uniQure NV

The year started well for the biotechnology industry. Steady dealmaking coupled with a rebound in initial public offerings helped lift investor confidence and, with it, drugmaker stocks.While that optimism has moderated somewhat since, a series of decisions by the Food and Drug Administration over the next three months could help the sector sustain its upturn. The agency may expand approvals for two cancer cell therapies and clear a third vaccine for respiratory syncytial virus. Sanofi and Regeneron await a decision on a lucrative new use for their top-selling Dupixent, while Geron could see decades of drug research finally pay off.Here are five FDA decisions to watch in the second quarter.CAR-T therapies for early multiple myeloma treatmentThe FDA this quarter could broaden the use of two cell therapies for multiple myeloma in decisions potentially worth billions of dollars to their respective developers.By April 5, the agency will decide whether to clear Johnson & Johnson and Legend Biotechs Carvykti for use in myeloma patients as early as after one relapse. That decision could be accompanied by a similar OK for Bristol Myers Squibbs Abecma and open up use of the therapies, which are currently only available in the U.S. for people with more advanced disease.European regulators have already endorsed earlier use of Abecma and Carvykti based on trial data showing they help people live longer without their disease progressing. But the FDA has appeared less supportive. The agency delayed a decision on Abecma last year and held a March advisory meeting to more closely scrutinize the data for it and Carvykti. In briefing documents, staff scientists flagged an apparent increase in the risk of death early on in the clinical trials of both therapies.The companies attributed that finding to limitations in the bridging therapies study participants could receive while waiting for CAR-T treatment. Panelists were convinced and backed both, agreeing that those issues likely wouldnt occur as frequently in practice.Nonetheless, the regulatory scrutiny suggests that the FDA might put new safety restrictions on Carvykti and Abecma if it chooses to clear them for earlier use. Jonathan GardnerModernas vaccine for RSVCOVID-19 vaccine makers Pfizer and Moderna have been facing the downside of their pandemic success, as falling sales have brought tough questions from investors.Moderna, for its part, aims to follow its COVID shot with several other messenger RNA-based vaccines, including one for respiratory syncytial virus thats now under FDA review.Respiratory syncytial virus, or RSV, can cause severe illnessin infants, older adults and people with compromised immune systems. Two vaccines, from GSK and Pfizer, are already approved to prevent RSV-related disease in adults over the age of 60. Sales of each have been strong, although GSK has established a clear edge.Moderna argues its vaccine, dubbed mRNA-1345, can compete. The shot is a pre-filled, single-dose vaccine, potentially offering more convenient administration.In Phase 3 testing, the vaccine was 84% effective at preventing infection with two or more symptoms in older adults. That efficacy does appear to wane over time, however.At a recent meeting, Centers for Disease Control and Prevention advisers reviewed known safety risks for GSKs and Pfizers shots, both of which were associated in very rare cases with Guillain-Barr syndrome, or GBS, a neurological condition affecting the nerves. Moderna has not reported any cases of GBS in Phase 3 testing of its shot.An FDA decision on mRNA-1345 is set for May 12. Delilah AlvaradoGerons imetelstat for myelodysplastic syndromesDrug development is a time-consuming, expensive process. Geron, a biotech founded 34 years ago to pursue stem cell research, knows this well.The companys long journey may finally reach an end by June 16, when the FDA will decide whether to approve a bone marrow disease drug Geron has been developing for nearly two decades. Called imetelstat, the drug treats myelodysplastic syndromes and some analysts believe it has blockbuster potential.Over the many years of Gerons research, imetelstat failed studies in multiple cancer types, and the company lost Johnson & Johnson as a partner along the way. But the drug finally succeeded in Phase 3 testing for myelodysplastic syndromes last year. And in March, it secured the endorsement of an FDA advisory panel despite skepticism from agency reviewers.Geron aims to challenge Bristol Myers Squibbs Reblozyl, first with a clearance in people with lower-risk disease and then in more people who have the condition. Stifel analyst Stephen Wiley estimated the drug could generate $2 billion in peak yearly sales, as it could be helpful in people who dont respond well to Reblozyl.Geron could be a buyout target, too, according to Wiley, though the company has said it plans to sell the drug on its