The most expensive drug on the market is called Glybera and is used to treat familial lipoprotein lipase deficiency, a hereditary disease found in only about 7,000 people worldwide
That's the issue with rare diseases/orphan drugs, the whole pharmaceutical platform as it is today is mostly unfit to treat them. It takes hundreds of millions to billions of dollars to develop a single new drug and then millions more to successfully file an NDA and begin manufacturing of the actual dosable drug. That's why the epigenetic platform has so much more potential in these areas, simple inhibitor molecules can turn on/off gene expression without nearly the side effects you see with traditional pharmaceuticals. Once the platform is more developed rare disease treatment will undergo a renaissance.
cool read
>The most expensive drug on the market is called Glybera and is used to treat familial lipoprotein lipase deficiency, a hereditary disease found in only about 7,000 people worldwide
That's the issue with rare diseases/orphan drugs, the whole pharmaceutical platform as it is today is mostly unfit to treat them. It takes hundreds of millions to billions of dollars to develop a single new drug and then millions more to successfully file an NDA and begin manufacturing of the actual dosable drug. That's why the epigenetic platform has so much more potential in these areas, simple inhibitor molecules can turn on/off gene expression without nearly the side effects you see with traditional pharmaceuticals. Once the platform is more developed rare disease treatment will undergo a renaissance.
cool read
That's the issue with rare diseases/orphan drugs, the whole pharmaceutical platform as it is today is mostly unfit to treat them. It takes hundreds of millions to billions of dollars to develop a single new drug and then millions more to successfully file an NDA and begin manufacturing of the actual dosable drug. That's why the epigenetic platform has so much more potential in these areas, simple inhibitor molecules can turn on/off gene expression without nearly the side effects you see with traditional pharmaceuticals. Once the platform is more developed rare disease treatment will undergo a renaissance.