Cysteamine Bitartrate is its name, and savin’ lives is its game.
It’s not often that you hear a drug referred to as “life-saving” (probably due to legal ramifications), but that’s just what researchers from Europe and India, The Quint reports, are claiming with the development of the cystinosis medication, Cysteamine Bitartrate.
So what is cystinosis anyway?
Cystinosis is a disease that slowly destroys vital organs, such as the kidneys, liver, eyes, muscles, and brain. And because there is such a small population of people who have the disease (only 11 children are known to have it in India), funding and research have remained limited.
But luckily, this new medication is in the final stages of development. One of the nephrologists working on the drug said its purpose is to remove amino acid cysteine that’s known to accumulate in cells.
However, because it’s not yet permitted in India, parents of cystinosis children import the drug once every three months from a pharmaceutical headquarters in Paris. And because it’s not approved, it’s very expensive for parents to acquire.
But, if the drug is approved in India, like many parents are hoping, it would lift a huge burden because it would drop the staggering costs quite a bit. In a typical case, it would be a long road to approval, especially because India isn’t considered an appropriate market with only 11 known patients.
But because it’s a “life-saving” drug, the high court of India has asked the controller to approve the drug without a clinical trial.
Though the fate of the drug is up in the air, all fingers are crossed that it reaches patients in India, sooner rather than later.