Cystic Fibrosis Disease

“Redefining Market for Cystic Fibrosis Disease: Where Strategies Meet Successful Achievements”

“Fighting Cystic Fibrosis Today for a Healthier Future”

“Genetic Mutations: The Door of Cystic Fibrosis”. There are no specific reasons available for mutations in the gene and approximately 12 million people are carriers of the abnormal CFTR gene. Scientists are researching on cause of mutation, early diagnosis, and accurate treatment for cystic fibrosis. Incredible innovations and revolutionary opportunities are transforming the diagnostic and treatment industry of cystic fibrosis.

However, based on CDC data, 1,000 cases of cystic fibrosis are diagnosed each year globally. However, around 70,000 people are suffering from cystic fibrosis disease globally out of which 40,000 patients are from the United States only 20,000 cases are from Europe and 3,000 patients have cystic fibrosis in Canada. In addition to that Ireland has a high incidence of cystic fibrosis, with one in every 1,400 live births.

Cystic fibrosis is a multi-system, autosomal-recessive, and monogenetic life-threatening disease in which exocrine glands of the body become dysfunctional. It has the potential to have substantial impacts on life expectancy as well as the standard of life.

Approximately 2,000 mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene cause cystic fibrosis disease which affects the lungs, pancreas, liver, intestine, reproductive organs, and sweat glands. However, because of improved care lifespan has increased up to 40 years for cystic fibrosis disease.

A symptom of cystic fibrosis varies from person to person and includes persistent coughing, Lung infections, nasal polyps, chronic sinus infection, and rectal prolapse.  However, according to the CFFPR report, cystic fibrosis affects 6% of people below the age of 18 and only 0.1% above the age of 30.

However, cystic fibrosis may lead to other illness like respiratory-related disorder, cancer, and lung-related diseases which causes the death of patients. The following chart shows the causes of death in percentage in the UK during the 2021 period.

As shown above, in the UK respiratory and cardiorespiratory-related infection causes major deaths accounting for 61.1%. However, 13% of deaths occur due to organ transplant-related cases and 7.2% and 6.9% of deaths are reported due to not known cases and trauma or suicide. In addition to that cancer, and liver disease measure 5.8%, and 3.2% death percentages.

The common risk factors for cystic fibrosis disease are age, family history with abnormal alleles for the CF gene, environment, and lifestyle. At the current time prevention of cystic fibrosis is not possible but it is prevented in the future by recognizing all carriers for cystic fibrosis.

Discovery in the preventive, diagnostic, and treatment fields has been identified as an important element of expanding the marketplace on a global scale for industry players. As a result, it contributes to the globalization of developing products.

DiseaseLandscape Insights assists the stakeholders by providing data related to the cystic fibrosis marketplace, knowledgeable choices in healthcare equipment, therapies, and diagnostic techniques where various new technologies and innovations are impacting the market of disease and driving higher expansion.

Cystic fibrosis is spreading throughout the world creating a drastic shift in an individual’s standard of life. The present condition of cystic fibrosis disease would propel the industries in the appropriate direction and provide emerging opportunities for a variety of market leaders in the domain of landscape insights.

Cystic Fibrosis Diagnostic Analysis-

“From Early Diagnosis to Healthy Perfection”

Diagnosis of Cystic Fibrosis is done by using a combination of tests. As cystic fibrosis disease damages various organ systems diagnostic tests related to all the possible organs are performed. There are a number of tests performed to diagnose cystic fibrosis which are listed below-

1. Blood Test- Blood tests are performed to check the level of a chemical called Immunoreactive Trypsinogen (IRT). An increased level of IRT indicates the dysfunction of the pancreatic gland which generally gets affected in cystic fibrosis disease. However, elevation in the level of IRT also occurs in premature or stressed infants so there is a need to conduct further tests to confirm the cystic fibrosis disease.

2. Sweat Test-Sweat test is used to measure the chlorine concentration in the patient’s body. A person having cystic fibrosis shows a high amount of chlorine because of abnormalities in the CFRT gene. The sweat test is done at the care centers which are cystic fibrosis Foundation-accredited to obtain accurate results.

