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Table 21: Cystic Fibrosis Agents


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Drug Category: Respiratory Agents

Medication Class/Individual Agents: Cystic Fibrosis Transmembrane Conductance Regulator Modulators

I. Prior-Authorization Requirements

 Cystic Fibrosis Agents – Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulators

Clinical Notes

Drug Details

Drug Generic Name

Drug Brand Name

PA
Status

Drug
Notes

elexacaftor / tezacaftor / ivacaftor Trikafta PD PA  
ivacaftor Kalydeco PD PA  
lumacaftor / ivacaftor Orkambi PD PA  
tezacaftor / ivacaftor Symdeko PD PA  

Please note: In the case where the prior authorization (PA) status column indicates PA, both the brand and generic (if available) require PA. Typically, the generic is preferred when available unless the brand-name drug appears on the MassHealth Brand Name Preferred Over Generic Drug List. In general, when requesting the non-preferred version, whether the brand or generic, the prescriber must provide medical records documenting an inadequate response or adverse reaction to the preferred version, in addition to satisfying the criteria for the drug itself.

ivacaftor

  • A potentiator of the CFTR protein thought to work by facilitating increased chloride transport by potentiating the channel-open probability of the CFTR protein.
  • Approved for individuals ≥ one month of age with cystic fibrosis (CF) and one of the FDA-approved mutations in the CFTR gene that is responsive to ivacaftor.
  • Strongly recommended by the CFF for individuals with CF and specific gene mutation noted above to improve lung function and quality of life, and to reduce exacerbations.1
  • Dosing for individuals one  to < two months of age and weight 3 kg or greater: 5.8 mg packet every 12 hours - mix with 5 mL soft food or liquid and administer with fat-containing food.*
  • Dosing for individuals two to < four months of age and weight 3 kg or greater: 13.4 mg packet every 12 hours - mix with 5 mL soft food or liquid and administer with fat-containing food.*
  • Dosing for individuals four months to < six months of age and weight 5 kg or greater: 25 mg packet every 12 hours - mix with 5 mL soft food or liquid and administer with fat-containing food.*
  • Dosing for individuals six months to < six years of age and weight < 7 kg: 25 mg packet every 12 hours - mix with 5 mL soft food or liquid and administer with fat-containing food.*
  • Dosing for individuals six months to < six years of age and weight 7 kg to < 14 kg: 50 mg packet every 12 hours - mix with 5 mL soft food or liquid and administer with fat-containing food.*
  • Dosing for individuals six months to < six years of age and weight 14 kg or greater: 75 mg packet every 12 hours - mix with 5 mL soft food or liquid and administer with fat-containing food.*
  • Dosing for individuals ≥ six years of age: 150 mg every 12 hours with fat-containing food.*

*Notes: Adjust dose for individuals with concomitant use of moderate and strong CYP3A inhibitors, moderate hepatic impairment, and use with caution in those with severe hepatic impairment. If possible, avoid concomitant use of CYP3A inducers (e.g., carbamazepine, rifampin, phenobarbital, phenytoin, St. John’s wort, etc).

 

lumacaftor/ivacaftor

  • A combination product that contains ivacaftor, a potentiator of the CFTR protein as well as lumacaftor, a CFTR corrector.
  • Approved for individuals ≥ one year of age with CF and two copies (homozygous) of the F508del mutation in the CFTR gene.
  • Dosing for individuals one to two years of age and weight seven kg to < nine kg: One packet (75 mg/94 mg) every 12 hours- mix with 5 mL of soft food or liquid and administer with fat-containing food.**
  • Dosing for individuals one to two years of age and weight nine kg to < 14 kg: One packet (100 mg/125 mg) every 12 hours- mix with 5 mL of soft food or liquid and administer with fat-containing food.**
  • Dosing for individuals one to two years of age and weight 14 kg or greater: One packet (150 mg/188 mg) every 12 hours- mix with 5 mL of soft food or liquid and administer with fat-containing food.**
  • Dosing for individuals two to five years of age and weight < 14 kg: One packet (100mg/125 mg) every 12 hours- mix with 5 mL soft food or liquid and administer with fat-containing food.**
  • Dosing for individuals two to five years of age and weight 14 kg or greater: One packet (150mg/188 mg) every 12 hours- mix with 5 mL soft food or liquid and administer with fat-containing food.**
  • Dosing for individuals six to < 12 years of age: Two tablets (100mg/125 mg) every 12 hours with fat-containing food.**
  • Dosing for individuals ≥ 12 years of age: Two tablets (200mg/125 mg) every 12 hours with fat-containing food.**

