Strensiq approved to treat rare genetic disorder HPP

Strensiq (asfotase alfa) recently became the first U.S. Food and Drug Administration (FDA)-approved treatment for perinatal, infantile and juvenile-onset hypophosphatasia (HPP), a rare, genetic, progressive, metabolic disease.

HPP leads to severe disability and life-threatening complications. Its symptoms include defective bone mineralization that can lead to rickets and softening of the bones that results in skeletal abnormalities. It can lead to muscle weakness with loss of mobility, seizures, pain, respiratory failure and premature death.

Severe HPP is diagnosed in an estimated 1 in 100,000 newborns; milder cases, occur more often.

“For the first time, the HPP community will have access to an approved therapy for this rare disease,” FDA Center for Drug Evaluation and Research Office of Drug Evaluation III Director Amy Egan said. “Strensiq’s approval is an example of how the Breakthrough Therapy Designation program can bring new and needed treatments to people with rare diseases.” 

Strensiq, manufactured by Alexion Pharmaceuticals, Inc., is administered by weekly injections.