Editing Cystic fibrosis (section)

===Other medication===
{{See also|Cystic fibrosis transmembrane conductance regulator#Drug target}}
Aerosolized medications that help loosen secretions include [[dornase alfa]] and [[Hypertonicity|hypertonic]] [[Saline (medicine)|saline]].<ref name="pmid16642591">{{cite journal | vauthors = Kuver R, Lee SP | title = Hypertonic saline for cystic fibrosis | journal = The New England Journal of Medicine | volume = 354 | issue = 17 | pages = 1848–51; author reply 1848–51 | date = April 2006 | pmid = 16642591 | doi = 10.1056/NEJMc060351 | s2cid = 26244542 }}</ref> Dornase is a [[Recombinant DNA|recombinant]] human [[deoxyribonuclease]], which breaks down DNA in the sputum, thus decreasing its viscosity.<ref name="pmid5694947">{{cite journal | vauthors = Lieberman J | title = Dornase aerosol effect on sputum viscosity in cases of cystic fibrosis | journal = JAMA | volume = 205 | issue = 5 | pages = 312–313 | date = July 1968 | pmid = 5694947 | doi = 10.1001/jama.205.5.312 }}</ref> Dornase alpha improves lung function and probably decreases the risk of exacerbations but there is insufficient evidence to know if it is more or less effective than other similar medications.<ref name="pmid33735508" /> Dornase alpha may improve lung function; however, there is no strong evidence that it is better than other hyperosmolar therapies.<ref name="pmid33735508">{{cite journal | vauthors = Yang C, Montgomery M | title = Dornase alfa for cystic fibrosis | journal = The Cochrane Database of Systematic Reviews | volume = 2021 | issue = 3 | pages = CD001127 | date = March 2021 | pmid = 33735508 | pmc = 8094421 | doi = 10.1002/14651858.CD001127.pub5 | collaboration = Cochrane Cystic Fibrosis and Genetic Disorders Group }}</ref>

[[Denufosol]], an investigational drug, opens an alternative chloride channel, helping to liquefy mucus.<ref name="pmid18276176">{{cite journal | vauthors = Kellerman D, Rossi Mospan A, Engels J, Schaberg A, Gorden J, Smiley L | title = Denufosol: a review of studies with inhaled P2Y(2) agonists that led to Phase 3 | journal = Pulmonary Pharmacology & Therapeutics | volume = 21 | issue = 4 | pages = 600–607 | date = August 2008 | pmid = 18276176 | doi = 10.1016/j.pupt.2007.12.003 }}</ref> Whether [[inhaled corticosteroids]] are useful is unclear, but stopping inhaled corticosteroid therapy is safe.<ref name="Inhaled corticosteroids for cystic">{{cite journal | vauthors = Balfour-Lynn IM, Welch K, Smith S | title = Inhaled corticosteroids for cystic fibrosis | journal = The Cochrane Database of Systematic Reviews | volume = 7 | issue = 7 | pages = CD001915 | date = July 2019 | pmid = 31271656 | pmc = 6609325 | doi = 10.1002/14651858.CD001915.pub6 }}</ref> There is weak evidence that corticosteroid treatment may cause harm by interfering with growth.<ref name="Inhaled corticosteroids for cystic"/> [[Pneumococcal vaccination]] has not been studied {{as of|2014|lc=y}}.<ref name="pmid25093421">{{cite journal | vauthors = Burgess L, Southern KW | title = Pneumococcal vaccines for cystic fibrosis | journal = The Cochrane Database of Systematic Reviews | volume = 8 | issue = 8 | pages = CD008865 | date = August 2014 | pmid = 25093421 | doi = 10.1002/14651858.CD008865.pub3 | veditors = Burgess L }}</ref> {{as of|2014}}, there is no clear evidence from randomized controlled trials that the [[influenza vaccine]] is beneficial for people with cystic fibrosis.<ref name="pmid24604671">{{cite journal | vauthors = Dharmaraj P, Smyth RL | title = Vaccines for preventing influenza in people with cystic fibrosis | journal = The Cochrane Database of Systematic Reviews | volume = 2014 | issue = 3 | pages = CD001753 | date = March 2014 | pmid = 24604671 | pmc = 7066935 | doi = 10.1002/14651858.CD001753.pub3 }}</ref>

