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- PMLiVE

rifampin

- PMLiVE

Ten new medicines recommended in Europe

Includes double approvals for rare disease specialist Alexion and Novartis

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

- 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

- PMLiVE

US approval for Astellas’ new antifungal drug Cresemba

Japanese firm co-markets the azole antifungal agent with Basilea

FDA launches drug shortages mobile app

USregulator offers the public and HCPs Android and iPhone versions of the app

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

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