**Notes: Adjust dose for individuals with hepatic impairment and concomitant use of strong CYP3A inhibitors (e.g., azole antifungals, clarithromycin, etc). If possible, avoid concomitant use of CYP3A inducers (e.g., carbamazepine, rifampin, phenobarbital, phenytoin, St. John’s wort, etc).

 

tezacaftor/ivacaftor

  • A combination product that contains ivacaftor, a potentiator of the CFTR protein, as well as tezacaftor, a CFTR corrector.
  • Approved for individuals ≥ six years of age with CF and two copies (homozygous) of the F508del mutation in the CFTR gene or who have at least one mutation in the CFTR gene that is responsive to tezacaftor/ivacaftor.
  • Dosing for individuals six to < 12 years of age weighing < 30 kg: One tablet (50mg/75 mg) every morning and one ivacaftor 75 mg tablet every evening with fat-containing food.***
  • Dosing for individuals six to < 12 years of age weighing ≥ 30 kg: One tablet (100mg/150 mg) every morning and one ivacaftor 150 mg tablet every evening with fat-containing food.***
  • Dosing for individuals ≥ 12 years of age: One tablet (100mg/150 mg) every morning and one ivacaftor 150 mg tablet every evening with fat-containing food.***

***Notes: Adjust dose for individuals with moderate or severe hepatic impairment or when coadministered with moderate or strong CYP3A inhibitors (e.g., azole antifungals, clarithromycin, etc).

 

elexacaftor/tezacaftor/ivacaftor

  • The newest agent that now contains two CFTR correctors, elexacaftor and tezacaftor as well as the potentiator, ivacaftor.
  • Approved for individuals aged two years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive to elexacaftor/tezacaftor/ivacaftor.
  • Dosing for individuals 2 to < 6 years of age and weighing < 14 kg is one elexacaftor 80 mg/ tezacaftor 40 mg/ ivacaftor 60 mg packet every morning and one ivacaftor 59.5 mg packet every evening.***
  • Dosing for individuals 2 to < 6 years of age and weighing ≥ 14 kg is one elexacaftor 100 mg /tezacaftor 50 mg/ ivacaftor 75 mg packet every morning and one ivacaftor 75 mg packet every evening.***
  • Dosing for individuals six to < 12 years of age weighing < 30 kg is two elexacaftor 50 mg/ tezacaftor 25 mg/ ivacaftor 37.5 mg tablets in the morning and one ivacaftor 75 mg tablet in the evening.***
  • Dosing for individuals six to < 12 years of age weighing ≥ 30 kg is two elexacaftor 100 mg/ tezacaftor 50 mg/ ivacaftor 75 mg tablets in the morning and one ivacaftor 150 mg tablet in the evening.***
  • Dosing for individuals ≥ 12 years of age is two elexacaftor 100 mg/ tezacaftor 50 mg/ ivacaftor 75 mg tablets in the morning and one ivacaftor 150 mg tablet in the evening.***

***Notes: Adjust dose for individuals with moderate hepatic impairment or when coadministered with moderate or strong CYP3A inhibitors (e.g., azole antifungals, clarithromycin, etc). Do not use in individuals with severe hepatic impairment or with concomitant strong CYP3A inducers (e.g., rifampin, rifabutin, phenobarbital, carbamazepine, phenytoin, etc).

References

1. Mogayzel PJ, Naureckas ET, Robinson KA, Mueller G, Hadjiliadis D, Hoag JB, et al. Cystic fibrosis pulmonary guidelines: chronic medications for maintenance of lung health. Am J Respir Crit Care Med. 2013 Apr 1;187(7):680-9.

 

 Cystic Fibrosis Agents – Mucolytics

Drug Details

Drug Generic Name

Drug Brand Name

PA
Status

Drug
Notes

dornase alfa Pulmozyme test  
mannitol inhalation powder Bronchitol PA  
Table Footnotes
PD Preferred Drug. In general, MassHealth requires a trial of the preferred drug or clinical rationale for prescribing a non-preferred drug within a therapeutic class.
 

