June 1: Tracking Florida COVID-19 Cases, Hospitalizations, and ...
Cystic Fibrosis Therapeutics Market Aims For A Remarkable US$ 116.42 Billion Valuation By 2033Future Market Insights,Inc.
In 2023, the global Cystic Fibrosis Therapeutics Market is expected to be worth US$ 12.5 billion. The overall demand for Cystic Fibrosis is expected to grow at a CAGR of 25.0% between 2023 and 2033, totaling around US$ 116.42 Billion by 2033, with the implementation of innovative treatment options for early detection.
The rising prevalence of CF indicates an increase in demand for treatment options, which will drive the Cystic Fibrosis Therapeutics Market in the near future. The key players in this market focus on the development of novel therapeutics for treatment in collaboration with nonprofit organisations. VX-445 + tezacaftor + ivacaftor, PTI-428, QR-010, AZD5634, and POL6014 are among the drugs in clinical trials.
Get a Sample Copy of the Report :https://www.Futuremarketinsights.Com/reports/sample/rep-gb-16252
The rising prevalence of cystic fibrosis, advancements in screening and treatment, and the availability of modified drug therapy and molecular prosthetics to cure cystic fibrosis are the major factors driving the revenue share of the cystic fibrosis therapeutics market during the forecast period. Furthermore, rising awareness about this progressive genetic disease, as well as the increasing adoption of drug therapies, are important factors that are expected to accelerate market growth over the forecast period.
Key Companies Profiled :
•Novartis AG•Vertex Pharmaceuticals Incorporated•AbbVie Inc.•Gilead Sciences, Inc.•Mylan N.V.•Pfizer Inc.•Bayer AG•Genentech, Inc.•AstraZeneca
Recent Developments:•In April 2018, Novartis Venture Fund along with Versant Ventures invested US$ 41 million for series B round in cystic fibrosis disease startup Enterprise Therapeutics. The financing will allow Enterprise Therapeutics to push three projects into clinical phase.
•In March 2018, US FDA approved product named Symdeko manufactured by Vertex Pharmaceuticals Incorporated. The product is a combination of Tezacaftor and Ivacaftor majorly used in the treatment of CF.
Key Segments Covered in the Cystic Fibrosis Industry Analysis:Cystic Fibrosis Therapeutics Market by Drug Class:•Cystic Fibrosis Transmembrane Conductance Regulator (CFTR)•Bronchodilators•Mucolytic•Pancreatic Enzyme Supplement•Others
Cystic Fibrosis Therapeutics Market by Route of Administration:•Oral•Parenteral
Cystic Fibrosis Therapeutics Market by Distribution Channel:•Retail Pharmacies & Drug Stores•Online Pharmacies•Hospital Pharmacies
Unlock Tailored Insights: Customize Your Report for Maximum Impact:https://www.Futuremarketinsights.Com/customization-available/rep-gb-16252
About Future Market Insights (FMI)
Future Market Insights, Inc. (ESOMAR certified, recipient of the Stevie Award, and a member of the Greater New York Chamber of Commerce) offers profound insights into the driving factors that are boosting demand in the market. FMI stands as the leading global provider of market intelligence, advisory services, consulting, and events for the Packaging, Food and Beverage, Consumer Technology, Healthcare, Industrial, and Chemicals markets. With a vast team of over 5000 analysts worldwide, FMI provides global, regional, and local expertise on diverse domains and industry trends across more than 110 countries.
Contact Us:
Nandini Singh Sawlani
Future Market Insights Inc.Christiana Corporate, 200 Continental Drive,Suite 401, Newark, Delaware – 19713, USAT: +1-845-579-5705
For Sales Enquiries: sales@futuremarketinsights.ComWebsite: https://www.Futuremarketinsights.ComLinkedInTwitterBlogsYouTube
How Does Cystic Fibrosis Affect The Lungs?
Cystic fibrosis affects many parts of the body, but its effects on the lungs can be especially harmful.
Cystic fibrosis is a chronic and progressive condition that causes the mucus in your lungs to be sticky and thick.
This makes it hard to breathe and leads to symptoms such as coughing and wheezing. It also causes bacteria to get trapped in the lungs, leading to infection and inflammation. These infections can be serious and lead to lung damage over time.
Treatment can't cure cystic fibrosis, but it can help reduce the severity of lung symptoms. Options include antibiotics, medications to clear airways, supplemental oxygen, and chest physical therapy.
Cystic fibrosis causes the mucus in your lungs to be overly sticky and thick. Since this mucus is so thick, it makes it harder to clear your lungs. This can make everyday breathing difficult. It causes mucus to fill up the windpipe, or trachea, several times a day. It also causes chronic coughing or wheezing that is often associated with cystic fibrosis.
