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- PMLiVE

Wakix

- 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

CHMP nod for Alexion, GSK drugs, revocation for Lartruvo

Opioid dependence therapy and Pfizer's PARP inhibitor also recommended

- PMLiVE

Orphan drugs market to soar to $224bn by 2024

Cell and gene therapies to fuel growth

- PMLiVE

Lilly to withdraw Lartruvo, a first for accelerated approval failures

Existing patients can use access programme

- PMLiVE

Sobi shows strong growth despite haemophilia competition

Looking to M&A for continued growth

- PMLiVE

AVEO warns EU approval of Fotivda could be at risk

Concerns arose with overall survival data

- PMLiVE

CHMP recommends new Keytruda dosing schedule

Will apply to eight indications across five tumour types

- PMLiVE

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

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

- PMLiVE

EMA could follow FDA’s Xeljanz blood clot warning

European agency in step with FDA after safety doubts

- PMLiVE

‘Inflexible’ NICE blocking access to rare disease drugs

Spinraza most high profile example of access problem

- PMLiVE

Big opportunities in big data, but EMA and industry just getting started

250m genomes sequenced, but data trapped in silos

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