This story was first published in digitalhealth.net
NHS strikes deal to make PKU drug available for all
The NHS is set to make a drug for the rare, debilitating disorder phenylketonuria (PKU) available to patients of all ages for the first time.
The health service will roll-out a national genetic testing and responsiveness programme for around 2,300 people with PKU across England in order to identify those patients who stand to benefit from the treatment. Around 50 people have already shown a positive response to the drug through testing and will be eligible to receive it on the NHS from next week.
A deal has been struck by the NHS to secure a non-branded ‘generic’ version of the drug, sapropterin dihydrochloride, for patients with the inherited condition, which means patients cannot eat protein. The genetic disorder prevents the breakdown of the amino acid phenylalanine and can lead to serious health issues, including brain damage, with patients forced to live on a highly restricted low-protein diet.
This is the latest in a string of deals agreed by NHS England to make life-changing, innovative treatments more readily available for patients, including risdiplam and Zolgensma for Spinal Muscular Atrophy, a first new treatment for sickle cell disease in two decades, and ‘5 minute’ breast cancer treatment, Phesgo, which has been rolled-out by the NHS in England faster than anywhere else in the World.
Stephen Powis, NHS Medical Director Professor, said: “This life-changing drug will make a huge difference to hundreds of people with PKU, allowing them to enjoy foods that were previously off limits and significantly improve their quality of life. It is fantastic the NHS has been able to use its commercial abilities to reach a deal to supply a non-branded version of sapropterin, to offer it all to all patients who are shown to gain a clinical response from the treatment.
“In the run-up to Christmas, after what I know has been a long and frustrating wait for many people, I am delighted the NHS has been able to reach this agreement with an alternative company that was ready to put patients first.”
Latest News
16/02/2024 - 12:56
Hammersmith and Fulham Council has created a new alliance intended to clean dirty air and improve residents' health.
16/02/2024 - 10:44
Two million more GP appointments a month are being delivered for patients compared to the same month before the pandemic as part of the NHS primary care access recovery plan according to new NHS data.
15/02/2024 - 10:26
The Welsh Government has provided innovation funding of £380,000 to support two projects utilising technology in domiciliary care.
15/02/2024 - 10:13
The new Scottish health secretary has met with frontline staff at Queen Elizabeth University Hospital in Glasgow.
14/02/2024 - 10:27
The North East London NHS Foundation Trust (NELFT) has appointed three new Executive Directors to the Trust Board.
Product Features
UK Building Regulations highlight toxic gas and smoke from layers of paint built up over multiple redecorations as a major cause of permanent ill health or death in a building fire.
Their concern rose with discovery the flame retardant paints most widely used paint along escape routes have been ones which to this day counter-productively use emission of heavy toxic gas to smother flames which rapidly spread along walls if layers of paint delaminate in a fire.
Supplier Profiles
Northwich’s Victoria Infirmary (VIN) Community Diagnostic Centre (CDC) has enabled more patients
Adveco, the commercial hot water specialist, announces the launch of live metering of domestic ho
Latest Features
Sarah Greenslade, public affairs and communications officer at the British Parking Association looks at some of the problems and innovations in healthcare parking
It’s easy to assume that the comms team is there to handle press enquiries and the occasional social media storm – but the reality is that strategic communications can make a measurable impact across the entire organisation, from operational to financial, when done properly
