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Renaissance in new drugs for rare diseases: Report in world's largest scientific socity magazine
Date:5/13/2013

with Alzheimer's disease both have high levels of a protein called tau.

  • Genzyme, Shire and BioMarin are examples of companies that work on rare diseases. Larger companies involved in the market are Pfizer and GlaxoSmithKline.

  • Some firms have even started funds specifically targeting rare diseases. Among the biggest moves was a partnership between Atlas Ventures and Shire to make early-stage investments in rare-disease opportunities. And just last month, New Enterprise Associates and Pfizer Venture Investments committed $16 million to Cydan, which will pluck rare disease projects from academia and start companies around the most promising ideas.

  • The Orphan Drug Act of 1983 fostered drug development for rare diseases by granting companies seven years of exclusivity, a waiver for FDA fees and tax credits. The incentives have worked the number of treatments for rare diseases has risen from 10 to over 400 since the ODA was created.

  • Last year, FDA regulations changed with the FDA Safety Innovation Act, making it easier to get these drugs through clinical trials an expensive and challenging hurdle.

  • Some patient advocates are simply raising awareness, but others have become more creative, funding researchers directly and founding companies themselves. For example, Jill Wood founded a company called Phoenix Nest to develop promising drug candidates for Sanfilippo syndrome if companies don't step up to support them.


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  • Contact: Michael Bernstein
    m_bernstein@acs.org
    202-872-6042
    American Chemical Society
    Source:Eurekalert

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