Prior Auth Protocol

FDA Approved Indications:

Treatment of cystic fibrosis (CF) in patients age 2 years and older who have one of the following mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R.
Treatment of CF in patients age 2 years and older who have an R117H mutation in the CFTR gene.

Health Net Approved Indications and Usage Guidelines:

Diagnosis of cystic fibrosis with one of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, R117H, S1251N, S1255P, S549N, or S549R.

Coverage is Not Authorized For:

Non-FDA approved indications, which are not listed in the Health Net Approved Indications and Usage Guidelines section, unless there is sufficient documentation of efficacy and safety in the published literature.
Patients with CF who are homozygous for the F508del mutation in the CFTR gene

General Information:

Reference values for sweat chloride are: < 40 mmol/L = CF unlikely; 40 to 60 mmol/L = borderline/ indeterminate; > 60 mmol/L = consistent with the diagnosis of CF.
If the patient's genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of a CFTR mutation followed by verification with bi-directional sequencing when recommended by the mutation test instructions for use.
The Cystic Fibrosis Foundation's Mutation Analysis Program (MAP) offers free and confidential genetic testing to patients with a confirmed diagnosis of cystic fibrosis. It can take up to 60 days to receive genotyping results, and additional time if further testing is needed. http://www.cfpaf.org/ResourceCenter/MutationAnalysisProgram/
Kalydeco is not effective in patients with CF who are homozygous for the F508del mutation in the CFTR gene.
It is recommended that ALT and AST be assessed prior to initiating Kalydeco, every 3 months during the first year of treatment, and annually thereafter. Dosing should be interrupted in patients with ALT or AST of greater than 5 times the upper limit of normal (ULN).
Data from the study of CF patients with nine CFTR mutations did not support approval of the drug in patients with the G970R mutation. As of 2014, it is estimated that there are about 10 people worldwide who have this mutation, including two in the United States.

Therapeutic Alternatives:

Drug	Dosing Regimen	Dose Limit/ Maximum Dose
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* Requires Prior Authorization

Recommended Dosing Regimen and Authorization Limit:

Drug	Dosing Regimen	Authorization Limit
Kalydeco	Adults and pediatric patients age 6 years and older: 150 mg PO Q12H with fat-containing food. Pediatric patients 2 to less than 6 years of age and less than 14 kg: 50 mg packet mixed with 1 teaspoon (5 mL) of soft food or liquid PO Q12H with fat-containing food. Pediatric patients 2 to less than 6 years of age and 14 kg or greater: 75 mg packet mixed with 1 teaspoon (5 mL) of soft food or liquid PO Q12H with fat-containing food.	Initial authorization: 6 months Re-authorization: Length of benefit with documentation of one of the following: improvement of predicted FEV1 of at least 7% or Sweat Chloride < 60 mmol/liter

Product Availability:

Tablet: 150 mg

Oral granules: Unit-dose packets containing 50 mg, 75 mg

References:

Kalydeco [Prescribing Information] Boston, MA: Vertex Pharmaceuticals Incorporated; March 2015.
LeGrys VA, Yankaskas JR, Quittell LM, et al. Diagnostic sweat testing: the Cystic Fibrosis Foundation guidelines. J Pediatr. 2007;151:85-9.
Ramsey BW, Davies J, McElvaney NG, et al. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med 2011;365:1663-72.
Micromedex^R Healthcare Series [Internet Database]. Greenwood Village, CO: Thomson Healthcare. Updated periodically. Accessed June 28, 2015.