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Achillion Pharma

- PMLiVE

Novartis shares positive late-stage results for Fabhalta in rare blood disorder PNH

Paroxysmal nocturnal haemoglobinuria affects approximately ten to 20 people per million worldwide

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Regeneron shares positive late-stage results for poze-cemdi in rare blood disorder PNH

Paroxysmal nocturnal haemoglobinuria affects up to 1.5 people per million in the US

- PMLiVE

AstraZeneca’s Voydeya recommended by NICE to treat rare blood disorder PNH

The drug has been recommended for use alongside the company’s C5 inhibitors

- PMLiVE

Roche’s injectable C5 inhibitor PiaSky recommended by NICE for rare blood disorder PNH

Paroxysmal nocturnal haemoglobinuria affects approximately 1.59 per 100,000 people in the UK

- 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

Alexion’s Ultomiris shows promise in neuromyelitis optica spectrum disorder phase 3 trial

Ultomiris-treated patients showed zero relapses with a median treatment duration of 73 weeks

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