Cerliponase alfa

This policy covers cerliponase alfa (Brineura) intraventricular enzyme replacement therapy to slow loss of ambulation and motor function in patients with neuronal ceroid lipofuscinosis type 2 (CLN2/TPP1 deficiency), primarily pediatric patients (FDA‑approved for ages ≥3 years with sBLA support for younger/presymptomatic children). Coverage requires documented TPP1 enzyme deficiency with confirmatory molecular testing, administration into the cerebrospinal fluid via a surgically implanted intraventricular access device by clinicians experienced in IVT infusion (label dosing, e.g., 300 mg every other week), and adherence to FDA/authoritative‑source guidance and plan‑specific benefit limits; uses inconsistent with evidence or labeling are not covered.

"Therapy is covered when proven effective for the relevant diagnosis or procedure per generally accepted standards of practice."