Orphan drug approvals of 2015: Europe and the United States

ABSTRACT

Introduction: The development of orphan drugs for the treatment of rare diseases has attracted increasing interest from the pharmaceutical industry. The level of European approvals remained high but approvals of novel orphan drugs in the US has declined sharply from the record level of 2014.

Areas covered: The review discusses all the orphan drug approvals of 2015. It firstly considers the 17 European approvals, which included 15 novel chemical entities. It then considers the 38 US approvals, which included 16 novel chemical entities. The discrepancies in approval timings between these markets are then discussed.

Expert Opinion: The steady increase in the number of orphan drug approvals is an encouraging trend. Also encouraging is the increasing use of expedited approval for those orphan drugs seeking marketing authorisations. In 2015 there also appear to be a general decrease in the time delay between US and European approvals. It is clearly desirable that new approvals target rare diseases for which there are no current treatments, or no effective treatments. It can thus be viewed as disappointing that many of the year’s orphan drug approvals were for the treatment of diseases were several treatment options are available.

KEYWORDS:

• EMA
• FDA
• orphan drugs
• rare diseases

Article highlights

• 2015 was another good year for orphan drug approvals in Europe and the US.

• Orphan drug approvals in 2015 included first approvals for 16 novel chemical entities.

• Orphan approvals in 2015 were heavily focused on oncology indications.

• Significant new approvals include treatments for hypophosphatasia, Leber hereditary optic neuropathy, Waldrom’s disease, homozygous familial hypercholesterolemia, Waldenstrom’s macroglobulinemia, and the first corrective therapy for cystic fibrosis patients expressing the (predominant) μF508 CFTR mutation.

• 2015 also saw the first stem cell therapy approved, but only in Europe.

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Declaration of interest

The author has no other 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.