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- PMLiVE

Lumena Pharmaceuticals

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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

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

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Watchdog's draft guidance does not recommend Celgene's drug for NHS use in England and Wales

Novartis sponsors rare disease patient community

Backs site for patients affected by pancreatic neuroendocrine tumours (PNET)

- 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

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