Concerted effort by governments, regulators, and industry is rapidly expanding development of new therapies for the more than 7,000 known rare diseases.

A new drug to treat a rare inherited bone disorder from Alexion Pharmaceuticals ALXN.O that costs more than $550,000 a year for each patient has been deemed too expensive by Britain’s health cost watchdog NICE. The case highlights the sky-high price of some medicines for treating rare diseases and the dilemma this poses for cash-strapped […]

Oct 23 (Reuters) – The U.S. Food and Drug Administration on Friday approved Alexion Pharmaceuticals Inc’s Strensiq, a treatment for a rare kind of metabolic bone disorder. The approval is the first in the United States for a treatment for perinatal, infantile and juvenile-onset hypophosphatasia (HPP). (1.usa.gov/1W9KuRH) HPP is a genetic and potentially fatal disorder […]