News First drug for rare disease APDS cleared for NHS use Pharming's Joenja has become the first disease-modifying drug that can be used by the NHS to treat APDS, an ultra-rare immunodeficiency disorder.
News Pharming brings first drug for rare disease APDS to US Joenja will launch in April and could make upwards of $200m a year in sales, say analysts.
News BioMarin ends year with $4.8bn play for Amicus BioMarin takes a leap towards its $4bn revenue target with a $4.8bn deal to acquire Amicus, which has two rare disease therapies on the market.
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