Zolgensma approval for child
A case was raised about a Killorglin infant with spinal muscular atrophy needing HSE approval for the drug Zolgensma. The Minister said it was a difficult, time-sensitive case and promised to follow it up.
I want to raise the case of Theo Whelan, a five-month-old Killorglin boy who has spinal muscular atrophy. Theo's parents, Shane and Stephanie Whelan, have contacted me to try to get the HSE to give approval to the so-called miracle drug, Zolgensma. I understand that two children, Theo and little 17-month-old Kate Mynard in Cork are the only known suitable recipients in Ireland for this drug, which can dramatically improve the quality of life with children with the condition if the drug is administered before the age of two years. Deputy Christopher O'Sullivan raised the case of Kate with the Taoiseach very recently. Time is crucial. I recall from the Orkambi campaign that this process can take far too long. Ireland is in negotiations with the company, together with Holland and Belgium, but while those negotiations are ongoing, Holland is administering the drug to children who need it right now before a long-term arrangement can be put in place. Can we pursue that? Can the Minister contact the Taoiseach's office to pursue the matter?
Comment on this
This sounds like a very difficult case, particularly when there are time considerations in respect of the application of the drug. I can understand why parents would be at their wits' end trying to get access to a drug they believe can make a life-changing difference to their child. It is the first that I have heard of this case but I will follow up on it. If the Deputy sends me the details I will speak to both the Taoiseach and the Minister for Health on it. There is a process that needs to be followed for the approval of new drugs, even for very rare conditions. I do not know whether it is possible to look at interim treatment but I will ask that question for the Deputy.