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NHS Strikes Deal to Make Phenylketonuria Drug Available for All

The NHS have struck a deal to secure a non-branded, generic, version of the drug Sapropterin dihydrochloride, for patients with PKU. The drug will be available to patients of all ages for the first time, including pregnant women.

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.

The drug works by stimulating a specific enzyme in the blood that helps to improve the breakdown of phenylalanine.

Around 50 people have already shown a positive response to the drug through testing. It became available on the NHS in December last year.

Clinical trials suggest that around four-in-10 people may benefit from Sapropterin, improving their quality of life significantly and reducing restrictions on the food they can eat.

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.

NHS Medical Director Professor Stephen Powis said: “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.”

The testing process can take between eight-to-10 weeks and the drug will be offered to every individual where a clinical benefit is observed.

The NHS is rolling out the testing programme to support the implementation of guidance from the National Institute for Health and Care Excellence (NICE). The original manufacturer of the branded drug had consistently refused to offer the drug at a fair price, meaning that NICE could only recommended its use in a much smaller patient population, with the drug being only available to patients up to age 22 because it was not deemed cost-effective by NICE.

Kim Innes, general manager of Teva UK and Ireland, said: “Doing the right thing by giving patients and the NHS access to affordable treatments is at the heart of everything we do at Teva. We’re working closely in partnership with NHS England in order to provide a cost-effective treatment for PKU patients that will give clinicians more options to help treat this rare but potentially serious inherited disorder.”

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