Overview
Cystic Fibrosis is treated with 8 medications in our database, including Trikafta, Kalydeco, BETHKIS, SYMDEKO, ORKAMBI, and 3 more. 4 of these have manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Vertex Pharmaceuticals, Chiesi, Vertex, Gilead Sciences, Viatris and others. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Cystic Fibrosis treatment below.
Also known as:
Clinical phenotype terms— hover any for plain English:
Autosomal recessive
Passed on when both parents carry the same gene change; often skips generations
Neonatal
Begins at or shortly after birth (first 4 weeks)
FDA & Trial Timeline
10 eventsThe Hospital for Sick Children
University of Kansas Medical Center — PHASE2
Anagram Therapeutics, Inc. — PHASE2
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
11 availableSYMDEKO
1 INDICATIONS AND USAGE SYMDEKO is indicated for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who are homozygous for the F508del mutation or who have at least one mutation …
1 INDICATIONS AND USAGE SYMDEKO is indicated for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who are homozygous for the F508del mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator ( CFTR ) gene that is responsive to tezacaftor/ivacaftor based on in vitro data and/or clinical evidence [see Clinical Pharmacology (12.1) and Clinical Studies (14) ] . 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. SYMDEKO is a combination of tezacaftor and ivacaftor, indicated for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who are homozygous for the F508del mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator ( CFTR ) gene that is responsive to tezacaftor/ivacaftor based on in vitro data and/or clinical evidence. ( 12.1 , 14 ) 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. ( 1 )
ALYFTREK
ALYFTREK is indicated for the treatment of cystic fibrosis (CF) in patients 6 years of age and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transm…
ALYFTREK is indicated for the treatment of cystic fibrosis (CF) in patients 6 years of age and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene
Kalydeco
1 INDICATIONS AND USAGE KALYDECO is indicated for the treatment of cystic fibrosis (CF) in patients aged 1 month and older who have at least one mutation in the CFTR gene that is responsive to ivacaft…
1 INDICATIONS AND USAGE KALYDECO is indicated for the treatment of cystic fibrosis (CF) in patients aged 1 month and older who have at least one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data [see Clinical Pharmacology (12.1) and Clinical Studies (14) ] . 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. KALYDECO is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator indicated for the treatment of cystic fibrosis (CF) in patients aged 1 month and older who have at least one mutation in the CFTR gene that is responsive to ivacaftor based on clinical and/or in vitro assay data. ( 12.1 , 14 ) 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. ( 1 )
Acetylcysteine
adjuvant therapy for patients with abnormal, viscid, or inspissated mucous secretions in pulmonary complications of cystic fibrosis
ORKAMBI
1 INDICATIONS AND USAGE ORKAMBI is indicated for the treatment of cystic fibrosis (CF) in patients aged 1 year and older who are homozygous for the F508del mutation in the CFTR gene. If the patient's …
1 INDICATIONS AND USAGE ORKAMBI is indicated for the treatment of cystic fibrosis (CF) in patients aged 1 year and older who are homozygous for the F508del mutation in the CFTR gene. If the patient's genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of the F508del mutation on both alleles of the CFTR gene. ORKAMBI is a combination of ivacaftor, a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, and lumacaftor, indicated for the treatment of cystic fibrosis (CF) in patients aged 1 year and older who are homozygous for the F508del mutation in the CFTR gene. If the patient's genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of the F508del mutation on both alleles of the CFTR gene. ( 1 ) Limitations of Use: The efficacy and safety of ORKAMBI have not been established in patients with CF other than those homozygous for the F508del mutation. ( 1 ) Limitations of Use The efficacy and safety of ORKAMBI have not been established in patients with CF other than those homozygous for the F508del mutation .
ALYFTREK
ALYFTREK is indicated for the treatment of cystic fibrosis (CF) in patients 6 years of age and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transm…
ALYFTREK is indicated for the treatment of cystic fibrosis (CF) in patients 6 years of age and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene
Trikafta
Indicated for CF in patients aged 2+ with at least one F508del mutation.
