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PMLiVE Top Pharma List

BioMarin

The Top Pharma List rankings are compiled from GlobalData's pharmaceutical revenue figures, which are based on prescription medicine sales, including generics drugs. They have been compiled below into an infographic of BioMarin's 2012 metabolic sales performance.

#OrkambiNow?

#OrkambiNow? But Orkambi is not the only orphan disease drug stuck in market access limbo in the England – Biogen’s Spinraza and BioMarin’s Kuvan are both currently suffering similar fates, and

Sangamo surges on data for haemophilia A gene therapy

Sangamo surges on data for haemophilia A gene therapy The new data would seem to put SB-525 firmly in contention with two other haemophilia A gene therapies that have been generating clinical data, namely valoctocogene roxaparvovec (valrox) from BioMarin ... therapies. BioMarin is currently thought to be

CHMP backs Lynparza in breast cancer, Dupixent in asthma

CHMP backs Lynparza in breast cancer, Dupixent in asthma Meanwhile, BioMarin got the nod for Palynziq (pegvaliase), a new enzyme replacement therapy for patients aged 16 and older with phenylketonuria, a rare but potentially serious inherited metabolic disease.

Roche buys gene therapy specialist Spark for $4.3bn

Roche buys gene therapy specialist Spark for $4.3bn It’s facing of competition in this area though, with BioMarin’s valoctocogene roxaparvovec also in phase 3 testing for haemophilia A and looking like it could be first to market

NICE knocks back BioMarin’s Batten disease drug

NICE knocks back BioMarin’s Batten disease drug England’s cost-effectiveness watchdog has today rejected BioMarin’s Battten disease therapy Brineura (cerliponase alfa) during final guidance. ... The decision, which was published under NICE’s Highly Specialised Technology (HST) committee, marks

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