The companies are plunking down substantial sums to increase their reach.
Price, market potential and emotional pull make this subcategory of orphan diseases one to watch, an analyst writes. Hint: it's not oncology.
Aegerion's Juxtapid is first to market for treating the ultrarare inherited cholesterol condition HoFH, or homozygous familial hypercholesterolemia.
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.
Corcept Therapeutics said its Cushing's syndrome drug will be launched three weeks earlier than expected.
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.