A significant advancement for people living with hereditary angioedema (HAE) arrived with the recent FDA approval of Ekterly (sebetralstat), the first oral on-demand therapy designed to treat acute HAE attacks in adults and children aged 12 and older. This new option, developed by KalVista Pharmaceuticals, marks a promising shift away from injectable treatments, offering greater convenience and flexibility for those managing this rare, potentially life-threatening condition.
Understanding HAE and the Need for New Therapies
Hereditary angioedema is a rare genetic disorder characterized by unpredictable and recurrent episodes of severe swelling, often affecting the skin, gastrointestinal tract, and airway. These attacks can be painful, disabling, and, in cases where the airway is involved, life-threatening. Until now, most approved on-demand treatments required intravenous or subcutaneous injection—a burden that complicates timely self-treatment and can increase anxiety for patients.
Ekterly: A Game-Changer in HAE Management
Ekterly’s approval is based on clinical trial results showing its efficacy in stopping acute HAE attacks when administered soon after symptoms begin. The oral medication acts by inhibiting plasma kallikrein, a key enzyme involved in the cascade that leads to swelling during attacks. In clinical testing, Ekterly provided rapid symptom relief and was well tolerated, with the most common side effects being mild and including headache and nausea.
The oral route is a major breakthrough for HAE patients. Instead of needing to mix, inject, or infuse medication—sometimes under stressful circumstances—patients can take a tablet as soon as they sense an attack starting. This not only increases the likelihood of prompt treatment but also offers greater independence and quality of life.
Implications for Patients and the HAE Community
The FDA’s approval of Ekterly is likely to have a substantial impact on the HAE community. For teenagers and adults who may be away from home when an attack occurs, the portability and ease of an oral tablet are especially valuable. The move to oral therapy also addresses needle phobia and reduces barriers to early intervention, which is critical for preventing attacks from progressing.
KalVista Pharmaceuticals has stated that it plans to make Ekterly available in the U.S. market promptly, working closely with healthcare providers and patient organizations to ensure access and education about the new therapy.
A Growing Arsenal Against HAE
Ekterly adds to the expanding options for HAE management, complementing existing preventative and emergency treatments. The approval underscores the importance of innovation in rare disease drug development and the ongoing commitment to improving patient experiences.
