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CAFICRESTAT
Generic Name: CAFICRESTAT
ABSTRACT -
Caficrestat (AT-007) is a potent, CNS-penetrant inhibitor of aldose reductase (AR), the enzyme responsible for conversion of glucose to sorbitol via the polyol pathway. It is under development by Applied Therapeutics for galactosaemia and other rare metabolic disorders where polyol pathway dysregulation leads to cellular damage. Phase I/II trials in classic galactosaemia demonstrate reduction in galactitol biomarkers.
DOSAGE -
Oral tablet or suspension; weight-adjusted dosing in paediatric and adult patients; once or twice daily per clinical protocol.
SIDE EFFECTS -
- Headache
- Nausea
- Fatigue
- Diarrhoea
- Dizziness
ALLERGIC REACTIONS -
These symptoms may indicate an allergic reaction and require immediate medical attention.
- Skin rash
- Urticaria
- Hypersensitivity
- Angioedema (rare)
DRUG INTERACTIONS-
- No significant CYP-based interactions identified to date
- Monitor with other drugs affecting polyol pathway metabolism
- Drug interaction data limited — full profile under investigation
PREAUTONS–
- Biomarker (galactitol) monitoring recommended to assess treatment response
- Not a substitute for dietary galactose restriction in galactosaemia
- Paediatric dosing requires weight-adjusted calculation
- Pregnancy safety not established
WARNINGS –
- Not curative — disease management only through biomarker reduction
- Long-term CNS effects of aldose reductase inhibition under study
- Use in clinical trial setting only until regulatory approval
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