“Advancements in Targeted and Prophylactic Therapies”
- A significant and accelerating trend in the global hereditary angioedema (HAE) therapeutic market is the shift toward targeted and long-term prophylactic treatments that significantly reduce the frequency and severity of HAE attacks, improving patient outcomes and quality of life
- For instance, therapies such as Takeda’s Takhzyro (lanadelumab), a subcutaneous monoclonal antibody, offer sustained prevention of HAE attacks with biweekly or monthly dosing, while BioCryst’s Orladeyo (berotralstat) provides the convenience of once-daily oral prophylaxis, appealing to patients seeking needle-free alternatives
- These advanced treatments target specific pathways such as plasma kallikrein inhibition or C1-esterase deficiency, offering a precision medicine approach that enhances treatment efficacy and minimizes side effects. The development of gene therapies is also gaining traction, with ongoing clinical trials exploring potential long-term or curative options for HAE patients
- Moreover, biopharmaceutical companies are actively investing in pipeline expansion and innovative delivery mechanisms such as subcutaneous self-administration and oral formulations to further simplify disease management
- This trend is fundamentally reshaping expectations for HAE care, with patients and providers increasingly prioritizing therapies that offer convenience, long-term protection, and improved safety. As a result, companies such as CSL Behring and Pharvaris are advancing next-generation HAE treatments that align with these evolving demands
- The growing preference for targeted, low-burden prophylactic therapies is rapidly transforming the therapeutic landscape, driving higher adoption rates and shaping the future direction of the hereditary angioedema market globally



