Drug Development Pipeline

We’re attacking CF from every angle.

Click on any drug in the interactive pipeline below to learn more about CF drugs in development or already in use by patients.

Click on any drug in the interactive pipeline below to learn more about CF drugs in development or already in use by patients.

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Drug Descriptions

Restore CFTR Function

  • Ivacaftor (Kalydeco®)

    Ivacaftor (Kalydeco®) is an oral medication that was the first drug available that targeted the underlying cause of CF – the defective CFTR protein.  Ivacaftor helps facilitate the opening of the chloride channel on the cell surface to allow chloride and sodium (salt) to move in and out of the cell. 

  • Lumacaftor + ivacaftor (Orkambi®)

    Lumacaftor and ivacaftor (Orkambi®) is a combination therapy combining lumacaftor, which is designed to fix the defective CFTR protein so that it can move to the proper place on the cell surface, with ivacaftor, which helps improve the function of the protein as a chloride channel on the cell surface.

  • Tezacaftor + ivacaftor (Symdeko®)

    Tezacaftor + ivacaftor (Symdeko®) is a combination therapy combining tezacaftor, a compound designed to move the defective CFTR protein to the proper place in the airway cell surface, with ivacaftor, which helps facilitate the opening of the chloride channel on the cell surface to allow chloride and sodium (salt) to move in and out of the cell.

  • VX-445 + tezacaftor + ivacaftor

    This program is testing VX-445 in combination with tezacaftor and ivacaftor. VX-445 and tezacaftor (VX-661) are new CFTR correctors. Correctors are drugs designed to fix the defective CFTR protein so that it can move to the proper place on the cell surface. Once CFTR protein reaches the cell surface, ivacaftor helps facilitate the opening of the chloride channel to allow chloride and sodium (salt) to move in and out of the cell. 

  • VX-659 + tezacaftor + ivacaftor

    This program is testing VX-659 in combination with tezacaftor and ivacaftor. VX-659 and tezacaftor (VX-661) are new CFTR correctors. Correctors are drugs designed to fix the defective CFTR protein so that it can move to the proper place on the cell surface. Once CFTR protein reaches the cell surface, ivacaftor helps facilitate the opening of the chloride channel to allow chloride and sodium (salt) to move in and out of the cell.

  • Ataluren

    Ataluren (Translarna™) is a novel, small molecule compound designed to enable production of a full-length and functional CFTR protein in individuals with CF who have nonsense mutations. The compound aims to help the body override a premature signal to stop production of the CFTR protein. 

  • QBW251

    QBW251 is a type of CFTR Modulator called a “potentiator”. Similar to the drug ivacaftor, this drug would help to facilitate the opening of the chloride channel on the cell surface. This compound is administered through an oral pill.

  • ABBV-2222 (formerly GLPG2222)

    ABBV-2222 (formerly GLPG2222) is a new CFTR corrector. Correctors are drugs designed to fix and restore the function of the defective CFTR protein. The corrected CFTR then moves to the cell surface, where it functions as a chloride channel and helps maintain the right balance of fluid in the airways.

  • FDL169

    FDL169 is a new CFTR corrector. Correctors are drugs designed to fix and restore the function of the defective CFTR protein. The corrected CFTR then moves to the cell surface, where it functions as a chloride channel and helps maintain the right balance of fluid in the airways.

  • PTI-428 (alone and in combination)

    PTI-428 is a type of CFTR modulator called an amplifier. Amplifiers increase the amount of CFTR protein in the cell. This makes more CFTR protein available for other therapies, such as ivacaftor and lumacaftor, to work on. When combined with other therapies, PTI-428 may increase CFTR function across multiple gene mutations.

    PTI-428 is currently being studied both alone as well as in combination with two other CFTR modulators, PTI-801 and PTI-808.

  • PTI-801 (alone and in combination)

    PTI-801 is a new CFTR corrector. Correctors are drugs designed to fix and restore the function of the defective CFTR protein. The corrected CFTR then moves to the cell surface, where it functions as a chloride channel and helps maintain the right balance of fluid in the airways.

