New Treatments on the Horizon
For new treatments to become accessible in Canada, they first need to go through a clinical trial in Canada. If you are interested in participating in a clinical trial, please visit our Clinical Trials page to learn about the current opportunities in Canada.
Adverum – ADVM-053: A gene therapy candidate being investigated as a potential single‑administration treatment to provide sustained levels of the C1 esterase inhibitor protein. Learn more here.
Alnylam Pharmaceuticals – AKN-F12: A RNA interference (RNAi) drug to knockdown factor XII. Learn more here.
Arrowhead – ARC-F12: An RNAi therapeutic inhibiting the production of Factor XII. Learn more here.
Attune Pharmaceuticals – ATN-249: An orally-administered plasma kallikrein inhibitor. Learn more here.
BioCryst – BCX7353: A once-daily oral treatment (capsule or liquid) inhibiting plasma kallikrein. Learn more here and find information on the clinical trial in Canada.
BioMarin Pharmaceutical Inc. – BMN 331: An investigational gene therapy intended to provide C1 esterase inhibitor to treat hereditary angioedema.
CSL Behring – CSL312: A humanised anti-factor XIIa monoclonal antibody for subcutaneous treatment. Learn more on the ClinicalTrials.gov website.
Ionis – IONIS-PKK-LRx: A Generation 2+ ligand-conjugated antisense drug designed to reduce prekallikrein production. Learn more on the Ionis website.
Pharvaris Netherlands B.V.: Dose-ranging Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema (RAPIDe-1). Learn more on the trial’s website and learn more on the ClinicalTrials.gov website.
Takeda Pharmaceuticals – TAK-743: Lanadelumab (Takhzyro), a fully human monoclonal antibody inhibitor of plasma kallikrein for the prevention of Hereditary Angioedema (HAE) Attacks in pediatric subjects 2-12 years of age. Learn more on Takeda’s website and learn more on the ClinicalTrials.gov website.