NICE recommends Biomarin’s Vimizim for patients with rare life-limiting metabolic disorder
Patients with a life-limiting skeletal condition welcome availability of elosulfase alfa on the NHS
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Tag: BioMarin
NICE ‘no’ for BioMarin’s Vimizin
The draft guidance has been published following an evaluation of new evidence on the use of the treatment
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BioMarin resubmits haemophilia A gene therapy to the EMA
Company initially withdrew a marketing authorisation application for the gene therapy last year
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EU review extended for BioMarin’s gene therapy Valrox
The EMA has asked for full data from an ongoing Phase III study of the treatment in patients with severe haemophilia A
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BioMarin submits EU marketing application for vosoritide
If approved, vosoritide would be the first medicine for the treatment of achondroplasia – the most common form of short stature – in Europe
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A&E attendances twice as high in deprived areas
Attendances for the 20% of the population living in the most deprived areas also account for 27% of all A&E attendances.
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BioMarin’s Brineura made available on NHS
The £500,000 per patient drug was deemed too costly for use in February 2018.
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EU approval for Palynziq in adult phenylketonuria
It’s the first substitution therapy approved in Europe to target the underlying cause of PKU by helping the body to break down Phe.
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Pfizer’s Talzenna cleared for breast cancer
US regulators have approved Pfizer’s PARP inhibitor Talzenna to treat patients with breast cancer carrying a certain mutation, identified by a companion diagnostic developed by Myriad.
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US clears first enzyme therapy for PKU
Biomarin’s Palynziq has been approved as the first enzyme therapy to treat the rare brain-threatening genetic disease phenylketonuria (PKU) in adults in the US.
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NICE deems Batten disease therapy too costly for NHS use
The National Institute for Health and Care Excellence has issued draft guidelines concluding that Biomarin’s Brineura is not a cost-effective treatment option for patients with the ultra rare disorder Batten disease.
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CROY – The Americas winners announced
PRA Health Sciences has been named Clinical Research Company of the Year at the PharmaTimes Clinical Researcher of the Year – The Americas competition.
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- Liverpool and McMaster University announce £300,000 strategic research partnership
- Study finds link between malaria parasites developing antimalarial drug resistance
- Researchers develop ‘digital twin’ heart models to better monitor PAH NHS patients
- Closed Loop and Pharmanovia investigate precision medicine combination in hypertension
- US study reveals interferon combinations cause lupus symptoms and affect treatment
