Category Archives: Orphan Drugs

J.P. Morgan: Pharma's Agenda for 2014

Presenters at the 32nd annual J.P. Morgan Healthcare Conference talked up value-based pricing, emerging market strategies, complex generics and new technology, from bedside devices and genetic sequencers to first-in-class mechanisms of action. Company executives honed in on their chosen therapeutic areas and made the case for aggressive spending in R&D to bring the next generation […]
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Ibrutinib's Breakthrough to Market

FDA’s new Breakthrough Therapies designation sped Pharmacyclics/J&J’s ibrutinib (brand name: Imbruvica) through regulatory review in four months, based on Phase 2 studies. Pricing and access issues, though, may not get resolved so quickly.
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PharmExec's Orphan Drug Pipeline Picks

Orphan drugs were given short shrift in PharmExec’s 2014 Pipeline Report, but a couple of pipeline candidates targeting small populations did make the list, and are expected to earn big dollars in the next few years. In the US, private and government payers haven’t yet had the nerve to balk at rare disease drug prices. […]
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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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