orphan drug
FDA awards orphan status to new brain cancer vaccine
August 9, 2017Research 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, 2016Research 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, 2016Research 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, …
Horizon receives European go-ahead for Ravicti
December 2, 2015Research 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, 2011Sales 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, 2011Research 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, 2010Research 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 …