3. Genetic Test-A genetic test is the most accurate test to diagnose cystic fibrosis disease because it identifies the exact type and place of mutation in the gene. NIH states that approximately 70 % of cystic fibrosis patient contains deltaF508 mutation. If the test is positive that means, there is ~99% chance that the patient is a carrier of cystic fibrosis.

4. Imaging Tests – An imaging test is the other important method to diagnose the disease, as well as the organ affected due to cystic fibrosis, and accurately, helps in understanding the severity of the cystic fibrosis disease. Following are the brief classifications of imaging tests.

• CT Scan – CT scan is used to take detailed images of the chest, abdomen, and sinus. This image helps to check for the level of Cystic Fibrosis by examining for aberrant mucus and dilated airways in the lungs and also for nasal polyps which all indicate the presence of Cystic Fibrosis Disease.
• Chest X-ray – A chest X-ray test is performed to evaluate the lung infection which needs to be treated with antibiotics. Chest X-ray tests are also useful to rule out other respiratory diseases such as pneumonia and to confirm that a patient is suffering from Cystic Fibrosis. 
• Magnetic Resonance Imaging – MRI is used to check detailed pictures of the digestive tract and lungs. MRI helps to analyze damage caused by Cystic Fibrosis. Instead of MRI, CT scans and chest X-rays are most commonly used for the diagnosis of Cystic Fibrosis because these tests give accurate results.

5. Lung Function Test – The liver function test is used to check the damage and infection to the liver due to Cystic Fibrosis. According to NIH, about 5 to 10% of people suffering from cystic fibrosis develop a liver-related disorder called multilobular cirrhosis. 

6. Sputum Culture Test –A Sputum culture test is performed to check the type of infecting bacteria in the patient. Analysis of sputum helps the clinician to advise proper antibiotics to the patient. The side effect of a sputum culture test is the patient may feel sore after the procedure.

Among all the tests listed above, the sweat test is the standard test and most widely used test for the diagnosis of cystic fibrosis. The reason behind this is that a sweat test does not cause any side effects and is comparatively cheaper than a genetic test. It also detects one of the major biomarkers of cystic fibrosis which is chlorine. More innovations in sweat test products are another field for market players to grow.

The global cystic fibrosis diagnostic market is highly competitive and continuously growing. Market players have scope in the development of various diagnostic kits by using new technologies. Along with that launching of cost-efficient and quick diagnostic tests is important in the diagnostic field of cystic fibrosis disease.

DiseaseLandscape Insights assists the industry players in the development of various diagnostic kits, the use of new technologies for improving the existing diagnostic method, and providing data on current market players and their products for more knowledge about market dynamics. By using the data from DLI, stakeholders choose the path of their business and implement strategies for the growth of the market.

The following table lists the names of the market leaders who are redefining healthcare with their forefront diagnostic innovations:

Cystic Fibrosis Treatment Analysis-

"Achieving Good Health: Reviewing Modern Advances in Cystic Fibrosis Treatment"

The number of cases of cystic fibrosis disease is rapidly growing. This has resulted in a massive increase in research to identify new medications to treat the condition. Despite all scientific efforts, there are presently no effective therapies for cystic fibrosis disease, although there is some alternative medicine to prevent and decrease the illness's symptoms. 

1. Medications- 

Antibiotics – Antibiotic treatment of chronic bacterial infections is recognized as the basis of cystic fibrosis therapy and has significantly contributed to patients' greatly improved life expectancy in recent decades. Antibiotics used to reduce bacterial infection are Levofloxacin, Colistin, Tobramycin, and the Beta-Lactam class of antibiotics.

• Anti-Inflammatory Medicines –Anti-inflammatory agents are used to reduce the inflammation caused by cystic fibrosis in the kidney, stomach, and lungs. Ibuprofen or corticosteroids are some of the anti-inflammatory medicines used currently but they show some side effects like bone thinning, increased BP and sugar, etc. 

• Bronchodilators-Bronchodilators are inhaled medications that help you breathe better by opening and relaxing the airways in the patients. 

• CFTR Modulators-The CFTR modulators medicine is given to patients suffering from cystic fibrosis. These medications help to improve the function of the mutated CFTR gene. They also help in reducing the salt content of sweat and improve lung and digestive tract function. The combined drug is the first licensed therapy that shows benefits for up to ~90% of cystic fibrosis patients reported by NIH.

