect of the drug on cardiovasc. morbid. and mortal. has not yet been determin.
Human Monoclonal Antibody. Evolocumab 140 mg/ml. PRE-FILL.SYR.: 1, 2, 3, 6. Prim. hypercholesterolem & mixed dyslipidem. in adult.: either 140 mg every two wks. or 420 mg once month.; both doses are clinic. equiv.
Homozyg. famil. hypercholesterolem. in adult. and adolesc. ≥ 12 yrs.: The init. recomm. dose is 420 mg once month. After 12 wks. of tmt., dose frequency can be up-titrated to 420 mg once every 2 wks. if a clinic. meaningful response is not achieved. Pts. on apheresis may initiate tmt. with 420 mg every two wks. to corresp. with their apheresis schedule.
Establish. atherosclerotic cardiovasc. dis. in adult.: 140 mg every two wks. or 420 mg once month.; both dosesare clinic. equiv.
Pts. with ren.impair.- pts with mild- moder. ren. impair.,- No dose adjust. is necessary in pts. with severe ren. impair. (eGFR<30 mL/min/1.73 m²).
Pts. with hep. impair.- No dose adjust. is necessary in pts. with mild hep. impair. See lit.
Hypercholesterolem. & and mixed dyslipidem., in adults with primary hypercholesterolem. (heterozygous famil. and non-famil.) or mixed dyslipidem., as an adjunct to diet: In comb. with a statin or statin with other lipid-lowering therap. in pts. unable to reach LDL-C goals with the max. tolerated dose of a statin or, alone or in comb. with other lipid-lower. therap. in pts. who are statin-intoler., or for whom a statin is contraindic.
Homozygous famil. hypercholesterolem.–adult. & adolesc. ≥12 yrs. and over with homozyg. famil. hypercholesterolem. in comb. with other lipid-lower. therap.
Establish. atherosclerotic cardiovasc. dis.-adults with established atherosclerotic cardiovasc. dis. (MI, stroke or peripher. arterial dis.) to reduce cardiovasc. risk by lower. LDL-C levels, as an adjunct to correct. of other risk factors: In comb. with the maxim. tolerated dose of a statin with/without other lipid-lower. therap.
Alone or in comb. with other lipid-lower. therap. in pts. who are statin-intoler., or for whom a statin is contraindic.
Alimentary Tract, Metabolism Enzymes. Asfotase Alfa 40 mg/ml, 100 mg/ml. VIAL: 1, 12×100mg, 1×40mg.
2 mg/kg of bdy. wt. admin. S.C. three times per week, or a dosage regimen of 1 mg/kg of bdy. wt. admin. S.C. six times per week. See lit.
Indicated for long-term enzyme replacement ther. in pts. with paediatric-onset hypophosphatasia to treat the bone manifestations of the dis.
C/I: Severe or life-threaten. hypersens. to the active subst. or to any of the excipients if hypersens. is not controllable.
Drug for musculo-skeletal system. Ataluren 125, 250, 1000 mg. SACHET: 30×125,250,1000 mg. Admin. oral. every day in 3 doses. The 1ST dose should be taken in the morn., the 2ND at midday, and the 3RD in the eve. Recommend. dosing intervals are 6 hrs. betwn. morn. and midday doses, 6 hours betwn. midday and eve. doses, and 12 hrs. betwn. the eve. dose and the first dose on the next day.
The recomm. dose is 10 mg/kg bdy. wt. in the morn. 10 mg/kg bdy. wt. at midday, and 20 mg/kg bdy. wt. in the eve. (for a total dly. dose of 40 mg/kg bdy. wt.). See Lit.
Tmt. of Duchenne muscul. dystrophy result. from a nonsense mutation in the dystrophin gene, in ambulatory pts. aged 2 yrs. & older. Efficacy has not been demonstrated in non-ambulat. pts.
The presence of a nonsense mutation in the dystrophin gene should be determined by genetic testing.
C/I: Hypersens. Concom. use of IV aminoglycosides.