NICE u-turn approves first therapy for children with XLH

Final Recommendation

We are absolutely thrilled to announce this morning that burosumab has been APPROVED for routine use on our NHS for children from 1 years up to young adults aged 16/17 years.

This news completely changes the landscape for the estimated 250 children living with XLH in the UK. Burosumab will be the first and only treatment that targets the cause of the hypophosphatemia in the body. The treatment helps reduce symptoms during childhood and by reducing bone damage during childhood growth it could mean the potential for a better quality of life for children later in their lives too.

We want to say a MASSIVE thank you to all those individuals who shared their experiences with us and with NICE so that decision-makers could understand what this treatment will really mean to patients and their families. Without all of your input this decision to approve burosumab may not have been made.

For those that can’t quite believe their eyes this morning, please visit NICE for the full guidance on children with XLH. If you have further questions about the treatment and whether it is right for your child then please do speak to their paediatrician that manages their XLH.

Since this news is so important, we’ve put together a press release for our site. (see attached)

Whilst access to burosumab on the NHS will just be for children for now, let me assure you that the fight for the adult license has begun.

Meanwhile please please do stay tuned and encourage other XLH’ers in the UK & their families to sign up with us because we’re so much stronger together.

XLH UK’s press release can be viewed here: