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Globe and Mail
20-05-2025
- Business
- Globe and Mail
Mucopolysaccharidosis Type I Pipeline Appears Robust With 8+ Key Pharma Companies Actively Working in the Therapeutics Segment
DelveInsight's, 'Mucopolysaccharidosis Type I Pipeline Insight 2025' report provides comprehensive insights about 8+ companies and 8+ pipeline drugs in Mucopolysaccharidosis Type I pipeline landscape. It covers the Mucopolysaccharidosis Type I pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Mucopolysaccharidosis Type I pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space. Discover the latest drugs and treatment options in the Mucopolysaccharidosis Type I Pipeline. Dive into DelveInsight's comprehensive report today! @ Mucopolysaccharidosis Type I Pipeline Outlook Key Takeaways from the Mucopolysaccharidosis Type I Pipeline Report In May 2025, JCR Pharmaceuticals Co. Ltd announced a JR-171-101 study (NCT04227600) and fulfill all eligibility criteria can be enrolled in this JR-171-102 study. In the JR-171-102 study, subjects will receive either 2.0 or 4.0 mg/kg/week of JR-171 intravenously at the same doses received at Week 12 of the JR-171-101 study. DelveInsight's Mucopolysaccharidosis Type I Pipeline report depicts a robust space with 8+ active players working to develop 8+ pipeline therapies for Mucopolysaccharidosis Type I treatment. The leading Mucopolysaccharidosis Type I Companies such as Orchard Therapeutics, JCR Pharmaceuticals, REGENXBIO and others. Promising Mucopolysaccharidosis Type I Pipeline Therapies such as Laronidase, Antihistamine, Antipyretic, JR-171, AGT-181, Cyclosporine A (CsA), Azathioprine (Aza), RGX-111, SBC-103, and others. Stay ahead with the most recent pipeline outlook for Mucopolysaccharidosis Type I. Get insights into clinical trials, emerging therapies, and leading companies with DelveInsight @ Mucopolysaccharidosis Type I Treatment Drugs Mucopolysaccharidosis Type I Emerging Drugs Profile OTL 203: Orchard Therapeutics OTL-203 is an investigational ex vivo autologous gene therapy being investigated for the treatment of MPS-I. It uses a modified virus to insert a functional copy of the IDUA gene into a patient's cells. OTL-203 is being developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. OTL-203 has received rare pediatric disease designation from the FDA. Through an ongoing proof-of-concept clinical trial, OTL-203 is being evaluated as a potential treatment for patients with the most severe form of MPS-I, known as Hurler syndrome. OTL-203 is an investigational therapy and has not been approved by any regulatory agency or health authority. Currently, the drug is in Phase III stage of its development for the treatment of Mucopolysaccharidosis Type I. JR 171: JCR Pharmaceuticals JR-171, is an investigational drug for the treatment of mucoplysaccharidosis type I (MPS I, or Hurler, Hurler-Scheie and Scheie syndrome). JR-171 is a blood-brain-barrier (BBB)-penetrating form recombinant α-L-iduronidase that was developed using JCR's proprietary J-Brain Cargo® BBB technology. JR-171 received the orphan drug designation from the US Food Drug Administration (FDA) in February 2021. The trial is also scheduled for enrolling patients in the US. Currently, the drug is in Phase I/II stage of its development for the treatment of Mucopolysaccharidosis Type I. The Mucopolysaccharidosis Type I Pipeline Report Provides Insights into The report provides detailed insights about companies that are developing therapies for the treatment of Mucopolysaccharidosis Type I with aggregate therapies developed by each company for the same. It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Mucopolysaccharidosis Type I Treatment. Mucopolysaccharidosis Type I Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects. Mucopolysaccharidosis Type I Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type. Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Mucopolysaccharidosis Type I market Explore groundbreaking therapies and clinical trials in the Mucopolysaccharidosis Type I Marketed and Pipeline Drugs. Access DelveInsight's detailed report now! @ New Mucopolysaccharidosis Type I Drugs Mucopolysaccharidosis Type I Companies Orchard Therapeutics, JCR Pharmaceuticals, REGENXBIO and others. Mucopolysaccharidosis type I (MPS I) pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as Oral Intravenous Subcutaneous Parenteral Topical Mucopolysaccharidosis Type I Products have been categorized under various Molecule types such as Recombinant fusion proteins Small molecule Monoclonal antibody Peptide Polymer Gene therapy Unveil the future of Mucopolysaccharidosis Type I Treatment. Learn about new drugs, pipeline