FDA Approves Medexus's Supplemental Biologics License Application for Ixinity to Treat Hemophilia B in Pediatric Patients
Toronto, Ontario and Chicago, Illinois-- March 26, 2024 - Medexus Pharmaceuticals (TSX: MDP) (OTCQX: MEDXF) today announced that the US Food and Drug Administration (FDA) recently approved Medexus ' s supplemental Biologics License Application (sBLA)... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - March 26, 2024 Category: Drugs & Pharmacology Source Type: news

The World ’s Most Expensive Drug Is Now a $4.25 Million Gene Therapy
A new gene therapy for an ultra-rare disease will have a wholesale cost of $4.25 million, making it the world’s most expensive drug. The one-time treatment, Lenmeldy, won U.S. regulatory approval on Monday to correct the underlying cause of a hereditary condition called early-onset metachromatic leukodystrophy, or MLD. MLD is a fatal disease in which infants sometimes start to lose the ability to walk and talk. Orchard Therapeutics said the drug’s price “reflects its clinical, economic and societal value” in a statement Wednesday.  [time-brightcove not-tgx=”true”] Read Mo...
Source: TIME: Health - March 21, 2024 Category: Consumer Health News Authors: Gerry Smith/Bloomberg Tags: Uncategorized healthscienceclimate wire Source Type: news

7 Biotech Stocks Ready to Ride the Sector ’s Resurgence
A rotation may be on the way Why do you want to consider biotech stocks? Simply, the narrative comes down to numbers and a possible rotation. First, the hard data supports bullishness in biotech stocks. According to Grand View Research, the underlying global industry reached a valuation of $1.55…#grandviewresearch #minneapolis #minnesota #biotechne #q4 #eps #hemophilia #cnta #q3 #westconshohocken (Source: Reuters: Health)
Source: Reuters: Health - March 19, 2024 Category: Consumer Health News Source Type: news

Why Novo Nordisk Stock Was a Winner Today
One of the healthcare stocks of the moment, Novo Nordisk (NYSE: NVO), had a good Tuesday on the market. Investors pushed its price 3% higher, more than double the percentage rate increase of the S&P 500 index, on the back of a positive move by an analyst. That action occurred late Monday…#novonordisk #nvo #argusresearch #jimkelleher #capitalonefinancial #denmark #hemophilia #ozempicwegovy #elililly #ericvolkman (Source: Reuters: Health)
Source: Reuters: Health - March 13, 2024 Category: Consumer Health News Source Type: news

[Ad hoc announcement pursuant to Art. 53 LR] Roche exceeds guidance and achieves sales growth of 1% (CER) for 2023 despite sharp COVID-19 sales decline
Group salesgrow by 1%1 at constant exchange rates (CER; -7% in CHF), more than offsetting the decline in COVID-19-related sales and biosimilar erosion, and thereby exceeding 2023 guidanceExcluding COVID-19 products,Group sales increase by 8%Pharmaceuticals Divisionsalesincrease by 6% (excluding COVID-19 medicine Ronapreve: +9%) due to ongoing high demand for newer medicines, with eye medicine Vabysmo continuing to be the top growth driver, followed by Ocrevus (multiple sclerosis), Hemlibra (haemophilia A) and Polivy (blood cancer)Diagnostics Division salesare 13% lower due to high demand for COVID-19 tests in 2022; strong ...
Source: Roche Media News - February 1, 2024 Category: Pharmaceuticals Source Type: news

[Ad hoc announcement pursuant to Art. 53 LR] Roche exceeds guidance and achieves sales growth of 1% (CER) for 2023 despite sharp COVID-19 sales decline
Group salesgrow by 1%1 at constant exchange rates (CER; -7% in CHF), more than offsetting the decline in COVID-19-related sales and biosimilar erosion, and thereby exceeding 2023 guidanceExcluding COVID-19 products,Group sales increase by 8%Pharmaceuticals Divisionsalesincrease by 6% (excluding COVID-19 medicine Ronapreve: +9%) due to ongoing high demand for newer medicines, with eye medicine Vabysmo continuing to be the top growth driver, followed by Ocrevus (multiple sclerosis), Hemlibra (haemophilia A) and Polivy (blood cancer)Diagnostics Division salesare 13% lower due to high demand for COVID-19 tests in 2022; strong ...
Source: Roche Investor Update - February 1, 2024 Category: Pharmaceuticals Source Type: news

