FDA Accepts and Boosts sBLA for Takeda’s HAE Treatment for Pediatric Patients

The United States Food and Drug Administration (FDA) accepted a supplemental Biologics License Application (sBLA) for the potential expanded use of TAKHZYRO® (lanadelumab-flyo). Currently, TAKHZYRO has approval for hereditary angioedema (HAE) attack prevention in people 12 years of age and older.

The sBLA intends the expanded use of TAKHZYRO to pediatric patients 2 to <12 years of age. The basis for the expansion is the SPRING Study. If approved, TAKHZYRO would be the first prophylaxis treatment for HAE patients younger than six.

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Preventing a Rare Disease’s Attacks

HAE is a rare inherited disorder wherein patients experience repeated instances of fluids accumulating outside of the blood vessels. This causes the blockage of normal blood flow, resulting in the swelling of the hands, feet, limbs, face, intestinal tract, or airway. Experts estimate that 50,000 – 150,000 people have this disease worldwide.

Treatments for the rare disease include BioCryst’s Orladeyo (berotralstat), an inhibitor of plasma kallikrein’s proteolytic activity. Plasma kallikrein plays a role in the body’s inflammatory response, specifically to do with the protein Factor XII.

TAKHZYRO is similarly a plasma kallikrein inhibitor. Initially gaining approval from the FDA in February of this year, Takeda’s inhibitor has since gained approvals in thirty other countries. However, Takeda returned to the FDA seeking extended approval for TAKHZYRO in October.

Expanding HAE Attack Prevention

Takeda submitted an sBLA to the FDA seeking the expansion of TAKHZYRO for the treatment of children younger than twelve. The basis of the sBLA was the SPRING Study, a Phase 3 clinical trial examining the effect of the inhibitor on HAE patients aged two to twelve.

The study demonstrated a 94.8% reduction in HAE attacks over a 52-week period of treatment. In addition, the efficacy and safety profile remained consistent with adult and adolescent groups in earlier studies. 

On the submission, Cheryl Schwartz, Senior Vice President, U.S. Rare Disease Business Unit at Takeda, said, “If TAKHZYRO is approved for this expanded use, children as young as two years old would have a treatment option which has shown proven effective prevention of HAE attacks in those over twelve years of age.”

The FDA accepted the sBLA, going so far as to grant it priority review. Takeda expects a decision within the first half of 2023. If approved, TAKHZYRO will be the first approved prophylaxis treatment for pediatric patients. 

Author

Max Heirich