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EC sets up expert group on rare diseases

Will revamp EUCERD to better handle development of orphan medicines

- PMLiVE

Boehringer: clinical trial set-up poses challenge in rare diseases

Says more support needed in regulatory process for orphan drugs

Actelion HQ Switzerland

Actelion to buy Ceptaris – if FDA backs its cancer treatment

Acquisition contingent on Valchlor approval in rare lymphoma

BTG extends orphan drug distribution partnership with Clinigen

Deal covers treatment for overexposure to chemotherapy

- PMLiVE

NICE demands more data from Roche for MabThera in rare autoimmune disease

Unable to recommend expanding drug’s indication to include vasculitis

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

Court orders EC to approve rejected orphan medicine

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

- PMLiVE

Revlimid faces NICE rejection for use in rare blood cancer

Watchdog's draft guidance does not recommend Celgene's drug for NHS use in England and Wales

- PMLiVE

Rare diseases among initial priorities of UK DNA mapping body

Genomics England will also help advance research into cancer and infectious diseases

- 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