Sebetralstat gets to keep orphan drug status for HAE in Europe
Decision on therapy's approval in EU expected by early October
The oral on-demand treatment candidate sebetralstat for hereditary angioedema (HAE) is getting to keep its orphan drug status in the European Union, developer Kalvista Pharmaceuticals announced.
According to a press release from the company, the European Medicines Agency (EMA) has decided to maintain the therapy’s orphan drug designation, first granted in 2022. The EMA’s decision secures exclusive marketing rights in the EU for Kalvista for 10 years, along with other regulatory advantages, should the therapy be approved for HAE.
A final decision from the European Commission regarding sebetralstat’s approval for this indication is now expected by early October.
“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. “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.”
Sebetralstat is already approved in the U.K and the U.S., where it’s sold under the brand name Ekterly.
To capitalize on the market exclusivity incentive included in orphan drug status, companies must submit an application for maintenance of the designation at the time they seek the therapy’s regulatory approval. This is a critical step that enables companies to protect their investment and ensure continued access to essential therapies for patients with rare diseases.
In July, the EMA’s Committee for Medicinal Products for Human Use (CHMP) recommended approval of sebetralstat for treating acute attacks in HAE patients ages 12 and older. If granted, sebetralstat will become the first and only oral on-demand HAE therapy available in the EU.
If approved, sebetralstat now keeps 10 years’ marketing exclusivity
The decision to maintain sebetralstat’s orphan drug status in the EU — a milestone attained by one other HAE therapy thus far, its developer noted — was based on findings that support the comparable efficacy of sebetralstat to injectable on-demand treatments.
The medication was developed to reduce both the severity and duration of swelling attacks associated with HAE. It works by inhibiting the activity of kallikrein, an enzyme involved in the production of bradykinin, the signaling molecule responsible for triggering swelling in HAE. By blocking kallikrein, sebetralstat aims to decrease bradykinin levels, thereby effectively controlling and managing swelling attacks.
This treatment approach differs from other on-demand HAE therapies, which are administered via subcutaneous (under-the-skin) or intravenous (into-the-vein) injections. Sebetralstat’s oral mode of administration may reduce the treatment burden for HAE patients who need lifelong access to on-demand medications.
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 … in a way that fits their lifestyle.
“Maintaining orphan designation for sebetralstat is a recognition of the meaningful impact this therapy can have on people living with HAE,” said Mauro Cancian, MD, PhD, head of the allergy division at the University of Padua in Italy.
According to Cancian, “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.”
The approvals of what is now Ekterly in the U.S. and Great Britian followed positive data from the global Phase 3 KONFIDENT trial (NCT05259917). Those results showed that treatment with sebetralstat led to significantly faster symptom relief, reductions in attack severity, and faster symptom resolution relative to a placebo.
Additional data from the KONFIDENT-S (NCT05505916) extension study showed that sebetralstat quickly alleviated swelling attacks, including those classified as severe or very severe, as well as attacks that could potentially impact the airways.
About the Author
