Orkambi — Medical Mutual
Cystic Fibrosis, Homozygous for the F508del Mutation in the CFTR Gene
Initial criteria
- Patient is age ≥ 1 year; AND
- Patient has TWO copies of the F508del mutation in the CFTR gene; AND
- Patient meets at least ONE of the following (i, ii, or iii): i. Positive cystic fibrosis newborn screening test; OR ii. Family history of cystic fibrosis; OR iii. Clinical presentation consistent with signs and symptoms of cystic fibrosis; AND
- Patient has evidence of abnormal cystic fibrosis transmembrane conductance regulator function as demonstrated by at least ONE of the following (i, ii, or iii): i. Elevated sweat chloride test; OR ii. Two cystic fibrosis-causing CFTR mutations; OR iii. Abnormal nasal potential difference; AND
- The medication is prescribed by or in consultation with a pulmonologist or a physician who specializes in the treatment of cystic fibrosis.
Reauthorization criteria
- Documentation of response to therapy is required for continuation of therapy.
Approval duration
initial 6 months, reauth 1 year