Medical Therapies for Enzyme Deficiencies – Commercial Medical Benefit Drug Policyopen_in_new

Covers specified enzyme replacement and related therapies for listed rare metabolic/lysosomal diseases but generally excludes combination use of disease‑modifying ERTs for the same disorder (Elfabrio is typically excluded) and certain products are not intended for CNS disease. Coverage requires laboratory or molecular diagnostic confirmation and clinical signs, dosing per FDA labeling, initial authorizations limited to 12 months with reauthorization only if prior receipt and documented clinical benefit; product‑specific limits include Pombiliti only for adults ≥40 kg used with Opfolda and after stopping prior ERT, and Revcovi limited to ADA‑SCID patients not suitable for/failed/awaiting HCT.

"Aldurazyme (laronidase) for treatment of mucopolysaccharidosis I (MPS I): Hurler variant (MPS IH), Hurler-Scheie variant (MPS IHS), or Scheie variant (MPS IS) when diagnosis is confirmed by deficie..."