Carbaglu® (carglumic acid) is a synthetic (or manmade) form of N-acetylglutamate (NAG)—a chemical compound your body needs to start the process of removing excess ammonia (a waste product your body produces when it breaks down proteins). High levels of ammonia are toxic to the body. Carbaglu helps to relieve symptoms by lowering high ammonia levels in people with NAGS deficiency.
Carbaglu is the only FDA-approved therapy for the treatment of high ammonia levels due to NAGS deficiency in people of all ages.
Other ammonia lowering therapies such as other medications, hemodialysis and limiting protein in the diet are recommended during an acute episode


Cystagon® (cysteamine bitartrate) Capsules for oral administration, contain cysteamine bitartrate, a cystine depleting agent which lowers the cystine content of cells in patients with cystinosis, an inherited defect of lysosomal transport. Cystagon® is the bitartrate salt of cysteamine, an aminothiol, beta-mercaptoethylamine.


Cystadrops® is indicated for the treatment of corneal cystine crystal deposits in adults and children from 2 years of age with cystinosis


Cystadane® is a methylating agent indicated for the treatment of homocystinuria to decrease elevated homocysteine blood levels. Included within the category of homocystinuria are deficiencies or defects in:
• Cystathionine beta-synthase (CBS)
• 5,10-methylenatetrahydrofolate reductase (MTHFR)
• cobalamin cofactor metabolism (cbl)


Kuvan® is the first and only FDA-approved medication for PKU to reduce blood Phe levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin (BH4-) responsive PKU. Kuvan is a pharmaceutical formulation of BH4, the natural cofactor for the PAH enzyme, which stimulates activity of the residual PAH enzyme to metabolize Phe into tyrosine. Kuvan is to be used in conjunction with a Phe-restricted diet


Ammonaps® is indicated as adjunctive therapy in the chronic management of urea cycle disorders, involving deficiencies of carbamylphosphate synthetase, ornithine transcarbamylase or argininosuccinate synthetase.
It is indicated in all patients with neonatal-onset presentation (complete enzyme deficiencies, presenting within the first 28 days of life). It is also indicated in patients with late-onset disease (partial enzyme deficiencies, presenting after the first month of life) who have a history of hyperammonaemic encephalopathy.


Orfadin® (nitisinone) is the only FDA approved therapy, for use with dietary restriction of tyrosine and phenylalanine, for the treatment of tyrosinemia type 1


Normosang® injection contains human hemin, which is a substance derived from human blood. It is used to treat a group of conditions called acute hepatic porphyrias. These are rare inherited metabolic disorders that cause problems with the production of a protein called haem.


Wilzin® is used to treat Wilson’s disease. Wilson’s disease is a rare inherited disorder where patients lack an enzyme that is needed to eliminate the copper contained in food from the body. This results in copper building up in the body, first in the liver, then in other organs such as the eye and the brain. This causes a variety of effects, including liver disease and damage to the nervous system.


Pedea® is used to treat ‘patent ductus arteriosus’ in newborn premature babies who were born six or more weeks too early (less than 34 weeks gestational age). Patent ductus arteriosus is a condition where the ductus arteriosus (the blood vessel that allows blood to bypass the baby’s lungs before birth) fails to close after birth. This causes heart and lung problems in the baby.


Vedrop® is used to treat or prevent vitamin-E deficiency (low vitamin-E levels). It is used in children up to the age of 18 years who have congenital or hereditary chronic cholestasis and who cannot absorb vitamin E from the gut. Congenital or hereditary chronic cholestasis is an inherited disease causing problems with the flow of bile from the liver to the gut. Bile is a fluid produced in the liver that helps to absorb fats from the gut

Pharmaceutical/ Parapharmaceutical






Metabolic & Rare Diseases


Orthopedic & Rheumatology




BroadHealth/ Food Supplements