Bacteria may get stuck in the sticky and thick mucus and can lead to infection. An infection can cause inflammation, which can cause the production of an even stickier mucus. Often, this means that infections that are minor and harmless for people without cystic fibrosis can be severe for people with cystic fibrosis.
People with cystic fibrosis might need to stay in the hospital for the duration of these infections to receive IV (intravenous) antibiotics and be monitored by healthcare professionals. Additionally, the lungs can become damaged by repeated infection and inflammation.
If you have a lung infection with cystic fibrosis, you may have symptoms such as:
There are multiple treatments to help treat the effect of cystic fibrosis on your lungs. This can include:
Cystic fibrosis is a chronic and progressive condition with no cure. Until recently, people with cystic fibrosis rarely lived past their teenage years. However, new treatments and screenings for earlier diagnosis are helping change the outlook for people with cystic fibrosis.
Today, people with cystic fibrosis are living decades longer than ever before.
People with cystic fibrosis born between 2018 and 2022 are predicted to live to an average age of 56, and it's predicted that over half of people with cystic fibrosis born in 2021 will reach the age of 65. Of course, these are estimates that represent wider trends. Your individual life expectancy will depend on a variety of factors.
Your doctor can help you understand your outlook and how your cystic fibrosis might progress over time.
Cystic fibrosis causes the mucus of your lungs to be too sticky and thick to function normally. As a result, people who have cystic fibrosis have breathing difficulties.
They typically have labored breathing and a chronic cough and need to clear mucus buildup from their trachea throughout the day. This mucus also puts people with cystic fibrosis at risk of lung infections. These infections are often significantly more serious than they would be for people without cystic fibrosis. They can also lead to inflammation, which can damage the lungs over time.
There's no cure for cystic fibrosis, but treatment can help manage symptoms. Primary treatment options include antibiotics to reduce and control infections, medications to keep airways clear, and chest physical therapy to remove mucus from the airways. Sometimes, treatments such as supplemental oxygen or a lung transplant can also be options.
ACS Celebrates Its 2023 Heroes Of Chemistry
Credit: EPNAC.Com
ACS president Judith C. Giordan announced the Heroes of Chemistry 2023 awards
Teams of chemical scientists from AbbVie and Enanta Pharmaceuticals, Honeywell, Incyte, Merck & Co., Vertex Pharmaceuticals, and ViiV Healthcare received 2023 Heroes of Chemistry Awards at a banquet in Alexandria, Virginia, on Oct. 12. The researchers were recognized for advancements in a number of areas: treating chronic hepatitis C, creating non-ozone-depleting greenhouse gas alternatives, developing a versatile treatment for rare blood cancers and graft-versus-host disease, designing a general anesthesia drug with reduced side effects, coming up with drugs that target and correct genetic abnormalities that cause cystic fibrosis, and improving HIV treatment.
Now in its 27th year, the Heroes of Chemistry Awards recognizes scientists whose use of chemistry acts as a service to society. The award is based on three criteria: technical merit, commercial impact, and benefit to humankind. It is the American Chemical Society's most prestigious distinction for industrial chemists and is supported by ACS's Board Committee on Corporation Associates.
"All of us who work in industrial science, and especially you who are being honored, provide new products and innovations, and you've improved all of our lives," ACS president Judith C. Giordan, who has been an industrial scientist her entire career, said at the event. "And that's what really matters."
This year's company teams and their commercialized products are:
▸ ABBVIE AND ENANTA PHARMACEUTICALS: MAVYRET
Credit: EPNAC.Com
ACS president Judith C. Giordan (far left) with the teams from AbbVie and Enanta Pharmaceuticals on stage at the Heroes of Chemistry 2023 awards event
Hepatitis C is an infectious liver disease that affects approximately 58 million people worldwide.
Teams from AbbVie and Enanta Pharmaceuticals discovered and developed two antiviral agents used in Mavyret, a drug that treats chronic hepatitis C in adults and children. The disease occurs after infection with the hepatitis C virus (HCV); depending on the genetic variation, it can cause short- or long-term effects that can vary in prevalence and aggressiveness.
Steve Elmore, vice president of drug discovery science and technology at AbbVie, described the discovery process of Mavyret as long and difficult. "It was only made possible through the combination of strong collaboration, remarkable persistence, and the courage to push the boundaries of both medicinal chemistry and synthetic chemistry."
The collaboration between the discovery teams at AbbVie and Enanta started in 2006. AbbVie, with a long history in virology, developed and commercialized the drug. The teams had to overcome a number of synthetic challenges before they could achieve a scalable, economical, and robust syntheses of the two active ingredients.
