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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.

HTA guidance from NICE, the SMC and the AWMSG

Here we present a feed of upcoming health technology assessment (HTA) decisions, covering appraisals by the National Institute of Health and Care Excellence (NICE), the Scottish Medicines Consortium (SMC) and the All Wales Medicines Strategy Group

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

BioMarin prepares for haemophilia A gene therapy data readout, filing

BioMarin prepares for haemophilia A gene therapy data readout, filing The company has rivals close behind it – Spark and Pfizer and Sangamo, but BioMarin could gain the upper hand this year if its data is robust. ... to $1bn.  The product was launched in the US last year and BioMarin expect a recommendation from

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