A father who lost his son to a rare genetic disorder has won his campaign to have the NHS fund a “wonder drug” for his baby daughter who also has the condition.
Shakil Malji’s nine-month-old daughter Maryam has type 1 spinal muscular atrophy (SMA), which affects eating, drinking and breathing. When she was diagnosed with the deadly muscle-wasting condition in December, doctors told her parents she could have less than a year to live.
The family, from Newham, were devastated to learn an access programme to the new drug Spinraza, the only approved medication for the disorder, had been stopped just weeks before Maryam’s diagnosis and the NHS drug approval body Nice was not recommending the treatment due to its “extremely high” cost.
Mr Malji set up a petition, which received more than 350,000 signatures, to get the drug funded, and led rallies outside Nice’s offices in London and Manchester with other campaigners, including religious leaders and MPs.
NHS England will now provide the treatment after “negotiating a deal” with US manufacturer Biogen. It will initially fund treatment for a limited time, allowing more data to be collected on its long-term effectiveness.
Mr Malji said: “I’m still in shock. It is such a relief. Maryam and all the other children who have this condition now have a chance at life. We have so many people to thank — locally, nationally and internationally — who helped with this campaign. When the news came I cannot describe how happy everyone was.”
Maryam’s brother Abdullah also had SMA1 and was one of the first children to receive Spinraza on a medical trial. He died aged one in December 2015 from an infection, but the overall trial was successful and led to the treatment being funded by governments in 24 European countries, including Scotland.
An access programme mainly paid for by Biogen was rolled out at Great Ormond Street Hospital in 2016 and showed extremely promising results. But it ended in October last year, leaving dozens of babies in England with the condition unable to receive the drug. At the time Spinraza cost £75,000 per dose. It is administered by four injections into the cerebral spinal fluid, with a top-up injection every four months.
West Ham MP Lyn Brown, who helped the family’s battle, said: “Months of campaigning by parents, councillors, imams, bishops and my fellow MPs has finally paid off … I’m overjoyed this decision has finally been taken.”
The treatment will be made available to the youngest and most severely affected patients immediately, Nice said. A spokesman added: “Where companies show appropriate flexibility, it is possible to find a way to provide important treatments to patients in a cost-effective way.”