Parents fight for rare achondroplasia drug access
SA family fights for five-year-old Frederick’s access to rare growth therapy, highlighting treatment barriers and costs.
POLOKWANE – At five years old, Frederick Hall should have nothing to worry about beyond playtime and laughter.
Most days, he does. On others, he is at ease in a hospital bed, with a drip in his arm and doctors examining him.
Frederick was born healthy and strong, but before his birth he was diagnosed with achondroplasia, a form of dwarfism.
His mom, Chantelle, explains that the condition is caused by a mutation in the FGFR3 gene, which impairs bone growth.
“Frederick’s early years were filled with medical complexity, from specialist care and long-distance travel to major surgery before his first birthday,” she says.
“Alongside learning how to be a parent, I had to learn about medicine, genetics and systems that were never designed with children like mine in mind.”
As Frederick grows older, the condition increasingly affects his body.
Chantelle says Voxzogo, developed by BioMarin Pharmaceutical, is the first FDA-approved treatment for achondroplasia.
The medication works by counteracting the overactive FGFR3 gene, stimulating natural growth pathways to help children gain height and reduce complications associated with short stature.
When the drug became available internationally, Chantelle felt hope.
“But in South Africa, hope quickly met reality. The cost is prohibitive, and access pathways are unclear,” she says.
Because the treatment is not yet available locally, families must import it at enormous expense.
A year’s treatment costs more than R6m per child, which is out of reach for most.
Early intervention is critical, as benefits decrease with delayed access. Treatment continues until growth plates close at around age 18.
Determined not to wait, Chantelle registered Frederick’s Growth Fund to raise money, advocate for access and campaign for compassionate pricing.
The immediate goal is to start two children on treatment for six months while longer-term solutions are pursued.
“This is not only about my child, but about every child who deserves the same chance,” she says.
To support the campaign, visit Frederick’s Growth Fund SA on Facebook.
