CHMP Backs First-in-Class Drug Emicizumab for Hemophilia A CHMP Backs First-in-Class Drug Emicizumab for Hemophilia A
The CHMP recommended marketing authorization for emicizumab. a first-in-class drug for patients of all ages with hemophilia A with factor VIII inhibitors.International Approvals (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - January 27, 2018 Category: Consumer Health News Tags: Hematology-Oncology News Alert Source Type: news

Roche wins EU panel's recommendation for hemophilia drug
ZURICH (Reuters) - A European Union panel on Friday recommended approval of Roche's Hemlibra to treat hemophilia in some patients after the Swiss drugmaker got the green light last year to begin selling the medicine in the United States. (Source: Reuters: Health)
Source: Reuters: Health - January 26, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

CHMP recommends EU approval of Roche ’s Hemlibra for people with haemophilia A with inhibitors
Roche today announced that the EU Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for Hemlibra ® (emicizumab) for routine prophylaxis of bleeding episodes in people with haemophilia A with factor VIII inhibitors. (Source: Roche Media News)
Source: Roche Media News - January 26, 2018 Category: Pharmaceuticals Source Type: news

CHMP recommends EU approval of Roche ’s Hemlibra for people with haemophilia A with inhibitors
Roche today announced that the EU Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for Hemlibra ® (emicizumab) for routine prophylaxis of bleeding episodes in people with haemophilia A with factor VIII inhibitors. (Source: Roche Investor Update)
Source: Roche Investor Update - January 26, 2018 Category: Pharmaceuticals Source Type: news

CHMP recommends EU approval of Roche ’s Hemlibra for people with haemophilia A with inhibitors
Roche today announced that the EU Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for Hemlibra ® (emicizumab) for routine prophylaxis of bleeding episodes in people with haemophilia A with factor VIII inhibitors. (Source: Roche Media News)
Source: Roche Media News - January 26, 2018 Category: Pharmaceuticals Source Type: news

CHMP recommends EU approval of Roche ’s Hemlibra for people with haemophilia A with inhibitors
Roche today announced that the EU Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for Hemlibra ® (emicizumab) for routine prophylaxis of bleeding episodes in people with haemophilia A with factor VIII inhibitors. (Source: Roche Investor Update)
Source: Roche Investor Update - January 26, 2018 Category: Pharmaceuticals Source Type: news

Local fallout from Sanofi ’s $11.6B Bioverativ deal: 3 things to know
Waltham-based Bioverativ, a hemophilia drugmaker that was spun out of Biogen last year, has around 275 employees in the state. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - January 22, 2018 Category: Biotechnology Authors: Max Stendahl Source Type: news

Sanofi digs deep to buy U.S. hemophilia group Bioverativ for $11.6 billion
PARIS (Reuters) - French healthcare group Sanofi has agreed to buy U.S. hemophilia specialist Bioverativ for $11.6 billion, its biggest deal for seven years and a major play to strengthen its presence in treatments for rare diseases. (Source: Reuters: Health)
Source: Reuters: Health - January 22, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

Biotech M & A takes off as Sanofi and Celgene spend $20 billion
LONDON (Reuters) - Biotech deal activity exploded on Monday with French drugmaker Sanofi and U.S.-based Celgene spending a combined total of more than $20 billion to add new products for hemophilia and cancer to their medicine cabinets. (Source: Reuters: Health)
Source: Reuters: Health - January 22, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

New drugs recast $10 billion hemophilia market as Sanofi swoops in
LONDON (Reuters) - Sanofi is placing a big bet on the $10 billion-a-year hemophilia market at a testing time, as scientific advances overhaul traditional approaches to treating the rare uncontrolled bleeding disorder. (Source: Reuters: Health)
Source: Reuters: Health - January 22, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

Sanofi, Facing Threat from Generics, Moves to Buy Hemophilia Drug Maker
The $11.6 billion deal for Bioverativ would bolster the French pharmaceutical giant ’ s portfolio as it faces falling sales for its diabetes drug, Lantus. (Source: NYT Health)
Source: NYT Health - January 22, 2018 Category: Consumer Health News Authors: CHAD BRAY Tags: Mergers, Acquisitions and Divestitures Drugs (Pharmaceuticals) Hemophilia Sanofi SA Brandicourt, Olivier Source Type: news

