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Batten disease

This page shows the latest Batten disease news and features for those working in and with pharma, biotech and healthcare.

NICE approves BioMarin’s Brineura after initial setback

NICE approves BioMarin’s Brineura after initial setback

First treatment for rare disease. BioMarin and NICE had a rocky start with Batten disease therapy Brineura, but the two have now come to an agreement. . ... The disease, known as neuronal ceroid lipfuscinosis type 2 (CLN2), is a very rare inherited

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  • BioMarin ticks over in Q2 ahead of gene therapy filing BioMarin ticks over in Q2 ahead of gene therapy filing

    A second-generation treatment for rare disease phenylketonuria (PKU), the newly launched drug reported $18.8m in sales. ... BioMarin will also hope to extend the commercial success of Brineura, its Batten disease therapy.

  • Amicus forms gene therapy manufacturing partnership with Catalent Amicus forms gene therapy manufacturing partnership with Catalent

    Amicus has one rare disease medicine already on the market, Galafold for Fabry disease, with an enzyme replacement therapy (AT-GAA) for Pompe disease in its late stage pipeline, to which ... capabilities. Thermo Fisher will supply Amicus with immediate

  • NICE knocks back BioMarin’s Batten disease drug NICE knocks back BioMarin’s Batten disease drug

    The committee initially agreed that Brineura shows substantial short-term benefits in slowing down the rate of Batten disease progression in draft guidance, but said BioMarin was unable to price the ... Results from a phase 1 and 2 clinical trial show

  • CHMP backs two rare disease therapies at April meeting CHMP backs two rare disease therapies at April meeting

    BioMarin got a green light for Brineura (cerliponase alfa), an enzyme replacement therapy for the ultra-rare neurodegenerative disease neuronal ceroid lipofuscinosis type 2 (CLN2), a form of Batten disease that ... There is no approved drug to treat the

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