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☆ FDA approves Pfizer’s first gene therapy for rare inherited bleeding disorder
▼ + stars: | 2024-04-26 | by ( Annika Kim Constantino | ) www.cnbc.com time to read: +3 min

The Food and Drug Administration on Friday approved Pfizer 's treatment for a rare genetic bleeding disorder, making it the company's first-ever gene therapy to win clearance in the U.S. The agency greenlit the drug, which will be marketed as Beqvez, for adults with moderate to severe hemophilia B who meet certain requirements. Without that protein, called factor IX, patients with hemophilia B bruise easily and bleed more frequently and for longer periods of time. The gene therapy will compete with Australia-based CSL Behring's Hemgenix, a similar treatment that won FDA approval for hemophilia B in 2022. The company is also developing a gene therapy for Duchenne muscular dystrophy, a genetic disorder that causes muscles to weaken gradually.

Persons: IX, hemophilia, Adam Cuker, Cuker Organizations: Drug Administration, Pfizer, U.S, Penn's, Spark Therapeutics, hemophilia Locations: U.S, Australia

☆ Insight: What happens when a $2 million gene therapy is not enough
▼ + stars: | 2023-08-12 | by ( Deena Beasley | ) www.reuters.com time to read: +13 min

Their experience raises broader questions around other high-cost gene therapies coming to market, sometimes after accelerated regulatory approvals, drug pricing experts said. Gene therapies work by replacing genes – the body's blueprint for its development. The gene Zolgensma delivers instructs the body to make a protein vital for muscle control. If gene therapies do fall short, it becomes harder to justify prices that researchers have argued are already poor value. More recently, the first hemophilia gene therapy approved by the U.S. Food and Drug Administration was priced by CSL Behring at $3.5 million; 26 more gene therapies are in late-stage development, according to IQVIA.

Persons: Elizabeth Kutschke, Ben, Zolgensma, Ben Kutschke, neurologists, Sitra Tauscher, Wisniewski, Ben's, Roger Hajjar, Brigham Gene, Kutschke, Vasant Narasimhan, Stacie Dusetzina, Roche's, Biogen, Roche, Maha Radhakrishnan, Steven Pearson, It's, Sree Chaguturu, Amanda Cook, Weston, Jackson, Cook, Elizabeth, Jerry Mendell, Russell Butterfield, –, Biogen's, Mendell, UMR, Spinraza, Eric Cox, Caroline Humer, Sara Ledwith Organizations: Reuters, U.S, Novartis, IQVIA Institute, Human Data, Novartis Gene Therapies, Mass, Cell Therapy, U.S . Food, Drug Administration, CSL Behring, CSL, Nashville's Vanderbilt University School of Medicine, Clinical, Economic, CVS Health, Aetna, SMA, Nationwide Children's Hospital, University of Utah Health, Children's, UnitedHealth, Thomson Locations: Oak Park, Berwyn , Illinois, Swiss, U.S, Lebanon , Virginia, United States, Columbus , Ohio, Russia, Kazakhstan, Chicago

☆ The most expensive drug in the world has a $3.5 million price tag. 2 potential patients share their hopes and fears for the one-time treatment.
▼ + stars: | 2022-12-05 | by ( Andrew Dunn | ) www.businessinsider.com time to read: +7 min

The FDA has approved a new gene therapy to treat hemophilia B, a genetic bleeding disorder. The drugmaker CSL Behring set a $3.5 million price for the one-time treatment. Lackey is learning to live with a severe case of hemophilia B, a rare genetic condition that keeps blood from clotting properly. But a critical unknown in judging CSL's $3.5 million price is the durability of the treatment. Lojewski said the $3.5 million price wasn't guided by other gene-therapy prices.

☆ The most expensive drug in the world just got approved at $3.5 million, and hemophilia patients are conflicted: 'Compared to what we pay, it doesn't seem too terrible.'
▼ + stars: | 2022-12-01 | by ( Andrew Dunn | ) www.businessinsider.com time to read: +7 min

The FDA has approved a new gene therapy to treat hemophilia B, a genetic bleeding disorder. Hemophilia patients told Insider they're excited about the new drug but worried about the price. Lackey is learning to live with a severe case of hemophilia B, a rare genetic condition that keeps blood from clotting properly. While the treatment breaks ground as the first approved gene therapy for hemophilia, its price is also unprecedented. Lojewski said the $3.5 million price wasn't guided by other gene-therapy prices.

☆ Regulators have approved the world's most expensive medicine that costs $3.5 million per patient
▼ + stars: | 2022-11-24 | by ( Ryan Hogg | ) www.businessinsider.com time to read: +3 min

The FDA approved a treatment costing $3.5 million, making it the world's most expensive medicine. Hemgenix effectively treated several patients with the blood condition Hemophilia B in trials. An independent study said a fair price for the drug would be about $2.9 million. download the app Email address By clicking ‘Sign up’, you agree to receive marketing emails from Insider as well as other partner offers and accept our Terms of Service and Privacy PolicyUS regulators have approved a hemophilia drug that will cost $3.5 million per patient, making it the world's most expensive medicine. However, the drug will have a list price of $3.5 million per dose, Managed Healthcare Executive reported, making it the world's most expensive medicine by some distance.

☆ FDA approves $3.5 million treatment for hemophilia, now the most expensive drug in the world
▼ + stars: | 2022-11-23 | by ( Deidre Mcphillips | ) edition.cnn.com time to read: +2 min

CNN —The US Food and Drug Administration on Tuesday approved Hemgenix, a new drug to treat hemophilia. Manufacturer CSL Behring set the price at $3.5 million per treatment, making it the most expensive drug in the world. In a recent cost-effectiveness analysis of the drug, weighing health benefits against offset costs, ICER suggested that a fair price for the drug to be between $2.93 million and $2.96 million. According to an analysis by GoodRx, the most expensive drug in the US previously was Zolgensma, which was approved by the FDA in 2019 to treat spinal muscular atrophy and priced at $2.1 million for a course of treatment. FDA approved Hemgenix based on safety and effectiveness evaluated in two studies of about 60 adult men.

Nov 22 (Reuters) - Australian drugmaker CSL Ltd (CSL.AX) on Tuesday set the list price of its one-time gene therapy for hemophilia B at $3.5 million, making it the world's most expensive treatment, following its approval by the U.S. health regulator. The first gene therapy for the rare genetic blood clotting disorder offers a long-term solution for patients as against current treatments from Biogen (BIIB.O), Pfizer (PFE.N) and others that focus on regular infusions. The gene therapy consists of an engineered virus carrying a gene expressed in the liver to produce clotting factor IX. While Zynteglo was priced at $2.8 million, Skysona had a wholesale cost of $3.0 million. CSL shares were up 1% at A$300.62 on the Australian stock exchange, while UniQure stock edged up 0.9% in extended trading.

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