- PMLiVE

FDA approves drugs for rare genetic liver condition and to reverse anticoagulation

Raptor’s Procysbi and CSL Behring’s Kcentra recommended in US

- PMLiVE

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 - Basel

Roche and Isis in $392m deal to research drugs for brain disorder

Will develop antisense drugs for Huntington's disease

- PMLiVE

Otsuka files new TB candidate in Japan as shortages worsen

Delamanid shows promise in multi-drug resistant TB but supplies of rifampin are low

- PMLiVE

Takeda files lymphoma drug brentuximab vedotin in Japan

Seeks approval for antibody-drug conjugate for two forms of the blood cancer

- PMLiVE

Novartis provides multimedia support to Rare Diseases Day

Collaborates with Eurordis on multimedia education campaign

- PMLiVE

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 building

Novartis’ Jakavi turned down by NICE for blood cancer

UK cost-effectiveness watchdog concerned about 'uncertainties' in data in draft guidance

- PMLiVE

FDA says approval of generic Doxil will ease cancer drug shortage

Allows Sun Pharma to manufacture its own version of the medicine

Sanofi reception

US approval for Sanofi’s Kynamro in genetic cholesterol disorder

Comes one month after drug was turned down in Europe due to safety concerns

- PMLiVE

Santhera’s Raxone turned down for rare eye condition

EMA concerned about drug's ability to improve eyesight

- PMLiVE

Pfizer acquires spinal muscular atrophy research programme

Gains rights to drug candidate RG3039 in deal with Repligen that could be worth up to $70m

- PMLiVE

FDA clears six new drugs in time for the new year

Brings 2012 tally to 39 – the highest for more than 15 years

- PMLiVE

Year-end FDA approvals for Novartis, GSK drugs

Agency gives go-ahead to Cushing’s disease treatment Signifor and raxibacumab for inhalational anthrax