Metabolic Diseases

8 Drugs classified under this therapeutic system

Enzyme Cofactor. Sapropterin (dihydrochloride) 100 mg.
SOL. TABS: 30, 120 x 100 mg. 5-20 mg/
kg/dly. See lit.
Tmt. HPA in adults, child over 4 yrs with
PKU, shown respons. to such tmt. Tmt.
HPA in adults, child over 4 yrs with BH4
defic., shown respons. to such tmt.
C/I: Hypersens.

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
and phenylalaline.
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.
C/I: Hypersens.

Human Monoclonal Antibody. Evolocumab 140 mg/ml.
PRE-FILL.SYR.or PEN:1, 2, 3, 6 X 1ml.
Prim. hypercholesterolem. & mixed dyslipidem. in adlts. and ped. pts. aged 10 and over: either 140 mg every two wks. or 420 mg once month. ; both doses
are clinic. equiv.
Homozyg. famil. hypercholesterolem. in adult. and ped. pts. ≥ 10 yrs.:
init. recomm. dose is 420 mg once month. After 12 wks. of tmt., dose freq. can be up-titrat. to 420 mg once every 2 wks. if clinic. meaningful resp. is not achieved. Pts. on apheresis may init. tmt. with 420 mg every two wks. to corresp. with their apheresis sched.
Establish. atheroscler. cardiovasc. dis. in adult.: 140 mg every two wks. or 420 mg once month.; both doses are clinic. equiv. See lit.
Hypercholesterolem. & and mixed dyslipidem. in adults with prim. hypercholesterolem. (heterozyg. famil. and non-famil.) or mixed dyslipidem. and in ped. pts. aged 10 and over with heterozyg. fam. hyperchol. as an adj. to diet:
in comb. with a statin or statin with other lipid-low. therap. in pts. unable to reach LDL-C goals with the max. toler. 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.
Homozyg.  famil. hypercholesterolem. –adult. & ped. pts. aged 10 and over- in comb. with other lipid-lower. therap.
Establish. atheroscl. cardiovasc. dis.-adults with MI, stroke or peripher. arterial dis.- to red. cardiovasc. risk by lower. LDL-C levels, as an adj. to correct. of other risk factors:
in comb.with the maxim. toler. dose of a statin with / w/o 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.
C/I: Hypersens.

Enzyme Cofactor. Sapropterin 100 mg.
Sol. Tabs. 30,120 X 100 mg
PKU (Phenylketonuria)

 

 starting dose in adult and paed. pts. is 10 mg/kg BW once daily. Dose to be adjusted, between 5 and 20 mg/kg/d, to maintain adequate blood phenylalanine levels.
BH4 deficiency
starting dose in adult and paed. pts. is 2 to5 mg/kg BW total daily dose. Doses may be adjusted up to 20 mg/kg/d.
Tmt.of hyperphenylalaninaemia (HPA) in adults and paed. pts. of all ages with phenylketonuria (PKU) who have been shown to be responsive to such tmt.
Tmt.of hyperphenylalaninaemia (HPA) in adults and paed.pts. of all ages with tetrahydrobiopterin (BH4) deficiency who have been shown to be responsive to such tmt.
CI: Hypersens.

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.