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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Restore CFTR Function Learn more ›
- Pre-clinical
- Phase One
- Phase Two
- Phase Three
- To Patients
- Elexacaftor + tezacaftor + ivacaftor (Trikafta®) › : To Patients ›
- Ivacaftor (Kalydeco®) › : To Patients ›
- Lumacaftor + ivacaftor (Orkambi®) › : To Patients ›
- Tezacaftor + ivacaftor (Symdeko®) › : To Patients ›
- Ataluren › : Phase Three ›
- VX-659 + tezacaftor + ivacaftor › : Phase Three ›
- QBW251 › : Phase Two ›
- ABBV-2222 (formerly GLPG2222) › : Phase Two ›
- ABBV-3067 › : Phase Two ›
- ELX-02 › : Phase Two ›
- Nesolicaftor (PTI-428) › : Phase Two ›
- Posenacaftor (PTI-801) › : Phase Two ›
- Dirocaftor (PTI-808) › : Phase Two ›
- VX-121 › : Phase Two ›
- VX-561 (formerly CTP-656) › : Phase Two ›
- Cavosonstat (N91115) › : Phase Two ›
- FDL169 › : Phase Two ›
- Riociguat › : Phase Two ›
- VX-152 + tezacaftor + ivacaftor › : Phase Two ›
- VX-440 + tezacaftor + ivacaftor › : Phase Two ›
- Eluforsen (QR-010) › : Phase One ›
- ABBV-191 › : Phase One ›
- MRT5005 › : Phase One ›
- Arcturus › : Pre-clinical ›
- Icagen › : Pre-clinical ›
- Reata › : Pre-clinical ›
- ReCode Therapeutics › : Pre-clinical ›
- Southern Research Institutes › : Pre-clinical ›
- Spirovant Sciences › : Pre-clinical ›
- 4D Molecular Therapeutics › : Pre-clinical ›
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Mucociliary Clearance Learn more ›
- Pre-clinical
- Phase One
- Phase Two
- Phase Three
- To Patients
- Dornase Alfa (Pulmozyme®) › : To Patients ›
- Hypertonic Saline › : To Patients ›
- Inhaled Mannitol (Bronchitol®) › : To Patients ›
- BI 1265162 › : Phase Two ›
- IONIS-ENaC-2.5 › : Phase Two ›
- OligoG › : Phase Two ›
- QBW276 › : Phase Two ›
- SPX-101 › : Phase Two ›
- VX-371 formerly (P-1037) › : Phase Two ›
- AZD5634 › : Phase One ›
- ETD002 › : Phase One ›
- Denufosol › : Phase One ›
-
Anti-Inflammatory Learn more ›
- Pre-clinical
- Phase One
- Phase Two
- Phase Three
- To Patients
- High dose ibuprofen for CF › : To Patients ›
- Lenabasum (JBT-101) › : Phase Two ›
- Acebilustat (CTX-4430) › : Phase Two ›
- Alpha 1 Anti-Trypsin › : Phase Two ›
- Docosahexaenoic Acid (DHA) › : Phase Two ›
- LAU-7b › : Phase Two ›
- GS-5745 › : Phase Two ›
- KB001-A › : Phase Two ›
- Sildenafil › : Phase Two ›
- CB-280 › : Phase One ›
- Lonodelestat (formerly POL6014) › : Phase One ›
- PUR 118 › : Phase One ›
-
Anti-Infective Learn more ›
- Pre-clinical
- Phase One
- Phase Two
- Phase Three
- To Patients
- Amikacin Liposome Inhalation Suspension (Arikayce®) › : To Patients ›
- Azithromycin › : To Patients ›
- Aztreonam (Cayston®) › : To Patients ›
- Inhaled Tobramycin › : To Patients ›
- Tobramycin Inhaled Powder (TOBI® Podhaler™) › : To Patients ›
- Inhaled Levofloxacin (Quinsair™) › : Phase Three ›
- Vancomycin Inhalation Powder (AeroVanc™) › : Phase Three ›
- Intravenous Gallium › : Phase Two ›
- AP-PA02 › : Phase Two ›
- Inhaled Nitric Oxide › : Phase Two ›
- SPI-1005 › : Phase Two ›
- Inhaled Molgramostim › : Phase Two ›
- ALX-009 › : Phase One ›
- Inhaled Gallium › : Phase One ›
- SNSP113 › : Phase One ›
- Inhaled Murepavadin › : Pre-clinical ›
- Matinas BioPharma › : Pre-clinical ›
- Microbion › : Pre-clinical ›
- Respirion › : Pre-clinical ›
- TB Alliance › : Pre-clinical ›
-
Nutritional-GI-Other Learn more ›
- Pre-clinical
- Phase One
- Phase Two
- Phase Three
- To Patients
Your search did not match any drugs. Please consider broadening your search.
