BioMarin aims to scoop up rare disease drugs as orphan field sizzles

Posted under BioMarin Pharmaceutical, Blog, Companies, Diagnostics, Funding, Genzyme, Medical Devices, Medical Supply, Pharmaceuticals, rare diseases, Startups, Universities, Videos by Ryan McBride

With the rare disease drug field as crowded as ever, BioMarin Pharmaceutical ($BMRN) is in the market to snap up smaller developers of the treatments to build on the company's established position in the market, CEO Jean-Jacques Bienaime told Bloomberg.

BioMarin has a track record of scooping up small rare disease players on the cheap. For example, it spent $22 million upfront for ZyStor in a deal that could be worth up to $115 million. Through that deal, BioMarin acquired ZyStor's Pompe disease drug (now called BMN-701). Bienaime told Bloomberg that the Pompe drug is due to enter Phase III in 2013, and it could compete with Genzyme's lucrative therapies for the rare ailment.

BioMarin, which already markets three drugs for rare diseases, has seen the number of players surge in recent years. Big Pharma has gotten in on the action, realizing the short time frames for developing orphan disease drugs, the high price tags of $200,000 or more for the therapies and other perks translate into a significant business opportunity. And the competition for gaining rights to assets in the rare disease field is fierce, with Shire, Pfizer ($PFE), Sanofi ($SNY), GlaxoSmithKline ($GSK) and others showing a willingness to bid for them.

Biotech companies are aiming to capitalize on the drugmakers' big appetites for rare disease drugs. For example, Alnylam ($ALNY) last week revealed that its RNA-interference drug for the rare disorder transthyretin-mediated amyloidosis is now one of its two lead programs. The company is attempting to translate its gene-silencing tech into commercially viable drugs.

While facing growing competition for licensing or buying rare disease drugs, BioMarin isn't relying exclusively on external sources for new products. The California biotech has 5 clinical programs under way, and its lead experimental candidate, called GALNS, is due to generate late-stage clinical date this year in patients with the genetic bone disease Morquio A syndrome, Bloomberg reports.

- get more in the Bloomberg article

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BioMarin perfects the popular rare disease drug model

Posted under BioMarin Pharmaceutical, Blog, Companies, Diagnostics, Funding, Medical Devices, Medical Supply, orphan drug status, Pharmaceuticals, rare diseases, Startups, Universities, Videos by John Carroll

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

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