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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

Gene therapy has allowed several children born with inherited deafness to hear. Globally, 34 million children have deafness or hearing loss, and genes are responsible for up to 60% of cases. Hereditary deafness is the latest condition scientists are targeting with gene therapy, which is already approved to treat illnesses such as sickle cell disease and severe hemophilia. Also, some people consider gene therapy for deafness ethically problematic. "This is real proof showing gene therapy is working,” Chen said.

Persons: ”, Zheng, Yi Chen, Dr, Yilai Shu, Chen, Eli Lilly, Akouos, he's, John Germiller, ” Germiller, “, Lawrence Lustig, Teresa Blankmeyer Burke, ” Chen Organizations: Children's Hospital of Philadelphia, Fudan University, Shanghai Refreshgene Therapeutics, Regeneron Pharmaceuticals, Decibel Therapeutics, Columbia, Gallaudet University, Associated Press Health, Science Department, Howard Hughes Medical Institute’s Science, Educational Media Group, AP Locations: China, Shanghai, Philadelphia, Spain

☆ Exclusive: Activist investor Elliott targets drugmaker BioMarin -sources
▼ + stars: | 2023-11-07 | by ( Svea Herbst-Bayliss | ) www.reuters.com time to read: +3 min

NEW YORK, Nov 7 (Reuters) - Activist investor Elliott Investment Management has built a stake in BioMarin Pharmaceutical (BMRN.O) and has been in discussions with the biotechnology company for months about its future, according to two people familiar with the matter. Elliott declined to comment, while a representative for BioMarin did not immediately respond to a request for comment. BioMarin shares rose 12% to $85.36 on the news in morning trading in New York on Tuesday. Prior to news of Elliott's engagement, BioMarin shares were down 24% year-to-date, significantly underperforming the iShares Biotechnology ETF (IBB.O), which is down 8.6%. Elliott also successfully pushed for drug makers Alexion Pharma (AZN.L) and Allergan (ABBV.N) to be sold.

Persons: Elliott, BioMarin, hemophilia, Jean, Jacques Bienaime, Alexander Hardy, Jefferies, Bienaime, Harvey, Richard Meier, Svea Herbst, Bayliss, Jamie Freed Organizations: Investment Management, BioMarin Pharmaceutical, Biotechnology, Alexion Pharma, Svea, Thomson Locations: BioMarin, San Rafael , California, West Palm Beach , Florida, New York

☆ A New Sickle Cell Treatment Will Change Lives — but How Many? - The New York Times
▼ + stars: | 2023-10-30 | by ( Daniela J. Lamas | ) www.nytimes.com time to read: +3 min

And though comprehensive sickle cell care — at dedicated centers with expert hematologists, social workers and pain management specialists — reduces hospitalizations, and is the standard for diseases like cystic fibrosis and hemophilia, which do not disproportionately affect Black people, these centers are few and far between for sickle cell. Into this complicated landscape enters the possibility of gene therapy. It’s important to note that this isn’t the first cure for sickle cell. is expected to review another gene therapy from the company Bluebird Bio that targets sickle cell disease but does not use CRISPR; this was the therapy Mr. Holmes received as part of the N.I.H. When she was 17 and hospitalized, facing the reality of her chronic illness, she told her mother that she was ready to pursue gene therapy.

Persons: Holmes, Elizabeth Ford, Ford Organizations: Vertex Pharmaceuticals, CRISPR Therapeutics, Bluebird

☆ AI, weight loss drugs and more: Portfolio manager names stocks to play 'new secular growth themes'
▼ + stars: | 2023-10-24 | by ( Amala Balakrishner | ) www.cnbc.com time to read: +4 min

And then you overlay on top of that the emergence of two or three brand new secular growth themes," he said. CNBC Pro takes a look at the stocks he likes in three "brand new" areas of growth: obesity drugs, reshoring and artificial intelligence. Reshoring infrastructure Ayer sees bright spots in the reshoring theme too. The portfolio manager named tech giant Amazon as a good play of the AI wave. The way Ayer sees it, more companies in the U.S. are foraying into AI right now, compared to those in other parts of the world.

