Sanofi reception

Sanofi gains accelerated review for rare disease drug

New pill for Fabry disease could be approved by the FDA before the end of the year

- PMLiVE

AstraZeneca gains FDA orphan status for new cancer drug

Gains a longer patent life for the investigational lung disease drug

EISAI

Eisai’s thyroid cancer drug Lenvima nears EU approval

Positive recommendation by the CHMP could see the orphan drug used in Europe within three months

Shire Basingstoke

Shire links with US hospital for rare disease research

Willwork with Cincinnati Children's Hospital Medical Centre novel therapies

- PMLiVE

FDA grants orphan drug status to Treeway’s ALS treatment

Amyotrophic Lateral Sclerosis drug TW001 gainsregulatory benefits

- PMLiVE

FDA clears first therapy for rare childhood cancer

United Therapeutics’ Unituxin approved for neuroblastoma

EU flag

Jinarc backed for European approval in rare kidney disease

Positive opinion for Otsuka comes just before rare disease awareness day

EU flag

Europe gains access to first stem cell drug

Chiesi's Holoclar has been given the green light for a rare eye condition

- PMLiVE

Imbruvica gets FDA OK for rare lymphoma

Cancer drug available in US to treat Waldenström's macroglobulinaemia          

- PMLiVE

NICE backs first ‘ultra-orphan’ drug Soliris

Final guidance backs Alexion’s treatment for very rare blood clot condition

- PMLiVE

Assessing drugs for ultra-rare conditions in the UK

Alexion’s Soliris has become the first drug to pass through NICE’s new highly specialised technology process

- PMLiVE

FDA approves Ipsen drug to treat rare tumours

Green light for Somatuline Depot in cancer of the neuroendocrine system

- PMLiVE

Celgene wins orphan drug award at PMEA

Company is recognised for providing early access to Imnovid in multiple myeloma

- PMLiVE

Vertex submits Kalydeco combo for cystic fibrosis

Seeks approval in both US and Europe

- PMLiVE

Orphan drug market to reach $176bn by 2020

New report suggests market will almost double in five years