    PTI-801 is currently being studied both alone as well as in combination with two other CFTR modulators, PTI-428 and PTI-808.

  • PTI-808 (alone and in combination)

    PTI-808 is a type of CFTR modulator called a potentiator. Potentiators help facilitate the opening of the chloride channel on the cell surface to allow chloride and sodium (salt) to move in and out of the cell.

    PTI-808 is currently being studied both alone as well as in combination with two other CFTR modulators, PTI-801 and PTI-428.

  • VX-561 (formerly CTP-656)

    VX-561 is an altered form of the potentiator ivacaftor (Kalydeco®). Potentiators are drugs that facilitate the opening of the chloride channel on the cell surface to allow chloride and sodium (salt) to move in and out of the cell. VX-561, or deuterated ivacaftor, may be more stable in the body than regular ivacaftor, which would allow it to be taken once a day.

  • Cavosonstat (N91115)

    Cavosonstat (N91115) is a compound that modulates the function of the defective CFTR protein and decreases inflammation in the lung. Cavosonstat is the first of a new class of compounds that increase levels of an important signaling molecule in the body, called S-nitrosoglutathione or GSNO. Levels of GSNO have been shown to be decreased in people with CF. These novel compounds have been shown to increase the amount of CFTR protein that reaches the cell surface and to stabilize CFTR protein so that its function can be improved. 

     

  • Riociguat

    Riociguat is a novel therapy that stimulates sGC, an enzyme in the cardiopulmonary system and the receptor for nitric oxide (NO). Preclinical data has shown evidence that riociguat can result in improved function of the CFTR protein as a chloride channel, moving salt and fluids in and out of cells.

     

  • VX-152 + tezacaftor + ivacaftor

    This program is testing VX-152 in combination with tezacaftor and ivacaftor. VX-152 and tezacaftor (VX-661) are new CFTR correctors. Correctors are drugs designed to fix the defective CFTR protein so that it can move to the proper place on the cell surface. Once CFTR protein reaches the cell surface, ivacaftor helps facilitate the opening of the chloride channel to allow chloride and sodium (salt) to move in and out of the cell.

  • VX-440 + tezacaftor + ivacaftor

    This program is testing VX-440 in combination with tezacaftor and ivacaftor. VX-440 and tezacaftor (VX-661) are new CFTR correctors. Correctors are drugs designed to fix the defective CFTR protein so that it can move to the proper place on the cell surface. Once CFTR protein reaches the cell surface, ivacaftor helps facilitate the opening of the chloride channel to allow chloride and sodium (salt) to move in and out of the cell.

  • Eluforsen (QR-010)

    Eluforsen (QR-010) is an oligonucleotide designed to repair CFTR-encoded mRNA, which could result in a normal CFTR protein in people who have one or two copies of the F508del mutation. It is delivered via inhalation.

  • MRT5005

    MRT5005 is a new drug designed to restore CFTR function by delivering correct copies of CFTR-encoded mRNA to the lungs. mRNA is a molecule that contains genetic instructions to make proteins. Delivery of CFTR-encoded mRNA would allow the lung cells to create normally functioning CFTR protein, regardless of an individual’s specific CFTR gene mutation. This drug is delivered via inhalation.

  • Arcturus

    This program is developing a potential inhaled therapy to deliver normal CFTR messenger RNA to the lungs. Delivery of CFTR-encoded mRNA would allow the lung cells to create normally functioning CFTR protein, regardless of an individual’s specific CFTR gene mutation. 

  • Editas

    This program is exploring ways to correct common and rare mutations in the CFTR gene using CRISPR/Cas approaches. These technologies aim to edit DNA so that the genetic defect that leads to a faulty CFTR protein is fixed. 

  • Genzyme/Sanofi

    This program is working to identify new therapies designed to fix the defective CFTR protein in people who have the F508del mutation. 

