FDA Approves New Breakthrough Medication for Cystic Fibrosis
Learning Objectives and CME/Disclosure Information
This activity is intended for healthcare providers delivering care to women and their families.
After completing this activity, the participant should be better able to:
1. Describe the underlying mechanisms of the newer medications to treat cystic fibrosis 2. Describe the findings from the recent trials using triple combination drug formulation used by the FDA to generate a priority review
Estimated time to complete activity: 0.25 hours
Susan J. Gross, MD, FRCSC, FACOG, FACMG
President and CEO, The ObG Project
Disclosure of Conflicts of Interest
Postgraduate Institute for Medicine (PIM) requires faculty, planners, and others in control of educational content to disclose all their financial relationships with ineligible companies. All identified conflicts of interest (COI) are thoroughly vetted and mitigated according to PIM policy. PIM is committed to providing its learners with high quality accredited continuing education activities and related materials that promote improvements or quality in healthcare and not a specific proprietary business interest of an ineligible company.
The PIM planners and others have nothing to disclose. The OBG Project planners and others have nothing to disclose.
Faculty: Susan J. Gross, MD, receives consulting fees from Cradle Genomics, and has financial interest in The ObG Project, Inc.
Planners and Managers: The PIM planners and managers, Trace Hutchison, PharmD, Samantha Mattiucci, PharmD, CHCP, Judi Smelker-Mitchek, MBA, MSN, RN, and Jan Schultz, MSN, RN, CHCP have nothing to disclose.
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Joint Accreditation Statement
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The FDA has approved a new triple therapy medication (elexacaftor/ ivacaftor/ tezacaftor) for patients with the most common cystic fibrosis (CF) mutation (approximately 90% of the CF population). The FDA granted this application Priority Review, in addition to Fast Track and Breakthrough Therapy Designation. Due to potential benefit to the CF community, the application was reviewed and approved approximately three months prior to the planned review date.
The medication is approved for the following
individuals with CF
At least one F508del mutation in the cystic
fibrosis transmembrane conductance regulator (CFTR) gene
Drug Mechanism of Action
With significant mutations such as F508del mutation, the CFTR protein undergoes two ‘hits’
Improper folding leading to entrapment in endoplasmic reticulum
Protein that does reach cell membrane does not become activated (i.e. fails to function correctly)
The drugs developed to treat CF are protein modulators
Correctors (elexacaftor and tezacaftor): Drugs that correct CFTR protein folding
Potentiator (ivacaftor): Activate function of the abnormal protein channel at the cell surface
Literature Review (see ‘Learn More – Primary Sources’ below)
Heijerman et al., Lancet 2019 (n=107)
Methods: Double-blind RCT (4 weeks) | homozygous
Elexacaftor plus tezacaftor plus ivacaftor or
Tezacaftor plus ivacaftor for 4 weeks
Primary outcome: Absolute change from baseline
in percent predicted forced expiratory volume in one second (ppFEV 1)
Primary outcome: Triple drug group had improved ppFEV
1 | Treatment difference 10.0% (95% CI, 7.4 to 12.6, p<0.0001)
Secondary outcomes: Triple drug group also
demonstrated improvement related to the following
Quality of life (Cystic Fibrosis Questionnaire)
Decreased sweat chloride concentration
Middleton et al., NEJM 2019 (n=403)
Methods: Double-blind RCT (24 weeks) | F508del mutation and a mutation on the second allele that results in either no CFTR protein or a CFTR protein that is not responsive to ivacaftor or tezacaftor/ivacaftor alone
Primary outcome: Absolute change from baseline in ppFEV 1
Primary outcome: Triple drug group had improved ppFEV 1 | Treatment difference 13.8% (95% CI, 12.1, 15.4; P<0.0001)
Other outcome improvements
Reduction in rate of pulmonary exacerbations
Decreased sweat chloride concentration
Increase in BMI
Similar between the groups
Many adverse events related to CF rather than medication effects
Rash and flu were more frequent in patients on triple therapy
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Disclosure of Unlabeled Use
This educational activity may contain discussion of published and/or investigational uses of agents that are not indicated by the FDA. The planners of this activity do not recommend the use of any agent outside of the labeled indications.
The opinions expressed in the educational activity are those of the faculty and do not necessarily represent the views of the planners. Please refer to the official prescribing information for each product for discussion of approved indications, contraindications, and warnings.
Participants have an implied responsibility to use the newly acquired information to enhance patient outcomes and their own professional development. The information
presented in this activity is not meant to serve as a guideline for patient management. Any procedures, medications, or other courses of diagnosis or treatment discussed or suggested in this activity should not be used by clinicians without evaluation of their patient’s conditions and possible contraindications and/or dangers in use, review of any applicable manufacturer’s product information, and comparison with recommendations of other authorities.
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