"This was at least a decades-long scientific journey that led to the discovery and enhancement of six distinct molecular entities across two companies with the approval of three HCV treatment regimes—culminating in Mavyret," Elmore said.
Mavyret can be considered the second generation of HCV treatment; it follows Viekira Pak and Technivie, which could treat only patients with certain mutations.
The World Health Organization is focused on eliminating hepatitis globally by 2030, and Mavyret meets the requirements of a treatment to be used on a large scale to eradicate HCV.
"We and others are committed to the eradication of HCV with a goal in the near future," Elmore said. In November 2018, AbbVie and the Medicines Patent Pool, a United Nations–backed public health organization, entered into a royalty-free licensing agreement to accelerate access to the drug in 99 low- and middle-income countries and territories.
"As a result of this and others' work, once considered a difficult-to-treat illness, HCV infection is one of the few diseases today that is effectively curable," Elmore said. "In 2021, we reached the remarkable milestone with the cure of 1 million HCV patients by treatment with Viekira Pak and Mavyret."
▸ HONEYWELL: SOLSTICE LBA
Credit: EPNAC.Com
ACS president Judith C. Giordan (far left) with the team from Honeywell on stage at the Heroes of Chemistry 2023 awards event
In 2007, the Montreal Protocol on Substances that Deplete the Ozone Layer agreed to regulate the consumption and production of close to 100 synthetic chemicals that are ozone-depleting substances. This included an amendment to accelerate the phaseout of hydrochlorofluorocarbons and, later, hydrofluorocarbons (HFCs). To answer this global imperative, a challenge was issued to industry.
"Without the perseverance and tenacity of this team, we wouldn't be here today," said Laura Reinhard, vice president and general manager at Honeywell Advanced Materials.
The Honeywell team developed Solstice Liquid Blowing Agent (LBA)—hydrochlorofluoroolefin-1233zd(E)—a next-generation LBA that replaces HFCs, a group of greenhouse gases, in applications such as refrigeration and insulating foam. Soltice LBA serves as a sustainable replacement for specific compounds with high global warming potential (GWP) with a range of 794–3,220. It achieves high performance and safety with a reduced environmental impact.
Previous alternatives did not thermally insulate effectively and were not energy efficient. Solstice LBA has a GWP of 1. It is nonflammable, non-ozone-depleting, highly insulating, thermally stable, and compatible with existing components; it also has low toxicity.
"Sustainability was not something that the world was talking about yet," Reinhard said. "This innovation was so well ahead of its time, so we needed to get early adopters to come together to allow us to be able to scale up and get leadership buy-in that this was the 'big bet' that they should be investing in. The one big concern that we had was, Is the world ready to pay to reduce climate change?"
Honeywell mobilized a cross-functional team to educate customers, regulators, and industries on the benefits of Solstice LBA.
Solstice LBA received final approval from the US Environmental Protection Agency in 2012. Demand has since grown rapidly—most likely as a result of a variety of rules (e.G., the Kyoto Protocol) implemented worldwide to help curb climate change. In turn, Honeywell has invested over $1 billion in research, development, and new capacity.
As of July 2022, the new Solstice technology discovered and developed in the commercial production process has helped avoid the potential release of the equivalent of more than 295 million metric tons of carbon dioxide into the atmosphere—the equivalent of eliminating the use of 688 million barrels of oil.
When people ask Reinhard why the team was so successful, she responds, "We were shaping the future. We knew this innovation was ahead of its time. . . .We assembled a team that would not accept defeat."
Credit: EPNAC.Com
ACS president Judith C. Giordan (far left) with the team from Incyte on stage at the Heroes of Chemistry 2023 awards event
The team from Incyte led the discovery and development of Jakafi, a versatile drug that can treat certain bone marrow and blood cancers in people who don't respond favorably to other chemotherapy treatments. It also can inhibit janus kinase (JAK), which are proteins involved in vital cell functions such as signaling, growth, and survival. Additionally, the US Food and Drug Administration granted it breakthrough therapy and orphan drug designation for treating graft-versus-host disease (GvHD), which occurs when donated or transplanted tissues attack the recipient's body cells.
"Every single step of this process was a team effort and would not be possible without the dedication and collaboration of our entire organization," said Xiaozhao Wang, head of medicinal chemistry at Incyte.
In 2002, the then-small team at Incyte began investigating JAK inhibitors—work that would continue into the 2010s. The active ingredient in Jakafi was synthesized in 2005 as part of the research and discovery.