  • Icagen

    This program is working to identify potential therapies for people with CF who have nonsense mutations. Nonsense mutations (also known as “x” or “stop” mutations) in the CFTR gene cause the production of CFTR protein to stop prematurely. Overriding these premature stop signals would allow full-length, functional CFTR protein to be made.

  • Pfizer

    This program is working to identify new therapies designed to fix the defective CFTR protein in people who have the F508del mutation.

  • Reata

    This program is working to expand the number of therapies designed to fix the defective CFTR protein in people who have the F508del mutation. 

  • ReCode Therapeutics

    This program is developing suppressor tRNA delivery to treat nonsense mutations. Nonsense mutations (also known as “x” or “stop” mutations) in the CFTR gene cause the production of CFTR protein to stop prematurely. Delivery of suppressor tRNA would allow lung cells to override these premature stop signals and make full-length, functional CFTR protein.

  • Southern Research Institutes

    This program is working to identify potential therapies for nonsense mutations in the cystic fibrosis-causing gene. 

  • Talee Bio

    This program is working to advance inhaled adeno-associated virus (AAV) delivery of the CFTR gene to the lungs. This would allow the lung cells to create normally functioning CFTR protein, regardless of an individual’s specific CFTR gene mutation. 

  • 4D Molecular Therapeutics

    This program is working to advance inhaled adeno-associated virus (AAV) delivery of the CFTR gene to the lungs. This would allow the lung cells to create normally functioning CFTR protein, regardless of an individual’s specific CFTR gene mutation. 

Mucociliary Clearance

  • Dornase Alfa (Pulmozyme®)

    Dornase alfa (Pulmozyme®) is an inhaled medication that thins and loosens mucus in the airways of people with CF.

  • Hypertonic Saline

    Hypertonic saline as an inhaled therapy is believed to increase hydration of airway surface liquid in people with CF, thereby improving mucus clearance.

  • Inhaled Mannitol (Bronchitol®)

    Bronchitol® is an inhaled dry powder form of mannitol, a naturally occurring osmotic agent, which works by drawing water into the airways. This helps to moisten and thin the sticky mucus found in the lungs of people with CF, making it easier to cough it out.

  • OligoG

    OligoG is a dry powder drug that has been shown to decrease the thickness of mucus in the lungs and may help individuals with cystic fibrosis clear mucus easier. OligoG may also help improve the effectiveness of some antibiotics. It is administered using a dry powder inhaler and also developed as a liquid for use with a nebulizer.

  • SPX-101

    SPX-101 is a compound designed to block the function of the sodium (Na+) channel found in the lungs. These channels move sodium and water away from the airway surface. Blocking these channels may help maintain fluid within the airways to improve mucus clearance. 

  • QBW276

    QBW276 is a compound designed to block the function of the sodium (Na+) channel found in the lungs. These channels move sodium and water away from the airway surface. Blocking these channels may help maintain fluid within the airways to improve mucus clearance. 

  • VX-371 formerly (P-1037)

    VX-371 is a compound designed to block the function of the sodium (Na+) channel found in the lungs. These channels move sodium and water away from the airway surface. Blocking these channels may help maintain fluid within the airways to improve mucus clearance. 

  • AZD5634

    AZD5634 is a compound designed to block the function of the sodium (Na+) channel found in the lungs. These channels move sodium and water away from the airway surface. Blocking these channels may help maintain fluid within the airways to improve mucus clearance. 

  • Denufosol

    This  alternative chloride channel activator has been removed from the pipeline due to lack of efficacy.

  • Ionis

    This program is studying a compound designed to block the function of the sodium (Na+) channel found in the lungs. These channels move sodium and water away from the airway surface. Blocking these channels may help maintain fluid within the airways to improve mucus clearance.

Anti-Inflammatory

  • Ibuprofen

    Ibuprofen is an oral, non-steroidal, anti-inflammatory medication.

  • Acebilustat (CTX-4430)

    Acebilustat (CTX-4430) is an oral anti-inflammatory drug that reduces production of leukotriene B4 (LTB4), a molecule that leads to inflammation and is known to be elevated in people with CF. Reduction of inflammation helps prevent permanent tissue damage in the lungs.

