Dáil Éireann Debate, Thursday - 30 July 2020
1443. Deputy Jennifer Murnane O'Connor asked the Minister for Health if ionafornid treatment in the rare disease progeria will be licensed here; if the State will be providing the financial burden of hospitals providing such treatment; and if he will make a statement on the matter. [20964/20]
View answerIn Ireland, a medicine must receive a marketing authorisation before being placed on the market. Marketing authorisations may be granted nationally by the national competent authority (in Ireland this is the Health Products Regulatory Authority) or by the European Commission, following a positive recommendation by the European Medicines Agency (EMA).
Lonafarnib is an investigational drug currently being studied for safety and efficacy for a number of conditions, including progeria syndrome. As such, Lonafarnib has yet to be approved for any condition.
In December 2018, following an application by the sponsoring company and a recommendation by the EMA, Lonafarnib was granted ‘orphan status’ by the European Commission, as a medicine which could potentially treat a rare disease such as progeria. Sponsors who obtain orphan designation benefit from protocol assistance, a type of scientific advice specific for designated orphan medicines, and market exclusivity once the medicine is on the market.
In the EU, medicines for rare diseases such as progeria must be authorised by the European Commission under the ‘centralised procedure’. It is therefore not open to Irish authorities to grant a marketing authorisation for Lonafarnib.
In May 2020 a marketing authorisation application for Lonafarnib was submitted to the EMA on behalf of the Commission under an accelerated assessment procedure. Evaluating a marketing-authorisation application under the centralised procedure can take up to 210 days, not counting clock stops when applicants have to provide additional information. Accelerated assessment reduces the timeframe for the EMA to review the marketing-authorisation application and can reduce the timeframe to 150 days, not including any clock stops. The EMA will follow the accelerated review timeline of the application and it will be up to the sponsoring company to respond promptly to requests for additional information or data to support its application.
As the second part of the question is a service matter, I have asked the Health Service Executive to respond to the deputy directly, as soon as possible.