The team had set out to develop a drug for patients with life-altering and life-threatening illnesses like myelofibrosis. Ruxolitinib quickly advanced to Phase I clinical trials. The trials were successful, and the need to scale up ruxolitinib production was recognized. With the team's diligence, Jakafi was approved by the FDA in 2011.
"This was of course an enormous milestone for the company, but also I think I can say this for all the folks out there in the field of drug discovery—that when you see your hard work and your creative ideas finally reach patients and make a difference in their lives, that's one of the proudest moments you can have as a scientist," Wang said.
Credit: EPNAC.Com
ACS president Judith C. Giordan (left) with the team from Merck on stage at the Heroes of Chemistry 2023 awards event
The Merck team pursued and proved a novel concept leading to the development of Bridion, a reversal agent, used during general anesthesia, for skeletal muscle relaxants that has reduced side effects.
Skeletal muscle relaxants help facilitate surgical procedures by blocking the physiological effects of neurotransmitters on striated muscle cells. The neuromuscular blockade is reversed to aid in a safe postoperative recovery of the patient. The most widely used reversal agent is an inhibitor that can lead to side effects such as lower heart rate, irregular heartbeat, and narrowed airways to the lungs.
"It makes me very proud to be recognized for the pivotal role of aspiration and creativity in chemistry," said Jonathan Bennett, Merck's vice president of discovery chemistry. "We recognize that it is absolutely essential to the delivery of these goals.
The Merck team came up with the novel idea that chemical encapsulation by an external host molecule of skeletal muscle relaxants helps it dissociate and reverses the neuromuscular blockade. Because this is an indirect action of the neurotransmitters usually inhibited by relaxants, it bypasses the less desirable side effects.
"There was a substantial amount of innovation required in discovery and development to be able to bring that to fruition," Bennett said.
Creating Bridion required chemically modifying sugar molecules known as cyclodextrins. In turn, that required a multidisciplinary chemistry team who could optimize synthetic, purification, and analytical techniques.
The US Food and Drug Administration approved Bridion in 2015, and patient demand for the drug spiked. After a 40% batch failure rate with the first generation synthesis of the chemical, a second generation was developed that focused on stabilizing the supply chain. That action contributed to the broader scientific community. These efforts have resulted in a 100% success rate on a development and commercial scale.
Bridion has been approved in over 95 countries for reversal of the effects of certain neuromuscular blocking agents in adults undergoing surgery and over 106 million vials have been distributed.
James Caldwell, a professor of anesthesia and perioperative care at the University of California, San Francisco, stated that "cyclodextrin-mediated reversal of neuromuscular block is potentially the greatest advance in neuromuscular pharmacology in the last 20 years."
Credit: EPNAC.Com
ACS president Judith C. Giordan (far left) with the team from Vertex on stage at the Heroes of Chemistry 2023 awards event
Cystic fibrosis (CF) affects more than 80,000 people around the globe. It is one of the most common severe single-gene disorders, and in 2017 it was reported that the estimated median age of people who died from CF in the US and Europe ranged from 29 to 32 years old.
The team from Vertex developed Trikafta, a combination of three therapies that target and correct the genetic abnormalities that cause CF in 93% of people with the disease.
About 90% of all CF patients in the US have the F508del mutation. In the drug discovery, the team faced an unprecedented challenge in developing a treatment that targeted F508del: inventing small molecules that promote the maturation to the cell surface of a misfolded protein.
The researchers had learned during previous CF drug development that they should find molecules that are both chemically and mechanistically distinct and that, in combination with other Vertex- developed drugs, they could be effective.
"It took 48,000 compounds to discover Trikafta, over 20 years of research," said Mark Bunnage, Vertex's global head of research.
The US Food and Drug Administration approved Trikafta in 94 days, one of the fastest regulatory approvals in history. The team took prior learnings and leveraged synthetic tools to create a product synthesis that has delivered dozens of batches of the drug and only one failed batch. The FDA has described the achievement of this Vertex team as "unique" and "groundbreaking."
About 16,000 patients have been treated with Trikafta. They have seen an 87% reduction in the risk of lung transplant, a 77% reduction in pulmonary exacerbations, and a 74% reduction in the risk of death.
"There are many of hundreds of colleagues who played a key role in getting this medicine all the way through discovery to commercialization, and it really is a true team sport," Bunnage said. "I would also like to thank the true heroes of our CF journey, and that is the patients and the families who have been with this team all the way."
▸ VIIV HEALTHCARE: RUKOBIA
Credit: EPNAC.Com
ACS president Judith C. Giordan (far left) with the team from ViiV Healthcare on stage at the Heroes of Chemistry 2023 awards event
Approximately 1.2 million people in the US have HIV, the virus that, if untreated, can lead to AIDS. In 2022 alone, it is estimated that 630,000 people worldwide died of HIV-related illnesses.
