First and only RNA-targeted prophylactic treatment for HAE approved in the US

A new medicine for HAE, called Dawnzera (donidalorsen) has been approved by the regulatory authorities in the USA, and can begin to be prescribed to people with HAE in the coming days.

The US Food and Drug Administration (FDA) approved donidalorsen to prevent attacks of HAE in adults and pediatric patients 12 years of age and older. Donidalorsen reduces the production of prekallikrein by turning off certain genetic messages in the body. This stops the body from overproducing bradykinin, preventing HAE attacks.

Donidalorsen 80mg is self-administered via subcutaneous autoinjector once every four (Q4W) or eight weeks (Q8W).

Brett P Monia PhD, Chief Executive Officer, Ionis, said: “Dawnzera represents a significant advance for people living with HAE who need improved treatment options. With strong and durable efficacy, convenient administration and the longest dosing option available, we believe Dawnzera will be the prophylactic treatment of choice for many people living with HAE. At Ionis, we are dedicated to turning groundbreaking science into life-changing medicines. To the patients, families, advocacy partners and investigators who helped make this moment a reality, we express our deepest gratitude.”

Anthony J Castaldo, CEO & Chairman of the Board, US Hereditary Angioedema Association (HAEA) and Hereditary Angioedema International (HAEi), said: “As the first FDA-approved RNA-targeted therapy for HAE, Dawnzera represents a welcome advance in therapeutic options for preventing attacks. Today’s approval gives people living with HAE and their physicians another important choice for aligning treatment with individual needs.”

Marc Riedl MD MS, Clinical Director, US HAEA Angioedema Center; University of California, San Diego; OASIS-HAE and OASISplus trial Investigator, said: “People living with HAE manage this condition for all their lives, and many continue to face unpredictable, painful and dangerous breakthrough attacks even with current treatments. Durable efficacy is essential in maintaining long-term disease control. Dawnzera is positioned to help meet patient needs, providing substantial and sustained reduction of HAE attacks, continued improvement over time and reduced burden of treatment.”

(Source: Ionis)