CSL's Andembry Gains FDA Approval, Set to Compete in HAE Market

CSL has secured FDA approval for Andembry, its new hereditary angioedema (HAE) preventative treatment, positioning the company to challenge established competitors in the U.S. market. This development marks a significant milestone in HAE treatment, offering patients a novel option with a unique mechanism of action.

Andembry: A New Contender in HAE Prevention

Andembry, a monoclonal antibody developed entirely by CSL, has received FDA approval for the prevention of HAE attacks in patients aged 12 and older. As the first prophylactic treatment that inhibits factor XIIa, Andembry represents a novel approach to managing this rare genetic condition.

HAE, affecting approximately 1 in 50,000 individuals, causes sudden and unpredictable episodes of severe swelling in various body parts, including the limbs, face, abdomen, and larynx. The disorder can be life-threatening when it affects the airways.

Dr. Tim Craig, a professor of medicine at Penn State University, highlighted the significance of this approval, stating, "We now have a new option to manage this condition through a new target, as it allows us for the first time to inhibit the top of the HAE cascade by targeting factor XIIa."

Clinical Efficacy and Market Positioning

In a phase 3 trial published in The Lancet in April 2023, Andembry demonstrated significant efficacy in reducing HAE attacks. The study revealed that 62% of patients receiving Andembry remained attack-free over a six-month period.

CSL's new treatment enters a competitive market currently dominated by Takeda's Takhzyro and BioCryst Pharmaceuticals' Orladeyo. Takhzyro, a blockbuster drug, generated sales of $1.2 billion last year, while Orladeyo reported sales of $438 million.

Andembry's monthly self-administration via a prefilled pen may offer a convenience advantage over Takhzyro's bi-weekly dosing schedule. However, cross-trial comparisons should be interpreted cautiously.

Market Impact and Availability

With Andembry's approval, CSL aims to recapture market share lost to newer therapies. The company's existing HAE treatments, Berinert and Haegarda, have faced competition from Takhzyro and Orladeyo in recent years.

Dr. Bill Mezzanotte, CSL's R&D chief, emphasized Andembry's potential impact, stating, "Andembry offers people living with this life-threatening condition long-term control over their disease along with a convenient administration method."

CSL has announced that Andembry will be available in the U.S. market by the end of this month, setting the stage for an intensified competition in the HAE treatment landscape.