  • Lenabasum (JBT-101)

    Lenabasum (JBT-101) is an oral drug that is aimed at promoting the resolution of inflammation. It is thought to increase production of anti-inflammatory molecules while reducing production of molecules that increase inflammation. Reduction of inflammation helps prevent permanent tissue damage in the lungs.

  • Alpha 1 Anti-Trypsin

    Alpha 1 Anti-trypsin is an aerosolized protease inhibitor with anti-inflammatory properties, derived from human plasma.

  • Docosahexaenoic Acid (DHA)

    Docosahexaenoic acid (DHA) is an omega-3 fatty acid with anti-inflammatory properties.

  • LAU-7b

    LAU-7b is an oral compound. It is a form of the retinoid fenretinide. Retinoids are a group of compounds related to vitamin A. Fenretinide may help reduce the inflammatory response in the lungs of people with CF.

  • GS-5745

    GS-5745 is an antibody that may help reduce inflammation in the lungs, leading to improved lung function in people with CF.

  • KB001-A

    KB001-A is a humanized monoclonal Fab fragment that targets a Pseudomonas aeruginosa virulence factor (Type III secretion system). 

  • Sildenafil

    Sildenafil is a phosphodiesterase inhibitor. Researchers at National Jewish Health in Denver examined whether sildenafil can lower markers of airway inflammation in people with CF.

  • POL6014

    This program is studying a compound designed to block the function of neutrophil elastase. Neutrophil elastase is a type of protein associated with inflammation in the lungs. Blocking the function of neutrophil elastase may minimize tissue destruction and decrease inflammation within the CF airway. 

  • PUR 118

    This compound is an inhaled dry powder formulation of a cationic airway liquid modulator.  It has anti-inflammatory properties and increases mucociliary clearance.

Anti-Infective

  • Amikacin Liposome Inhalation Suspension (Arikayce®)

    Amikacin liposome inhalation suspension (Arikayce®) is an antibiotic to treat chronic lung infections caused by nontuberculous mycobacteria (NTM). 

  • Azithromycin

    Azithromycin is an oral antibiotic with anti-inflammatory properties.

  • Aztreonam (Cayston®)

    Inhaled aztreonam (Cayston®) is an antibiotic that has been approved for people with CF who are chronically infected with Pseudomonas aeruginosa.

  • Inhaled Tobramycin

    TOBI® and  BETHKIS® are aerosolized forms of the antibiotic tobramycin, which is used to treat people with CF who are chronically infected with Pseudomonas aeruginosa. 

  • Tobramycin Inhaled Powder (TOBI® Podhaler™)

    The TOBI® Podhaler™ is the powder form of the inhaled antibiotic tobramycin and is used to treat people with CF who have Pseudomonas aeruginosa. It is taken with an inhaler, making it a convenient alternative to TOBI®, which must be taken using a nebulizer.

  • Inhaled Levofloxacin (Quinsair™)

    Inhaled levofloxacin (Quinsair™) is a formulation of the antibiotic levofloxacin for the management of chronic lung infections caused by Pseudomonas aeruginosa and other bacteria.

  • Vancomycin Inhalation Powder (AeroVanc™)

    Vancomycin inhalation powder (AeroVanc™) is an inhaled dry powder version of the antibiotic vancomycin for the treatment of methicillin-resistant Staphylococcus aureus (MRSA) airway infection in people with CF.

  • Inhaled Nitric Oxide

    Nitric oxide is a gas with antimicrobial properties that may reduce lung infections when inhaled, leading to improved lung function in people with CF.

  • Intravenous Gallium

    Gallium is a molecule, nearly identical to iron, that disrupts iron-dependent biological processes and has been shown to kill antibiotic-resistant strains of Pseudomonas aeruginosa in laboratory research. Gallium has already been approved by the FDA for intravenous use in people and is now being studied for its safety and effectiveness in controlling P. aeruginosa in people with CF.

