CAMBRIDGE, Mass., and SALISBURY, England — The European Medicines Agency’s Committee for Orphan Medicinal Products has confirmed the maintenance of orphan designation for sebetralstat, KalVista Pharmaceuticals’ experimental treatment for hereditary angioedema (HAE).
Sebetralstat, an oral plasma kallikrein inhibitor, recently received a positive opinion from the EMA’s Committee for Medicinal Products for Human Use for treating acute HAE attacks. A final decision from the European Commission is expected by early October. If approved, the orphan designation will grant the drug 10 years of market exclusivity in the European Union.
“We are pleased with the decision to maintain EU orphan designation for sebetralstat, which is an uncommon achievement that reflects the significant benefits sebetralstat offers,” said Ben Palleiko, CEO of KalVista. He noted that the status provides regulatory and financial advantages that will support commercialization efforts.
The EMA’s decision was based on findings that sebetralstat offers comparable efficacy to injectable on-demand treatments while providing a “major contribution to patient care” by reducing the impact of HAE attacks. KalVista said the drug is now one of only two HAE therapies to retain orphan designation in the EU.
Mauro Cancian, M.D., Ph.D., head of the Allergy Division at the University of Padua in Italy, said the oral formulation could address challenges faced by patients using injectable therapies. “With currently available injectable therapies, many patients delay or avoid treatment due to the hassle and fear of injection-site reactions, often suffering through attacks longer than they need to,” he said. “Oral sebetralstat has the potential to change that, giving patients the ability to treat attacks early, with confidence, in a way that fits their lifestyle.”
