Last week, Deputy Broughan wrote about access to Spinraza for SMA sufferers.  Another treatment for a rare disease that he has consistently been raising has been access to Kuvan for people with PKU (Phenylketonuria).

Today, Deputy Broughan received the following welcome response from the HSE following his most recent Parliamentary Question on the matter:

“Sapropterin (Kuvan) is an orphan medicine administered orally for the treatment of Phenylketonuria.

 Sapropterin has been approved by the HSE Senior leadership Team for use in specific circumstances in line with Start-Stop Guidelines prepared by expert clinicians. The HSE has agreed improved but commercially confidential terms with Biomarin in relation to the pricing of this medicine.

Patient and clinician engagement was of significant assistance in the development of these guidelines which have enabled reimbursement.

Reimbursement will be in place from 1 July 2019 under the High Tech arrangements.”

Deputy Broughan says “I am delighted to see that some people with PKU will be able to access Kuvan from later this year.  I have concerns about the “specific circumstances” phrase used in the reply but I will await word from the PKU Association to see if this is something that we need to follow-up with.  In the meantime, I would like to commend the people with PKU and their families who have fought so hard for access to this treatment for many years.”