[[Ivacaftor]] is a medication taken by mouth for the treatment of CF due to several specific mutations responsive to ivacaftor-induced CFTR protein enhancement.<ref name=Whit2014/><ref name="pmid25148205">{{cite journal | vauthors = Wainwright CE | title = Ivacaftor for patients with cystic fibrosis | journal = Expert Review of Respiratory Medicine | volume = 8 | issue = 5 | pages = 533–538 | date = October 2014 | pmid = 25148205 | doi = 10.1586/17476348.2014.951333 | s2cid = 39537446 }}</ref> It improves lung function by about 10%; however, {{as of|2014|lc=y}} it is expensive.<ref name="Whit2014">{{cite journal | vauthors = Whiting P, Al M, Burgers L, Westwood M, Ryder S, Hoogendoorn M, Armstrong N, Allen A, Severens H, Kleijnen J | title = Ivacaftor for the treatment of patients with cystic fibrosis and the G551D mutation: a systematic review and cost-effectiveness analysis | journal = Health Technology Assessment | volume = 18 | issue = 18 | pages = 1–106 | date = March 2014 | pmid = 24656117 | pmc = 4780965 | doi = 10.3310/hta18180 }}</ref> The first year it was on the market, the list price was over $300,000 per year in the United States.<ref name=Whit2014/> In July 2015, the U.S. Food and Drug Administration approved [[lumacaftor/ivacaftor]].<ref name="FDA-2015">{{Cite web|url=https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm453565.htm|title=Press Announcements - FDA approves new treatment for cystic fibrosis |website=FDA |date=2 July 2015 |language=en|access-date=16 January 2017|url-status=dead |archive-url=https://web.archive.org/web/20170118091335/https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm453565.htm|archive-date=18 January 2017}}</ref> In 2018, the FDA approved the combination [[ivacaftor/tezacaftor]]; the manufacturer announced a list price of $292,000 per year.<ref name="The Boston Globe-2018">{{cite web|url=https://www.bostonglobe.com/business/2018/02/12/fda-approves-another-vertex-drug-for-treatment-cystic-fibrosis/uU5Jfnirrwv57kH1aj2W5N/story.html|title=FDA approves another Vertex drug for treatment of cystic fibrosis |date=12 February 2018 |website=[[The Boston Globe]] |url-access=subscription |url-status=live |archive-url=https://archive.today/20231226081346/https://www.bostonglobe.com/business/2018/02/12/fda-approves-another-vertex-drug-for-treatment-cystic-fibrosis/uU5Jfnirrwv57kH1aj2W5N/story.html |archive-date= 26 December 2023 }}</ref> [[Tezacaftor]] helps move the CFTR protein to the correct position on the cell surface, and is designed to treat people with the [[F508del]] mutation.<ref name="Cystic Fibrosis News Today">{{cite web|url=https://cysticfibrosisnewstoday.com/tezacaftor-vx-661-for-cystic-fibrosis|title=Tezacaftor (VX-661) for Cystic Fibrosis |website=Cystic Fibrosis News Today|access-date=23 December 2018|archive-url=https://web.archive.org/web/20180929042325/https://cysticfibrosisnewstoday.com/tezacaftor-vx-661-for-cystic-fibrosis|archive-date=29 September 2018|url-status=dead}}</ref>

