BioMarin Announces Advancements in FDA Review of ROCTAVIAN(TM) (Valoctocogene Roxaparvovec) for Adults with Severe Hemophilia A
FDA No Longer Plans to Hold an Advisory Committee Meeting, as Previously Planned, to Discuss the Biologics License Application (BLA)
BioMarin Remains on Track to Host Scheduled Manufacturing Inspections by FDA in the Coming Weeks
SAN RAFAEL, Calif., ... Biopharmaceuticals, FDA BioMarin Pharmaceutical, ROCTAVIAN, hemophilia A, gene therapy (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - November 23, 2022 Category: Pharmaceuticals Source Type: news
$3.5M gene therapy for hemophilia gets FDA approval
U.S. health regulators have approved the first gene therapy for hemophilia, a blood-clotting disorder with few treatment options (Source: ABC News: Health)
Source: ABC News: Health - November 23, 2022 Category: Consumer Health News Tags: Health Source Type: news
CSL Behring receives FDA approval for first hemophilia B gene therapy to treat adults
The global hemophilia treatment market size was estimated at $12.8 billion in 2021, and is projected to reach $26.9 billion by 2031 (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - November 23, 2022 Category: Biotechnology Authors: John George Source Type: news
U.S. Food and Drug Administration Approves CSL's Hemgenix(R) eEtranacogene dezaparvovec-drlb), the First Gene Therapy for Hemophilia B
This historic approval provides a new treatment option that reduces the rate of annual bleeds, reduces or eliminates the need for prophylactic therapy and generates elevated and sustained factor IX levels for years after a one-time infusion
With the app... Biopharmaceuticals, FDA CSL, HEMGENIX, etranacogene dezaparvovec-drlb, hemophilia (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - November 22, 2022 Category: Pharmaceuticals Source Type: news
FDA approves first gene therapy to treat adults with Hemophilia B
Today, the U.S. Food and Drug Administration approved Hemgenix (etranacogene dezaparvovec), an adeno-associated virus vector-based gene therapy for the treatment of adults with Hemophilia B (congenital Factor IX deficiency) who currently use Factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes. (Source: World Pharma News)
Source: World Pharma News - November 22, 2022 Category: Pharmaceuticals Tags: Featured FDA Regulatory Affairs Source Type: news
FDA Approves Hemgenix (etranacogene dezaparvovec-drlb) Gene Therapy for Hemophilia B
This historic approval provides a new treatment option that reduces the rate of annual bleeds, reduces or eliminates the need for prophylactic therapy and generates elevated and sustained factor IX levels for years after a one-time infusion
With... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - November 22, 2022 Category: Drugs & Pharmacology Source Type: news
Rapid development of lower leg compartment syndrome following firearm injury in a patient with moderate hemophilia B [letter] - Bodrozic J, Lekovic D, Koncar I, Sulovi ć Dzelatović N, Miljic P.
TO THE EDITOR: Hemophilia is the most common inherited bleeding disorder caused by factor VIII (hemophilia A) or factor IX (FIX) (hemophilia B) deficiency. The incidence of hemophilia A is 1 in 5,000, and that of hemophilia B is 1 in 30,000 live male b... (Source: SafetyLit)
Source: SafetyLit - November 14, 2022 Category: International Medicine & Public Health Tags: Economics of Injury and Safety, PTSD, Injury Outcomes Source Type: news
Roche to present data at ASH 2022 showcasing strength of haematology portfolio and expanding into new areas to address more patient needs
Interim data from phase III HAVEN 7 study reinforceHemlibra ’s efficacy and safety in infants with severehaemophilia A without factor VIII inhibitors1New and updated data support use ofPolivy in diffuse large B-cell lymphoma, including its potential as a treatment option for previously untreated patients2New and updated data for innovative CD20xCD3 T-cell engaging bispecific antibodiesLunsumio andglofitamab further enhance their potential as effective, off-the-shelf, fixed-duration treatment options for people with lymphoma3,4,5,6,7First phase III data forcrovalimab show the co-primary efficacy endpoints were met, with s...
