Fresh from the designation pipeline: orphan drugs recently designated in the EU (June–July 2014)

Abstract

Introduction: The increasing awareness of rare diseases and their unmet therapeutic need prompted the development of various innovative treatments such as gene therapies, chelating agents for abnormal molecules, or regenerative medicine approaches.

Areas covered: Gene therapies intended for various forms of retinopathies and for lysosomal storage disease, chelating agents for light-chain amyloidosis and a restorative therapy for epidermolysis bulosa are among the orphan drugs designated in the EU between June and July 2014.

Expert opinion: Among the drugs discussed in this paper, two recently designated orphan drugs intended for AL amyloidosis may be promising therapeutic candidates if the future clinical data are supportive.

Keywords::

• AL amyloidosis
• gene therapy
• monoclonal antibodies
• orphan drugs
• rare disease