Baby With Fatal Condition Gets World's Most Expensive Drug After Parents' Battle

An 11-month-old baby with a fatal genetic condition has received the world's most expensive drug after a campaign fought by his parents.

Edward Willis-Hall, from Colchester, England, has severe spinal muscular atrophy (SMA), which causes paralysis, muscle weakness and progressive loss of movement.

He received the gene therapy Zolgensma, which contains a replica of the missing gene SMN1 and is administered in a single dose, at Sheffield Children's Hospital last weekend.

Edward's mother Megan Willis said she was "so excited and relieved" her son had received the drug, the BBC reported. "For so long we have been trying to fight for this drug. I think I am in shock, I can't believe the day is here," she said.

Babies born with severe SMA Type 1 have a life expectancy of just two years, according to NHS England. There is no cure, but NHS England said in studies Zolgensma has helped babies breathe without a ventilator, sit up on their own and crawl and walk after a single treatment.

The drug has a reported list price of £1.79 million ($2.47 million) per dose, but NHS England said it had negotiated a "substantial confidential discount" on the price when it approved the drug for use in March.

However, guidelines set by the National Institute for Health and Care Excellence (NICE) said the drug should only be used for babies under six months who were not already being treated.

Edward, who was diagnosed at seven weeks, was receiving injections of another drug called Spinraza. Spinraza slows degeneration, but patients need regular injections in their spine for life.

NICE said decisions on whether to use Zolgensma on babies like Edward would be made on a case-by-case basis.

Willis, 29, previously told the BBC that they were fighting "a race against time" to get the drug for her son.

She and her partner, John Hall, had tried to raise £1.2 million ($1.66 million) last year to get the drug in the U.S. The U.S. Food and Drug Administration approved Zolgensma for use in 2019.

On a website for their son, the couple said they were "grateful" to have Spinraza available to them, but Zolgensma "will give Edward the best chance to live a fulfilling life."

"Our world has been turned upside down, in the most extreme way," they added. "Edward has brought purpose to our life and makes each day shine in the brightest way possible. He is the most beautiful, happy baby and it breaks our heart that he has this condition."

Now that her son has received Zolgensma, Willis said the family can look forward to celebrating his first birthday.

"I feel like the first year of his life has been robbed from us, but he is one next month and we can move on as a family, now that we know he is having the very best treatment," she said.

Willis and Sheffield Children's Hospital have both been contacted for additional comment.

Sheffield Children’s Hospital
An exterior view of Sheffield Children's Hospital on July 12, 2021 in Sheffield, England. Sheffield Children’s Hospital/Getty Images