"ViiV Healthcare's commitment is, quite simply, to leave no person with HIV behind, and it's a mission that we've been on for almost 15 years," said Eric Gillis, senior director, head of chemistry at ViiV Healthcare.
The ViiV team discovered and developed Rukobia, a first-in-class attachment inhibitor that prevents HIV-1 from interacting with immune cells. The drug was developed for people who have run out of other HIV treatment options.
The discovery of Rukobia was complex and challenging. It required systematic mapping, the development of new synthetic methodologies, and innovation. A series of more than 15 new synthetic methodologies were developed in this process.
To address certain limitations, ViiV decided upon an oral delivery approach to control the concentration of the parent drug when measured in plasma. The success of Rukobia also required the development of an innovative slow-release formulation.
"People living with HIV have benefited from innovative medicines that have transformed diagnosis of this serious disease from a death sentence to one in which millions can live their lives today on their own terms," Gillis said.
HIV is constantly evolving, meaning that it has the potential of becoming resistant to certain classes of medicine after years of use.
"To stay ahead of this, we need new medicines that can attack the virus in novel ways," Gillis said. "From 2007 until 2020 there were no new drug approvals for oral HIV medicines that could do this, and it would have been longer if it had not been for the incredible team of scientists that we're honoring."
The US Food and Drug Administration approved Rukobia in July 2020. This came after the FDA designated Rukobia a breakthrough therapy and granted it fast-track and priority review status. The drug is now approved in the US and Europe.
Those honored include:
ABBVIE▸ Alessandra Mattei▸ Brian Kotecki▸ Clifford Mitchell▸ Daniel Mack▸ David Hill▸ Jean-Christophe Califano▸ Jeffrey Kallemeyn▸ John Randolph▸ Jordan Stobaugh▸ Keith McDaniel▸ Kirill Lukin▸ Laurie Mlinar▸ Mark Matulenko▸ Mathew Mulhern▸ Matthew Pelc▸ Michael Abrahamson▸ Moiz Diwan▸ Kenneth Engstrom▸ Nandkishor Nere▸ Pamela Donner▸ Rolf Wagner▸ Russell Cink▸ Seble Wagaw▸ Shashank Shekhar▸ Shuang Chen▸ Timothy Towne▸ Warren Kati▸ Westin MorrillENANTA (In collaboration with AbbVie)▸ Guoqiang Wang▸ Jun Ma▸ Yat Sun Or
HONEYWELL▸ Daniel Merkel▸ David Williams▸ Haiyou (Hugh) Wang▸ Hang Pham▸ Jim Ling▸ Joshua Close▸ Mary Bogdan▸ Michael Van Der Puy▸ Rajiv Singh▸ Ryan Hulse
INCYTE▸ Bhavnish Parikh▸ Dave Meloni▸ Greg Hollis▸ Haisheng (Jason) Wang▸ Jack Shi▸ James Rodgers▸ Jiacheng Zhou▸ Kris Vaddi▸ Laurine Galya▸ Lixin Shao▸ Maryanne Covington▸ Mei Li▸ Nikoo Falahatpisheh▸ Peggy Scherle▸ Pingli Liu▸ Ryan McGee▸ Stacey Shepard▸ Swamy Yeleswaram▸ Thomas Maduskuie▸ Trupti Sheth▸ Yanlong Li▸ Yong Ni▸ Yongchun Pan▸ Yvonne Lo
MERCK & CO.▸ David Rees▸ Jamie McCabe Dunn▸ Jonathan Bennett▸ Michael Ward▸ Ming-Qiang Zhang▸ Ronnie Palin▸ Yu He
VERTEX PHARMACEUTICALS▸ Alexander Abela▸ Bérénice Lewandowski▸ Billie Kline▸ David Siesel▸ Fabrice Pierre▸ Hari Khatuya▸ Jason McCartney▸ Jeremy Clemens▸ Jinglan Zhou▸ Johnny Uy▸ Mark Miller▸ Sabine Hadida▸ Thomas Cleveland▸ Vito Melillo▸ Yanwei Zhang
VIIV HEALTHCARE▸ Chungping Herman Chen▸ David Conlon▸ John Kadow▸ Jonathan Brown▸ Martin Eastgate▸ Nicholas Meanwell▸ Peter Timmins▸ Pin-Fang Lin▸ Richard Colonno▸ Tao Wang▸ Timothy Connolly▸ Yasutsugu Ueda
Chemical & Engineering News
ISSN 0009-2347
Copyright © 2023 American Chemical Society
Comments
Post a Comment