EU Orphan Designations Fall Sharply In 2017

The number of medicines awarded orphan designation in the EU has fallen for the first time since 2013. In 2017 there were 144, considerably fewer than in 2016 when there were 209. In the meantime, orphan drug advocacy group Eurordis says radical change is needed if patients are to have broader access to these important but often highly priced drugs.

pills-tablets-glass-container_69512212_1200
Orphan Drug Designations Fall in the EU in 2017

The number of products awarded an orphan drug designation by the European Medicines Agency in 2017 fell by almost a third. In 2017, 144 designations were awarded compared with 209 in 2016, a fall of 31%.

The decline in designations by the EMA’s Committee for Orphan Medicinal Products (COMP) in 2017 bucks a trend that saw...

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Pink Sheet for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required

More from Europe

EMA’s PRIME Scheme Explored: Growing Designations - But Are Accelerated Assessments Slipping?

 

While the first six months of the year saw eight new drugs targeting a range of diseases enter the European Medicines Agency’s priority medicines scheme, and three PRIME-designated treatments go on to win EU marketing approval, use of the accelerated assessment mechanism appears to be limited.

EU Medical Countermeasures Strategy Promises More AMR Pull Incentives

 

A new EU strategy to ensure access to essential medicines will boost the development of critical medical counter measures, including vaccines and therapies, according to one pharmaceutical industry group.

Animal Testing: US And EU Regulatory Support Grows For Alternatives, But Validation A Hurdle

 

In this first of a two-part series of articles, US Pharmacopeia CEO Ronald T. Piervincenzi tells the Pink Sheet how the use of alternatives to animal testing in drug development is gaining traction and discusses the challenges that exist.

AI And Inertia: The Disruptors Keeping EU Regulators Awake At Night

 

Data is “the new oil,” but its use will challenge regulators, while inertia is the biggest disrupter of all for European regulators, according to European Medicines Agency chiefs past and present.

More from Geography

EMA’s PRIME Scheme Explored: Growing Designations - But Are Accelerated Assessments Slipping?

 

While the first six months of the year saw eight new drugs targeting a range of diseases enter the European Medicines Agency’s priority medicines scheme, and three PRIME-designated treatments go on to win EU marketing approval, use of the accelerated assessment mechanism appears to be limited.

Global Pharma Guidance Tracker - June 2025

 

Stay up to date on regulatory guidelines from around the world with the Pink Sheet’s Guidance Tracker. The complete Global Pharma Guidance Tracker, with sortable and searchable listings going back to 2014, is available online.

US FDA’s CRL Release More Incremental Than ‘Radical’ Transparency

 

The transparency initiative collects CRLs already made public in approval packages on its drugs@fda site to post on the openFDA public portal.