The global cystic fibrosis therapeutics market size was valued at USD 6.33 billion in 2021 and is expected to reach USD 13.88 billion in 2030 expanding at a CAGR of 9.12%.
Cystic fibrosis is a genetic condition that mostly impacts the lungs but can also impact other organs and systems in the body such as the pancreas, liver, and kidneys. This condition is brought on by a mutation in the CFTR gene, which stands for cystic fibrosis transmembrane conductance regulator. This gene, which is found on chromosomal number 7, is important for regulating perspiration, mucus, and other bodily fluids. Its location is on chromosome number 7. However, mutation of this gene leads to absence of CFTR function, which in turn leads to loss of chloride and changes the structure of the protein. This is a consequence of the chain reaction that begins when this gene is mutated. Patients suffering from this condition have trouble breathing, the development of mucus, the pathway of the pancreas, the bile duct, the gut, and the salivary gland are all affected by the difficulties. The sweat test and genetic testing are the two primary methods for diagnosing cystic fibrosis. The treatment of cystic fibrosis includes the use of physical therapy, which assists in the decrease of mucus formation and fights against infections of an organ. [Cystic fibrosis] In addition, the usage of medications like Pulmozyme, Kalydeco, and a variety of others is an essential part of the therapy for cystic fibrosis. Several methods, including inhalation and oral administration, are available for delivering these medications to the patient
A significant element that is anticipated to promote the advancement of the market is the growing awareness in developing nations regarding the oral medication therapies for diseases like cystic fibrosis, such as pancreatic enzyme supplements and CFTR modulators, and the cost affordability of these medicines. Cystic fibrosis is a condition that can cause damage to the lungs and other organs in the body. To improve the overall state of public health, a great number of healthcare organisations and medical institutes are working to raise knowledge about the treatment of cystic fibrosis. Through the use of social media, the Cystic Fibrosis Foundation helped raise awareness of cystic fibrosis by sharing personal experiences about being diagnosed, the different therapies available, and many other topics.
The industry has been sparked by a rise in the number of cystic fibrosis patients worldwide. Cystic fibrosis pharmacotherapy has led to a number of advancements in the treatment of this condition, which is positively affecting market growth. The industry's rise is also being fuelled by market trends that are now being used in the treatment of the ailment. This is because a growing number of candidates for therapeutic treatment are being produced by important corporations and are now in the pipeline. As a result, pharmaceutical firms are being urged to create novel therapies that target flaws in the CFTR protein. Additionally, the launch of Ivacaftor/Lumacaftor, a potentiator that improves quality of life for individuals who suffer from the ailment, is also assisting in the market's growth.
In addition, the expansion of research and development activities as well as technical advancements made by key firms are contributing to the growth of this market via the introduction of new products. For instance, the National Research Council has developed a diagnostic smart patch for cystic fibrosis that may be worn to detect the ailment at an earlier stage. This patch makes use of wireless technologies to provide digital power.
CF is quickly becoming the most prevalent genetic illness in every region of the world. This frequently influences several organ systems across the body. According to the Cystic Fibrosis Patient Registry, it is believed that more than 30,000 persons in the United States are now afflicted with this ailment, and it is projected that around 70,000 people throughout the world are afflicted with this condition. In addition to this, it is also mentioned that each year there are roughly 1,000 new cases of the condition that are identified. As a result, it is anticipated that the growing frequency of respiratory issues, gastrointestinal complications, and reproductive diseases in patients with CF would also contribute to an increase in the demand for the treatment of the illness throughout the course of the projection period.
In addition, the implementation of innovative treatment options for early detection is expanding the patient population for better treatment, which is expected to have an impact on the expansion of this market throughout the course of the projected period.
The imminent arrival of new medications for the treatment of cystic fibrosis (CF) is a welcome development that is contributing to the expansion of the market. During the time period in question, the development of new pharmaceuticals that are targeting the deficiency in cells of people who are afflicted with illnesses is expected to boost the growth of the market. The fact that this treatment is expected to be transformative for a great number of patients is driving the expansion of the market. In addition, a growth in the number of people who are affected by the condition is believed to be the most important element driving the market. Because of this, the launch of freshly developed pipeline pharmaceuticals that are necessary for the treatment of very ill patients is gaining momentum. In addition to this, many possible candidates are now in their last phases of development and are forecasting the expansion of the market in the near future. For instance, Vertex Pharmaceuticals Incorporated recently revealed that its phase Ill clinical research on inhaled mannitol suggested for the treatment of mucociliary clearance had yielded favourable findings. In addition to this, technologically enhanced devices utilised for a variety of critical situations such as airway clearing procedures, mucous thinners, and others are prepared to spike the cystic fibrosis therapeutics market over the projection period.
