Forest Gate baby with rare genetic disorder is denied life-saving drug because of its cost
PUBLISHED: 11:51 24 January 2019 | UPDATED: 12:01 24 January 2019
A couple from Forest Gate who lost a son to a rare genetic disorder are pleading with the NHS to provide a "wonder drug" for their baby daughter, who also has the condition.
Health chiefs are refusing to recommend the potentially life-saving drug for Maryam Malji because of its ‘extremely high’ cost however it is available in Scotland.
The five-month-old has type 1 spinal muscular atrophy (SMA), which affects walking, eating, drinking and breathing and doctors have told her parents, Zainab and Shakil, she could have just months to live.
Maryam’s brother Abdullah also had SMA1 and was one of the first children to receive the drug Spinraza when he was placed on a medical trial.
He died aged one in December 2015, from an infection, but the trial was successful and led to Spinraza being government-funded in 24 European countries.
An access programme mainly funded by US drug firm Biogen was rolled out at Great Ormond Street Hospital in 2016.
It ended in October - six weeks before Maryam’s diagnosis- after NHS approval body NICE said it was not recommending Spinraza due to its “extremely high” cost.
A petition set up by Mr Malji to get the drug funded for Maryam, the two other children diagnosed since October, and future sufferers has more than 130,000 signatures.
He said: “When Abdullah started on the trial we had no hope. But his improvement was incredible. He was very weak and it was an infection which killed him.
“Doctors told us he would have to spend the last weeks of his life on a ventilator, but because of the drug that never happened. When we were told it was too late for [Maryam] to get Spinraza we were heartbroken. It’s so unfair.”
Children on Spinraza continue to receive it. SMA affects motor functions in the spinal cord and lower brain stem, resulting in severe and progressive muscle wastage.
Spinraza, also known as Nusinersen, is the first approved treatment for the disease.
It costs £75,000 per dose and is administered by four injections into the cerebral spinal fluid, followed by a top-up injection every four months.
Mr Malji’s campaign has been backed by charities, local councillors and Labour MP Lyn Brown.
She said: “I believe Biogen have a moral duty to find a way forward for Maryam. Abdullah was involved in the trials that proved Spinraza works.”
Forst Gate South councillor Mas Patel added: “Its heartbreaking for any parent to see their child suffer, but for Mariams parent’s it’s even more painful having to go through the whole excruciating experience again. We cannot allow drug companies such as Biogen to abdicate their responsibility and having used Abdullah during his final moments to test the drug, it’s only right that they now provide his sister with the drug to save her life.”
Mr Malji set up the Newham-based charity Abdullah Aid, which works in countries across the world, in honour of his son. But he is considering moving the family to Scotland.
He added: “With the help of communities and a relative, Islamic scholar Ismail Menk, we managed to reach over 130,000 signatures with our petition, the main purpose of this was to allow Maryam and many other children have access to Spinraza to treat a cruel disease which is taking lives of children.
“We humbly ask the public to continue signing the petition to make a larger impact to save lives of babies like Maryam.”
Meindert Boysen, director of the centre for health technology evaluation at NICE, said talks between NHS England and Biogen were ongoing.
“The committee was willing to be flexible because of the nature of the condition, but the very high cost of Nusinersen meant it could not recommend the drug,” he added.
A spokesman for Biogen said it is “working towards a positive outcome in negotiations”.
To see the petition and sign visit change.org/p/biogen-idec-spinraza-on-nhs-for-sma.
For more information about Abdullah Aid visit abdullahaid.org.uk.