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- PMLiVE

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- PMLiVE

FDA approves BioMarin’s Roctavian as first gene therapy for severe haemophilia A

Approximately 2,500 patients are expected to be eligible to receive the one-time therapy

Biomarin

BioMarin’s application for haemophilia A gene therapy accepted by FDA

If approved, the treatment would be the first gene therapy in the US for this indication

- PMLiVE

EC approves BioMarin’s gene therapy Roctavian for severe haemophilia A

Approximately 20,000 adults are affected by haemophilia A across more than 70 countries in Europe, the Middle East and Africa

Biomarin

FDA keeps BioMarin’s gene therapy for phenylketonuria on hold

The decision follows data suggesting BioMarin’s experimental gene therapies may have the potential to progress cancers

Biomarin

FDA pauses BioMarin gene therapy trial

The study for BMN 307 in genetic disease phenylketonuria will be paused while malignancies in mice are explored

- PMLiVE

Presidential hopeful Bernie Sanders criticises BioMarin’s gene therapy price

Vowed to break patent if company goes ahead with proposed price

- PMLiVE

Sangamo steps up BioMarin chase with haemophilia gene therapy data

Updated results presented at ASH 2019

Biomarin

NICE approves BioMarin’s Brineura after initial setback

First treatment for rare disease

Biomarin

BioMarin ticks over in Q2 ahead of gene therapy filing

Haemophilia candidate valrox will be a game-changer for company

- PMLiVE

ACPs to lead gene therapies in haemophilia market growth

New therapies to hit existing players

Biomarin

BioMarin eyes haemophilia therapy filing, but data scares investors

Doubts remain on longer term efficacy

Biomarin

EU nod for BioMarin’s second PKU therapy Palynziq

Palyniq is significant, but gene therapy pipeline most anticipated

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