II. Therapeutic Uses

FDA-approved, for example:

  • treatment of cystic fibrosis (CF)

Note: The above list may not include all FDA-approved indications.

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III.  Evaluation Criteria for Approval

Please note: In the case where the prior authorization (PA) status column indicates PA, both the brand and generic (if available) require PA. Typically, the generic is preferred when available unless the brand-name drug appears on the MassHealth Brand Name Preferred Over Generic Drug List. In general, when requesting the non-preferred version, whether the brand or generic, the prescriber must provide medical records documenting an inadequate response or adverse reaction to the preferred version, in addition to satisfying the criteria for the drug itself.

  • All PA requests must include clinical diagnosis, drug name, dose, and frequency.
  • Dispensing in a 90-day supply of medication may be mandated or allowable for agents in this therapeutic class (designated by M90 or A90, respectively, in the Drug Notes section above). Applicable quantity limits are described below as units per day, per month, per 28 days, or as clinically appropriate, and may be extrapolated for fills of longer day supply. 
  • A preferred drug may be designated for this therapeutic class. In general, MassHealth requires a trial of the preferred drug or clinical rationale for prescribing a non-preferred drug within a therapeutic class. Additional information about these agents, including PA requirements and preferred products, can be found within the MassHealth Drug List at www.mass.gov/druglist.
  • For recertification requests, approval may require submission of additional documentation including, but not limited to, documentation of: some or all criteria for the original approval; response to therapy; clinical rationale for continuation of use; status of member’s condition; appropriate diagnosis; appropriate age; appropriate dose, frequency, and duration of use for requested medication; complete treatment plan; current laboratory values; and member’s current weight.
  • Additional criteria may apply depending upon diagnosis and/or requested medication (see below). Other factors, including rebate and FDA-approval status, may change independently of scheduled MassHealth updates, which may result in additional restrictions.

 

Bronchitol

  • Documentation of all of the following is required:
    • appropriate diagnosis; and
    • member is ≥ 18 years of age; and
    • member has passed the Bronchitol Tolerance Test; and
    • inadequate response, adverse reaction, or contraindication to both of the following: Pulmozyme, sodium chloride for inhalation; and
    • appropriate dosing. 

 

Kalydeco

  • Documentation of all of the following is required:
    • appropriate diagnosis; and
    • member is ≥ one month of age; and
    • requested quantity is ≤ two units/day; and
    • baseline body mass index (BMI); and
    • for members > six years of age, baseline percent predicted forced expiratory volume in one second (ppFEV1). 
  • For recertification, documentation of positive response to therapy (e.g., improvement in BMI, ppFEV1, decrease in clinical exacerbations) is required. 

 

Orkambi

  • Documentation of all of the following is required:
    • appropriate diagnosis; and
    • member is ≥ one year of age; and
    • one of the following:
      • for tablets, requested quantity is ≤ four tablets/day; or
      • for granules, requested quantity is ≤ two packets/day; and
    • baseline BMI; and 
    • for members > six years of age, baseline ppFEV1.
  • For recertification, documentation of positive response to therapy (e.g., improvement in BMI, ppFEV1, decrease in clinical exacerbations) is required. 

 

Symdeko

  • Documentation of all of the following is required:
    • appropriate diagnosis; and
    • member is ≥ six years of age; and
    • requested quantity is ≤ two tablets/day; and
    • baseline BMI; and 
    • for members > six years of age, baseline ppFEV1.
  • For recertification, documentation of positive response to therapy (e.g., improvement in BMI, ppFEV1, decrease in clinical exacerbations) is required. 

 

Trikafta

  • Documentation of all of the following is required:
    • appropriate diagnosis; and
    • member is ≥ two years of age; and
    • one of the following:
      • for tablets, requested quantity is ≤ three tablets/day; or
      • for granules, requested quantity is ≤ 2 packets/day; and
    • baseline BMI; and 
    • for members > six years of age, baseline ppFEV1.
  • For recertification, documentation of positive response to therapy (e.g., improvement in BMI, ppFEV1, decrease in clinical exacerbations) is required. 


Original Effective Date: 04/2003

Last Revised Date: 03/2024


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Last updated 05/16/24

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