Below is the name of CFTR modulators which has been approved by the FDA to treat cystic fibrosis by targeting mutated gene-

• Trikafta- It is a combination of Elexacaftor, Ivacaftor, and Tezacaftor drug which is used to treat Cystic Fibrosis patients aged 12 years and above. 
• Symdeko- The formulation of Symdeko is a mixture of Lumacaftor and Ivacaftor drugs. It is approved for patients aged 6 years and above.  
• Orkambi- It is a combination of lumacaftor and ivacaftor drugs that are used to treat patients having the 2 years and above age group. 
• Kalydeco- It contains Ivacaftor drug which is used for 6 months and older babies. 

2. Surgery 

• Lung Transplant- In severe cases of cystic fibrosis lung transplant surgery is performed to remove a diseased or infected lung and replace it with a healthy lung. However, a lung transplant does not cure cystic fibrosis but extends the life of patients by 5 to 10 years. Some patients live more than 15 years also. Furthermore, there is a waiting for a transplant of the lung which is about 18 months so there might be chances that the patient may die in the waiting period.

3. Airway Clearance Therapy-Airway clearance therapy is done by using a device that helps to clear airway secretions by shaking the mucus in the airways. Unfortunately, due to a variety of treatment hurdles, retention of Cystic Fibrosis patients to airway clearance therapy is roughly 40-47%.

DiseaseLandscape Insights supports the market participants to transform and expand in the treatment domain of cystic fibrosis disease in order to improve patient retention, explore innovations in the treatment area, and achieve new business heights.

DLI equips market players to be one step ahead of the competition with our thorough analysis of treatment options, service contributions, inventory of products, and market competition. As a devoted partner to the pharmaceutical sector, we offer extensive support in arranging, carrying out, and evaluating clinical studies for the development of novel new drugs.

The worldwide cystic fibrosis disease market is highly fragmented and owes a prominent market player with a global presence.  The market leaders in the table below are reinventing the landscape and pushing innovation to achieve better health outcomes.

Services Provided by DiseaseLandscape Insights (DLI) in the continuously Expanding Cystic Fibrosis Disease Market-

"Converting Records into Opportunities for Market Expansion and Establishing the Cystic Fibrosis-Free Population."

DiseaseLandscape Insights (DLI) helps to develop and set up commercial cystic fibrosis disease services for prominent worldwide players. DLI assists key players by providing market analysis of product launch expansion, research, and development in the field of drug discovery and launch phases. DLI provides services in the placement of resources, risk evaluation, and safety of drugs, as well as insights for long-term planning into disease dynamics.

Additionally, DLI offers data for clinical and drug safety, medication-related papers, as well as regulatory body-related documents. Stakeholders access journal articles, publication abstracts, marketing materials, the websites of major medical organizations to discover even more, and data related to various preventive action plans provided by global organizations to keep the stakeholders alert and clear.

The business element of cystic fibrosis disease focuses on a variety of services and goods, ranging from management and prevention to further therapy and research. One of the services provided to make people aware of prevention of cystic fibrosis disease is given below:

• The Cystic Fibrosis Foundation aims to shape public policy to aid its efforts for the cure of cystic fibrosis disease and allow the chance to live full, healthy lives to all individuals suffering from cystic fibrosis by educating elected officials and other government decision-makers about the needs of the cystic fibrosis community

Regulatory Framework for Cystic Fibrosis Disease-

"Regulatory Adherence: The Route to a Sustainable Business Environment"

DiseaseLandscape Insights considers the regulatory ecosystem of different countries for cystic fibrosis disease. DLI provides data about risk assessment laws, import/export legislation, and surveillance to the key player. By choosing DLI services market players will definitely prevent and respond to cystic fibrosis outbreaks for the safety and well-being of the impacted population.

However, there are different programs that have been organized to spread awareness about cystic fibrosis disease. To restart the Adult Cystic Fibrosis Assistance Program (ACFAP) in legislation and financing, the CF Foundation wrote a letter to the New York State Assembly Health Committee in February 2023. 

In addition to that, considering public health the CF Foundation requested the Advisory Committee on Immunization Practices (ACIP) in September 2021, seeking clarification on eligibility for Comirnaty booster doses and assurance that persons with cystic fibrosis disease are included in the classification of patients at high risk for severe COVID-19 disease.