In 2019, the [[combination drug]] [[elexacaftor/ivacaftor/tezacaftor]], marketed as Trikafta and described as modulator therapy,<ref name="Cystic Fibrosis Foundation"/> was approved for CF patients over the age of 12 in the United States,<ref name=Drugs2019>{{cite web |title=Trikafta (elexacaftor, ivacaftor and tezacaftor) FDA Approval History |url=https://www.drugs.com/history/trikafta.html |website=Drugs.com |language=en}}</ref><ref name="Office of the Commissioner-2020a">{{Cite web | author = Office of the Commissioner |date=24 March 2020 |title=FDA approves new breakthrough therapy for cystic fibrosis |url=https://www.fda.gov/news-events/press-announcements/fda-approves-new-breakthrough-therapy-cystic-fibrosis |access-date=28 April 2022 |website=FDA |language=en}}</ref> extended to age 6 in 2021.<ref name="www.cff.org-2021">{{Cite web |title=FDA Accepts Vertex Application for Expansion of Trikafta to Include Children ages 6-11 {{!}} Cystic Fibrosis Foundation |url=https://www.cff.org/news/2021-01/fda-accepts-vertex-application-expansion-trikafta-include-children-ages-6-11 |access-date=28 April 2022 |website=www.cff.org |date=26 January 2021 |language=en}}</ref> In Europe this drug was approved in 2020 and marketed as Kaftrio.<ref name="NHSEngland">{{cite web |title=NHS England » Landmark NHS deal to open up access to life-changing cystic fibrosis drug |url=https://www.england.nhs.uk/2020/08/landmark-nhs-deal-to-open-up-access-to-life-changing-cystic-fibrosis-drug/ |website=www.england.nhs.uk |date=21 August 2020 |access-date=8 August 2021}}</ref>  It is used in those who have a f508del mutation, found in about 90% of patients with cystic fibrosis.<ref name=Drugs2019/><ref name="Office of the Commissioner-2020">{{Cite web | author = Office of the Commissioner |date=24 March 2020|title=FDA approves new breakthrough therapy for cystic fibrosis|url=https://www.fda.gov/news-events/press-announcements/fda-approves-new-breakthrough-therapy-cystic-fibrosis|access-date=12 August 2020|website=FDA|language=en}}</ref> According to the [[Cystic Fibrosis Foundation]], "this medicine represents the single greatest therapeutic advancement in the history of CF, offering a treatment for the underlying cause of the disease that could eventually bring modulator therapy to 90 percent of people with CF."<ref name="Cystic Fibrosis Foundation">{{Cite press release| location = Bethesda, Md. | publisher = Cystic Fibrosis Foundation |title=Statement on FDA approval of Trikafta, the first triple-combination therapy for the most common CF mutation|url=https://www.cff.org/About-Us/Media-Center/Press-Releases/Cystic-Fibrosis-Foundation-statement-on-FDA-approval-of-TRIKAFTA-the-first-triple-combination-therapy-for-the-most-common-CF-mutation/|access-date=12 August 2020| date = 21 October 2019 |language=en}}</ref> In a clinical trial, participants who were administered the combination drug experienced a subsequent 63% decrease in pulmonary exacerbations and a 41.8&nbsp;mmol/L decrease in sweat chloride concentration.<ref name="Elexacaftor-Tezacaftor-Ivacaftor fo">{{cite journal | vauthors = Middleton PG, Mall MA, Dřevínek P, Lands LC, McKone EF, Polineni D, Ramsey BW, Taylor-Cousar JL, Tullis E, Vermeulen F, Marigowda G, McKee CM, Moskowitz SM, Nair N, Savage J, Simard C, Tian S, Waltz D, Xuan F, Rowe SM, Jain R | title = Elexacaftor-Tezacaftor-Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele | journal = The New England Journal of Medicine | volume = 381 | issue = 19 | pages = 1809–1819 | date = November 2019 | pmid = 31697873 | pmc = 7282384 | doi = 10.1056/NEJMoa1908639 }}</ref> By mitigating a repertoire of symptoms associated with cystic fibrosis, the combination drug significantly improved quality-of-life metrics among patients with the disease as well.