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

Abstract

Introduction: Many rare diseases could benefit from a therapeutic point of view; examples include targeted gene therapies or ‘borrowing’ therapies from other diseases based on their mechanism of action, which is similar to that of the rare disease in question.

Areas covered: This paper discusses five of the most recent orphan drug designations in the EU. Their corresponding compounds are to treat severe combined immune deficiency, Leber’s congenital amaurosis, Charcot–Marie–Tooth 1A disease or bronchopulmonary dysplasia.

Expert opinion: Most of the therapies discussed are currently still at an experimental stage and the demonstration of a comparable efficacy in clinical stages of development will be essential for market access.

Keywords::

• bronchopulmonary dysplasia
• Charcot–Marie–Tooth
• Leber’s congenital amaurosis
• orphan drugs
• severe combined immune deficiency