Despite the fact that it has been observed that the growth of the market will be projecting over the years due to an increase in awareness of the treatment options, a rise in the prevalence of the disease across the globe, an increase in the launch of new drugs, and other developments, there are some factors that are negatively influencing the growth of the market. The fact that the items might cause undesirable side effects including infections of the upper respiratory tract or passages, difficulties with the liver, chest discomfort, and a rise in blood pressure, among other issues, may impede the expansion of the market. In addition to this, the high expense of therapy among CF patients who are in need of a lung transplant, liver transplant, or any of the other types of transplants is another factor that is preventing the market from expanding. In addition, some of the reasons that are having a negative impact on the cystic fibrosis market are extended FDA approvals, product recalls, severe regulations, and others.
The global cystic fibrosis therapeutics market is segmented by route of administration, drug class, and region.
On the basis of the Drug class, the market may be broken down into four subcategories: mucolytics, bronchodilators, pancreatic enzyme supplements, and CFTR modulators. Maintaining lung function while controlling respiratory infection and cleaning mucus from airways, regulating nutritional therapy, and limiting complications are the basic concerns in the management of cystic fibrosis (CF). In the year 2012, the pharmaceutical company Kalydeco became the first company to commercialise a CFTR modulator medication. It was the sole CFTR modulator medication available for purchase as of the year 2014.
As a result of the introduction of the brand-new medication Orkambi in 2016, sales of CFTR modulators brought in the most money, or around 47.3 percent of total sales. The ineffective protein that is produced because of the CF gene can have its function restored thanks to this medication. This kind of medication treats the ailment's root cause in a way that is both effective and safe. One of the most important things that may be considered for its part is the product launch that took place in 2015. The market for CFTR modulators would expand at a profitable rate of development of 24.1 percent throughout the duration of the projection.
In 2016, the oral route had the biggest share—nearly 63.7 percent—due to the benefits of its administration, including simplicity and ease of consumption without the assistance of a healthcare expert. One of the key reasons projected to drive the need for these medications is growing understanding of the advantages of oral medications, such as CFTR modulators and pancreatic enzyme supplements.
CFTR modulators like KALYDECO and ORKAMBI and pancreatic enzyme supplements like Creon/Zenpep, PERTZYE, Ultresa, and Viokace are a few of the authorised therapies that are taken orally. The oral medication sector is anticipated to be driven by these treatments' high commercial availability and enormous demand for CF treatment. According to the U.S. Patient Registry 2015, 41.9 percent of children between the ages of 3 and 5 and 21.5 percent of children between the ages of 0 and 3 were given hypertonic saline prescriptions. One of the main causes of the rising need for oral medication formulations is the rising prevalence of CF in new-born infants.
One of the major reasons projected to drive the market is an increase in clinical studies to broaden the patient population. Additionally, the market is anticipated to develop at a profitable rate of 20.1 percent during the projected period. Another factor that is anticipated to increase demand for oral medications is the rising number of private and public entities investing in this market.
The market is divided into five regions based on geography: North America, Europe, Asia Pacific, Latin America, and the Middle East and Africa. In 2016, North America brought in the most money, $2,396.4 million. The majority of those affected by CF are of Caucasian heritage, making North America the region where CF is most common. Additionally, one of the main factors that may be attributed for its highest share is actions conducted by the CF Foundation and Cystic Fibrosis Canada.
With a profitable growth rate of 18.8 percent, Asia Pacific is anticipated to expand at the quickest rate. Due to underdiagnosis and a lack of country-specific CF patient registries, the incidence of CF is less frequently reported in the Asia Pacific region. Nevertheless, over the course of the forecast period, rising awareness about CF disorders and treatment options is anticipated to drive this segment. Over the course of the projected period, the market is anticipated to grow because of this region's economic development and improved healthcare facilities.
One of the key reasons projected to drive the market in the approaching years is the growing awareness among healthcare professionals and the patient population about innovative therapies in the market & its advantages over the current medication portfolio. Drugs specifically for CF are not readily available now, but rising product introductions and approvals in this market are expected to stimulate growth.
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