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- 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

Cancer drugs lead SMC recommendations

New treatments from Roche, Lilly and Celgene backed for use in Scotland

- PMLiVE

SMC fails to recommend Roche’s Perjeta

Says drug is not a cost-effective option in breast cancer

- PMLiVE

Extra stomach cancer indication for Cyramza in US

FDA backs Lilly drug in combination with paclitaxel chemotherapy

- 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

- PMLiVE

FDA approves Baxter drug for rare form of haemophilia

Obizur is derived from pig proteins

- PMLiVE

Orphan drugs lead CHMP recommendations

Clinuvel's Scenesse is first drug set for European approval to treat rare intolerance to sunlight

- PMLiVE

EMA offers orphan drug benefits for Ebola research

Incentives to encourage development of new treatments for the virus

- PMLiVE

FDA awards $19m in rare disease grants

US regulator supports 15 orphan drug projects

- PMLiVE

NICE backs Celgene’s Revlimid in bone marrow disorder

Recommends that drug can be used on NHS to treat myelodysplastic syndromes

Genentech’s Sunil Agarwal joins rare disease firm

He will serve as chief medical officer at Ultragenyx

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