Two large studies are underway to determine whether a drug will be the first to get the go-ahead in the US for treating a syndrome related to autism.
The studies will establish if zatolmilast remains in line to become the first drug to gain approval for treating Fragile X syndrome.
Dr Elizabeth Berry-Kravis, who is leading research on zatolmilast as a treatment for Fragile X syndrome
Neurologist Dr Elizabeth Berry-Kravis, of Rush University Medical Centre in Chicago, is leading the research for biotechnology firm Tetra Therapeutics.
Genetic cause of autism
Zatolmilast is thought to promote connections between neurons. These connections are impaired in people with Fragile X.
The condition is the most common known genetic cause of autism.
Many of its features, such as poor eye contact, repetitive behaviours and social and communication difficulties, are among the core symptoms of autism.
In an email, Berry-Kravis wrote of how she hoped the drug would help with “language-related cognition, and an improvement in ability to do functional activities”.
She added: “This is under the broad category of cognitive improvement. Behaviour may improve secondary to cognitive improvement.”
Path to prescription?
Berry-Kravis added that she expects to complete the trials next year. If they are successful, the drug may become available in 2026.
But this would depend on its path through the US Food and Drug Administration, she added.
The research involves trials focused on two separate groups.
One includes young and adolescent boys aged between nine and 18.
The other involves men between 18 and 45.
‘We will need to await the outcome’
Research suggests that Fragile X is around twice as common in boys as girls.
Across the two studies, an estimated 300 men and boys with the condition will participate in the research.
Following these trials, there will be a two-year extension study to look at the long-term safety and tolerability of the drug.
Pippa Sargent is chief executive of The Brain Charity, which helps people with all neurological conditions.
She said though medication is not suitable for everyone, the drug may offer hope for those looking for a pharmacological solution.
She added: “We will need to await the outcome of the studies to understand its potential impact further.”
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Published: 11 November 2024
