orphan drug

Showing 7 posts of 7 posts found.


FDA awards orphan status to new brain cancer vaccine

August 9, 2017
Research and Development, Sales and Marketing FDA, life sciences, orphan drug, pharma, pharmaceuticals

The FDA has announced that it has awarded orphan drug status to the vaccine SurVaxM for patients with glioblastoma, the …


GW Pharmaceuticals says Phase III trials for Epidiolex to treat rare epilepsy in children shows positive results

June 27, 2016
Research and Development, Sales and Marketing Epidiolex, GW Pharmaceuticals, NDA, US FDA, drug trial, orphan drug

GW Pharmaceuticals (Nasdaq: GWPH) said late-stage trials for its Epidiolex (cannabidiol or CBD) to treat Lennox-Gastaut syndrome (LGS), a rare …


AZ drug gets orphan status for rare inflammatory disease

March 9, 2016
Research and Development AstraZeneca, MEDI-551, MedImmune, orphan drug

AstraZeneca’s biologics R&D arm MedImmune has been granted Orphan Drug Designation by the FDA for its investigational anti-CD19 monoclonal antibody, …

EU flags

Horizon receives European go-ahead for Ravicti

December 2, 2015
Research and Development, Sales and Marketing Horizon Pharma, Ravicti, horizon, orphan drug, rare disease, urea cycle disorders

The European Commission has approved Ravicti for rare urea cycle disorders.  Ravicti, (glycerol phenylbutyrate), is an oral liquid developed by Horizon …

First idiopathic pulmonary fibrosis drug approved

March 4, 2011
Sales and Marketing Esbriet, IPF, InterMune, Orphan Drugs, idiopathic pulmonary fibrosis, orphan drug

The first ever treatment for the potentially fatal lung disease idiopathic pulmonary fibrosis (IPF) has been approved in the EU. …

Bayer’s regorafenib gains orphan status for GIST

February 4, 2011
Research and Development, Sales and Marketing Bayer, FDA, GIST, gastrointestinal stromal tumours, orphan drug, regorafenib

Bayer’s investigational compound regorafenib has been granted orphan drug status for the treatment of patients with gastrointestinal stromal tumours (GIST) …

Novartis blood cancer drug impresses in late stage trial

December 22, 2010
Research and Development, Sales and Marketing INC424, Novartis, blood cancer, myelofibrosis, orphan drug

Novartis has released positive phase III results of its myelofibrosis drug INC424 showing it met its primary endpoint of reducing …

Latest content