Fresh from the designation pipeline: orphan drugs recently designated in the European Union (August–October 2014)

Abstract

Introduction: This paper features new therapies that have recently received orphan drug designation in the European Union (EU) for various rare diseases. As in the previous papers of this series, orphan drugs designated for diseases with no therapy authorized in the EU are discussed.

Areas covered: From repurposed therapies such as retinol for the prevention of bronchopulmonary dysplasia or acamprosate calcium for Fragile-X syndrome to gene therapy for enzyme deficiencies or monoclonal antibodies for inherited metabolic disorders, a large range of diseases and therapies are discussed.

Expert opinion: Inherited conditions can be tackled with various approaches, including ‘etiologic’ therapies (i.e., treating the cause or origin) or with ‘pathogenic’ therapies (i.e., inhibiting pathogenic pathways). In this paper, this is best exemplified by two very spectacular therapies, a gene therapy to treat a mitochondrial disorder (mitochondrial neurogastrointestinal encephalomyopathy) and a monoclonal antibody for X-linked hypophosphatemia. In both cases, the preclinical evidence supports the feasibility of the approaches but clinical efficacy data are needed to enable these therapies to be authorized on the market.

Keywords:

• EMA
• orphan drug
• orphan drug designation
• rare diseases

Declaration of interest

The author has no relevant affiliations or financial involvement with any organization 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.