Sanofi to buy Bioverativ, Biogen's hemophilia spinout, for $11.6B
Paris-based Sanofi SA, the state ’s largest biotech employer, said Monday that it will buy hemophilia drugmaker Bioverativ for $11.6 billion, one year after the Waltham company was spun out of Biogen. Sanofi (NYSE: SNY), the parent company of Cambridge-based Sanofi Genzyme, said it will pay $105 per share in cash for Bioverativ (Nasdaq: BIVV), which spun out of Biogen (Nasdaq: BIIB) last January. The price represents a 63 percent premium to Bioverativ’s share price as of Friday. The deal is… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - January 22, 2018 Category: Pharmaceuticals Authors: Max Stendahl Source Type: news

Sanofi to buy US biotech group Bioverativ for $11.6bn
Purchase of haemophilia drugmaker fulfils French group’s long quest for a deal (Source: FT.com - Drugs and Healthcare)
Source: FT.com - Drugs and Healthcare - January 22, 2018 Category: Pharmaceuticals Source Type: news

Gene therapy comes of age
After almost 30 years of promise tempered by setbacks, gene therapies are rapidly becoming a critical component of the therapeutic armamentarium for a variety of inherited and acquired human diseases. Gene therapies for inherited immune disorders, hemophilia, eye and neurodegenerative disorders, and lymphoid cancers recently progressed to approved drug status in the United States and Europe, or are anticipated to receive approval in the near future. In this Review, we discuss milestones in the development of gene therapies, focusing on direct in vivo administration of viral vectors and adoptive transfer of genetically engi...
Source: ScienceNOW - January 11, 2018 Category: Science Authors: Dunbar, C. E., High, K. A., Joung, J. K., Kohn, D. B., Ozawa, K., Sadelain, M. Tags: Medicine, Diseases review Source Type: news

Sanofi to market new hemophilia drug in deal with Alnylam
PARIS (Reuters) - French pharmaceutical group Sanofi said it has obtained the right to develop and sell a new hemophilia drug in a restructuring of its partnership with U.S. Alnylam Pharmaceuticals. (Source: Reuters: Health)
Source: Reuters: Health - January 8, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

Making History for Hemophilia!
Mountaineer and Save One Life board Member Chris Bombardier is set to scale the Last of the Seven Summits, Mt. Vinson, becoming the first person with hemophilia in history to climb all Seven Summits!(PRWeb December 22, 2017)Read the full story at http://www.prweb.com/releases/2017/12/prweb15038100.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - December 22, 2017 Category: Pharmaceuticals Source Type: news

Stunning gene therapy breakthrough driven by great dedication and graft | Robin McKie
We need more than ever to celebrate advances in medical science – though they may take years to emergeThere has been a surprising outbreak of the use of the c-word among medical researchers over the past few days. Normally cautious in their language, they have nevertheless been wielding the term “cure” when discussing the long-term potential of two separate treatments for inherited ailments that were announced last week. Such enthusiasm is striking.In one case, scientists based at St Bartholomew ’s, London – who have been working on the inherited bleeding disorder haemophilia A –outlined...
Source: Guardian Unlimited Science - December 17, 2017 Category: Science Authors: Robin McKie Tags: Medical research Science Genetics Biology Cystic fibrosis Society Huntington's disease Alzheimer's Dementia Source Type: news

Roche, Shire court fight escalates over haemophilia drug
ZURICH (Reuters) - Roche's patent dispute with London-listed Shire over the Swiss drugmaker's new haemophilia drug Hemlibra has escalated, with Shire filing a new motion in a U.S. court that Roche says aims to stop some patients from getting its medicine. (Source: Reuters: Health)
Source: Reuters: Health - December 16, 2017 Category: Consumer Health News Tags: healthNews Source Type: news

Alnylam gets FDA OK to restart hemophilia drug trial after patient's death
Three months after Alnylam Pharmaceuticals halted trials of a hemophilia drug due to a patient's death, the Cambridge biotech has gotten permission from the FDA to resume studies. Alnylam (Nasdaq: ALNY), one of the state’s largest and most valuable biotechs, stopped the Phase 2 and Phase 3 studies of fitusiran in September after one patient died of brain swelling triggered by a blood clot. The news sank shares of Alnylam, shaving $1 billion from its market cap. On Friday , though, Alnylam said… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - December 15, 2017 Category: Pharmaceuticals Authors: Max Stendahl Source Type: news

