Tag: Spinraza

Wythenshawe family win fight for”miracle” drug for baby Haris

vid00009_dvd.originalA 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.

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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.”

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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.”

Campaigning family continues fight for”miracle” drug for baby Haris

A Wythenshawe dad of a terminally ill baby says time is running out for the NHS to provide a “miracle” drug which could massively improve the quality of his son’s life.

Eight-month-old Haris, from Newall Green, who has Spinal Muscular Atrophy was given three months to live when he was diagnosed with the condition in February.

Until February last year, the wonder drug Spinraza which can prolong and dramatically improve quality of life was available on the NHS, but was withdrawn on cost grounds.

Haris’ mum and dad Renata and Shakeel have been campaigning for the NHS restore funding for the drug, which is available in a number of countries including Scotland.

And in March the family and their supporters lobbied a meeting of the National Institute for Health and Care Excellence, the body which makes the recommendations for the funding of drugs on the NHS.

Following the meeting, NICE’s Chief Executive, Sir Andrew Dillon, wrote to Wythenshawe MP Mike Kane saying they were hopeful of  positive outcome with the drug company Biogen, who manufacture the drug.


But nearly two months after their protest, there is still no decision on the funding of the drug which is deemed too expensive.

With Haris is now back home with his family in Newall Green, Shakeel says the NHS, NICE and Biogen all share responsibility for the failure to provide the drug with will have a massive impact on his son’s quality of life.

Funding for the drug will be considered again at meeting of NICE next week (Wednesday May 8) and NICE have released this statement:

Following the committee meeting on the 6 of March 2019, the appraisal committee asked NHS England and Biogen to enter into commercial discussions to see whether a managed access arrangement could be agreed for Spinraza consistent with NICE’s framework for determining cost effectiveness.

We have been informed by the parties that no agreement has yet been reached. Nevertheless, we consider it necessary to return to the appraisal committee next week in case an agreement is reached, or if no agreement is reached, for the committee to consider the consequences for guidance development.

This topic will be considered by the committee on Wednesday 8 May. Given the nature of the discussion, this will be held in a private.

Shakeel  says the fight will continue. Boxer Amir Khan is supporting the campaign and Shakeel says he has received tremendous support from the local community and from the charity New Life which has funded a special cot costing £4,000 and a buggy costing £3170. Haris also has a Just Giving page to raise funds in the event that the final decision is to reject funding the drug and the family has to go abroad to get treatment.

Spinal muscular atrophy is caused by dysfunctional or missing gene that affects the production of a protein essential for motor neurons. This condition is manifested by a shrinking spinal cord.

One in 40 people are a carrier of the faulty gene and in the most severe cases, such as Haris, children rarely live beyond two years, making SMA the leading cause of death in babies and toddlers in the country.

But Spinraza has been known to have significant effects on the length and quality of life for patients.

Manfuacturer’s Biogen markets Spinraza at $750,000 dollars in the USA during the first year and $375,000 per subsequent year. The company offered the drug at £450,000 to the NHS and an undisclosed discount, but it still was not enough.

One report has said Biogen’s growth is heavily dependent upon SMA medication.


Family of Wythenshawe baby fight for right to “miracle drug”

This is Haris Khan from Newall Green, Wythenshawe. He is six months old and desperately needs a drug currently not available on the NHS

Doctors say Haris, who suffers from Spinal Muscular Atrophy, say he may not make it till his first birthday without Spinraza, a drug no longer available in England but used in Scotland.

Today (March 6) his family and supporters protested at a meeting of the National Institute for Health and Care Excellence (NICE) and pleaded with them to make the drug available.

NICE have avoided recommending the drug because of its £400,000 a year cost. But the drug is available in Scotland.

Haris’ dad, Shakeel Khan, told ITV News: “There is a treatment out there called Spinraza that can help save his life.

“But it’s not available on the NHS here in England, we are absolutely disgusted at this.

“Apparently it’s too expensive to treat Harris.”

While the drug is not seen as a cure families in England are pleading for it be made available on the NHS.

Spinal muscular atrophy is a genetic condition that weakens the muscles and gets worse with time.

The manufacturers of the drug said today they will lower the cost of the medication and Haris’ supporters have set up a Just Giving page to fund their campaign. https://www.justgiving.com/crowdfunding/littleharis