own. Ben FidlerSanofi and Regenerons Dupixent for COPDDupixent is one of the pharmaceutical industrys most widely used and lucrative products. Approved for asthma, eczema and three other inflammatory conditions, the drug earned the companies more than $11 billion in sales last year.By June 27, the FDA could add another major indication to Dupixents list of approved uses. The regulator is currently reviewing the injectable medicine as a treatment for a type of chronic obstructive pulmonary disease, or COPD.If cleared by the FDA, Dupixent would be the first biologic drug for people with moderate-to-severe COPD who also have evidence of whats called Type 2 inflammation. A pair of late-stage studies, which enrolled nearly 1,900 people, showed Dupixent could cut the rate of COPD attacks by about one-third. Analysts therefore expect the FDA to issue an approval and forecast billions of dollars in additional yearly sales. Ned PagliaruloPfizers gene therapy for hemophilia BSome time during the second quarter, the FDA will decide whether to clear a gene therapy from Pfizer for hemophilia B, the less common form of the blood clotting disorder.Reaching this point has been a long time coming for Pfizer, which licensed the therapy from Spark Therapeutics a decade ago. In subsequent testing, treatmentlowered bleeding rates and reduced the need for regular blood transfusions powerful benefits meant to last many years with a single infusion.But its unclear whether Pfizer will find a market if the FDA chooses to approve its therapy. A rival genetic medicine, from CSL Behring and UniQure, won U.S. approval for hemophilia B in November 2022. However, few patients have been treated to date, sluggish uptake that could reflect the breadth of effective options available.The story is similar with Roctavian, a gene therapy BioMarin Pharmaceutical sells for the more common A form of hemophilia. Only three patients received Roctavian last year: two in Germany and one in the U.S.Pfizers therapy, which in January won Canadian approval, could provide another data point. But the company seems to be downplaying its potential. In recent presentations for investors, a once-weekly shot called marstacimab has gotten higher billing. Ned Pagliarulo '

Dive Brief:Orchard Therapeutics said Wednesday it will offer a new gene therapy to children with a rare, devastating disease at a record-setting wholesale price of $4.25 million.The therapy, Lenmeldy, won Food and Drug Administration approvalon Monday to treat patients with early-onset metachromatic leukodystrophy, or MLD. The disease, which most often attacks infants between six months and two years of age, robs patients of the ability to walk, talk and function in the world, killing most of its earliest victims within five years of onset.Lenmeldys price tag will leapfrog those of the two most expensive gene therapies available in the U.S. Sarepta Therapeutics sells its Elevidys treatment for Duchenne muscular dystrophy for $3.2 million, while CSL and UniQures hemophilia treatment Hemgenix costs $3.5 million.Dive Insight:Gene therapy makers like Orchard are counting on insurers to recognize the value proposition of their products. Unlike traditional pharmaceuticals that might be taken regularly for the rest of a persons life, gene therapies carry the promise of a long-lasting treatment that can alter the course of a disease and offer significant savings for the care needed to treat patients.The argument doesnt always work. Difficulty winning reimbursement forced gene therapy maker Bluebird bio to pull out of Europe in 2021. More recently, BioMarin has struggled with the launch of its hemophilia gene therapy, scaling back projections significantly. Orchard itself gave up on a gene therapy called Strimvelisin 2022.But Orchard sees a different trajectory for Lenmeldy, in part because of its long-term data. Researchers have 12 years of follow-up on the earliest patients treated with Lenmeldy, the longest duration of any gene therapy launched in the U.S. Orchard also plans to develop outcomes- and value-based agreements designed to share risks with payers.In studies, Orchard found that all patients with pre-symptomatic late infantile MLD treated with Lenmeldy were still alive at age six, compared with just 58% of such children historically. In addition, 71% of treated children could walk at age five without assistance and 85% had normal language and performance IQ scores. That has not been seen with untreated children.MLD places an enormous emotional and economic burden on families and caregivers who face substantial wage loss and added expenses each year as the disease progresses all while dealing with the unquantifiable anguish of losing their child, Bennett Smith, Orchards senior vice president and general manager of North America, said in the companys statement. Gene therapy can be transformative, he said.In Wednesdays release, Orchard highlighted the findings of the Institute for Clinical and Economic Review, which found that Lenmeldy had the highest value-based price of any treatment it has evaluated. Still, Orchard priced its therapy above the $2.3 million to $3.9 million cost-effectiveness threshold determined by that group.Orchard, which was acquired by Kyowa Kirin this year, is setting up five treatment centers across the U.S. to administer Lenmeldy. The first, in Minnesota, is in the final stages of qualification and has already treated several children on a compassionate use basis. Four others, in Atlanta, Philadelphia, San Francisco and Texas, are in the process of qualification. '