Persons: Sanjay Ayer, Ayer, He's bullish, —, Christina Cheddar Berk, Carmen Reinicke, Julie Coleman Organizations: CNBC Pro, WCM Investment Management, Equity Fund, Nordisk, Novo Nordisk, U.S . Food, Drug Administration, Tetra Tech, Canadian, Canadian Pacific, U.S Locations: U.S, China, Japan, India

☆ Here are Friday's biggest analyst calls of the day: Tesla, Ford, Apple, Amazon, Disney, Nike, FedEx and more
▼ + stars: | 2023-09-29 | by ( Michael Bloom | ) www.cnbc.com time to read: +6 min

Bank of America reiterates Disney as buy Bank of America said it's standing by its buy rating on the stock. Morgan Stanley reiterates Ford as overweight Morgan Stanley said it's standing by its overweight rating on shares of Ford despite the United Auto Workers strike. Canaccord reiterates Tesla as buy Canaccord said its standing by its buy rating on Tesla ahead of its upcoming delivery numbers report. Morgan Stanley reiterates Apple as overweight Morgan Stanley said it's standing by its overweight rating on shares of Apple. Morgan Stanley reiterates Amazon as overweight Morgan Stanley said Amazon continues to be the "dominant" player in e-commerce.

Persons: Jefferies, Edward Jones downgrades Berkshire Hathaway, Edward Jones, Morgan Stanley, Ford, it's, Canaccord, Tesla, we've, Goldman Sachs, Raymond James, Bud Light, Apple, Amazon, Brinker, Stifel, Huntington Organizations: Jefferies, Ball Corporation, Bank of America, Disney, DIS, CYE23, Texas, 3Q23, Brands, Burger King, Ford, United Auto Workers, HSBC, FedEx, UPS, DHL, Nike, Immunology, " Bank of America, Anheuser, Busch InBev, of America, Apple, Citi Locations: Texas, QSR, North America, Asia, ZION

☆ 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

☆ New $2.9 Million Gene Therapy Promises to Remake Hemophilia Treatment
▼ + stars: | 2023-06-30 | by ( Jared S. Hopkins | ) www.wsj.com time to read: 1 min

This copy is for your personal, non-commercial use only. Distribution and use of this material are governed by our Subscriber Agreement and by copyright law. For non-personal use or to order multiple copies, please contact Dow Jones Reprints at 1-800-843-0008 or visit www.djreprints.com. https://www.wsj.com/articles/new-2-9-million-gene-therapy-promises-to-remake-hemophilia-treatment-da254ef4

Persons: Dow Jones

☆ US FDA approves BioMarin's gene therapy for hemophilia A
▼ + stars: | 2023-06-29 | by ( Nathan Gomes | ) www.reuters.com time to read: +3 min

It priced the one-time therapy, Roctavian, at $2.9 million. BioMarin said most study participants continued to respond to the gene therapy through year three and beyond, and it would continue to monitor them for 15 years. BioMarin's therapy will compete for market share with Roche's (ROG.S) Hemlibra, an antibody drug which mimics the function of the blood clotting protein missing in hemophilia A patients. In April, BioMarin cut its annual sales forecast range for Roctavian to $50 million to $100 million, from $100 million to $200 million. There are about 16,000 patients in the United States with hemophilia A in which they are missing the factor VIII clotting protein.

Persons: BioMarin, Joel Beatty, Robert W Baird, Roctavian, Beatty, Khushi Mandowara, Bhanvi Satija, Nathan Gomes, Akash Sriram, Deena Beasley, Krishna Chandra Eluri, Maju Samuel, David Gregorio Our Organizations: U.S . Food, Drug Administration, European Union, Los, Thomson Locations: U.S, California, Novato , California, hemophilia, United States, Bengaluru, Los Angeles

☆ Stocks making the biggest moves midday: Dollar Tree, Amazon, FedEx and more
▼ + stars: | 2023-06-21 | by ( Alex Harring | ) www.cnbc.com time to read: +4 min

People walk by a Dollar Tree store on December 11, 2018 in the Brooklyn borough of New York City. FedEx — The delivery company fell 1.7% after quarterly revenue missed expectations and announced CFO Mike Lenz would retire on July 31. Adjusted earnings were better than expected at $4.94 per share against the anticipated $4.89, while forward guidance was around flat. Advanced Micro Devices — Shares of the chipmaker pulled back nearly 5%, on track for their biggest intraday loss in two weeks. Dollar Tree — Shares of Dollar Tree popped more than 3% after the discount retailer reiterated its fiscal second-quarter 2023 earnings guidance.