Medical News Today: Hemophilia cure? Gene therapy trial shows dramatic results
A year after gene therapy treatment, people with hemophilia A are showing normal levels of clotting factor and big reductions in bleeding. (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - December 15, 2017 Category: Consumer Health News Tags: Blood / Hematology Source Type: news

FDA allows Alnylam to restart hemophilia treatment trials
(Reuters) - The U.S. Food and Drug Administration has allowed Alnylam Pharmaceuticals Inc to restart clinical trials on a drug to treat patients with a rare bleeding disorder, the company said on Friday. (Source: Reuters: Health)
Source: Reuters: Health - December 15, 2017 Category: Consumer Health News Tags: healthNews Source Type: news

Hemophilia trial finds promising cure
A gene-therapy trial by London researchers has seen remarkable success in finding a long-term cure for hemophilia A. (Source: CNN.com - Health)
Source: CNN.com - Health - December 14, 2017 Category: Consumer Health News Source Type: news

Shire taps Rani Therapeutics ’ oral delivery tech for hemophilia therapy
Shire (NSDQ:SHPG) said this week that it is collaborating with Rani Therapeutics to combine the company’s Rani Pill technology for the delivery of factor VIII therapy in patients with hemophilia A. According to the terms of the deal, Shire has the exclusive option to negotiate a license to develop and commercialize the technology as a delivery mechanism for FVIII therapy after the companies conduct feasibility studies. Shire also made an equity investment into Rani Therapeutics as part of the collaboration. Get the full story at our sister site, Drug Delivery Business News. The post Shire taps Rani The...
Source: Mass Device - December 14, 2017 Category: Medical Devices Authors: Sarah Faulkner Tags: Biotech Drug-Device Combinations Pharmaceuticals Research & Development Wall Street Beat ranitherapeutics Shire Plc. Source Type: news

Gene therapy trial could be a cure for haemophilia
Jake Omer, 29 from Billericay, Essex, who took part in the Bars Health NHS Trust trial, revealed the gene therapy changed his life and that he now has 'hope for the future'. (Source: the Mail online | Health)
Source: the Mail online | Health - December 14, 2017 Category: Consumer Health News Source Type: news

Haemophilia: Sufferers one step closer to cure after ’mind-blowing' trial results
A CURE for haemophilia is one step closer after “mind-blowing results” from a gene therapy trial. (Source: Daily Express - Health)
Source: Daily Express - Health - December 14, 2017 Category: Consumer Health News Source Type: news

Is THIS a cure for haemophilia?
The trial, led by Barts Health NHS Trust, saw 13 patients injected with a copy of the missing gene that puts them at risk of excessive bleeding, which allowed cells to produce the missing factor. (Source: the Mail online | Health)
Source: the Mail online | Health - December 14, 2017 Category: Consumer Health News Source Type: news

Groundbreaking gene therapy trial set to cure hemophilia
A 'cure' for haemophilia is one step closer, following results published in the New England Journal of Medcine of a groundbreaking gene therapy trial led by the NHS in London. Clinical researchers at Barts Health NHS Trust and Queen Mary University of London have found that over one year on from a single treatment with a gene therapy drug, participants with haemophilia A (the most common type) are showing normal levels of the previously missing protein, and effectively curing them. (Source: World Pharma News)
Source: World Pharma News - December 14, 2017 Category: Pharmaceuticals Tags: Featured Research Research and Development Source Type: news

Groundbreaking gene therapy trial set to cure hemophilia
(Queen Mary University of London) A 'cure' for hemophilia is one step closer, following results of a groundbreaking gene therapy trial led by Queen Mary University of London and the NHS in London. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - December 14, 2017 Category: International Medicine & Public Health Source Type: news

Haemophilia patient: How gene therapy has changed my life
How pioneering gene therapy has given a haemophilia patient a new lease of life. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - December 14, 2017 Category: Consumer Health News Source Type: news

Haemophilia A trial results 'mind-blowing'
Doctors use gene therapy to correct the defect that causes haemophilia A. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - December 14, 2017 Category: Consumer Health News Source Type: news

Leap forward towards gene therapy cure for haemophilia A
Scientists around the world congratulate the team that has made a major advance in finding a cure for the life-threatening blood disorderScientists believe they are on the way to finding a cure for haemophilia A, the bleeding disorder that currently requires sufferers to inject themselves every other day to avoid life-threatening complications.One dose of a gene therapy given experimentally to 13 patients by NHS doctors in the UK has allowed them all to come off treatment. These were men – most sufferers are – who would not only bleed without stopping from an injury but would bleed into their joints even in the...
Source: Guardian Unlimited Science - December 14, 2017 Category: Science Authors: Sarah Boseley Health editor Tags: Science Health & wellbeing Life and style Society Source Type: news

