uniQure recently announced that its novel gene therapy, AMT-130 (ifezuntirgene inilparvovec), demonstrated statistically significant disease slowing in a Huntington’s disease (HD) pivotal trial. The results indicate that AMT-130 holds blockbuster potential in the disease space, according to GlobalData, leading data and analytics company.

Pippa Salter, Managing Neurology Analyst at GlobalData, comments: “In a market where the only available therapies are for the symptomatic management of chorea, the efficacy results of AMT-130 are unprecedented. Key opinion leaders (KOLs) previously interviewed by GlobalData consistently highlighted the significant unmet need in the HD market for a therapy that can modify the disease, and slow or prevent progression, with AMT-103 poised to become the first treatment option for HD that meets this need giving it blockbuster potential.”

However, despite the significant results for AMT-130, barriers remain for uptake of the gene therapy, if approved.

Salter continues: “Firstly, as a gene therapy, AMT-130 is expected to be extremely expensive, which may pose reimbursement and access challenges for patients. Additionally, AMT-130 requires invasive, complex MRI-guided, convection-enhanced stereotactic neurosurgical delivery, which not all patients may be eligible for or want to undergo. This cumbersome route of administration will also pose significant operational burden for AMT-130.”

Furthermore, once administered the gene therapy cannot be removed, although it has been well tolerated, with no serious adverse events observed since the trial was temporarily paused in 2022 and additional monitoring procedures were put in place following the neurosurgery.

Some KOLs remained cautious about the longer-term safety of a gene therapy, given that the pivotal trials have only enrolled a small number of patients with only 12 patients in the high-dose AMT-130 group having reached the 36 month point thus far.

Salter concludes: “As AMT-103 will likely not be available for all patients with HD, there remains room for other developers pursing alternative potentially disease-modifying options for the disease. Many KOLs noted that if an oral drug could demonstrate significant efficacy, it would face the fewest access barriers for patients. Overall, GlobalData expects significant growth in the HD market over the next 10 years, with uniQure’s AMT-130 kickstarting the paradigm shift in how the disease can be treated.”

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