Aqneursa — Medica

Initial criteria

• age ≥ 4 years
• patient weighs ≥ 15 kg
• patient has one or more neurologic symptom(s) of Niemann-Pick disease type C (e.g., loss of motor function, difficulty swallowing, speech or cognitive impairment)
• diagnosis established by genetic test showing biallelic pathogenic variants in NPC1 or NPC2 gene
• medication prescribed by or in consultation with a geneticist, endocrinologist, metabolic disorder subspecialist, neurologist, or a physician specializing in the treatment of Niemann-Pick disease type C or related disorders

Reauthorization criteria

• according to prescriber, patient has derived benefit from treatment defined as disease stabilization, slowed progression, or improvement
• medication prescribed by or in consultation with a geneticist, endocrinologist, metabolic disorder subspecialist, neurologist, or a physician specializing in the treatment of Niemann-Pick disease type C or related disorders

Approval duration

initial: 6 months; reauth: 1 year