Rare Diseases

Shire, Salix buy way to expansion

The companies are plunking down substantial sums to increase their reach.

Analyst says smart money is on drugs for certain orphan diseases


Price, market potential and emotional pull make this subcategory of orphan diseases one to watch, an analyst writes. Hint: it's not oncology.

Drug for ultrarare lipid disorder OK'd by FDA

Drug for ultrarare lipid disorder OK'd by FDA


Aegerion's Juxtapid is first to market for treating the ultrarare inherited cholesterol condition HoFH, or homozygous familial hypercholesterolemia.

Company news: smartphones, Janssen, FDA

Smartphones will influence 19% of in-store sales in four years, Janssen acquires an experimental heart treatment, and the FDA can now fast-track rare disease medications.

Company news: Corcept Therapeutics and Daiichi Sankyo

Corcept Therapeutics said its Cushing's syndrome drug will be launched three weeks earlier than expected.

Shire exits Fabry's market, citing costs as FDA weighs more research


Shire has pulled its drug, Replagal, from FDA review. The company was set to go before the agency next month, but preliminary talks indicated the FDA was going to require more testing.

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