Persons: Mike Lenz, MicroStrategy, Tesla, Rivian, Goldman, OneSpaWorld, Walt Disney, Needham, Laura Martin, Bob Iger, Raymond James, Uniqure, hemophilia, Yun Li, Michelle Fox, Jesse Pound, Sarah Min, Brian Evans Organizations: Federal Trade Commission, Amazon, FedEx, Securities and Exchange Commission, Barclays, GlaxoSmithKline —, Petrobras —, Walt Disney —, AMD Locations: Brooklyn, New York City, bitcoin, Netherlands

☆ I make about $6,500 donating plasma over 100 times a year, but I don't do it for the money
▼ + stars: | 2023-03-19 | by ( Emily Swaim | Jessica Orwig | ) www.businessinsider.com time to read: +4 min

Patrick Herdener donates plasma twice a week, every week and makes between $50-$70 per donation. Herdener said he started donating for money but the spirit of being helpful motivates him now. To be honest, when I first started donating plasma, the main thing that motivated me was money. I started donating more often when I learned where my plasma was going. Patrick Herdener has been donating plasma twice a week for 13 years.

People often write off donating plasma as a side hustle for college kids, but my husband and I think the extra money can be worth it. How donating plasma worksDonating plasma isn't the same thing as donating blood. How to earn money donating plasmaDonating plasma can be a relatively low-effort side gig. Donating plasma: Frequently asked questionsWhat makes you ineligible to donate plasma? What are the side effects of donating plasma?

☆ Roche flags 2023 earnings decline on slump in Covid products
▼ + stars: | 2023-02-02 | by ( ) www.cnbc.com time to read: +2 min

A view of the Roche towers, designed by architects Herzog and de Meuron, the headquarters of the Swiss pharma giant Roche, in Basel. Roche warned of a decline in 2023 earnings, as revenue growth from new drugs including haemophilia treatment Hemlibra and multiple sclerosis drug Ocrevus would not make up for a steep demand drop for Covid treatments and diagnostic testing. Sales and core earnings per share were expected to decrease at a "low single-digit" percentage this year, the Swiss drugmaker said in a statement on Thursday. Last year, group revenue edged 1% higher to 63.3 billion Swiss francs ($69.78 billion), the company reported, beating market expectations of 63.2 billion francs, while core operating profit gained 1% to 22.2 billion Swiss francs, just shy of the average analyst estimate of 22.4 billion francs. The decline equates to 5 billion Swiss francs, in his estimate.

☆ Evercore ISI's top picks for 2023 include a tech comeback story and a car racing play
▼ + stars: | 2023-01-12 | by ( Michelle Fox | ) www.cnbc.com time to read: +4 min

Finding opportunities amid the market volatility is "mission critical," and there are several to be had this year, according to Evercore ISI. The Wall Street firm is predicting an economic and earnings recession, catalyzing a "cathartic" volatility spike in 2023. "Alpha opportunities are surfacing in 2022's wreckage from inflation's breakout resulting in record tightening, catalyzing a stock/bond correlated decline. With that in mind, Evercore came up with its top stock picks for 2023. The streaming company should enjoy a comeback this year, after losing 51% in 2022, according to analyst Mark Mahaney.

☆ U.S. new drug price exceeds $200,000 median in 2022
▼ + stars: | 2023-01-05 | by ( Deena Beasley | ) www.reuters.com time to read: +3 min

The median annual price of the 17 novel drugs the U.S. Food and Drug Administration (FDA) approved since July 2022 is $193,900, down from $257,000 in the first half of 2022, Reuters found. For full year 2022, the median was $222,003. Reuters Graphics Reuters GraphicsIn 2021, the median annual price was $180,000 for the 30 drugs first marketed through mid-July, according to a study published recently in JAMA. Health insurers and other payers often demand discounts and rebates for prescription drugs once competing treatments become available. As patents expire, lower-cost generics also mitigate prescription drug price inflation, which in the 12 months through November 2022 was 1.9%, according to the Bureau of Labor Statistics.

