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- PMLiVE

Spectrum Pharmaceuticals

- PMLiVE

Speedy approval for Pfizer’s Onpattro rival

Approved in two different formulations

Biomarin

EU nod for BioMarin’s second PKU therapy Palynziq

Palyniq is significant, but gene therapy pipeline most anticipated

- PMLiVE

Orphan drugs market to soar to $224bn by 2024

Cell and gene therapies to fuel growth

- PMLiVE

Sobi shows strong growth despite haemophilia competition

Looking to M&A for continued growth

- PMLiVE

Unruffled by ‘outlier’ claims, Vertex CEO upbeat on Orkambi breakthrough

NHS England going public with price makes deal impossible, says Leiden

- PMLiVE

‘Inflexible’ NICE blocking access to rare disease drugs

Spinraza most high profile example of access problem

- PMLiVE

Afinitor and Uptravi gain funding from NHS England

Rare disease treatments navigate tricky market access route

- PMLiVE

Chugai gains FDA breakthrough status for rare CNS treatment

Adds to company's BTD roll-call

- PMLiVE

Roche gains EU PRIME status for Spinraza rival

Oral treatment joins Novartis' gene therapy challenger

- PMLiVE

NICE says no to rival Alnylam and Akcea amyliodsis drugs – but deal could be done

Room for manoeuvre after initial rejection

- PMLiVE

Biogen picks up another drug from wide-ranging Ionis alliance

Aims to follow success of Spinraza

- PMLiVE

Orphan drugs from Biogen and Amicus win UK Prix Galien

New medical technology category added for the first time

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