Drug Descriptions
Restore CFTR Function
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Elexacaftor + tezacaftor + ivacaftor (Trikafta®)
Elexacaftor + tezacaftor + ivacaftor (Trikafta®) is a combination therapy combining three CFTR modulators. Elexacaftor and tezacaftor are CFTR correctors, a type of modulator designed to fix the defective CFTR protein so that it can move to the proper place on the cell surface. Ivacaftor is a potentiator. Once CFTR protein reaches the cell surface, potentiators help facilitate the opening of the chloride channel to allow chloride and sodium (salt) to move in and out of the cell.
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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.
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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.
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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.
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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.
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VX-659 + tezacaftor + ivacaftor
This program tested VX-659 in combination with tezacaftor and ivacaftor. VX-659 and tezacaftor (VX-661) are 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.
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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.
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ABBV-2222 (formerly GLPG2222)
ABBV-2222 (formerly GLPG2222) is a type of CFTR modulator called a 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.
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ABBV-3067
ABBV-3067 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.
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ELX-02
ELX-02 is a compound designed to restore CFTR function in 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. ELX-02 is intended allow lung cells to override these premature stop signals and make full-length, functional CFTR protein.
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Nesolicaftor (PTI-428)
Nesolicaftor is a new type of modulator called an amplifier. Amplifiers increase the amount of CFTR protein in the cell. This makes more CFTR protein available for other therapies, such correctors and potentiators, to work on.
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Posenacaftor (PTI-801)
Posenacaftor is a type of CFTR modulator called a 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.
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Dirocaftor (PTI-808)
Dirocaftor 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.
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VX-121
VX-121 is a 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.
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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.
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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.
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FDL169
FDL169 is a 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.
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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.
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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.
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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.
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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.
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ABBV-191
This program is studying ABBV-191, a type of CFTR modulator called a potentiator.
Once CFTR protein reaches the cell surface, potentiators help facilitate the opening of the chloride channel to allow chloride and sodium (salt) to move in and out of the cell. This helps maintain the right balance of salt and water on the airway surface.
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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.
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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.
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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.
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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.
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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.
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Southern Research Institutes
This program is working to identify potential therapies for nonsense mutations in the cystic fibrosis-causing gene.
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Spirovant Sciences
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.
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4D Molecular Therapeutics
This program is working to advance a gene delivery vehicle that targets cells in the lung. 4D-710 is a customized adeno-associated virus (AAV) vector designed to deliver a healthy CFTR gene specifically to cells in the lungs of people with CF. This would allow the lung cells to create normally functioning CFTR protein, regardless of an individual’s specific CFTR gene mutation.
Mucociliary Clearance
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Dornase Alfa (Pulmozyme®)
Dornase alfa (Pulmozyme®) is an inhaled medication that thins and loosens mucus in the airways of people with CF.
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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.
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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.
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BI 1265162
BI 1265162 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.
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IONIS-ENaC-2.5
IONIS-ENaC-2.5Rx 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.
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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.
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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.
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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.
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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.
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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.
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ETD002
ETD002 is a compound designed to increase the activity of TMEM16A, a chloride channel found on the surfaces of airway cells. Similar to the CFTR channel, the TMEM16A channel helps regulate the amount of salt and fluids on the airway surface. Increasing the activity of TMEM16A may help increase the amount of fluid within the airways to improve mucus clearance.
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Denufosol
This alternative chloride channel activator has been removed from the pipeline due to lack of efficacy.
Anti-Inflammatory
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High dose ibuprofen for CF
Ibuprofen is an oral, non-steroidal, anti-inflammatory medication.
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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.
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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.
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Alpha 1 Anti-Trypsin
Alpha 1 Anti-trypsin is an aerosolized protease inhibitor with anti-inflammatory properties, derived from human plasma.
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Docosahexaenoic Acid (DHA)
Docosahexaenoic acid (DHA) is an omega-3 fatty acid with anti-inflammatory properties.
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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.
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GS-5745
GS-5745 is an antibody that may help reduce inflammation in the lungs, leading to improved lung function in people with CF.
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KB001-A
KB001-A is a humanized monoclonal Fab fragment that targets a Pseudomonas aeruginosa virulence factor (Type III secretion system).
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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.
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CB-280
CB-280 is an oral drug designed to increase the amount of arginine in the lungs. Arginine is a molecule that occurs naturally in the body, and is important for the lungs to produce nitric oxide, a gas that helps the lungs fight infection.
Sputum from people with CF has been shown to contain lower amounts of arginine and nitric oxide than normal. Lower nitric oxide levels are associated with worsened lung function and increased infection. Increasing arginine levels may increase the production of nitric oxide, which would strengthen the immune response, reduce inflammation and improve lung function.
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Lonodelestat (formerly POL6014)
Lonodelestat is 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.
