Biotech Values

[DewDiligence]

BMRN/SRPT are the cover story of today’s Barron’s:

http://www.barrons.com/articles/biotechs-biggest-battle-1448083775?alg=y

The biggest battle in biotechnology is playing out as two companies, Sarepta Therapeutics and BioMarin Pharmaceutical, seek regulatory approval for drugs that could slow the progression of Duchenne muscular dystrophy, a fatal, inherited muscle-wasting disease that afflicts about 15,000 to 20,000 boys in the U.S., a similar number in Europe, and tens of thousands more in the rest of the world.

The stakes are high since there is now no effective treatment for the disease, which affects one in 3,500 boys, putting them in wheelchairs by their teens or earlier and leading to death, usually between ages 20 and 30, mostly from cardiac and lung problems.

The drama over…DMD involves innovative science, an impassioned patient-advocacy community, skeptical regulators at the U.S. Food and Drug Administration, dueling companies, and a huge market opportunity for investors. Not since the AIDS crisis of 1980s and early 1990s has there been so much patient pressure on the FDA to approve a drug.

For those who have followed the stories of the DMD drugs, the Barron’s article won’t tell you anything you didn’t know; however, the Barron’s piece is a pretty good ‘ReadMeFirst’ primer for those who have not.