- PMLiVE

Gilead’s seladelpar granted conditional EC approval to treat rare liver disease PBC

Primary biliary cholangitis affects approximately 163,000 people in Europe

Biogen Idec building

Biogen gains rights to Stoke’s Dravet syndrome candidate in deal worth $550m

The rare genetic epilepsy affects up to 38,000 people in the US, UK, EU and Japan

- PMLiVE

Novo Nordisk invests $1.2bn to establish rare disease production facility in Denmark

The site will be designed to accommodate multiple product types within rare diseases

regeneron headquarters

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

FDA approves BridgeBio Pharma’s Attruby to treat rare heart disease ATTR-CM

Transthyretin amyloid cardiomyopathy affects up to 500,000 people worldwide

- PMLiVE

Merck KGaA/Abbisko share positive phase 3 results for pimicotinib in rare tumour type

The companies entered into a licensing agreement for the candidate in December last year

- PMLiVE

NICE recommends Blueprint Medicines’ Ayvakyt to treat rare blood disorder

Systemic mastocytosis affects approximately one in every 10,000 people in the UK

- PMLiVE

AstraZeneca’s Fasenra approved by EC to treat rare inflammatory disease EGPA

Eosinophilic granulomatosis with polyangiitis affects approximately 118,000 people globally

- 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

Ipsen’s Iqirvo recommended by NICE to treat rare liver disease primary biliary cholangitis

The disease affects approximately 25,000 people in the UK, the majority of whom are women

- PMLiVE

AstraZeneca/Ionis’ Wainzua receives positive CHMP opinion to treat rare disease ATTRv-PN

The drug can be self-administered via an auto-injector and is designed to lower the production of TTR protein at its source

- PMLiVE

AstraZeneca receives MHRA approval for Wainzua in rare nerve disease ATTRv-PN

The therapy is designed to reduce the effects of the disease by lowering the production of TTR protein

- PMLiVE

Sanofi and Orano Med partner to advance radioligand therapies for rare cancers

The companies will invest in a new entity that will operate under the Orano Med brand

- PMLiVE

NHS England begins newborn screening programme to identify rare genetic conditions

The Generation Study is aiming to screen 100,000 newborns for more than 200 diseases

- 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