NEW YORK, March 18, 2024 /PRNewswire/ -- Technavio categorizes the global gene therapy market size as estimated to increase by USD 4,613.9 million between 2022 and 2027. The market's growth momentum will be progressing at a CAGR of 19.91% during the forecast period. During the forecast period, North America is anticipated to drive 39% of global market growth, as per Technavio's analysts. The US, particularly, emerges as a promising investment hub within the region, supported by substantial funding from entities like the National Institutes of Health (NIH) and the Biomedical Advanced Research and Development Authority (BARDA) to emerging players in the market. This influx of government and private sector investment is propelling the development of innovative gene therapies, with 18 clinical trials and 17 biotech startups underway. Such financial backing is poised to significantly stimulate market demand in the coming years. Continue Reading Technavio has announced its latest market research report titled Global Gene Therapy Market 2023-2027 Discover some insights on market size historic data (2017 to 2021) and Forecast (2023 to 2027) before buying the full report - Request a sample report Technavio has segmented the market based on therapy areas (oncology, CNS, ophthalmology, rare diseases, and others), and delivery mode (in vivo and ex vivo). The market growth in the oncology segment will be significant during the forecast period. The global oncology drugs market has a huge unmet demand for the treatment of various indications. Also, the increasing prevalence of cancer indications has led pharmaceutical companies and research institutions to conduct extensive research to develop drugs for these indications. The lack of efficacy of small molecules and biomolecules has led companies to move towards effective therapeutic options such as gene therapy. Such factors will increase the segment growth during the forecast period. For insights on global, regional, and country-level parameters with growth opportunities from 2017 to 2027 - Download a Sample Report Key Companies The global gene therapy market is fragmented, with the presence of several global as well as regional vendors. A few prominent vendors that offer gene therapy in the market are Abeona Therapeutics Inc., Adaptimmune Therapeutics plc, Adverum Biotechnologies Inc., Amgen Inc., Astellas Pharma Inc., Biogen Inc., bluebird bio Inc., Editas Medicine Inc., ElevateBio, F. Hoffmann La Roche Ltd., Generation Bio Co., Gilead Sciences Inc., Novartis AG, Orchard Therapeutics Plc, Poseida Therapeutics Inc., Sangamo Therapeutics Inc., Sibiono GeneTech Co. Ltd., Syncona Ltd., uniQure NV, and Voyager Therapeutics Inc. and others. Abeona Therapeutics Inc.: The company offers gene therapy solutions such as Phase 3 VIITAL Clinical Trial of EB 101. Amgen Inc.: The company offers gene therapy solutions such as CAR T cell immunotherapies. Astellas Pharma Inc.: The company offers gene therapy solutions such as AT132, AT845, AT466. Biogen Inc.: The company offers gene therapy solutions such as GTxAU. The growth of the market is driven by the increase in special drug designations. Most gene therapy programs have been granted orphan drug designation, fast track designation, or breakthrough designation by the U.S. FDA, EMA, and other regulatory bodies for the treatment of certain diseases. In addition, orphan drug status will also help the company with tax incentives in its next research. Such advantages of specialty drug designations will drive the growth of the market during the forecast period. Growing research in gene therapy for CVDs and orphan diseases is one of the key market trends that is contributing to the market growth. High treatment cost is one of the factors hindering the gene therapy market growth. Driver, Trend & Challenges are the factor of market dynamics which states about consequences & sustainability of the businesses, find some insights from a sample report! Analyst Review Gene therapy stands at the forefront of medical innovation, offering promising solutions for a myriad of genetic disorders ranging from atrophy to muscular dystrophy. As the world grapples with health challenges like the COVID-19 