Category Archives: Orphan Drugs

Swedish Firm Honored for Rare Disease Treatments

Swedish Orphan Biovitrum AB (Sobi) was last night presented with a EURORDIS Award for its commercial and development portfolio in the field of rare diseases. The award also recognized the company’s track record on access to drugs and its collaboration with patient organizations. EURORDIS is Europe’s largest patient organization in rare diseases. Its CEO, Yann […]
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Europe: Expectations High for Rare Disease Day

Next week Europe will celebrate Rare Disease Day. This is an interesting example – one might almost say a rare example – of a successful attempt by a European interest group to capture public attention. It is now an annual awareness-raising event co-ordinated by the European rare diseases lobby group, EURORDIS. For this year’s celebration, […]
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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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