Experimental Gene Therapy Allows Kids with Inherited Deafness to Hear
Gene therapy has allowed several children born with inherited deafness to hear. A small study published Wednesday documents significantly restored hearing in five of six kids treated in China. On Tuesday, the Children’s Hospital of Philadelphia announced similar improvements in an 11-year-old boy treated there. And earlier this month, Chinese researchers published a study showing much the same in two other children. [time-brightcove not-tgx=”true”] So far, the experimental therapies target only one rare condition. But scientists say similar treatments could someday help many mor...
Source: TIME: Health - January 25, 2024 Category: Consumer Health News Authors: LAURA UNGAR/AP Tags: Uncategorized wire Source Type: news

Health Canada approves Pfizer's gene therapy for treatment of hemophilia
Pfizer said on Wednesday that Canada's health regulator approved its gene therapy for the treatment of a rare inherited bleeding disorder called hemophilia B ahead of a U.S. decision. (Source: CBC | Health)
Source: CBC | Health - January 3, 2024 Category: Consumer Health News Tags: News/Health Source Type: news

Gene Therapy for Hemophilia Becomes a Reality
(MedPage Today) -- Earlier this year we reported on FDA's approval of valoctocogene roxaparvovec (Roctavian) for adults with severe hemophilia A. In this story, we provide an update on what has happened since. Two gene therapies have now been... (Source: MedPage Today Hematology/Oncology)
Source: MedPage Today Hematology/Oncology - December 29, 2023 Category: Hematology Source Type: news

Synergy Billing Corp. Welcomes WellPha Specialty Pharmacy to its Customer Portfolio
Revenue cycle management and consulting firm pairs a national reach with a boutique approach to deliver custom solutions for WellPha in the Medicare hemophilia patient population HIGH POINT, N.C., Dec. 12, 2023 /PRNewswire-PRWeb/ -- Synergy Billing Corp., a boutique consulting and... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - December 12, 2023 Category: Pharmaceuticals Tags: LIC Source Type: news

Novel Monoclonal Antibody Reduces Bleeds in Hemophilia A and B
(MedPage Today) -- SAN DIEGO -- The investigational monoclonal antibody marstacimab reduced the rate of treated bleeds in patients with hemophilia A or B without inhibitors to factor VIII or factor IX, the phase III BASIS trial showed. The annualized... (Source: MedPage Today Hematology/Oncology)
Source: MedPage Today Hematology/Oncology - December 12, 2023 Category: Hematology Source Type: news

FDA and EMA Accept Marstacimab Regulatory Submissions for the Treatment of Hemophilia A and B
Submissions based on positive data from the Phase 3 BASIS trial, which were presented this past weekend at the American Society of Hematology (ASH) Annual Meeting If approved in the U.S. and EU, marstacimab could become the first once-weekly... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - December 11, 2023 Category: Drugs & Pharmacology Source Type: news

Emicizumab Safe, Effective, in Infants With Hemophilia A
(MedPage Today) -- SAN DIEGO -- Administering emicizumab (Hemlibra) to previously untreated or minimally treated infants with severe hemophilia A without factor VIII inhibitors is effective and safe, according to results from research presented... (Source: MedPage Today Hematology/Oncology)
Source: MedPage Today Hematology/Oncology - December 10, 2023 Category: Hematology Source Type: news

New data reinforce the benefit of early preventative treatment with Roche ’s Hemlibra for babies with severe haemophilia A
Phase III HAVEN 7 primary data presented at ASH 2023 provide additional confidence in the favourable efficacy and safety profile of subcutaneous Hemlibra given soon after birth1At nearly two years median follow-up in the descriptive, single-arm study, no babies experienced spontaneous bleeds requiring treatment, and all treated bleeds were as a result of trauma1Safety results were consistent with previous studies of Hemlibra, with no new safety signals observed1The HAVEN 7 study was developed in collaboration with the haemophilia A community, to generate additional evidence for the prophylactic treatment of infants with ha...
Source: Roche Media News - December 9, 2023 Category: Pharmaceuticals Source Type: news

New data reinforce the benefit of early preventative treatment with Roche ’s Hemlibra for babies with severe haemophilia A
Phase III HAVEN 7 primary data presented at ASH 2023 provide additional confidence in the favourable efficacy and safety profile of subcutaneous Hemlibra given soon after birth1At nearly two years median follow-up in the descriptive, single-arm study, no babies experienced spontaneous bleeds requiring treatment, and all treated bleeds were as a result of trauma1Safety results were consistent with previous studies of Hemlibra, with no new safety signals observed1The HAVEN 7 study was developed in collaboration with the haemophilia A community, to generate additional evidence for the prophylactic treatment of infants with ha...
Source: Roche Investor Update - December 9, 2023 Category: Pharmaceuticals Source Type: news