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sebelipase alfa

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

Alexion adds to pipeline rebuild with $855m Wilson buy

Alexion adds to pipeline rebuild with $855m Wilson buy

A $1bn purchase of Enobia in 2011 gave it an enzyme therapy for the ultra-orphan disease hypophosphatasia (HPP) - Strensiq (asfotase alfa) - which made $340m in sales last year but still ... However, its 2015 takeover of Synageva for $8.4bn has been less

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  • NICE broadens access to Alexion's rare disease drug Strensiq NICE broadens access to Alexion's rare disease drug Strensiq

    New draft guidance from NICE means that the wider population of infants with HPP - not just those with the most severe form - will be eligible for treatment with Strensiq (asfotase alfa), ... NICE also turned down Alexion's other new enzyme replacement

  • NICE blocks second Alexion rare disease drug in a week NICE blocks second Alexion rare disease drug in a week

    A final ruling from the UK's cost-effectiveness watchdog has concluded that the high cost of Kanuma (sebelipase alfa) - at almost £500, 000 per patient per year - cannot be justified ... Throughout this extremely complicated evaluation we have given the

  • AZ, BMS and Boehringer among UK Prix Galien finalists AZ, BMS and Boehringer among UK Prix Galien finalists

    Janssen. MCL, CLL, WM. sebelipase alfa. Kanuma. Alexio. Lysosomal acid lipase deficiency (LAL-D). ... asfotase alfa. Strensiq. Alexion. paediatric-onset hypophosphatasia (HPP) - bone manifestations. Real World Evidence Award 2016 finalists.

  • Rare disease therapies head the ‘class of 2015’ Rare disease therapies head the ‘class of 2015’

    These were Kanuma (sebelipase alfa), the first-ever treatment for the rare, inherited disorder lysosomal acid lipase deficiency - and Strensiq (asfotase alfa), the first enzyme replacement therapy for patients with infantile- ... EU. sebelipase alfa.

  • NICE wants more data on Alexion's orphan drug Kanuma NICE wants more data on Alexion's orphan drug Kanuma

    data on whether [sebelipase alfa] completely reversed LAL-D over the long term and prevented complications of the condition". ... Even based on more optimistic assumptions of long-term treatment effect, the cost of sebelipase alfa would be very high,"

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  • Pharma deals in May 2015 Pharma deals in May 2015

    This agreement brings access to Kanuma (sebelipase alfa) which is in registration for the treatment of lysosomal acid lipase (LAL) deficiency. ... Corporate acquisition. Rare diseases portfolio including Kanuma (sebelipase alpha) for LAL deficiency.

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