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- PMLiVE

Orfadin

Enabling access for patients with rare diseases

When it comes to meeting patient needs, the well-known counsel to ‘think globally, act locally’ rings true

EC consults on orphan drug application guidelines

Also seeks input on the transfer of designations from one sponsor to another

- PMLiVE

Analysts question value of rumoured $20bn Roche bid for Alexion

Shares in orphan drug specialist slide back after earlier surge

- PMLiVE

Court orders EC to approve rejected orphan medicine

Says CTRS should be allowed to market Orphacol for the treatment of rare bile condition

- PMLiVE

FDA grants orphan status to Activartis’ brain cancer immunotherapy

Regulator says AV0113 has potential in rare cancer malignant glioma

- PMLiVE

Celgene wins expanded EU approval for cancer drug Revlimid

Approved for use in patients with rare blood disorder

- PMLiVE

Celgene’s Revlimid wins US lymphoma approval

FDA recommends extra indication for blockbuster cancer drug

- PMLiVE

FDA gives priority review status to BMS/ AZ’s metreleptin

Former obesity prospect has potential in the rare disease lipodystrophy

- PMLiVE

Orphan status in US for Astellas/ Basilea antifungal

Isavuconazole is being developed as a treatment for invasive aspergillosis

- PMLiVE

FDA gives Alexion’s metabolic drug breakthrough status

Reduces requirements for registration, potentially speeding up approval

Healthcare at Home and QED partner on orphan diseases

OrphanReach alliance will see companies provide product lifecycle services

Novartis building

Novartis gets FDA green light for new Ilaris indication

Immunotherapeutic wins US approval for severe form of childhood arthritis

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