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- PMLiVE

CTRS

Novartis building

NICE backs Novartis’ Jetrea in rare eye condition

Recommends drug be available for NHS use to treat vitreomacular traction

- PMLiVE

ReNeuron wins orphan status for stem cell therapy

ReN003 is being assessed as a treatment for rare eye disorder retinitis pigmentosa

- PMLiVE

Chiesi acquires Zymenex and mulls new rare disease unit

Deal adds late-stage orphan drug to Italian pharma company's pipeline

Shire extends rare genetic disease alliance

Agrees option with Santaris to nominate additional discovery targets

- PMLiVE

Novartis orphan drug wins ‘breakthrough’ status from FDA

Bimagrumab could be first treatment for rare life-threatening muscle-wasting condition

- PMLiVE

FDA panel turns down Otsuka’s drug for rare kidney disease

Unable to recommend expanded use of tolvaptan

EU flag

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

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