Not long after acquiring Shire to add to its orphan-drug portfolio, the drugmaker scored an orphan designation from both FDA and EMA last week.
Innovation is still valued in the orphan-drug space, but the reimbursement bar is rising. NPS Pharma's Eric Pauwels, who's launched five ultra-rare disease products, explains how these biotech brands can demonstrate their value. Marc Iskowitz reports
Kynamro's slow start, coupled with competition, has prompted Sanofi to add sales reps.
Regulatory Focus discusses how the expanding US population may make it harder for a condition to qualify as rare.
The drugmaker announced Friday that the DOJ wants documents regarding sales and marketing of Juxtapid, its drug for an ultra-rare lipid disorder.
PR firm Burson-Marsteller launched a new healthcare subsidiary, Orexigen's net loss widened in Q2, and Corcept closed out its first full quarter with orphan treatment Korlym.