NHS England has announced that it will make sapropterin dihydrochloride available to all patients with phenylketonuria (PKU) after agreeing a deal for the generic version of the drug.
Branded Drug 'Not Cost Effective' for All
Recent guidance from the National Institute for Health and Care Excellence (NICE) restricted treatment with the branded version of sapropterinin (Kuvan, BioMarin) to patients under the age of 22, on the grounds that the branded drug was too expensive to be cost-effective for older adults.
NHS England has now struck a deal with Teva Pharmaceuticals, backed by NICE, to make the first generic version of the drug.
Prof Stephen Powis, NHS England's medical director, 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."
National Testing Programme
Following the agreement, sapropterin will be rolled out to more than 2300 people with PKU across England as part of a national testing programme to identify patients who could 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.
Pregnant women who have PKU will also be offered the drug immediately.
NHS England says clinical trials have suggested that 4 in 10 people with PKU may benefit from sapropterin, improving their quality of life significantly and reducing restrictions on the food they can eat.
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."
Commenting on the announcement, a spokesperson for the National Society for Phenylketonuria (NSPKU) said: "People with PKU and their families have worked hard with NSPKU to get to this announcement.
"PKU is a lifelong condition, and we welcome the news that sapropterin will be available to all adults."
The NSPKU says it will be in contact with the Welsh and Scottish Governments about plans for extending access in Wales and Scotland. Northern Ireland usually approves NICE guidance.
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Cite this: Peter Russell. All Phenylketonuria Patients in England to be Offered Generic Sapropterin - Medscape - Dec 17, 2021.
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