Fresh from the designation pipeline: orphan drugs recently designated in the European Union (March – April 2013)

Abstract

Introduction: This paper continues the series devoted to the discussion of the orphan drugs newly designated in the EU for rare diseases for which no authorised medication exists on the market.

Areas covered: In this paper few designations for diseases such as Churg Strauss or Staargard's disease. Many of the orphan drugs discussed hereby are intended to reduce the inflammation which is the main pathogenic event driven by various genetic or acquired abnormalities.

Expert opinion: All orphan drug designations were granted based on the supportive data coming from preclinical studies. It is hoped that these preliminary results are going to be backed up by clinical results.

Keywords::

• Churg Strauss syndrome
• designation
• Duchenne's muscular dystrophy
• mepolizumab
• orphan drugs
• peptidase
• ramiprilat
• recombinant human tripeptidyl
• Staargard's disease