Nulibry (fosdenopterin) — Medical Mutual

Initial criteria

• Will not be used in combination with other substrate replacement therapy (e.g., recombinant cyclic pyranopterin monophosphate, etc.); AND
• Must be prescribed by, or in consultation with, a specialist in medical genetics or pediatric neurology; AND
• Patient has a diagnosis of MoCD Type A confirmed by a mutation in the MOCS1 gene suggestive of disease as identified on molecular genetic testing; OR
• Patient has biochemical features suggestive of MoCD Type A (i.e., elevated sulfites in urine, low serum uric acid, elevated urinary xanthine and hypoxanthine) and will be treated presumptively while awaiting genetic confirmation; AND
• Patient has a baseline value for the following: urinary s-sulfocysteine (SSC) normalized to creatinine; AND
• Clinical notes regarding signs and symptoms of disease which may include, but are not limited to, seizure frequency/duration, growth, and developmental milestones

Reauthorization criteria

• Patient continues to meet universal and other indication-specific relevant criteria such as concomitant therapy requirements (not including prerequisite therapy), performance status, etc. identified in section III; AND
• Absence of unacceptable toxicity from the drug. Examples of unacceptable toxicity include severe phototoxicity, clinically significant infection, etc.; AND
• Disease response compared to pre-treatment baseline as evidenced by the following: reduction in urinary SSC normalized to creatinine; AND stabilization or improvement in one or more signs and symptoms of disease including, but not limited to, seizure frequency/duration, growth, achievement of developmental milestones; OR
• Patient initiated therapy as an inpatient based upon a presumptive diagnosis of MoCD Type A which was subsequently confirmed by genetic testing; AND patient is responding to therapy compared to one or more pre-treatment baseline parameters which prompted the workup for MoCD

Approval duration

initial 6 months, renewal 12 months