Pharmafile Logo
T40 2022 Cover
Diversity, Equity and Inclusion
Communiqué 53 Cover Image
- PMLiVE

Orphan Drugs

- 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

- PMLiVE

UK market access: The importance of a local value story

Meeting the needs of the health service’s new stakeholders

- PMLiVE

Novartis orphan drug wins ‘breakthrough’ status from FDA

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

- PMLiVE

Outcomes shape the new European payer networks

In the new commercial reality of cost-constrained healthcare environments the payer is everywhere

- PMLiVE

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

Unable to recommend expanded use of tolvaptan

Roche's Perjeta pertuzumab

Roche’s breast cancer drug too expensive for NHS use

But pharma company says UK Government dithering on Perjeta patient access scheme

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

- PMLiVE

Building and delivering market access capabilities in emerging markets

Local skills gaps must be addressed if the potential of the BRIC countries is to be realised

Subscribe to our email news alerts

Latest jobs from #PharmaRole

Latest content

Latest intelligence

Quick links