Children like little Rory need Kuvan drug now!
HEALTH chiefs must seize the opportunity to end a 12- year farce and strike a deal over a drug for sufferers of a rare genetic condition, say MPs.
Those with Phenylketonuria, or PKU, must live on a severely restricted diet as too much protein can cause brain damage unless they take Kuvan.
The NHS has failed to make the lifechanging drug available for free. Now its maker will in the new year lose their exclusive EU patent – allowing rivals to enter the market with cheaper generic versions of the drug.
Campaigners for Kuvan have met NHS England with the backing of over a dozen MPs, urging that they finally strike a deal with drug maker BioMarin. It follows the Daily Express crusade to ensure Kuvan is available to all on the NHS.
If BioMarin refuses to lower its price, they insist that NHSE negotiates a deal with cheaper generic drug makers instead.
Kuvan costs around £ 45,000 per adult per year, but as low as £ 4,000 for a toddler. The NHS wants a significant discount for buying in bulk.
PKU sufferers must replace real protein with a foul- tasting synthetic and avoid meat, fish, eggs, pulses, dairy and some vegetables.
Kuvan regulates blood levels when a person with PKU eats tiny amounts of protein- rich foods, such as milk, cheese, nuts or meat. The medicine, which could help 600 UK sufferers lead a normal life, has been available since 2009, but not on the NHS. Meanwhile, 26 other European countries including Latvia, Bulgaria and Estonia, all have it.
A cross- party letter to Health Secretary Matt Hancock was the brainchild of Labour MP for Blaydon, Liz Twist, who said: “Families need Kuvan now! We are deeply anxious PKU sufferers who are suitable for this life- changing drug have been ignored for too long.”
The letter was signed by 14 peers and MPs, including Lord Austin; Tory MPs Peter Aldous, Richard Drax, Jeremy Hunt and Nigel Mills; LibDem Tim Farron and Labour MPs Christina Rees, Marie Rimmer, John McDonnell, Kerry McCarthy, Emma Lewell- Buck, Catherine McKinnell and Sharon Hodgson.
Kuvan is BioMarin’s brand name for what is an older drug called sapropterin but their patents have until now stopped others from making it in Europe. BioMarin’s patents expire in January, meaning in 2021 rival firms can make their own, cheaper version which the NHS could also buy.
But post- Brexit they will have to get licensed by the Medicines and Healthcare products Regulatory Agency ( MHRA) and MPs want that fast- tracked.
The letter to Mr Hancock stresses: “It is encouraging generic manufacturers can prepare to license generic sapropterin from December 2020. Manufacturers will now need to apply for a separate UK licence from MHRA.
“MHRA can take potentially more than a year to issue a licence. The Government must act to ensure swift access. Further delay is agonising to patients and their families. More delay will risk harming people.”
Swiss- based firm Dipharma has told the Daily Express it wants to offer a PKU treatment solution. Chairman Marc- Olivier Geinoz said: “Provided it would be economically viable, Dipharma would be interested to submit its generic sapropterin in the UK.”
Campaigner Kate Learoyd, 46, is mum to PKU sufferer 12- year- old Alex. She was with campaigners at last week’s meeting with Blake Dark, the NHSE Commercial Medicines director. Kate said: “The NHS systems have failed to sort this out for years. MPs have seen the heartache PKU families have lived with and want to see a solution fast.”
NHSE said generic sapropterin must be approved by drugs appraiser National Institute for Health and Care Excellence ( Nice) before any deal can be approved. A spokesman said: “The NHS has repeatedly urged BioMarin to offer the drug at a fair price. We have already committed to offering the immediate rollout of a generic v ersion if agreed by Nice.”