ASH: High - Dose Gene Transfer Beneficial in Severe Hemophilia A
Factor VIII gene transfer tied to sustained normalization of factor VIII activity levels (Source: The Doctors Lounge - Oncology)
Source: The Doctors Lounge - Oncology - December 11, 2017 Category: Cancer & Oncology Tags: Internal Medicine, Oncology, Conference News, Source Type: news

New CDC head faces questions about financial conflicts of interest
ATLANTA — After five months in office, President Trump’s new director of the Centers for Disease Control and Prevention has been unable to divest financial holdings that pose potential conflicts of interest, hindering her ability to fully perform her job. Brenda Fitzgerald, 71, who served as the Georgia public health commissioner until her appointment to the […]Related:Surge in gun sales after Sandy Hook led to spike in accidental gun deaths, study saysA mother got the flu from her children — and was dead two days laterGene therapy makes a big advance treating hemophilia B blood disorder (Sour...
Source: Washington Post: To Your Health - December 11, 2017 Category: Consumer Health News Source Type: news

Why Spark Therapeutics stock price dropped by 40% this morning
Spark Therapeutics ’ stock price plunged more than 40 percent Monday morning after the Philadelphia-based gene therapy company provided an update of its experimental hemophilia A treatment. Preliminary clinical data showed the gene therapy treatments produced significant reductions in bleeding rates, but yielded mu ch lower increases in levels of factor VIII — the clotting protein missing in hemophilia A patients. California-based BioMarin Pharmaceutical released study results on Saturday of… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - December 11, 2017 Category: Pharmaceuticals Authors: John George Source Type: news

Spark Shadows BioMarin In Hemophilia Gene Therapy Race
Spark Therapeutics, the company likely to launch the first gene therapy in the U.S., is presenting data on both of its gene therapies for hemophilia this morning at the annual meeting of the American Society of Hematology in Atlanta. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - December 11, 2017 Category: Pharmaceuticals Authors: Matthew Herper, Forbes Staff Tags: NASDAQ:ONCE NASDAQ:BMRN Source Type: news

New therapies improve outlook for bleeding and clotting disorders
(American Society of Hematology) In three studies being presented today during the 59th American Society of Hematology (ASH) Annual Meeting and Exposition in Atlanta, researchers report remarkable benefits from new, more easily administered therapies for bleeding and clotting disorders, including hemophilia A and people with cancer experiencing venous thromboembolism. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - December 11, 2017 Category: International Medicine & Public Health Source Type: news

Prophylactic Tx Stems Bleeds in Hemophilia A with Inhibitors (CME/CE)
(MedPage Today) -- HAVEN 2 trial results in pediatric patients called " outstanding and unusual " (Source: MedPage Today Hematology/Oncology)
Source: MedPage Today Hematology/Oncology - December 10, 2017 Category: Hematology Source Type: news

Emicizumab'Life-Changing' for Hemophilia A With Inhibitors Emicizumab'Life-Changing' for Hemophilia A With Inhibitors
Emicizumab prophylaxis, recently approved for adult and pediatric patients with hemophilia A with inhibitors to factor VIII, has additional support from the HAVEN 2 study in pediatric patients.Medscape Medical News (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - December 10, 2017 Category: Consumer Health News Tags: Hematology-Oncology News Source Type: news

' Promise of a Cure': Gene Therapy for Hemophilia A'Promise of a Cure': Gene Therapy for Hemophilia A
A gene-therapy-based approach reports that an adenovirus-based cassette was able to carry and deliver the culprit missing gene for hemophilia A: factor VIII.Medscape Medical News (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - December 10, 2017 Category: Consumer Health News Tags: Hematology-Oncology News Source Type: news

Excitement Builds Around Gene Therapy Cures For Hemophilia
New therapies are creating excitement for the treatment of a terrible disease. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - December 9, 2017 Category: Pharmaceuticals Authors: Matthew Herper, Forbes Staff Tags: NASDAQ:BMRN NASDAQ:ONCE NASDAQ:SHPG NYSE:NVO NYSE:PFE Source Type: news

