Fresh from the designation pipeline: orphan drugs designated in the European Union (November – December 2014)

Abstract

Introduction: In this article, designations for acute respiratory distress syndrome, hereditary haemorrhagic telangiectasia, fibrodysplasia ossificans progressiva, xeroderma pigmentosum, warts, hypogammaglobulinemia, infections, myelokathexis syndrome, amyotrophic lateral sclerosis, multiple system atrophy, dermatomyositis, polymyositis, neurotrophic keratitis and epidermolysis bullosa are discussed.

Areas covered: Orphan drugs designated between November and December 2014 within the EU are discussed.

Expert opinion: Most of the drugs discussed have been repurposed, and in most of the cases this is based on the identification of therapeutic targets which are common for the original and the orphan therapeutic indications. The most relevant example is that of plerixafor.

Keywords:

• orphan drugs
• rare disease
• repurposing
• therapeutic targets

Declaration of interest

The author has no relevant affiliations or financial involvement with any organisation or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.