KalVista Pharmaceuticals' Sebetralstat Maintains Orphan Designation in EU, Aims to Transform HAE Treatment

KalVista Pharmaceuticals' sebetralstat maintains orphan designation in the EU, paving the way for market exclusivity and improved treatment of hereditary angioedema. The drug's oral administration offers a novel approach to treating acute attacks, potentially enhancing patients' quality of life. Sebetralstat is one of only two HAE therapies maintaining orphan status in the EU.

KalVista Pharmaceuticals, Inc. (Nasdaq: KALV) has received a significant boost as the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) confirmed the maintenance of orphan designation for sebetralstat. This designation underscores the critical unmet need that sebetralstat addresses in the European Union (EU) and paves the way for market exclusivity and improved treatment of hereditary angioedema (HAE).

Sebetralstat, a novel oral plasma kallikrein inhibitor, has received a positive CHMP opinion for the treatment of acute attacks of hereditary angioedema (HAE). The European Commission (EC) is expected to make a final decision by early October. The decision to maintain orphan designation was based on a finding of comparable efficacy to injectable on-demand treatments while offering a major contribution to patient care by reducing the morbidity of HAE attacks.

Maintaining orphan designation provides several important regulatory and financial benefits, including 10 years of market exclusivity in the EU following approval. Notably, sebetralstat is now one of only two HAE medicines to have maintained orphan designation in the EU, highlighting its distinctive position within the HAE treatment landscape.

Ben Palleiko, CEO of KalVista, stated, "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. Orphan status provides benefits that will support the commercialization of sebetralstat in the EU, including 10 years of market exclusivity, as we continue our efforts to bring this important new treatment option to people living with HAE around the world."

Mauro Cancian, MD, PhD, Head of the Allergy Division at the University of Padua in Padova, Italy, added, "Maintaining orphan designation for sebetralstat is a recognition of the meaningful impact this therapy can have on people living with HAE. 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. 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."

Hereditary angioedema (HAE) is a rare genetic disease resulting in deficiency or dysfunction in the C1 esterase inhibitor (C1INH) protein and subsequent uncontrolled activation of the kallikrein-kinin system. People living with HAE experience painful and debilitating attacks of tissue swelling in various locations of the body that can be life-threatening depending on the area affected. Treatment guidelines recommend treating attacks as early as possible to prevent progression of swelling and shorten the time to attack resolution, and to consider treatment for all attacks, regardless of anatomic location or severity.

Sebetralstat is a novel plasma kallikrein inhibitor approved in the United States and United Kingdom under the brand name EKTERLY® for the treatment of HAE attacks in people 12 years of age and older. With ongoing studies exploring its use in children aged two to 11 and multiple regulatory applications under review in key global markets, sebetralstat has the potential to become the foundational therapy for HAE management worldwide.

KalVista Pharmaceuticals, Inc. is a global biopharmaceutical company dedicated to developing and delivering life-changing oral therapies for individuals affected by rare diseases with significant unmet needs. In the U.S., KalVista markets EKTERLY®, the first and only oral on-demand treatment for hereditary angioedema (HAE).

The maintenance of orphan designation for sebetralstat is a significant milestone for KalVista, as it positions the company to offer a novel, convenient treatment option for patients suffering from HAE. This development is likely to attract investor interest and could potentially drive stock performance, given the company's focus on delivering life-changing oral therapies for rare diseases.

References:
[1] https://www.stocktitan.net/news/KALV/kal-vista-pharmaceuticals-announces-maintenance-of-orphan-6yyci9w8yhs1.html
[2] https://www.biospace.com/press-releases/kalvista-pharmaceuticals-announces-maintenance-of-orphan-designation-for-sebetralstat-in-european-union