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- PMLiVE

Wilson Therapeutics

- PMLiVE

Alexion launches ‘colourUp4RARE challenge’ to mark Rare Disease Day 2024

The campaign is aimed at raising awareness of how to improve quality of life for patients

- PMLiVE

AstraZeneca’s rare disease unit receives NICE recommendation for Wolman disease therapy in infants

The enzyme replacement therapy will become the first treatment available on the NHS for the rare genetic disease

- PMLiVE

AstraZeneca latest to sue US government over Medicare drug price negotiation plan

The programme already faces court challenges from drugmakers and industry groups

- PMLiVE

AstraZeneca’s rare disease unit to acquire Pfizer’s early-stage gene therapies for $1bn

The transaction could potentially see staff associated with the portfolio move to Alexion

- PMLiVE

AstraZeneca announces major expansion of Canadian research footprint

The investment includes the creation of 500 jobs and a new rare disease research hub

- PMLiVE

AstraZeneca progresses application for $360m Ireland manufacturing site

The investment is expected to create around 100 highly skilled jobs

- PMLiVE

Roche reports positive results for crovalimab as rare blood disease treatment

The drug was shown to be as effective as current treatment, with less frequent dosing

- PMLiVE

bluebird bio sells second FDA priority review voucher for $95m

The company sold its first voucher to Dutch biotech Argenx for $102m

- PMLiVE

FDA approves Ferring’s Adstiladrin as first gene therapy for bladder cancer

Non-muscle-invasive bladder cancer represents around 75% of all new bladder cancer cases

- PMLiVE

Roche’s COVID-19 antibody receives FDA approval for hospitalised adults

The decision makes Actemra the first FDA-approved monoclonal antibody to treat COVID-19

- PMLiVE

FDA lifts partial clinical hold on bluebird bio’s sickle cell gene therapy studies

The company can now resume the enrolment and treatment of patients aged two to 17 years

- PMLiVE

FDA approves updated COVID-19 boosters for children aged from six months

The Moderna and Pfizer/BioNTech boosters both target the BA.4/BA.5 omicron subvariants

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