Latest news with #Zynteglo
Yahoo
5 days ago
- Business
- Yahoo
As Interest Grows for New Agents to Treat Thalassemia, Hematologists Begin Early Identification of Ideal Patients for Gene Therapies Vs. New Therapeutic Options
Spherix Global Insights' new patient audit on transfusion-dependent thalassemia reveals strong physician interest in emerging treatments like mitapivat and etavopivat, and provides insight into ideal candidates for curative gene therapies. EXTON, PA, June 04, 2025 (GLOBE NEWSWIRE) -- Despite the promise of new curative strategies for beta thalassemia, such as Casgevy (Vertex) and Zynteglo (bluebird bio), most transfusion-dependent thalassemia patients remain tethered to long-term transfusion support. Transfusions, while providing symptom relief, in turn create logistical barriers and their own clinical challenges. New data from Spherix Global Insights' independent chart audit, Patient Chart Dynamix™: Transfusion Dependent Thalassemia 2025 (US), is based on insight from 81 real-world patient charts submitted by 49 US-based hematologists. These data paint a complex picture of therapeutic inertia, growing anticipation for emerging treatments, and deep-seated frustration with limitations of current treatment options. Most transfusion-dependent beta thalassemia patients follow some regular transfusion schedule. That said, many receive them at relatively extended intervals: often once a month or even less frequently, though a small subset still require more frequent transfusions. This variation in treatment patterns, along with persistent symptoms and unmet needs highlighted in the chart audit, points to a strong opportunity for new therapies to improve care for a broad range of patients. Among surveyed hematologists, mitapivat (Agios) currently holds a slight edge in name recognition over etavopivat (Novo Nordisk). Interestingly, despite this difference in familiarity, physicians identified slightly more patients as potential candidates for etavopivat, hinting at a broader perceived clinical fit once awareness and understanding of the agent catch up. Chart audit data allows users to probe further into specific patient types which physicians perceive as better fits for either agent, as well as those who they do not perceive as candidates. This growing interest in emerging oral therapies reflects a broader desire among hematologists for more accessible, lower-burden treatment options. While most patients are theoretically eligible for curative approaches like gene therapy, the market still favors lower-barrier alternatives. These alternatives include oral therapies that do not require referral to specialized centers or extensive pre-conditioning, and brands with which they are more familiar given other indications such as Reblozyl (Bristol Myers Squibb). Disease-modifying therapies that are easier to prescribe and integrate into routine practice have the promise to significantly improve the lives of patients who would not qualify or want gene therapy. Access barriers compound the issue, with over half of physicians voicing frustration over payer restrictions that make it difficult to initiate optimal care. These challenges highlight a critical need – not just for clinical innovation, but also for strong manufacturer support in navigating reimbursement and expanding access. As the beta thalassemia landscape continues to evolve, hematologists are increasingly looking toward emerging therapies that are easier to prescribe, carry fewer logistical burdens than gene therapy, and offer clear reductions in transfusion dependency. Products like mitapivat and etavopivat are well-positioned to address this gap, assuming familiarity, confidence, and access grow in tandem – but the goal to cure patients of hemoglobinopathies still carries heavy weight, with desire to treat with gene therapy consistently growing. Patient Chart Dynamix™ is an independent, data-driven service unveiling real patient management patterns through rigorous analysis of large-scale patient chart audits. Insights reveal the 'why' behind treatment decisions, include year over year trending to quantify key aspects of market evolution, and integrate specialists' attitudinal & demographic data to highlight differences between stated and actual treatment patterns. About Spherix Global Insights Spherix is a leading independent market intelligence and advisory firm that delivers commercial value to the global life sciences industry, across the brand lifecycle. The seasoned team of Spherix experts provides an unbiased and holistic view of the landscape within rapidly evolving specialty markets, including dermatology, gastroenterology, rheumatology, nephrology, neurology, ophthalmology, and hematology. Spherix clients stay ahead of the curve with the perspective of the extensive Spherix Physician Community. As a trusted advisor and industry thought leader, Spherix's unparalleled market insights and advisory services empower clients to make better decisions and unlock opportunities for growth. To learn more about Spherix Global Insights, visit or connect through LinkedIn. For more details on Spherix's primary market research reports and interactive dashboard offerings, visit or register here: NOTICE: All company, brand or product names in this press release are trademarks of their respective holders. The findings and opinions expressed within are based on Spherix Global Insight's analysis and do not imply a relationship with or endorsement of the companies or brands mentioned in this press release. CONTACT: Sarah Hendry, Hematology Franchise Head Spherix Global Insights 4848794284 in to access your portfolio
Yahoo
13-05-2025
- Health
- Yahoo
Top 10 most expensive prescription drugs in the US by price and by sales volume
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. Read On The Fox News App "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. 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. "This gene therapy can help patients make healthy red blood cells on their own and reduce how often they need treatment," said Dubinsky. 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. 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 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." Greg Norman of Fox News Digital contributed article source: Top 10 most expensive prescription drugs in the US by price and by sales volume
CBS News
28-04-2025
- Health
- CBS News
