Health Canada has expanded the approval of Evkeeza (evinacumab) to include children as young as six months old with homozygous familial hypercholesterolemia (HoFH). This approval makes Evkeeza the first and only medication available for this age group in Canada for HoFH, a rare genetic disorder causing extremely high LDL cholesterol. The decision is based on model-based extrapolation analysis predicting similar or better LDL-C reduction in infants compared to older patients and supportive data from compassionate use cases.
This approval is crucial for infants with HoFH, as early intervention is essential to mitigate the severe cardiovascular risks associated with the disease. High LDL cholesterol levels, if untreated from infancy, can lead to premature heart disease and even death in childhood. The availability of Evkeeza offers a critical treatment option to manage these risks from a much earlier age, potentially significantly improving long-term outcomes and quality of life for these young patients.
The expanded approval is backed by data showing that infants aged six months to less than five years are projected to experience similar or greater LDL-C reductions at 24 weeks compared to older children and adults receiving the same dosage. Data from a compassionate use program involving five patients aged one to four years further supports these findings, demonstrating clinically meaningful LDL-C reductions consistent with older patients. The safety profile in younger infants is also expected to be similar to that observed in older patients, with no new safety concerns identified. Evkeeza is currently reimbursed and available in several countries, including Canada (through private and public drug plans), the UK, the US, and others, and is accessible through early access programs in several more.
This expanded indication represents a significant advancement in the treatment of HoFH. It allows healthcare providers to intervene earlier in the disease progression, offering hope for improved cardiovascular outcomes for the youngest and most vulnerable HoFH patients. This development underscores the ongoing progress in developing targeted therapies for rare diseases and highlights the potential for improving the lives of patients with previously limited treatment options.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
