FDA grants priority review to lanadelumab-flyo for pediatric hereditary angioedema attacks

The FDA has granted priority review to a supplemental biologics license application for the potential expanded use of lanadelumab-flyo to prevent hereditary angioedema attacks in patients aged 2 to younger than 12 years.

There is no currently approved prophylaxis treatment for children aged younger than 6 years with hereditary angioedema (HAE), which would make lanadelumab-flyo (Takhzyro, Takeda) the first treatment available for this population if approved, according to a Takeda press release.

“Unpredictable, debilitating and potentially life-threatening HAE swelling attacks can cause a physical and emotional toll on those living with this rare disorder,” Cheryl Schwartz, MBA, senior vice president of the U.S. rare disease business unit at Takeda, said in the press release.

Cheryl Schwartz

“If Takhzyro is approved for this expanded use, children as young as 2 years old would have a treatment option which has shown proven effective prevention of HAE attacks in those over 12 years of age,” Schwartz said.

Treatment with lanadelumab-flyo involves subcutaneous injection of 300 mg/2 mL or 150 mg/1 mL doses every 2 or 4 weeks, based on the age of the patient. With training by a health care professional, doses may be administered by the patient or by a caregiver.

Takeda based its application on the results of its SPRING study, where the drug reduced the rate of HAE attacks among children aged 2 to younger than 12 years by a mean of 94.8% compared with baseline. Further, 76.2% of patients remained attack-free during the 52-week treatment period.

No deaths or serious treatment-emergent adverse events were reported.

Takeda expects the FDA to make a decision about the application in the first half of 2023.

Reference:

• Takeda’s Takhzyro® (lanadelumab) demonstrated positive results in the prevention of HAE attacks in first and only open-label phase 3 trial in children ages 2 to <12 years. https://www.takeda.com/newsroom/newsreleases/2022/phase-3-spring-study-data-presented-at-eaaci/. Published June 30, 2022. Accessed Oct. 5, 2022.