Navenibart Receives FDA Orphan Drug Designation to Treat Hereditary Angioedema

The FDA has granted navenibart (STAR-0215; Astria Therapeutics) orphan drug designation to treat hereditary angioedema (HAE)—an autosomal dominant disease that occurs due to a lack of C1-inhibitor protein or dysfunctional C1-inhibitor protein. As a monoclonal antibody inhibitor of plasma kallikrein, navenibart could provide extensive attack prevention for HAE, according to study authors.^1,2

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“Receiving orphan drug designation for navenibart is an important affirmation of our belief that there is a significant unmet need for people living with HAE,” Jill C Milne, PhD, CEO of Astria Therapeutics, said in a news release. “We believe navenibart has the potential to be the market-leading HAE treatment because of its trusted mechanism and modality, efficacy observed to date, and low treatment burden with infrequent dosing, and think that navenibart could change the way that people live with their HAE.”¹

HAE is known to cause painful and abrupt swelling in parts of the body, defined as attacks that can occur randomly lasting for a few days. While the swelling can occur in any part of the body, it most commonly arises on the abdomen, face, throat, genital, hands, and feet, presenting difficulties to complete daily tasks. Study authors noted that HAE attacks are often triggered by emotional stress, minor injury, surgery, or dental procedure, hereditary-angioedema, hormonal influences, or other physical activities. An individual could experience a non-raised, non-itchy rash before the swelling occurs, indicating symptoms of an attack. If not treated the HAE attacks could last for a 48 to 72-hour period.^2,3

Individuals with HAE Type 1 and 2 typically have low functioning C1 esterase inhibitors (C1-INH), along with low levels of plasma, presented in HAE Type 1 or normal/elevated levels in HAE Type 2. This elicits overactivation from a plasma kallikrein protein that leads to overproduction of bradykinin that forms the attacks, according to study authors.³

The study authors noted that navenibart was developed to aid treatment of HAE to provide swift and sustained HAE attack prevention through administration every 3 to 6 months. The role of navenibart is to block plasma kallikrein even in the absence of the C1-inhibitor, reducing bradykinin production and preventing pain.^1,4

The orphan drug designation was granted based on initial results from the phase 1b/2 ALPHA-STAR clinical trial that displayed a favorable safety and tolerability profile, reducing monthly attack rates by 90% to 96% when treated with a dose once or twice over 6 months. The researchers noted that following the positive results from the phase 1b/2 study, they plan to conduct a phase 3 trial with results expected by the end of 2026.^1,4

The company noted that they are determined to develop a treatment that will allow individuals that experience HAE attacks to focus their time and energy on what matters most to them, emphasizing their commitment to further treatment options.⁴

REFERENCES

1. Astria Therapeutics Receives FDA Orphan Drug Designation for Navenibart (STAR-0215) for the Treatment of Hereditary Angioedema. Business Wire. News release. September 30, 2024. Accessed October 1, 2024. https://www.businesswire.com/news/home/20240930717552/en

2. Hereditary Angioedema. StatPearls. News release. May 1, 2023. Accessed October 1, 2024. https://www.ncbi.nlm.nih.gov/books/NBK482266/

3. What is hereditary angioedema? PienseAEH. News release. Accessed October 1, 2024. https://www.pienseaeh.com.co/en-colom-dsbl/patient/what-is-hae/signs-symptoms-and-causes#:~:text=signs%20and%20symptoms%20of%20hereditary,di%EF%AC%83cult%20to%20perform%20everyday%20tasks

4. Navenibart (STAR-0215). Astria Therapeutics. News release. Accessed October 1, 2024. https://astriatx.com/our-science/star-0215