☆ Pfizer's hemophilia B gene therapy succeeds in late-stage study
▼ + stars: | 2022-12-29 | by ( ) www.reuters.com time to read: +1 min

Dec 29 (Reuters) - U.S. drugmaker Pfizer Inc (PFE.N) said on Thursday its experimental gene therapy for the treatment of hemophilia B, a rare inherited blood disorder, met its main goal in a late-stage study. The drugmaker licensed its hemophilia B gene therapy from Roche's (ROG.S) Spark Therapeutics unit in 2014 for a $20-million upfront payment. Pfizer plans to discuss the late-stage data with regulatory authorities in Europe and the United States and share additional data for the experimental therapy at a scientific conference in early 2023. According to government data, the estimated prevalence of hemophilia in the United States is 12 cases per 100,000 males for hemophilia A and 3.7 cases per 100,000 males for hemophilia B. In November, the U.S. health regulator approved the first gene therapy, CSL Ltd and uniQure's Hemgenix, to treat hemophilia B.Pfizer is also testing other experimental gene therapies in late-stage trials as potential treatments for the bleeding disorder hemophilia A and muscular disorder Duchenne muscular dystrophy.

☆ Drug Prices Reach New High—in the Millions
▼ + stars: | 2022-12-26 | by ( Peter Loftus | ) www.wsj.com time to read: 1 min

The most recent gene therapy approved in the U.S. set a price record: $3.5 million for CSL’s Hemgenix, a treatment for hemophilia B. A new era of expensive drugs has arrived: medicines priced in the millions of dollars a patient. Since August, U.S. or European health regulators have approved four new products intended as one-time treatments for rare genetic diseases that carry list prices of at least $2 million a patient, including two from Bluebird Bio Inc.

☆ Health-care stocks are looking good for 2023 and not just because the sector is a 'safe haven'
▼ + stars: | 2022-12-21 | by ( Christina Cheddar Berk | ) www.cnbc.com time to read: +9 min

Investors looking for cover this year would have done well if they sought a safe haven in large-cap pharmaceutical stocks, a trend that is likely to continue into 2023. The Inflation Reduction Act provided some clarity around drug pricing that should help health-care stocks. The average price target, according to FactSet, is $62, or nearly 40% above the stock's closing price on Tuesday. Gene therapies in focus Investors will be closely watching the progress of several gene therapies, according to Phipps. "Yes, these are expensive therapies," Phipps said.

☆ 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.

☆ Morgan Stanley's top biotech picks for 2023. One 'transformative' stock promises 50% upside
▼ + stars: | 2022-12-02 | by ( Samantha Subin | ) www.cnbc.com time to read: +3 min

Morgan Stanley views biotechnology as an area of the market that abounds with opportunity heading into 2023. It's been a painful year for the investors — and once high-flying growth stocks — as the Federal Reserve's rate hikes lead to fears of a slowing economy. Morgan Stanley also expects mergers and acquisitions activity to pick up in 2023 given the piles of cash some companies have on hand. Here are some of the stocks Morgan Stanley recommends heading into the new year: Harrison named BeiGene among the bank's biotech stock picks heading into the new year. "We expect 2023 to finally be the year that commercial upside can drive BMRN higher," Harrison wrote.

☆ 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.

☆ A top Wall Street analyst names 6 major events that could send biotech stocks soaring between now and the end of 2022
▼ + stars: | 2022-11-14 | by ( Yeji Jesse Lee | ) www.businessinsider.com time to read: +5 min

Cantor Fitzgerald analysts named six big events to mark on your calendar. While the majority of the expected events are set to come from smaller biotech companies, bigger companies — like Roche, Gilead, and Alnylam — have their own events coming through the pipeline. Here are the six major events from big biotech companies that could send stocks soaring:1. Cantor Fitzgerald analysts give Gilead's stock a neutral rating and they have an overweight rating on Arcus. Lenacapavir "could become a much bigger piece of the HIV growth narrative over the next few years," according to Cantor Fitzgerald analysts.

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