Category Archives: Orphan Drugs

Lessons from Biotech: Tweaking the Imperfections

An increasing number of small biotech companies are being hatched to focus on a single disease or condition. The strategy goes against the diversified portfolio approach most big pharma companies have embraced, and instead puts all the eggs in one basket. Dart Therapeutics—one such company—has taken on Duchenne Muscular Dystrophy (DMD), a disease found in […]
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The Little Orphan That Could: How Alexion is Going Global

Since the initial FDA approval of its flagship orphan drug Soliris in 2007, Alexion Pharmaceuticals has done well for itself as a small biotech firm that focuses on ultra-rare disease. So well in fact, that instead of partnering, the company has taken global expansion into its own hands. Apart from a pipeline that includes asfotase […]
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The List So Far: FDA Breakthrough Designations

With announcements pouring in since the beginning of this year, FDA’s fourth and newest expedited approval pathway for medicines has begun to bear fruit. Established in 2012 within the FDA Safety and Innovation Act (FDASIA), Breakthrough Therapy designations have so far totaled 25 as of the end of July. With a separate senior management team […]
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Orphan Drug Laws: A Pharmacist's Perspective

Orphan Drug Laws:  A Pharmacists’ Perspective Guest blogger Dr. Albert Wertheimer Professor and Director, Health Services Research Temple University Philadelphia PA Pharm Exec’s sister organization CBI hosted its annual Orphan Drug Innovation Summit in Philadelphia on July 17-18.  This is a topic that I have professional interest in, as well as a general curiosity.  I […]
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Orphan Drug Adoption: the More Regulation, the Better

New survey research indicates that a country with a strong regulatory commitment for advancing drugs for rare diseases does better in getting orphan drugs to patients than countries with lesser regulatory frameworks. In his study, ‘The Effect of Market-Based Economic Factors on the Adoption of Orphan Drugs Across Multiple Countries,’ John Matthews, Associate Director of […]
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