A Wythenshawe family have won their battle for NHS approval of a “miracle drug”, which will massively boost the quality of life of their terminally ill son.
Shakeel Khan and his wife Renata have been fighting for months to have drug Spinraza, funded by the NHS to treat their son Haris who suffers from Spinal Muscular Atrophy. The drug which is used a number of countries around the world, including Scotland, was not available on NHS in England because of cost.
But this week, following a meeting of the National Institute for Health and Clinical Excellence, the family finally got the result they had been campaigning tirelessly for and were told the drug, manufactured by Biogen, will now be available to treat their son.
And it means families across the country will also be able to benefit from the drug which has a massive impact on the quality of life of sufferers.
Shakeel said he was delighted with the decision and has praised the local community in Wythenshawe for their support as well as local businesses and sports stars including footballers Jermaine Defoe and Riyad Mahrez, and boxers Tyson Fury and Amir Khan, who helped raise funds for their campaign.
Former England international Defoe, whose Jermaine Defoe Foundation helps children in the UK, bought a carry-cot for the family and delivered a personal message to Shakeel and Renata when news of their victory came through.
Mahrez, whose goal helped secure Manchester City’s fourth Premier League title on the last day of the season, has also supported the family and visited them at their home in Newall Green where he met Haris’ brother, 10-year-old Blues fan Maryus. The club has also donated a signed shirt.
Wythenshawe boxing legend Tyson Fury and Bolton boxer Amir Khan both donated signed gloves.
Shakeel has also thanked Piece of Cake by Neelam Burto, Creations by Rocky and the local One Stop Shop whose Carriers for Causes initiative has raised cash for Haris.
Shakeel told the Reporter: “I can’t thank these people enough. They have been amazing. And I want to thank all my neighbours who have been incredible, especially Angela and Lee.”
The fund raising events are continuing and Shakeel is to buy a specially adapted vehicle for Haris and other disabled children in Wythenshawe with plans for days out for disabled youngsters in the area.
Donations can be made via the Just Giving page.
NICE, Biogen and NHS England say they have agreed to make Spinraza available for children, young people and adults with SMA Types 1, 2 and 3 through a scheme known as a Managed Access Agreement (MAA). This means that patients will be able to get Spinraza while more long-term data on its effectiveness is gathered.
NHS England says the treatment will be made available to the youngest and most severely-affected (SMA Type 1) patients immediately by Biogen, with NHS England offering funding on NICE’s publication of final guidance.
It says that for older babies, children and young adults with SMA Types 2 and 3, the NHS will begin to provide Spinraza shortly after NICE’s guidance is published, once the services to deliver them are established. This is not expected to take more than a few weeks.
The charity, Muscular Dystrophy UK welcomed the news, but said on their website: “Our work here is not done. The lengthy, frustrating delays which we have seen throughout this process must not be allowed to happen again.”
And Doug Henderson, Managing Director of Spinal Muscular Atrophy UK, said: “At last the SMA community has the answer it has been asking for since NICE started its appraisal almost 17 months ago. Our thanks to NICE, NHS England and Biogen for this great news and to all the SMA community who worked so hard on this journey. The clinical evidence was there; our voices were finally heard.
“We are only sorry that it took so long when time matters so much; for the families with infants with SMA Type 1 who have had no access to treatment since November 2018; for families and adults who have desperately wanted to have the opportunity to see what potential this treatment might have for them; for the clinicians who have been so frustrated by their lack of power to offer it.
“We will now do all we can to support the smooth implementation of the Managed Access Agreement. We will also continue to work to advocate for change to the NICE process for appraising access to orphan medicines so that future treatments are more rapidly and appropriately assessed.”