Category Archives: Orphan Drugs
By Pharm Exec | Published: March 21, 2014
The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorization for Sylvant (siltuximab) for adult patients with multicentric Castleman’s disease.
By Pharm Exec | Published: February 26, 2014
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 […]
By Guest Blogger | Published: February 24, 2014
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, […]
By Ben Comer | Published: January 17, 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 […]
By Ben Comer | Published: November 25, 2013
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.