Overall, regulatory regulations have a considerable influence on the cystic fibrosis disease sector, and DLI offers an in-depth evaluation of several regional standards that are now in place, as well as prospective future changes in the cystic fibrosis disease market.

Competitive Analysis-

“Maintaining Competitive Edge in the Cystic Fibrosis Market"

Several international industries are concentrating on manufacturing new products and enhancing the use of technology to improve current processes to increase their level of competition. Industries are also concentrating on ways to expand their product offerings, most commonly through collaborations, regulatory approval for medical research and devices, and an emphasis on cooperation.

However, Competitors like Gilead Sciences International Ltd. have found out that, persons with cystic fibrosis develop chronic pseudomonal airway infection which is the leading cause of disease and mortality. Gilead Sciences International Ltd. sent the New Drug application to the FDA for Aztreonam lysine medicine. 

In addition to that, Eloxx Pharmaceuticals has launched ELX-02 medicine which has the capacity to restore the CFTR activity. A clinical trial supports the use of ELX-02 in Cystic Fibrosis Patients twice weekly. The overall revenue of Eloxx Pharmaceuticals has increased due to the innovation in the treatment field of cystic fibrosis.

Research and developments of existing industries have increased the awareness of their products as well as diseases in the market. Manufacturing and marketing authority of such products has increased the overall revenue of the companies. However, it enhanced the collaborations in the industry and established another standard for other lead players.

DiseaseLandscape Insights helps the industry player by providing intense knowledge about all the existing market players, their innovations, strategies, and collaborations and helps in selecting the correct marketplace to grow exponentially. 

Market Trends-

“Reviewing Market Dynamics to Accelerate the Business”

The market is rising as a result of the development of numerous medications for the treatment of cystic fibrosis condition, which cannot cure the condition entirely. To develop a fully curative treatment for cystic fibrosis disease, a lot of research is necessary. Market participants have several prospects in both the diagnostic and therapy domains.

However, to improve patient health and make people aware of the disease, the Cystic Fibrosis Foundation introduces Path to a Cure, a $500 million research portfolio designed to advance therapies for all individuals living with Cystic Fibrosis, including those who do not currently have a breakthrough therapy. Market players have numerous opportunities in the treatment area of cystic fibrosis disease.

Moreover, many researchers are working on Induced Pluripotent Stem Cell Therapy to cure cystic fibrosis disease. In laboratory studies, researchers have used induced pluripotent stem cells and achieved 20% repair in the mutated cells. Key players have the scope to innovate new sources of stem cells, cost-efficient technique development, and other therapies and inventions to completely cure cystic fibrosis disease.

Also, improvements in research and development for the treatment and diagnosis of cystic fibrosis disease have expanded the chances for numerous market participants. New medicine production, novel therapy, and diagnostic kit manufacture have raised market size and hence the income of existing important players. Innovative technology, regulatory approval, government initiatives for patient health, and the introduction of laws and regulations in the research sector have expanded the market's influence.

Clinical Trial Data Assessment-

The crucial role of clinical trials in the practice of evidence-based medicine and health care reform is highlighted by the government's increasing concentration on comparing the effectiveness of studies. One of the main goals of reforming healthcare is achieved by the market participants accurately examining medical therapy with the use of clinical data. 

The table below includes the study names of the ongoing clinical trials as well as the stages at which they are taking place.

Conclusion-

DiseaseLandscape Insights (DLI) assists industry players in developing and implementing viable treatment plans for preventing and managing cystic fibrosis disease occurrences. Furthermore, there is an increasing need for diagnostic tools, clinical evaluations, and cystic fibrosis surgery as a result of greater awareness and predicted epidemics.

DiseaseLandscape Insights (DLI) provides the necessary information to key players who are involved in the manufacturing of treatment products. Support from DiseaseLandscape Insights makes it easy for market participants to organize and carry out clinical trials for innovative medicines and pharmaceuticals, patient recruiting tactics, and regulatory compliance. This enables businesses to conduct concentrated R&D, learn about contract manufacturing groups, find raw material sources, and ensure legal compliance.  All in all, DLI assists all key players in gaining a stronger foothold in the cystic fibrosis disease domain.