Mylan Orphan Drug Approved In USA

4 September 1994

Mylan has received approval from the US Food and Drug Administration to market its Cystagon (cysteamine bitartrate) for the 200 or so patients in the USA who suffer from nephropathic cystinosis. Cystagon was one of the first drugs to be designated under the Orphan Drug Act of 1983.

Nephropathic cystinosis is characterised by short stature, weak bones and progressive renal failure caused by the accumulation of the amino acid cystine in kidney tubules. Cystine is also deposited in the connective tissue and the cornea, where crystals are visible and serve as a diagnostic marker for the condition. Death occurs early as a consequence of the persistent uremia associated with the disorder.

Cysteamine bitartrate was developed with $900,000 in grants from the FDA's Office of Orphan Product Development, reports the Pink Sheet. Studies which included most of the nephropathic cystinosis patients in the USA, have showed that treatment with cysteamine bitartrate reduces the incidence of kidney failure. It does not affect stature.

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