Products
Mercaptamine was approved in many countries in 2019 in the form of hard capsules (Procysbi). The active ingredient is also known as cysteamine or cysteamines. In 2020, eye drops were approved for the treatment of cystine deposits in the cornea (Cystadrops). This article refers to the capsules.
Structure and properties
Mercaptamine (C2H7NS, Mr = 77.15 g/mol) is an aminothiol present in in the drug as mercaptamine tartrate or mercaptamine hydrochloride. Mercaptamine is a degradation product of the sulfur-containing amino acid cysteine.
Effects
Mercaptamine (ATC A16AA04) reduces the accumulation of cystine in certain cells (eg, leukocytes, muscle, and liver cells) and delays the development of renal failure. It is transported into lysosomes and leads to the degradation of cystine to cysteine and cysteine-mercaptamine disulfide, which are removed from lysosomes. The half-life is approximately 4 hours.
Indications
For the treatment of established nephropathic cystinosis. This is a lysosomal storage disease caused by a mutation in a cystine transporter. This leads to an accumulation of cystine in the lysosomes.
Dosage
According to the SmPC. Capsules are usually taken in the morning and evening (every 12 hours). The exact information on taking with food in the professional and patient information should be followed!
Contraindications
- Hypersensitivity
- Hypersensitivity to penicillamine
- Lactation
Full details of precautions and interactions can be found in the drug label.
Adverse effects
The most common possible adverse effects include lack of appetite, vomiting, nausea, diarrhea, weakness, and fever.
