The FDA Just Approved Its Most Expensive Drug
The Food and Drug Administration just approved its most expensive drug ever — a hemophilia B gene therapy from Carlisle Companies (CSL) and UniQure (QURE) — and QURE stock popped Wednesday.X Meanwhile, BioMarin (BMRN) shares broke out after the FDA said it wouldn't hold an advisory committee…#genetherapy #qure #uniqure #therapy #poppedwednesdayx #hemophilia #gene #expensivedrug #csl #carlisle (Source: Reuters: Health)
Source: Reuters: Health - November 23, 2022 Category: Consumer Health News Source Type: news
Why Shares of uniQure Rose 14.61% on Wednesday
What happened Shares of uniQure (QURE 14.61%) climbed 14.61% on Wednesday. The stock closed on Tuesday at $23, then opened on Wednesday at $23.76. It climbed as high as $26.38 before closing the day at $26.36. The stock has a 52-week low of $12.52 and a 52-week high of $30.41; it is up more than…#therapy #hemophilia #uniqure #genetherapy #hemgenix #csl #gene #disease #fda #qure (Source: Reuters: Health)
Source: Reuters: Health - November 23, 2022 Category: Consumer Health News Source Type: news
One hurdle down? FDA won't hold adcomm for BioMarin's hemophilia gene therapy after all
One hurdle down? FDA won't hold adcomm for BioMarin's hemophilia gene therapy after all The FDA may be taking its time reviewing BioMarin’s gene therapy for hemophilia A, but at least the biotech now has one fewer thing to worry about. While the agency has previously planned to convene an external…#genetherapy #readingendpoints #fda #endpoints #biomarin #hurdle #gene #therapy #roctavian #hemophilia (Source: Reuters: Health)
Source: Reuters: Health - November 23, 2022 Category: Consumer Health News Source Type: news
A Gene Therapy for Hemophilia, Hemgenix, That Costs $3.5 Million Gets FDA Approval
WEDNESDAY, Nov. 23, 2022 -- People with one form of the genetic blood disorder hemophilia now have a one-time treatment with a $3.5 million price tag.
The U.S. Food and Drug Administration approved the new gene therapy Hemgenix on Nov. 22. Soon... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - November 23, 2022 Category: General Medicine Source Type: news
The FDA Just Approved The Most Expensive Drug Ever
The Food and Drug Administration just approved its most expensive drug ever — a hemophilia gene therapy from Carlisle Companies (CSL) and UniQure (QURE) — and QURE stock popped Wednesday.X The infusion is approved for hemophilia B patients currently on a prevention regimen called Factor IX…#qure #poppedwednesdayx #uniqure #expensivedrug #carlisle #therapy #genetherapy #hemophiliagene #hemophilia #csl (Source: Reuters: Health)
Source: Reuters: Health - November 23, 2022 Category: Consumer Health News Source Type: news
If you have hemophilia, you can now get a one-time gene therapy for $3.5 million
U.S. health regulators on Tuesday approved the first gene therapy for hemophilia, a $3.5 million one-time treatment for the blood-clotting disorder. The Food and Drug Administration cleared Hemgenix, an IV treatment…#genetherapy #hemophilia #treatment #gene #fda #cslbehring #genetherapies #bleeding #therapy #clotting (Source: Reuters: Health)
Source: Reuters: Health - November 23, 2022 Category: Consumer Health News Source Type: news
FDA Approves First Gene Therapy for Hemophilia B FDA Approves First Gene Therapy for Hemophilia B
Etranacogene dezaparvovec is the first gene therapy option for adults with hemophilia B who use factor IX prophylaxis and have repeated, spontaneous bleeding episodes. This one-time gene therapy has a list price of $3.5 million.MDedge News (Source: Medscape Hematology-Oncology Headlines)
Source: Medscape Hematology-Oncology Headlines - November 23, 2022 Category: Cancer & Oncology Tags: Hematology-Oncology News Alert Source Type: news
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
