Following the conclusion of International Gaucher Disease Awareness Month, key opinion leaders (KOLs) interviewed by GlobalData believe pipeline gene therapies will significantly impact the Gaucher disease landscape. Gene therapy has the curative potential to provide patients with a healthy GBA1 copy, disrupting the Gaucher drug market. However, much groundwork remains to be covered, says GlobalData.
Gaucher disease, a rare inherited metabolic disorder caused by defects in the GBA1 gene, results in harmful glucocerebroside accumulation, which may affect the spleen, liver, and nervous system.
Sulayman Patel, Analyst at GlobalData, comments, “Our findings indicate that KOLs are aligned in recognising the significant potential for gene therapy to displace the current standard of care; enzyme replacement therapy (ERT) and substrate reduction therapy (SRT). Despite this, our KOLs have expressed both optimism and caution with respect to the early-stage nature of these therapies”.
Currently approved therapies, namely ERTs and SRTs, aim to resolve systemic manifestations, thereby leaving neurological symptoms unaddressed. This affects types 2 and 3 patients, who can experience seizures, cognitive impairment, and poor vision.
Addressing this shortfall, Prevail Therapeutics (a subsidiary of Eli Lilly) and Freeline Therapeutics are developing PR-001 and FLT-201, which are in Phase I/II development, respectively. In turn, these pipeline therapies aim to facilitate glucocerebroside breakdown, offering a targeted, long-lasting approach.
Patel comments, “KOLs have emphasised the promise of gene therapy, identifying these treatments as game-changers for the future of Gaucher disease management. While in preliminary stages, these offer a paradigm shift from existing treatments, which offer no resolution for neurological manifestations. Indeed, our experts have highlighted the need for more safety data. However, this will be explored as we await further developments.”
Gene therapy has previously demonstrated significant success in efficacy and safety for treating spinal muscular atrophy and hemophilia patients, which provides compelling evidence for the use of gene therapy in Gaucher disease patients.
Patel concludes, “These insights have underlined the potential gene therapy has to impact the Gaucher disease landscape. Providing a long-term solution that addresses the underlying root cause of the disease, gene therapy poses a strong threat to existing treatments, which currently fail to ameliorate neurological manifestations and create a high financial burden for payers.”