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.
This article is accessible to registered users, to continue reading please register for free. A free trial will give you access to exclusive features, interviews, round-ups and commentary from the sharpest minds in the pharmaceutical and biotechnology space for a week. If you are already a registered user please login. If your trial has come to an end, you can subscribe here.
Login to your accountTry before you buy
7 day trial access
Become a subscriber
Or £77 per month
The Pharma Letter is an extremely useful and valuable Life Sciences service that brings together a daily update on performance people and products. It’s part of the key information for keeping me informed
Chairman, Sanofi Aventis UK
Copyright © The Pharma Letter 2024 | Headless Content Management with Blaze