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- PMLiVE

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Europe gains access to first stem cell drug

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

- PMLiVE

NICE backs first ‘ultra-orphan’ drug Soliris

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

- PMLiVE

Boehringer drug approved in EU for rare lung disease

Nintedanib is already available as cancer treatment

Shire Basingstoke

Shire signs deal to buy NPS for $5.2bn

Firm hopes to boost its presence in rare disease sector

- 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

‘Major step forward’ as EMA recommends first stem cell therapy

But wants more clinical trial data to shore up orphan drug's marketing licence

- 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

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