  • SPI-1005

    SPI-1005 is an oral form of the drug ebselen. Ebselen is a molecule designed to mimic the function of a key enzyme found in the inner ear, glutathione peroxidase (GPx). Regular use of aminoglycoside antibiotics can cause damaging side effects in the inner ear. By mimicking the function of GPx, SPI-1005 may protect the inner ear from damage.

  • ALX-009

    ALX-009 is a combination of two substances found naturally in the body, hypothiocyanite (OSCN-) and lactoferrin. Both of these molecules are part of the body’s natural immune system, but they are deficient in the airway surface liquid of people with CF. By adding both OSCN- and lactoferrin to the airways, ALX-009 may increase the ability of people with CF to fight bacteria without harming their body’s own cells. ALX-009 is delivered via inhalation.

  • SNSP113

    SNSP113 is a new inhaled glycopolymer, a type of molecule being developed to treat infection and inflammation in the lungs. When bacteria infect the lungs of people with CF, it can form structures called biofilms. Biofilms make bacteria more resistant to antibiotics. SNSP113 helps break up biofilms in the lungs, allowing antibiotics to fight bacteria more effectively. SNSP113 may also make mucus thinner and less sticky, reducing inflammation and improving airway clearance. 

  • Inhaled Gallium

    This program is studying an inhaled formulation of gallium. Gallium is a molecule, nearly identical to iron, that disrupts iron-dependent biological processes and has been shown to kill antibiotic-resistant strains of Pseudomonas aeruginosa in laboratory research. Intravenous (IV) gallium has already been approved by the FDA for use in people and is now being studied for its safety and effectiveness in controlling P. aeruginosa in people with CF.

Nutritional-GI-Other

  • AquADEKs

    AquaADEKs® is an oral antioxidant vitamin formulation specifically for people with CF.

  • Pancrelipase Enzyme Products

    These are digestive enzymes that are prepared using pancreases from pigs.

  • RELiZORB®

    RELiZORB® is a digestive enzyme cartridge that connects to enteral tube feeding systems. It is designed to mimic normal pancreatic function by helping to break down the fats in enteral tube feeding formula. By breaking down these fats before the formula is ingested, RELiZORB® may allow for delivery of increased absorbable calories and simplify the use of enzymes in overnight feedings. 

  • Liprotamase

    Liprotamase (Sollpura) is a pancreatic enzyme replacement that is not prepared from animal sources.

  • Burlulipase

    Burlulipase is a liquid solution developed to treat pancreatic insufficiency caused by CF. It is a bacterial lipase that has been shown to be more resistant against inactivation by gastric acid, giving the drug the potential to be more effective than existing pancreatin products derived from pigs. 

  • Glutathione

    Glutathione is an antioxidant that is important to the normal functioning of the intestine and lungs. Glutathione levels have been shown to be lower in people with CF. Oral glutathione may improve growth and decrease gut inflammation in children with CF.

  • MS1819-SD

    MS1819-SD is a non-porcine (not pig-derived) enzyme for individuals with CF who have exocrine pancreatic insufficiency. It is a man-made version of the lipase enzyme from the yeast Yarrowia lipolytica. This drug does not contain any animal products.

To advance drug development and a search for a cure, Cystic Fibrosis Foundation (CFF) has contractual agreements with several companies to receive royalties related to drugs that are developed as a result of CFF funding, see “How Drugs Get on the Pipeline.”

Drug Development Pipeline 101

  • How an Experimental Drug Becomes an Approved Therapy

    Ever wondered what it takes for a potential cystic fibrosis drug to become approved by the U.S. Food and Drug Administration? Here's an explanation of the four phases of clinical research.

  • About the Drug Development Pipeline

    The Cystic Fibrosis Foundation facilitates the development of promising new drugs, and the drug development pipeline enables you to track the progress of these potential therapies.

  • How Drugs Get on the Pipeline

    The Drug Development Pipeline features an extensive list of drugs that are in development or approved for treating cystic fibrosis. For a drug or program to be shown on the pipeline, it must meet certain conditions.

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