One of the big trends in drug development over the past three years has been the growing popularity of new treatments for rare diseases. And if you read through Bloomberg's lengthy feature on Novato, CA-based BioMarin's program for achondroplasia--a rare bone growth disorder that causes dwarfism--you'll see why. A drug program targeting a tiny number of patients can be wrapped relatively quickly; developers can achieve special orphan drug marketing status for the program and there's a steady stream of revenue to look forward to.
BioMarin ($BMRN) has been focused on enzyme replacement therapies, with several successful programs wrapping up after an average of four years. Its R&D group--which prepared the IND filed for this program, its seventh in the clinic, at the beginning of this month--believes they can match that track record for achondroplasia. And once on the market analysts at Baird analysts estimate that the company can reap $900 million a year by charging a rare disease-average $200,000 to $400,000 a year.
BioMarin's BMN-111 is just entering human trials. It's designed to correct the genetic defect through daily injections. And while the treatment is intended to correct the height of children after they are initially diagnosed, investigators believe that it can also help add to the height of more mature subjects.
"Our hope is that it will be profoundly effective," if not a complete cure, Hank Fuchs, chief medical officer, told Bloomberg.
- here's the article from Bloomberg
Special Report: BioMarin Pharmaceutical - Biotech's Biggest Spenders 2011