A Belgian toddler whose family successfully crowdfunded millions for the world’s most expensive drug has been administered the shot which could potentially rid her from a life-threatening condition.
“Last Wednesday was one of the most important days of our lives. Pia received the gene therapy,” Ellen de Meyer, the toddler’s mother, said in a press conference. “We are now 48 hours later and she has not had any side effects so far.”
The family of 10-month-old Pia, who suffers from spinal muscular atrophy, made the headlines in Belgium and abroad after successfully crowdfunding the €1.9 million needed to afford Zolgensma, which is the most expensive drug in the world.
Spinal muscular atrophy is a rare genetic disease which progressively leads to muscular failure and sees most children diagnosed with it die in early infancy.
The toddler’s mother said their achievement would have “never happened without the help of the Belgian population,” adding that her daughter’s story had been “one of hope,” according to HLN.
The crowdfunding campaign prompted a massive outpour of solidarity from across the country, with the family reaching their million-euro goal in only two days.
“The drug must now do its work,” De Meyer said, adding that the first signs of success would be that Pia manages to sit down on her own. “Then she could sit in a wheelchair and lead a beautiful and independent life.”
Upon release, Zolgensma was hailed as a breakthrough in the treatment of the disease, since it is the only drug capable of halting the muscular deterioration and allowing a sick child to develop normally.
“We now want to be especially careful and give the drug time,” De Meyer said, adding that they had been in touch with a German family who had seen “spectacular progress” after giving the shot to their child.
The family’s crowdfunding achievement to afford Zolgensma, manufactured by pharmaceutical giant Novartis, sparked a conversation about the staggering costs of treatment for rare diseases.
Their campaign was mirrored by other families in Belgium, equally unable to face the often elevated costs of treatment for their children.
“We are well aware that there are many more children who are in a similar situation,” De Meyer said, adding that a conversation about pricing in the pharmaceutical industry was long overdue.
While industry actors have said that Zolgensma’s price was justified by its results, which could “dramatically transform” the lives of patients and their families, the plight of Pia’s family has prompted calls for structural solutions to ensure wider access to treatments for rare diseases.