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- PMLiVE

siltuximab

- PMLiVE

Boehringer eyes rare disease use for respiratory blockbuster

Would help support respiratory franchise

- 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

Sobi shows strong growth despite haemophilia competition

Looking to M&A for continued growth

- PMLiVE

Janssen looks to expand Darzalex in Europe

Regimen reduced risk of disease progression or death by 44%

- PMLiVE

NICE partially clears Darzalex combo in blood cancer

Approved in second-line use under  CDF

- 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

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