<ref name="Elexacaftor-Tezacaftor-Ivacaftor fo"/><ref name="Cystic Fibrosis Foundation"/> The combination drug is also known to interact with CYP3A inducers,<ref name="pmid9131486">{{cite journal | vauthors = Wilkinson GR | title = Cytochrome P4503A (CYP3A) metabolism: prediction of in vivo activity in humans | journal = Journal of Pharmacokinetics and Biopharmaceutics | volume = 24 | issue = 5 | pages = 475–490 | date = October 1996 | pmid = 9131486 | doi = 10.1007/BF02353475 | s2cid = 30289771 }}</ref> such as carbamazepine used in the treatment of bipolar disorder, causing elexafaftor/ivacaftor/tezacaftor to circulate in the body at decreased concentrations. As such, concurrent use is not recommended.<ref name="pmid32265602">{{cite journal | vauthors = Ridley K, Condren M | title = Elexacaftor-Tezacaftor-Ivacaftor: The First Triple-Combination Cystic Fibrosis Transmembrane Conductance Regulator Modulating Therapy | journal = The Journal of Pediatric Pharmacology and Therapeutics | volume = 25 | issue = 3 | pages = 192–197 | date = 1 April 2020 | pmid = 32265602 | pmc = 7134581 | doi = 10.5863/1551-6776-25.3.192 }}</ref> The list price in the US is going to be $311,000 per year;<ref name="Reuters-2019">{{cite web |title=Vertex prices cystic fibrosis combo treatment at $311,000-per-year |url=https://www.reuters.com/article/us-vertex-pharms-fda/vertex-prices-cystic-fibrosis-combo-treatment-at-311000-per-year-idUSKBN1X024U |website=Reuters |access-date=23 October 2019 |language=en |date=21 October 2019}}</ref> however, insurance may cover much of the cost of the drug.<ref name="Good News Network-2019">{{Cite web |date=3 November 2019|title=FDA Approves the First New Cystic Fibrosis Treatment in Decades|url=https://www.goodnewsnetwork.org/fda-approves-the-first-new-cystic-fibrosis-treatment-in-decades/|access-date=12 August 2020|website=Good News Network|language=en-US}}</ref>

[[Ursodeoxycholic acid]], a [[Bile acid|bile salt]], has been used; however, a 2021 study aimed at evaluating whether the incidence of severe liver disease differed between CF centers routinely prescribing or not prescribing UDCA found no reduction in portal hypertension.<ref>{{cite journal |display-authors=5| last1=Colombo | first1=Carla | last2=Alicandro | first2=Gianfranco | last3=Oliver | first3=Mark | last4=Lewindon | first4=Peter J | last5=Ramm | first5=Grant A | last6=Ooi | first6=Chee Y. | last7=Alghisi | first7=Federico | last8=Kashirskaya | first8=Nataliya | last9=Kondratyeva | first9=Elena | last10=Corti | first10=Fabiola | last11=Padoan | first11=Rita | last12=Asherova | first12=Irina | last13=Evans | first13=Helen | last14=de Monestrol | first14=Isabelle | last15=Strandvik | first15=Birgitta | last16=Lindblad | first16=Anders | title=Ursodeoxycholic acid and liver disease associated with cystic fibrosis: A multicenter cohort study | journal=Journal of Cystic Fibrosis | volume=21 | issue=2 | date=2022 | doi=10.1016/j.jcf.2021.03.014 | pages=220–226}}</ref>

The combination [[vanzacaftor/tezacaftor/deutivacaftor]] (Alyftrek) was approved for medical use in the United States in December 2024.<ref>{{cite press release | title=Vertex Announces US FDA Approval of Alyftrek, a Once-Daily Next-in-Class CFTR Modulator for the Treatment of Cystic Fibrosis | publisher=Vertex | via=Business Wire | date=20 December 2024 | url=https://www.businesswire.com/news/home/20241220133127/en/ | access-date=24 December 2024}}</ref>