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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
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Amikacin Liposome Inhalation Suspension (Arikayce®)
Amikacin liposome inhalation suspension (Arikayce®) is an antibiotic to treat chronic lung infections caused by nontuberculous mycobacteria (NTM).
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Azithromycin
Azithromycin is an oral antibiotic with anti-inflammatory properties.
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Aztreonam (Cayston®)
Inhaled aztreonam (Cayston®) is an antibiotic that has been approved for people with CF who are chronically infected with Pseudomonas aeruginosa.
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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.
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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.
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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.
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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.
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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. It is currently being studied for its effectiveness in treating infections in people with CF.
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AP-PA02
This program is studying AP-PA02, a type of phage therapy designed to fight Pseudomonas aeruginosa infections in people with CF. Bacteriophages are specialized viruses that kill very specific bacterial strains. They are found in the environment and are the predators of bacteria in nature. “Phage therapy” refers to the use of these bacteriophages to treat an infection in a person.
AP-PA02 is a mix of multiple different types of phages that is also known as an inhaled cocktail. In lab tests AP-PA02 was able to kill more than 80 percent of Pseudomonas strains from people with CF.
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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.
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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.
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Inhaled Molgramostim
Inhaled molgramostim is a form of granulocyte macrophage-colony stimulating factor (GM-CSF). GM-CSF is a protein that occurs naturally in the human immune system and plays an important role in activating the immune system to kill bacteria and other infections. Because inhaled mogramostim helps to stimulate the part of the immune system that fights Nontuberculous mycobacteria (NTM), it may be able to help the body clear NTM infection.
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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.
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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.
Gallium can also be administered intravenously. 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 infections in people with CF.
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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.
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Inhaled Murepavadin
This program is studying an inhaled version of murepavadin, an antibiotic that targets multi-drug resistant Pseudomonas aeruginosa infections in people with cystic fibrosis. An inhaled version could make it easier for someone with a Pseudomonas infection to take the drug from home. In addition, the drug, which targets the outer membrane of bacteria, has the potential to be a once-a-day treatment.
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Matinas BioPharma
This program is studying MAT2501, an oral version of the drug amikacin. Amikacin is an antibiotic that targets nontuberculous mycobacteria (NTM) infections in people with cystic fibrosis. An oral formulation of the drug may reduce the risk of known amikacin side effects such as hearing loss and kidney problems. An oral version would also make it easier for someone with an NTM infection to take the drug from home.
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Microbion
This program is studying pravibismane, a new inhaled antibiotic. In the lungs of people with CF, bacteria may form protective layers -- known as biofilms -- that make them more difficult to kill. Pravibismane may help break down biofilms and kill drug-resistant bacteria, such as multi-drug resistant Pseudomonas aeruginosa and nontuberculous mycobacteria.
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Respirion
This program is developing and testing a new antibiotic therapy for cystic fibrosis. This therapy is a combination of two existing approved drugs: the antibiotic tobramycin and another compound, calcium EDTA. When bacteria infect the lungs of people with CF, they can form structures called biofilms. Biofilms make bacteria more resistant to antibiotics. The compound calcium EDTA may help break down biofilms in the lungs, allowing antibiotics to fight bacteria more effectively.
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TB Alliance
This program will identify and test potential treatments for infections caused by Mycobacterium abscessus (M. abscessus) and Mycobacterium avium complex (MAC), two types of nontuberculous mycobacteria (NTM).
Nutritional-GI-Other
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AquADEKs
AquaADEKs® is an oral antioxidant vitamin formulation specifically for people with CF.
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Pancrelipase Enzyme Products
These are digestive enzymes that are prepared using pancreases from pigs.
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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.
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Liprotamase
Liprotamase (Sollpura) is a pancreatic enzyme replacement that is not prepared from animal sources.
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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.
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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.
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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 a lipase enzyme taken from the yeast Yarrowia lipolytica. This drug does not contain any animal products.
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Synspira
This program is studying SNSP003, a non-porcine (not pig-derived) enzyme replacement therapy for individuals with CF who have exocrine pancreatic insufficiency. It is a man-made version of a lipase enzyme taken from bacteria, and it does not contain any animal products.
SNSP003 may improve dosing convenience by reducing pill burden compared to current porcine-based pancreatic enzyme replacement therapies (PERT). SNSP003 is also being developed in a formulation for pediatric and adult patients who are unable to swallow capsules.
To advance drug development and a search for a cure, the Cystic Fibrosis Foundation (CFF) has contracts with several companies to help fund the development of potential treatments and/or cures for cystic fibrosis. Pursuant to these contracts, CFF may receive milestone based payments, equity interests, royalties on the net sales of therapies, and/or other forms of consideration. Resulting revenue received by CFF is used in support of our mission. See “How Drugs Get on the Pipeline.” for more.
Drug Development Pipeline 101
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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.
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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.
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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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