developments, and key companies with DelveInsight's expert analysis @ Mucopolysaccharidosis Type I Market Drivers and Barriers Scope of the Mucopolysaccharidosis Type I Market Report Coverage- Global Mucopolysaccharidosis Type I Companies- Orchard Therapeutics, JCR Pharmaceuticals, REGENXBIO and others. Mucopolysaccharidosis Type I Pipeline Therapies- Laronidase, Antihistamine, Antipyretic, JR-171, AGT-181, Cyclosporine A (CsA), Azathioprine (Aza), RGX-111, SBC-103, and others. Mucopolysaccharidosis Type I Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination Mucopolysaccharidosis Type I Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Get the latest on Mucopolysaccharidosis Type I Therapies and clinical trials. Download DelveInsight's in-depth pipeline report today! @ Mucopolysaccharidosis Type I Companies, Key Products and Unmet Needs Table of Contents Introduction Executive Summary Mucopolysaccharidosis type I (MPS I): Overview Pipeline Therapeutics Therapeutic Assessment Mucopolysaccharidosis type I (MPS I)– DelveInsight's Analytical Perspective Late Stage Products (Phase III) OTL 203: Orchard Therapeutics Drug profiles in the detailed report….. Mid Stage Products (Phase II) Drug Name: Company Name Drug profiles in the detailed report….. Early Stage Products (Phase I) JR 171: JCR Pharmaceuticals Drug profiles in the detailed report….. Preclinical and Discovery Stage Products Drug name : Company name Drug profiles in the detailed report….. Inactive Products Mucopolysaccharidosis type I (MPS I) Key Companies Mucopolysaccharidosis type I (MPS I) Key Products Mucopolysaccharidosis type I (MPS I)- Unmet Needs Mucopolysaccharidosis type I (MPS I)- Market Drivers and Barriers Mucopolysaccharidosis type I (MPS I)- Future Perspectives and Conclusion Mucopolysaccharidosis type I (MPS I) Analyst Views Mucopolysaccharidosis type I (MPS I) Key Companies Appendix About Us DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve. Media Contact Company Name: DelveInsight Business Research LLP Contact Person: Yash Bhardwaj Email: Send Email Phone: 09650213330 Address: 304 S. Jones Blvd #2432 City: Las Vegas State: NV Country: United States Website:
Fox News
13-05-2025
- Health
- Fox News
Top 10 most expensive prescription drugs in the US by price and by sales volume
Print Close By Melissa Rudy Published May 13, 2025 On the heels of President Donald Trump's Monday announcement of an executive order that will slash prescription drug prices in the U.S., the spotlight is on current costs and how much Americans could save. The president's order calls for "most favored nations drug pricing" — which means "the lowest price paid for a drug in other developed countries, that is the price that Americans will pay," he said. "Some prescription drug and pharmaceutical prices will be reduced almost immediately by 50 to 80 to 90%," Trump said. PRESIDENT TRUMP TAKES ON 'BIG PHARMA' BY SIGNING EXECUTIVE ORDER TO LOWER DRUG PRICES Katy Dubinsky, a New York pharmacist and founder and CEO of Vitalize, applauded the move to reduce prescription prices, noting that Trump's order tackles a long-standing problem. "But this will not be simple to accomplish," she told Fox News Digital. "The executive order doesn't reduce costs immediately," she said. "It directs government agencies to start drafting the rules, which may take months." Here are the five most expensive prescription drugs in the U.S. by price — followed by five by volume. 5 most expensive prescription drugs in the US Dubinsky detailed some of the most expensive prescription drugs in the country today and what conditions they treat. 1. Lenmeldy (atidarsagene autotemcel) by Orchard Therapeutics – $4.25 million This medication is used to treat metachromatic leukodystrophy (MLD), a rare genetic disorder that damages the nervous system, Dubinsky said. "It is given once and is supposed to stop or slow down the disease in young kids," she noted. TOP 10 'ALLERGY CAPITALS' OF THE US, PLUS 4 TIPS TO MANAGE SYMPTOMS 2. Hemgenix (etranacogene dezaparvovec-drlb) by CSL Behring – $3.5 million This medication is prescribed for people with hemophilia B, a bleeding disorder. "This one-time treatment helps the body make its own clotting factor, so patients don't need regular infusions," said Dubinsky. 3. Elevidys (delandistrogene moxeparvovec-rokl) by Sarepta Therapeutics – $3.2 million This prescription medication, intended for young boys, treats Duchenne muscular dystrophy (DMD), a condition that weakens muscles over time. "It aims to slow down how fast the disease progresses," Dubinsky said. 4. Skysona (elivaldogene autotemcel) by Bluebird Bio – $3 million "This medication is used for cerebral adrenoleukodystrophy (CALD), a serious brain disease in boys," said Dubinsky. "This therapy tries to slow the damage before symptoms get worse." 