More than 20 Irish patients with severe lung disease are facing an uncertain future as their access to a “life-changing” drug ends this week.
Some of the patients, who have genetic emphysema, have been on the drug, Respreeza, for up to a decade.
However, the manufacturer, CSL Behring, is ending access under its compassionate use programme at the end of September.
An application to have Respreeza, which costs more than €80,000 per patient per year, provided for free in the Irish health service was rejected by the HSE in August.
CSL Behring decided to withdraw the drug after talks with the HSE failed to reach agreement.
The drug is the only treatment that slows the progression of the condition by decreasing the frequency and severity of chest infections and associated hospital admissions.
The patients on Respreeza have alpha 1 antitrypsin deficiency - also known as alpha-1 - a hereditary condition that is unusually common in Ireland.
Many were put on clinical trials from 2006 and, when these ended in 2010, a compassionate use programme.
The company repeatedly threatened to end the programme but delayed the execution of this threat up to now.
Many of the patients involved have been on the therapy for more than ten years and have no other therapy option, according to the Alpha One Foundation, which runs a HSE-funded screening programme for the condition in the hospital.
An additional 40 patients who were diagnosed after the trial ended were waiting to be put on what the foundation says is a “life-changing” therapy.
Geraldine Kelly, chief executive of Alpha One, said the affected patients were "distraught".
“We simply don’t know for certain what the impact will be, but we do know that any lung damage that occurs cannot be reversed. Patients are likely to incur chest infections and other complications.”
Last week, Taoiseach Leo Varadkar said it would be "inappropriate and unethical" for CSL Behring to cut off supply of the drug to Irish patients.
Backing the HSE stance, he said there was insufficient evidence to suggest patients would derive a clinically meaningful benefit from the treatment.
Ms Kelly asked why the Taoiseach would call on the company not to withdraw the drug if he didn’t think it was beneficial.
“He also says the Government cannot interfere with decision-making process for drugs but this is precisely what happened with Orkambi [the cystic fibrosis treatment].”