Health Canada recent approvals in rare disease

by Brigitte Leonard, Ph.D

In 2024, Health Canada approved 9 products targeting several rare disorders. This approval rate demonstrate the combining efforts of government policies, research capabilities and pharmaceutical companies deployed to address these conditions.

New hope for people with sickle cell disease or beta-thalassemia

Health Canada approved Casgevry for commercialization in September 2024 to treat two rare genetic blood disorders: sickle cell disease and beta-thalassemia Casgevy (exa-cel: exagamglogene autotemcel) is a gene therapy developed by Vertex Pharmaceuticals and CRISPR Therapeutics. Casgevy reduce significantly pain crises and hospitalizations in sickle cell disease by reaching normal or near-normal adequate hemoglobin levels.

Good new for anemic patients diagnosed with myelofibrosis

Myelofibrosis is a rare cancer characterized by an overproduction of abnormal blood cells and inflammation markers called cytokines. Treatment of myelofibrosis has improved significantly since the 1st JAK inhibitor, Jakavi, was approved in 2011. However, anemia, often present at diagnosis, represents a limiting factor to treat patients with a typical JAK inhibitor. Ojjaara is as efficient as other JAK inhibitors. However, unlike other therapies, it can improve anemia by facilitating the production of blood cells. SIMPLIFY trials have demonstrated that patients can reach healthier blood levels and become transfusionindependent. Some patients could even delay complex procedures such as transplantation due to this treatment.

What about a rare pulmonary condition called arterial hypertension ?

Health Canada approved WINREVAIR in August 2024, respectively. Merck demonstrated the safety and efficacy of WINREVAIR in pulmonary arterial hypertension (PAH) with the STELLAR trial (NCT04576988). PAH is a rare, progressive and life-threatening disease in which blood vessels in the lungs thicken and narrow, causing significant strain on the heart [?]. WINREVAIR, added to background therapy, increases physical capability and reduces the risk of death and clinical events related to PAH with an acceptable safety profile.