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- PMLiVE

Wakix

- PMLiVE

Rivals rise as safety issue mars Pfizer’s DMD gene therapy trial

Company to press ahead with phase 3 trial

- PMLiVE

CHMP blocks UCB/Amgen’s Evenity approval for osteoporosis

Another obstacle for drug

- PMLiVE

Alexion’s Soliris adds new approval in rare disease NMOSD

First ever drug approved for rare autoimmune disease

- PMLiVE

Acer Therapeutics hit by FDA filing rejection

Shares plunge as regulator calls for new trial

- PMLiVE

NHS England wants money-back guarantee on Orkambi

Vertex resisting real-world data proposal

- PMLiVE

Boehringer eyes rare disease use for respiratory blockbuster

Would help support respiratory franchise

- PMLiVE

Brexit “having impact on EU drug development”

EMA and UK regulator both affected

- 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

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