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- PMLiVE

Lumena Pharmaceuticals

National Institute for Health and Care Excellence NICE logo

Amicus gets NICE green light for Fabry drug

Oral therapy alternative Galafold approved for routine NHS use

- PMLiVE

NICE blocks second Alexion rare disease drug in a week

Says high cost and “considerable uncertainties” over long-term benefits prohibit routine NHS use

ABPI London offices

UK pharma slams new drug evaluation plans

ABPI and BIA oppose "arbitrary" proposal to impose £100,000 QALY limit on rare disease therapies

ABPI London offices

UK pharma slams new drug evaluation plans

ABPI and BIA oppose "arbitrary" proposal to impose £100,000 QALY limit on rare disease therapies

- PMLiVE

A rare talent

Shire’s Sebastian Stachowiak on dealing with change and working for a fit-for-purpose orphan drug environment

Shire Basingstoke

Shire pays record $350m to settle US kickback allegations

Charges centred around diabetic foot ulcer treatment Dermagraft, which it sold in 2014

Biogen Idec building

FDA approves Biogen’s Spinraza for spinal muscular atrophy

Analysts predict blockbuster sales as drug is priced at $750,000 for first year of treatment

- PMLiVE

Intercept gets EU nod for rare liver disease therapy

Ocaliva approved as a combination treatment for primary biliary cholangitis

- PMLiVE

Shire to focus rare disease R&D with new US facility

Cambridge, Massachusetts hub to drive firm’s push to become leading rare disease specialist

- PMLiVE

Orphan drugs: Leading the way in patient-centricity

What lessons about optimal patient engagement can big pharma learn from those operating in rare disease?

National Institute for Health and Care Excellence NICE logo

NICE backs Amicus Therapeutics’ Galafold

Recommends oral alternative for treating Fabry disease

- PMLiVE

Access to orphan drugs for rare cancers in EU28

Although the economic burden imposed by rare cancers has not yet been adequately assessed, the treatment burden posed by rare cancers on individuals, societies and healthcare systems make them a...

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