First patient to receive gene therapy treatment developed at Children's Hospital of Philadelphia living pain-free
A new gene therapy developed at the Children's Hospital of Philadelphia has been approved by the FDA and is being used to transform lives. The first patient to receive this gene therapy after it was FDA cleared is now living pain-free for the first time. Bike riding is fun now for 12-year-old Rahemeen Nabeel, who lives in Cherry Hill, New Jersey. "I do lots of sports, and now that I have like more energy and stuff, I actually joined clubs and stuff," Nabeel said. Energy is something that was in short supply for most of her life. Nabeel was born with an inherited blood disorder called beta thalassemia. "It was just, like, so painful," she said. "Like I was in constant pain." And there were constant blood transfusions — a struggle for the family, who lived in Dubai. "So we took her to Paris, we took her to Italy, Rome, Switzerland, and then we took her to Turkey, Korea," said Zainab Nabeel, the 12-year-old's mother. They finally ended up at the Children's Hospital of Philadelphia, which had done extensive research on a gene therapy where the patients' own stem cells are reprogrammed to fix the blood disorder. "It was like a nightmare, but also like a really big blessing," Rahemeen Nabeel said. The treatment includes chemotherapy that allows the gene therapy to work. "It was a long, stressful journey, but we are really, really happy," Zainab Nabeel said. It's been two years since Rahemeen Nabeel became the first patient to receive the FDA-approved treatment. Her mother said she's considered cured now. No longer needing transfusions, Rahemeen Nabeel is back to enjoying her pet bird, Mango, and is doing well in 6th grade. "Now I'm a bit too energized," she said. The new gene therapy treatment, called Zynteglo, is being called a game changer. "To see a therapy like this move into being widely available for patients, this is why we do what we do," said CHOP's Dr. Timothy Olson. CHOP has about 17 patients in various stages of treatment with the new gene therapy. Rahemeen Nabeel, who led the way, said she eventually wants to do something that combines science with her love for animals.
Yahoo
17-04-2025
- Business
- Yahoo
Bluebird bio reaffirms Carlyle deal as Ayrmid misses deadline
Bluebird bio (bluebird) has reaffirmed its support for a proposed acquisition by Carlyle and SK Capital after rival bidder Ayrmid failed to deliver a binding offer or secure financing by the extended deadline. The announcement marks the second time Ayrmid has initiated discussions with bluebird without producing a fully financed proposal. Bluebird said it had engaged with Ayrmid for three weeks following the private equity firm's unsolicited bid in March, including extending the original deadline to allow more time for due diligence and deal structuring. However, Ayrmid did not submit a definitive proposal by the agreed-upon 15 April cut-off date and has yet to secure the necessary capital to proceed. As a result, bluebird's board of directors is reiterating its unanimous support for the acquisition agreement with Carlyle and SK Capital, which was first announced in February 2025. The board is urging shareholders to tender their shares by the updated deadline of 2 May, extending the previous expiration date of 18 April. bluebird's board chairman Mark Vachon said: 'Ayrmid's proposal remains highly conditional. bluebird has engaged with Ayrmid on two separate occasions—neither of which has resulted in a binding or fully-financed offer. The board unanimously reaffirms its support of the previously announced agreement with Carlyle and SK Capital in the strongest possible terms.' Bluebird has been under significant financial pressure for several years. The company currently faces the risk of defaulting on a $175m loan from Hercules Capital, secured in 2024 to support ongoing operations. Without a new capital injection, the board said, the company's liquidity position remains precarious. Carlyle and SK Capital's proposal offers $3 per share in cash, along with a potential additional payment of $6.84 per share if certain clinical and commercial milestones are met. Ayrmid's offer, disclosed last month, proposed $4.50 per share upfront and the same milestone-based payout structure. However, bluebird confirmed that Ayrmid has not demonstrated access to committed funding. bluebird noted that Ayrmid is continuing to seek financing and may provide further updates in the coming days. bluebird's financial position has deteriorated over time, despite achieving several key regulatory milestones. The company currently markets three US Food and Drug Administration (FDA)-approved gene therapies – Zynteglo (betibeglogene autotemcel) for transfusion-dependent beta-thalassemia, Skysona (elivaldogene autotemcel) for cerebral adrenoleukodystrophy, and Lyfgenia (lovotibeglogene autotemcel) for sickle cell disease – but has struggled with slow uptake, high costs, and reimbursement hurdles. In 2023, the company suffered a major setback when the FDA denied a priority review voucher (PRV) for Lyfgenia. Bluebird had already arranged to sell the voucher to Novartis for $103m, but the agency ruled that the therapy's active ingredient had previously been used in Zynteglo, for which the company had already received a PRV in 2022. The loss of the expected funds deepened bluebird's financial strain. To fill the gap, bluebird pursued emergency financing through a $150m public stock offering underwritten by Goldman Sachs and JP Morgan, as well as up to $100m in receivables financing from Alterna Capital Solutions. The company has also undergone repeated cost-cutting efforts, including workforce reductions of 30% in 2022 and an additional 25% last year. Despite these efforts, bluebird's therapies continue to face stiff competition. Lyfgenia is priced at $3.1m per patient, while a competing treatment, Casgevy (exagamglogene autotemcel) from Vertex Pharmaceuticals and CRISPR Therapeutics, is listed at $2.2m. Both therapies were approved on the same day in December 2023 to treat sickle cell disease. Going public in 2013, bluebird has seen its stock decline sharply in recent years. Following the 16 April update, shares closed down by 7%. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. "Bluebird bio reaffirms Carlyle deal as Ayrmid misses deadline" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site.