pandemic, companies like Applied Genetic Technologies Corporation and Benitec Biopharma in countries like Australia and Belgium are actively engaged in pioneering gene therapy research to combat such conditions. Beta thalassemia, sickle cell disease, and spinal muscular atrophy represent just a fraction of the genetic issues that gene therapy aims to address. With the advent of breakthrough treatments like betibeglogene autotemcel and onasemnogene abeparvovec, patients suffering from these debilitating conditions now have newfound hope. The gene therapy market, bolstered by advancements in CRISPR Therapeutics and lentivirus technology, is witnessing exponential growth. As companies like Pfizer, Cellectis, and Novartis Gene Therapies invest in research and development, the market is projected to expand further, especially in regions like California and Massachusetts, known for their biotechnology hubs. However, this growth isn't without challenges. Regulatory bodies like ClinicalTrials.gov, the FDA, and the International Organization for Standardization (ISO 9000) play pivotal roles in ensuring the safety and efficacy of gene therapies. Striking a balance between innovation and compliance with regulations such as the California Consumer Privacy Act and the General Data Protection Regulation remains paramount. One of the critical factors driving the gene therapy market is the increasing prevalence of genetic disorders worldwide. From hereditary illnesses to congenital diseases, the demand for innovative treatments continues to rise. Moreover, with advancements in gene editing and therapeutic gene delivery systems, the potential to target specific cells and protein molecules offers new avenues for treatment. Venture capital financing and partnerships between academia and industry players further fuel research efforts. Companies like Lonza Group and Oxford Biomedica contribute to the manufacturing and logistics aspects of gene therapy, ensuring efficient drug delivery and scalability. In conclusion, the gene therapy market holds immense promise in addressing genetic disorders and improving patient outcomes. As the field continues to evolve, collaboration, adherence to regulations, and a focus on patient-centric approaches will be essential in unlocking the full potential of gene therapy to tackle the most challenging medical conditions of our time. Download a Sample Report Market Overview The Gene Therapy Market is witnessing exponential growth globally, with companies like Applied Genetic Technologies Corporation and Benitec Biopharma leading the charge. This revolutionary approach offers hope for conditions such as atrophy and beta thalassemia, providing targeted solutions to genetic disorders. Recent advancements include Betibeglogene autotemcel, a promising treatment for beta-thalassemia pioneered by CRISPR Therapeutics. Biomedicine is at the forefront of this transformative field, driving innovation and clinical trials. Amidst the COVID-19 pandemic, gene therapy research continues unabated, with countries like Australia and Belgium actively contributing to the global effort. As the market expands, gene therapy emerges as a beacon of hope for patients worldwide, offering personalized treatments and potential cures. Download a Sample Report Related Reports: The cell and gene therapy market size is expected to increase by USD 9.97 billion from 2021 to 2026, and the market's growth momentum will accelerate at a CAGR of 20.15%. The cell therapy market size is expected to increase by USD 21.061 billion from 2021 to 2026, and the market's growth momentum will accelerate at a CAGR of 56.79%. TOC Executive Summary Market Landscape Market Sizing Historic Market Size Five Forces Analysis Market Segmentation by Therapy Areas market Segmentation by Delivery Mode Customer Landscape Geographic Landscape Drivers, Challenges, and Trends Company Landscape Company Analysis Appendix About US Technavio is a leading global technology research and advisory company. Their research and analysis focuses on emerging market trends and provides actionable insights to help businesses identify market opportunities and develop effective strategies to optimize their market positions. With over 500 specialized analysts, Technavio's report library consists of more than 17,000 reports and counting, covering 800 technologies, spanning across 50 countries. Their client base consists of enterprises of all sizes, including more than 100 Fortune 500 companies. This growing client base relies on Technavio's comprehensive coverage, extensive research, and actionable market insights to identify opportunities in existing and potential markets and assess their competitive positions within changing market scenarios. Contact Technavio Research Jesse Maida Media & Marketing Executive US: +1 844 364 1100 UK: +44 203 893 3200 Email: [email protected] Website: SOURCE Technavio