FDA gives Alexion’s metabolic drug breakthrough status
Reduces requirements for registration, potentially speeding up approval
Healthcare at Home and QED partner on orphan diseases
OrphanReach alliance will see companies provide product lifecycle services
Novartis gets FDA green light for new Ilaris indication
Immunotherapeutic wins US approval for severe form of childhood arthritis
FDA approves drugs for rare genetic liver condition and to reverse anticoagulation
Raptor’s Procysbi and CSL Behring’s Kcentra recommended in US
European drug applications ‘stable but more complex in 2012’
Despite austerity measures, EMA's annual report shows signs of biopharma resilience
Rare disease patients face 5-7 year wait for diagnosis
Doctors often lack time and resources to manage patients
Roche and Isis in $392m deal to research drugs for brain disorder
Will develop antisense drugs for Huntington's disease
Otsuka files new TB candidate in Japan as shortages worsen
Delamanid shows promise in multi-drug resistant TB but supplies of rifampin are low
Takeda files lymphoma drug brentuximab vedotin in Japan
Seeks approval for antibody-drug conjugate for two forms of the blood cancer
Novartis provides multimedia support to Rare Diseases Day
Collaborates with Eurordis on multimedia education campaign
Shining a light on orphan medicines
Orphan diseases represent one of the most exciting and emotive areas in modern medicine and need a communications strategy that covers a spectrum of issues
Novartis’ Jakavi turned down by NICE for blood cancer
UK cost-effectiveness watchdog concerned about 'uncertainties' in data in draft guidance
FDA says approval of generic Doxil will ease cancer drug shortage
Allows Sun Pharma to manufacture its own version of the medicine
US approval for Sanofi’s Kynamro in genetic cholesterol disorder
Comes one month after drug was turned down in Europe due to safety concerns
Santhera’s Raxone turned down for rare eye condition
EMA concerned about drug's ability to improve eyesight