Source: Roche Media News - November 3, 2022 Category: Pharmaceuticals Source Type: news
Roche to present data at ASH 2022 showcasing strength of haematology portfolio and expanding into new areas to address more patient needs
Interim data from phase III HAVEN 7 study reinforceHemlibra ’s efficacy and safety in infants with severehaemophilia A without factor VIII inhibitors1New and updated data support use ofPolivy in diffuse large B-cell lymphoma, including its potential as a treatment option for previously untreated patients2New and updated data for innovative CD20xCD3 T-cell engaging bispecific antibodiesLunsumio andglofitamab further enhance their potential as effective, off-the-shelf, fixed-duration treatment options for people with lymphoma3,4,5,6,7First phase III data forcrovalimab show the co-primary efficacy endpoints were met, with s...
Source: Roche Investor Update - November 3, 2022 Category: Pharmaceuticals Source Type: news
Advocacy in Hemophilia: Building the Future by Design
Advances in the treatment and care of hemophilia have accelerated in recent years. And, according to Jack Uldrich, global futurist and author, the field could be on the precipice of even more disruptive changes, coming at an accelerated pace.Uldrich explains that multiple innovations could potentially advance the field. These new frontiers include gene therapy and many other novel treatment approaches, such as mobile and decentralized blood-testing technology, artificial intelligence, nanomaterials for the delivery of novel treatments, advancements in battery technology (enabling drone-blood-delivery services), synthetic b...
Source: EyeForPharma - November 3, 2022 Category: Pharmaceuticals Authors: Clare Jackson Source Type: news
[Ad hoc announcement pursuant to Art. 53 LR] Roche records solid results for the first nine months of 2022
Group salesup 2%[1] at constant exchange rates (CER) and 1% in Swiss francs; as expected, significantly lower COVID-19-related sales in both divisions in the third quarterSales in the Pharmaceuticals Divisionat the previous year ’s level with significantly lower sales of COVID-19-related products (Ronapreve and Actemra/RoActemra) and losses to biosimilars, offset by strong growth of newer medicinesSales in the Diagnostics Divisionrise 6%; base business remains strong; as expected, demand for COVID-19 tests sharply down in third quarterHighlights in the third quarter:EU approval forVabysmo (severe eye diseases)US approval...
Source: Roche Investor Update - October 18, 2022 Category: Pharmaceuticals Source Type: news
[Ad hoc announcement pursuant to Art. 53 LR] Roche records solid results for the first nine months of 2022
Basel, 18 October 2022Group salesup 2%1 at constant exchange rates (CER) and 1% in Swiss francs; as expected, significantly lower COVID-19-related sales in both divisions in the third quarterSales in the Pharmaceuticals Divisionat the previous year ’s level with significantly lower sales of COVID-19-related products (Ronapreve and Actemra/RoActemra) and losses to biosimilars, offset by strong growth of newer medicinesSales in the Diagnostics Divisionrise 6%; base business remains strong; as expected, demand for COVID-19 tests sharply down in third quarterHighlights in the third quarter:EU approval forVabysmo (severe eye ...
Source: Roche Media News - October 18, 2022 Category: Pharmaceuticals Source Type: news
While waiting for FDA decision on hemophilia gene therapy, BioMarin to lay off 120
The cuts are intended to reduce costs, streamline operations and increase profitability for BioMarin, frequently tapped as a likely takeover target. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - October 7, 2022 Category: Pharmaceuticals Authors: Ron Leuty Source Type: news
BioMarin Resubmits Biologics License Application (BLA) for Valoctocogene Roxaparvovec AAV Gene Therapy for Severe Hemophilia A to the FDA
BLA Includes Substantial Body of Data from Pivotal Phase 3 and Ongoing Phase 1/2 Studies
If Approved, Would Be 1st Gene Therapy in U.S. for Treatment of Severe Hemophilia A
SAN RAFAEL, Calif., Sept. 29, 2022 /PRNewswire/ -- BioMarin... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - September 29, 2022 Category: Drugs & Pharmacology Source Type: news
How Long Does A Cephalohematoma Take to Resolve?
Discussion
Cephalomatomas occur relatively commonly in 0.2-3% of newborn infants. They are blood collections in the subperiosteal skull bones, usually in the parietal area. They are usually unilateral but can be bilateral. They do not transilluminate. They are felt to be caused by pressure or other trauma and occur in vaginal and cesarean deliveries, with presumed periosteal disruption leading to externally located bleeding (not on the brain side of the bone). The blood fills the space with some pressure building up and the blood acts to tamponade itself. The blood coagulates, slowly organizes and is reabsorbed. If reabsor...
Source: PediatricEducation.org - September 12, 2022 Category: Pediatrics Authors: Pediatric Education Tags: Uncategorized Source Type: news