5. Zynteglo (betibeglogene autotemcel) by Bluebird Bio – $2.8 million Zynteglo is for beta-thalassemia, a blood condition that usually requires regular transfusions. "Some prescription drug and pharmaceutical prices will be reduced almost immediately by 50 to 80 to 90%." "This gene therapy can help patients make healthy red blood cells on their own and reduce how often they need treatment," said Dubinsky. Top 5 most expensive drugs by sales volume John Stanford, executive director of Incubate, a Washington-based coalition of early-stage life-science investors, shared his thoughts on the top five most expensive drugs by sales volume. "Typically, when the government is focused on the most expensive drugs, they're focused on the metric based on sales volume rather than, for instance, a rare disease therapy with a high list price but smaller patient pool," he told Fox News Digital. "Often, officials are focused on total drug spending by Medicare or other government programs." 1. Keytruda (pembrolizumab) by Merck — $25 billion revenue (2023) Keytruda is an immunotherapy medication used to treat a variety of cancers, including melanoma, non-small cell lung cancer, liver cancer and others. "Keytruda has become Merck's crown jewel, helping the company expand its cancer treatment portfolio with more than 1,000 active clinical trials," Stanford told Fox News Digital. TERMINAL COLON CANCER PATIENT SAVED BY BREAKTHROUGH TREATMENT 2. Eliquis (apixaban) by Bristol Myers Squibb and Pfizer — $18.95 billion Eliquis (apixaban) is an "anchor drug" for both BMS and Pfizer, according to Stanford. Apixaban is prescribed to prevent the formation of blood clots and to treat deep vein thrombosis and pulmonary embolism (a blood clot in the lungs). 3. Ozempic (semaglutide) by Novo Nordisk — $13.93 billion Prescribed for type 2 diabetes, the semaglutide medication Ozempic has become widely popular for its weight-loss effects and other health benefits. "Ozempic's sales are powering Novo Nordisk's broader foray into GLP-1s for obesity, heart disease and liver conditions — all areas with high development costs and uncertain scientific outcomes," Stanford told Fox News Digital. "The money has gone toward scaling up production to meet demand for GLP-1s and avoid supply shortages." 4. Humira (AbbVie) — $14.4 billion (U.S. 2023 revenue) "Humira has been one of the highest-grossing drugs in history, generating over $200 billion during its exclusivity period," Stanford said. The injectable medication, which contains the active ingredient adalimumab, is used to treat rheumatoid arthritis and other inflammatory conditions. CLICK HERE TO SIGN UP FOR OUR HEALTH NEWSLETTER 5. Biktarvy by Gilead — $11.85 billion Biktarvy is an HIV treatment that includes the three ingredients bictegravir, emtricitabine and tenofovir alafenamide. "Biktarvy isn't just a leading HIV treatment — it's the financial backbone for Gilead's move into cancer research," Stanford said. Which drugs could be affected? Dr. Jacob Glanville, CEO of Centivax, a San Francisco biotechnology company, said vaccines and most generic drugs would not likely be changed by the executive order. "Most vaccines that Americans take cost less than a hundred dollars, while generic drugs are often less than a dollar a pill," he told Fox News Digital. What would be affected, Glanville predicted, are newer brand-name drugs still under IP exclusivity, antibody therapies, cellular therapies, gene therapies and personalized cancer vaccines. "Some of these are excruciatingly expensive — $100,000 to $500,000 for a treatment course for a patient. However, they are also often the most effective treatments for certain cancers, autoimmune disorders or rare diseases." The price of innovation? The pharmaceutical industry might argue that lowering the prices on these medicines will result in a "dramatic reduction of investment" in creating such breakthroughs, said Glanville. The industry may also argue that these medicines eventually become generic — at which point the prices drop, according to the expert. For more Health articles, visit "From a patient's perspective, the price of medical care in the United States is unsustainable, and extremely expensive medicine is part of that," he said. But "the insurance system and the hospital business also contribute." "If the prices of new medicines are capped, then effort should be made to reduce the cost of clinical trials and drug GMP manufacturing. Otherwise, we will lose a lot of innovation." CLICK HERE TO GET THE FOX NEWS APP Greg Norman of Fox News Digital contributed reporting. Print Close URL