Roche ’s Hemlibra continued to substantially reduce bleeds in people with haemophilia A with inhibitors
Roche announced today that new data from the ongoing Hemlibra ® (emicizumab) clinical development programme were presented at the 59th American Society of Hematology (ASH) Annual Meeting. (Source: Roche Media News)
Source: Roche Media News - December 9, 2017 Category: Pharmaceuticals Source Type: news

Roche ’s Hemlibra continued to substantially reduce bleeds in people with haemophilia A with inhibitors
Roche announced today that new data from the ongoing Hemlibra ® (emicizumab) clinical development programme were presented at the 59th American Society of Hematology (ASH) Annual Meeting. (Source: Roche Investor Update)
Source: Roche Investor Update - December 9, 2017 Category: Pharmaceuticals Source Type: news

A 2nd Gene Therapy Breakthrough Against Hemophilia
Coming just days after reports of a gene therapy that pushed the bleeding disorder hemophilia B into remission, new research suggests the same could be true for adults with the "A" form of the disease. (Source: WebMD Health)
Source: WebMD Health - December 9, 2017 Category: Consumer Health News Source Type: news

Another Gene Therapy Breakthrough Against Hemophilia
Coming just days after reports of a gene therapy that pushed the bleeding disorder hemophilia B into remission, new research suggests the same could be true for adults with the "A" form of the disease. (Source: WebMD Health)
Source: WebMD Health - December 9, 2017 Category: Consumer Health News Source Type: news

First Successful Gene Therapy for Hemophilia A (CME/CE)
(MedPage Today) -- Experts suggest potential cure in sight (Source: MedPage Today Pediatrics)
Source: MedPage Today Pediatrics - December 9, 2017 Category: Pediatrics Source Type: news

Another Gene Therapy Breakthrough Against Hemophilia
SATURDAY, Dec. 9, 2017 -- Coming just days after reports of a gene therapy that pushed the bleeding disorder hemophilia B into remission, new research suggests the same could be true for adults with the " A " form of the disease. That's significant... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - December 9, 2017 Category: General Medicine Source Type: news

Gene Therapy Sparks Significant Factor IX Activity in 10 Men With Hemophilia B Gene Therapy Sparks Significant Factor IX Activity in 10 Men With Hemophilia B
Experimental gene therapy designed to restore production of factor IX has left all 10 hemophiliac volunteers without the need for baseline prophylaxis and halted nearly all excess bleeding, according to a December 6 online report of a small phase 1-2a study in The New England Journal of Medicine.Reuters Health Information (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - December 8, 2017 Category: Consumer Health News Tags: Hematology-Oncology News Source Type: news

Surge in gun sales after Sandy Hook shooting led to spike in accidental gun deaths, study says
In the days after the horrific shooting at Sandy Hook Elementary School, gun enthusiasts rushed to buy millions of firearms, driven by fears that the massacre would spark new gun legislation. Those restrictions never became a reality, but a new study concludes that all the additional guns caused a significant jump in accidental firearm deaths. […]Related:A mother got the flu from her children — and was dead two days laterGene therapy makes a big advance treating hemophilia B blood disorderAn Illinois doctor traded drugs for sex and cash. He just pleaded guilty. (Source: Washington Post: To Your Health)
Source: Washington Post: To Your Health - December 7, 2017 Category: Consumer Health News Source Type: news

Surge in gun sales after Sandy Hook led to spike in accidental gun deaths, study says
In the days after the horrific shooting at Sandy Hook Elementary School, gun enthusiasts rushed to buy millions of firearms, driven by fears that the massacre would spark new gun legislation. Those restrictions never became a reality, but a new study concludes that all the additional guns caused a significant jump in accidental firearm deaths. […]Related:A mother got the flu from her children — and was dead two days laterGene therapy makes a big advance treating hemophilia B blood disorderThe moral differences between pro- and anti-vaccine parents (Source: Washington Post: To Your Health)
Source: Washington Post: To Your Health - December 7, 2017 Category: Consumer Health News Source Type: news

Gene Therapy Shows Beneficial Effect in Men With Hemophilia B
Reduction in annualized bleeding rate and in factor use with infusion of viral vector with transgene (Source: The Doctors Lounge - Oncology)
Source: The Doctors Lounge - Oncology - December 7, 2017 Category: Cancer & Oncology Tags: Oncology, Pathology, Journal, Source Type: news