HAE treatment Navenibart named EC orphan medicinal product
Move follows U.S. FDA orphan drug designation
The European Commission has granted orphan medicinal product designation (OMPD) to navenibart, an experimental long-acting therapy for hereditary angioedema (HAE).
The designation is aimed at providing incentives for the development of therapies that are meant to diagnose, prevent, or treat rare diseases, or those affecting fewer than 10,000 people in the EU. Developers may receive European Medicines Agency assistance in optimizing the therapy’s clinical development, as well as regulatory and financial incentives.
The EC’s decision on Astria Therapeutics’ navenibart comes on the heels of the U.S. Food and Drug Administration’s (FDA) granting of similar orphan drug status to the treatment.
“We are very pleased to receive Orphan Medicinal Product Designation for navenibart in Europe,” Jill C. Milne, PhD, CEO of Astria, said in a company press release. “We believe that OMPD status, in combination with the FDA’s designation of navenibart as an orphan drug in the United States, reinforces both the global unmet need for people with HAE and the potential for navenibart to change the way that people live with HAE.”
HAE is a chronic genetic disease marked by recurrent swelling attacks affecting the deeper layers of the skin or mucus membranes. It’s driven by the overproduction of bradykinin, a signaling molecule that regulates blood pressure and inflammation by promoting blood vessel widening.
HAE treatment for swelling attacks
Navenibart, formerly known as STAR-0215, is designed to block the activity of kallikrein, the enzyme that mediates bradykinin’s production, preventing swelling attacks.
The investigational therapy is similar to Takhzyro (lanadelumab), an approved HAE treatment that is given every other week or once monthly via a subcutaneous, or under-the-skin, injection. Navenibart is administered in the same way, but at a much lower frequency — one subcutaneous injection every three to six months — to prevent swelling attacks.
ALPHA-STAR (NCT05695248) is an ongoing Phase 1b/2 clinical trial that’s evaluating three different dosing regimens of navenibart in adults with HAE type 1 or 2.
After an eight-week period to gauge the rate of pre-treatment swelling attacks, some participants received a 450 mg injection of navenibart, while others received one 600 mg injection followed by a second 300 mg dose three months later. A third group of patients received two 600 mg injections one month apart.
Interim data released in March showed the rate of swelling attacks dropped by 90% to 96% when navenibart was dosed once or twice over the course of six months. No serious treatment-related side effects were reported.
ALPHA-SOLAR (NCT06007677) is an open-label extension study that aims to collect long-term safety and efficacy data on navenibart in participants who completed the ALPHA-STAR study. Those who choose to join the extension will receive navenibart injections every three months (300 mg) or every six months (600 mg).
A Phase 3 trial that will further evaluate the safety and efficacy of navenibart is planned for early 2025, according to Astria.
“We look forward to the expected progression of navenibart into a Phase 3 trial in the first quarter of 2025 and continuing our efforts to bring a new treatment option to people living with HAE around the world,” Milne said.
The FDA has granted navenibart fast-track designation, a status designed to speed the development and regulatory review of therapies that have the potential to address unmet needs.
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