The drug approval for "Spheragen Syrup," which had been developed as a fundamental treatment for autism spectrum disorder, has fallen through. As the field is one where global big pharma companies have also repeatedly failed, attention is turning to the results of domestic biotechs continuing related research.
According to the pharmaceutical and biotech industry on the 2nd, the Central Pharmaceutical Affairs Advisory Committee of the Ministry of Food and Drug Safety last month determined that it was difficult to acknowledge the efficacy of Astrogen's autism treatment candidate "AST-001 (Spheragen Syrup)." The company had initially aimed to develop a fundamental treatment for autistic children aged 2 to 7, but failed to prove efficacy in clinical trials.
It subsequently narrowed the indication to improving motor skills in autistic children aged 2 to 5 and applied for drug approval. Regarding this, the Central Pharmaceutical Affairs Advisory Committee explained, "In children aged 2 to 5, motor skills can develop in response to external stimuli even without any particular treatment," adding, "For approval, only pre-planned statistical analysis results are recognized as confirmatory evidence, but it appears meaning was found through post-hoc analysis." It further added, "Additional clinical trials are needed to prove efficacy."
Globally, no drug that treats autism spectrum disorder itself has been developed. This is because the mechanism of onset is highly complex, with gene mutations, neurodevelopmental pathways, and environmental factors acting in different combinations for each patient. Currently, only drugs that control accompanying symptoms such as attention deficit hyperactivity disorder (ADHD) or problematic behaviors are being used. Last year, U.S. President Donald Trump mentioned leucovorin as an autism treatment, but after negative results emerged from a large-scale clinical trial in January this year, the U.S. Food and Drug Administration (FDA) only added approval for the indication of cerebral folate deficiency caused by FOLR1 mutation, an ultra-rare genetic disorder.
Due to such difficulties, global big pharma and biotechs have also repeatedly halted development of fundamental treatments for autism spectrum disorder. Switzerland's Roche discontinued development in 2020 after the Phase 3 trial failure of its candidate balovaptan, and the following year France's second-largest pharmaceutical company Servier withdrew development of bumetanide after it failed to prove efficacy against placebo in a Phase 3 trial. Switzerland's Novartis also ultimately failed in developing mavoglurant, a treatment for fragile X syndrome, which is highly associated with autism.
Nevertheless, the challenges of domestic biotechs continue. Nature Cell obtained FDA approval for a Phase 1 clinical trial of "Astrostem-A," an autologous adipose-derived mesenchymal stem cell treatment for adult autism patients, and has been conducting the trial since April this year. Neuroventi also completed a domestic Phase 2 clinical trial of "NV01-A02" in February this year and is collecting data. SK Biopharm, known for its epilepsy treatment cenobamate, also launched joint research and development with Interon last November to discover a treatment candidate for autism spectrum disorder utilizing neuroimmune system regulation.
An industry official said, "Autism spectrum disorder is one of the most difficult diseases to prove consistent efficacy for in clinical trials because of the great heterogeneity of the patient group," but forecast, "As it is a representative area of unmet medical need with no fundamental treatment, if a company emerges that proves efficacy, it could change the market landscape."
