Strensiq asfotase alfa

Drug Class

Tissue non-specific alkaline phosphatase replacement therapy

Mechanism

Replaces deficient tissue non-specific alkaline phosphatase (TNSALP), preventing inorganic pyrophosphate accumulation and promoting bone mineralization.

Approved Indication

Treatment of patients with perinatal/infantile- and juvenile-onset hypophosphatasia (HPP).

What This Means For Patients

Strensiq (asfotase alfa) is an enzyme replacement therapy approved by the FDA and EMA for the treatment of hypophosphatasia (HPP), a rare genetic bone disorder. It helps prevent bone deformities, fractures, and systemic complications by restoring alkaline phosphatase activity. Under special regulatory pathways, patients can access this treatment in the Boao Lecheng pilot zone.

Regulatory Approvals

• FDA — approved (2015) — label
• EMA — approved (2015) — source

Pivotal Clinical Evidence

• ENB-002-08/ENB-003-08 — Whyte et al., N Engl J Med 2012 (NCT01176266) — DOI

Sources

• https://www.accessdata.fda.gov/drugsatfda_docs/label/2015/125513s000lbl.pdf
• https://www.ema.europa.eu/en/medicines/human/EPAR/strensiq