BioMarin's Reversal of Fortune

FDA advisory panels can be pretty dull. They feature a numbing onslaught of data, ages of power point slides, and hours of jargon-dense back-and forth-between the FDA and drug companies. That wasn't the case for a review this week of drisapersen, a BioMarin Pharmaceutical drug for a rare, fatal disease called Duchenne Muscular Dystrophy, which nearly always affects young boys. It included moving testimony from patients and their parents. At one point, people trying to watch online overloaded the FDA's livestream.

This was a more difficult vote than most for the FDA panel, particularly in front of people suffering from the disease. There's no approved therapy for the drug, and DMD is fatal, so families and patients accept a high level of risk for even a small benefit. There's been an intense lobbying effort to get approval.