TOBI
Detailed copay and financial assistance information is not publicly available for this medication at this time. Please consult your pharmacist or the manufacturer's official patient support program fo
Cayston
* Patient Copay Amount: $10 per fill * Maximum Annual Benefit Limit: $8,000 per calendar year (up to 7 fills) * Core Eligibility Restrictions: Must have commercial (private) insurance; not eligible if
Pulmozyme
* Patient Copay Amount: As little as $30 per 30-day supply * Maximum Annual Benefit Limit: $10,000 per calendar year * Core Eligibility Restrictions: Must have commercial (private) insurance. Not va
BETHKIS
Detailed copay and financial assistance information is not publicly available for this medication at this time. Please consult your pharmacist or the manufacturer's official patient support program fo
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Treatment Centers
8 centersNational Institutes of Health Clinical Center
📍 Bethesda, Maryland
👤 Payal P Khincha, M.D.
👤 Christopher Grunseich, M.D.
Children's Hospital Colorado
📍 Aurora, Colorado
👤 Zachary Grinspan, MD
Boston Children's Hospital
📍 Boston, Massachusetts
Massachusetts General Hospital
📍 Boston, Massachusetts
👤 Matthew Frigault, MD
👤 Janssen Research & Development, LLC Clinical Trial
Children's Hospital of Orange County
📍 Orange, California
👤 Richard Neibeger, MD
Cincinnati Children's Hospital Medical Center
📍 Cincinnati, Ohio
Ann & Robert H. Lurie Children's Hospital of Chicago
📍 Chicago, Illinois
University of Arkansas for Medical Sciences
📍 Little Rock, Arkansas
Financial Resources
13 resourcesGood Days — Cystic fibrosis
Good Days
Cystic fibrosis
Patient Services Inc — Cystic fibrosis
Patient Services Inc
Cystic fibrosis
NORD Patient Assistance — Cystic fibrosis
NORD Patient Assistance
Cystic fibrosis
Patient Advocate Foundation — Cystic fibrosis
Patient Advocate Foundation
Cystic fibrosis
The Assistance Fund — Cystic fibrosis
The Assistance Fund
Cystic fibrosis
HealthWell Foundation — Cystic fibrosis
HealthWell Foundation
Cystic fibrosis
PAN Foundation — Cystic fibrosis
PAN Foundation
Cystic fibrosis
6 travel grants are also available for Cystic fibrosis patients — see Travel Grants below ↓
Travel Grants
6 grantsBoston Children's Hospital Cystic Fibrosis Patient Resources
Eligibility generally requires a diagnosis of cystic fibrosis, with many programs specifically requiring residency in Massachusetts or treatment at a Massachusetts-based care center. Specific age requirements vary by program, with some focused on children under 18 and others catering to adults or family caregivers.
BC Cystic Fibrosis Subsidy for Medications and Travel Program
Kitabis Copay Assistance
Community
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5 articlesCaregiver Resources
NORD Caregiver Resources
Support, advocacy, and financial assistance for caregivers of rare disease patients.
Mental Health Support
Rare disease caregiving can be isolating. Connect with counseling and peer support.
Family & Caregiver Grants
Financial assistance programs specifically for caregivers of rare disease patients.
Social Security Disability
Learn how rare disease patients may qualify for SSDI/SSI benefits.
Common questions about Cystic fibrosis
What is Cystic fibrosis?
Cystic Fibrosis is treated with 8 medications in our database, including Trikafta, Kalydeco, BETHKIS, SYMDEKO, ORKAMBI, and 3 more. 4 of these have manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Vertex Pharmaceuticals, Chiesi, Vertex, Gilead Sciences, Viatris and others. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Cystic Fibrosis treatment below.
How is Cystic fibrosis inherited?
Cystic fibrosis follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
At what age does Cystic fibrosis typically begin?
Typical onset of Cystic fibrosis is neonatal. Age of onset can vary across affected individuals.
Are there clinical trials for Cystic fibrosis?
Yes — 20 recruiting clinical trials are currently listed for Cystic fibrosis on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Cystic fibrosis?
25 specialists and care centers treating Cystic fibrosis are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.
What treatment and support options exist for Cystic fibrosis?
8 FDA-approved treatments and 13 patient support programs are currently tracked on UniteRare for Cystic fibrosis. See the treatments and support programs sections for copay assistance, eligibility, and contact details.