Health Canada recent approvals (con’t)

Patients treated with standard treatment can continue to experience severe anemia and need frequent blood transfusions. As demonstrated in the ALPHA trial, adding Voydeya to standard therapy allows patients to improve their anemia, reduce their transfusion need and reduce their fatigue compared to placebowithanacceptablesafetyprofile.

In July 2024, HC approved ZILBRYSQ for treating generalized myasthenia gravis (gMG)[PM]. The FDA (USA) and EMA (Europe) approved the product in October and December 2023, respectively [?]. gMG is a rare autoimmune disorder. The immune system attacks the connection between nerves and muscles, leading to muscle weakness. This condition occurs later in life between 40 to 60 years old. This progressive disease is debilitating and life-threatening. The initial symptoms can be eyelid drooping and difficulty keeping the mouth closed. Over time, more severe symptoms appear, like difficulty swallowing, leg paralysis and difficulty breathing. UCB Pharma sponsored the development of ZILBRYSQ, which interferes with the immune system to prevent damage to these connections. In the RAISE study, patients improved functionality over standard care with an acceptable safety profile. The improvement is significant at 12 weeks and continues to improve over time.

In June 2024, HC approved WAINUA to treat hereditary transthyretinmediated amyloidosis (hATTR) [PM]. The FDA (USA) approved the product in December 2023 [Press]. hATTR is a rare genetic debilitating disease that leads to peripheral nerve damage. A mutation in the gene transthyretin (TTR) causes the production of a dysfunctional TTR protein. These TTR proteins form aggregations and cause nerve damage. Within five years of diagnosis, patients present with motor disabilities. Without treatment, the genetic disorder is generally fatal within a decade [Cortese]. AstraZeneca and Ionis Pharma sponsored the development of WAINUA, which reduces the production of TTR proteins. In the NEURO-TTRansform study, patients treated with Wainua demonstrated consistent and sustained significant clinical benefits with an acceptable safety profile. The three significant clinical benefits are reduced serum TTR concentration, reduced nerve impairment and improved quality of life.

In June 2024, HC approved EBGLYSS to treat eczema if standard prescription skin products don’t work [PM]. This product has been approved in the USA (FDA) and Europe (EMA) since September 2023 [Press]. Several patients still struggle to control their eczema with currently available therapies. Many experience poor long-term disease control, and severe itch can significantly impact their daily lives [Chiesa]. The program included three clinical studies and 1062 patients: ADvocate 1, ADvocate 2, and Adhere. Patients treated with Ebglyss experienced a significant improvement in their skin condition compared to placebo or standard treatments, and the safety profile was acceptable.

In May 2024, HC approved APRETUDE to reduce the risk of sexually acquired HIV-1 infection. The company that sponsored the submission was ViiV Healthcare ULC. Pre-exposure prophylaxis (PrEP) to reduce the risk of sexually acquired HIV is a strategy that has gained popularity in the last ten years to end the HIV epidemic. Products are already on the market, but the high level of adherence needed is not always respected. It is hoped that the availability of a long-acting injectable PrEP option will increase PrEP uptake and adherence in targeted groups. The trial showed that participants who took Apretude had 69% less risk of getting infected with HIV when compared to participants who took the current standard of care. Apretude has been on the USA market since December 2021 and has shown an acceptable safety profile.

In July 2024, HC approved TIBSOVO to treat two types of cancers positive for a specific mutation called IDH1: acute myeloid leukemia (AML) and cholangiocarcinoma. Patients with IDH1-positive AML have an inferior outcome when treated with current therapy. Servier sponsored the submission to Health Canada. During the clinical trial in AML, adding TIBSOVO to standard treatment increased the odds of responding by almost six times and reduced the odds of progressing by 67%. Similar results were observed in the cholangiocarcinoma study, where the time without progression doubled versus the placebo group, and the odds of progress were reduced by 63%.