The European Medicines Agency (EMA) looks set to give approval for the extension of Kalydeco to people with CF who have the R117H gene alteration and who are aged between 6 months and 18 years.

This will likely make this important drug therapy available in Ireland for about 50 young people and children with CF in the near future. 

At present, only those aged 18 and over of this defined group can access Kalydeco in Ireland.

Once final EMA approval has been given, this drug extension will be automatically extended to Ireland. 

Vertex today (1 May, 2020) announced the Committee for Medicinal Products for Human Use (CHMP) – a sub committee of the EMA - has today made a recommendation to the full EMA to extend Kalydeco to under 18’s.

The full press release from Vertex is available to view by clicking here.

Thanks to the innovative deal agreed between the Irish Government and Vertex in 2017, the 50 or so children and young people with CF in Ireland eligible for this extension, will be automatically entitled to this extension once it is finally approved by the EMA. 

The Role of the CHMP

The CHMP plays a vital role in the authorisation of medicines in the European Union (EU). The CHMP is responsible for:

Conducting the initial assessment of EU-wide marketing authorisation applications;

Assessing modifications or extensions ('variations') to an existing marketing authorization (as in this case)

Considering the recommendations of the Agency's Pharmacovigilance Risk Assessment Committee on the safety of medicines.

The CHMP is a sub committee of the The European Medicines Agency.

The EMA is an agency of the European Union in charge of the evaluation and supervision of medicinal products.