Morning Break: Monthly HIV Tx Effective; Curing Hemophilia; Elephants and Cancer
(MedPage Today) -- Health news and commentary from around the Web gathered by the MedPage Today staff (Source: MedPage Today Psychiatry)
Source: MedPage Today Psychiatry - August 15, 2018 Category: Psychiatry Source Type: news

They Thought Hemophilia Was a ‘ Lifelong Thing. ’ They May Be Wrong.
Experimental gene therapies have yielded promising results in early trials. But the drugs have left some patients wary, worried that success will not last. (Source: NYT Health)
Source: NYT Health - August 13, 2018 Category: Consumer Health News Authors: GINA KOLATA Tags: Hemophilia Genetics and Heredity Blood Proteins Drugs (Pharmaceuticals) Clinical Trials Biotechnology and Bioengineering Source Type: news

RCSI and Bayer enter research collaboration to improve hemophilia treatment
(RCSI) RCSI and Bayer have today announced a research collaboration that aims to improve treatments for people with severe hemophilia. The project will explore new treatments that can be tailored to the severity of each individual's condition in order to safely and effectively promote blood clotting in people with hemophilia. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - August 9, 2018 Category: International Medicine & Public Health Source Type: news

Why Spark's stock price is down 30 percent
Despite posting its first-ever profitable quarter, shares of Spark Therapeutics were trading down 30 percent Tuesday morning after the Philadelphia gene therapy company reported two patients had an adverse immune response to its experimental hemophilia A therapy. Spark said the immune response problem was addressed and it is moving forward with late-stage testing of the treatment, known as SPK-8011 that will begin by the end of the year. Hemophilia A is a genetic bleeding disorder caused by missing… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - August 7, 2018 Category: Biotechnology Authors: John George Source Type: news

Shire’s immunology unit shines in Q2 but haemophilia sales stall
(Source: FT.com - Drugs and Healthcare)
Source: FT.com - Drugs and Healthcare - July 31, 2018 Category: Pharmaceuticals Source Type: news

Balance, falls, and exercise: beliefs and experiences in people with hemophilia: a qualitative study - Flaherty LM, Schoeppe J, Kruse-Jarres R, Konkle BA.
BACKGROUND: Prior research has established that falls are commonplace in adults with hemophilia, and advises that physical therapy and exercise are successful in fall prevention. Recognizing obstacles and catalysts to physical therapy and exercise in peopl... (Source: SafetyLit)
Source: SafetyLit - July 28, 2018 Category: International Medicine & Public Health Tags: Ergonomics, Human Factors, Anthropometrics, Physiology Source Type: news

Pfizer begins late-stage testing of Spark's hemophilia B gene therapy
Pfizer Inc. said Monday it has started late-stage clinical testing of an experimental gene therapy treatment, originally developed by Spark Therapeutics in Philadelphia, for patients with hemophilia B. The transfer of the gene therapy program to Pfizer from Spark is now complete, the companies said. Under the terms of a product license agreement signed in December 2014, Pfizer has now assumed sole responsibility for all subsequent pivotal studies, all regulatory activities, manufacturing and gl obal… (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - July 16, 2018 Category: American Health Authors: John George Source Type: news

Pfizer begins late-stage testing of Spark's hemophilia B gene therapy
Pfizer Inc. said Monday it has started late-stage clinical testing of an experimental gene therapy treatment, originally developed by Spark Therapeutics in Philadelphia, for patients with hemophilia B. The transfer of the gene therapy program to Pfizer from Spark is now complete, the companies said. Under the terms of a product license agreement signed in December 2014, Pfizer has now assumed sole responsibility for all subsequent pivotal studies, all regulatory activities, manufacturing and gl obal… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - July 16, 2018 Category: Biotechnology Authors: John George Source Type: news

Pfizer initiates pivotal Phase 3 program for investigational hemophilia B gene therapy
Pfizer Inc. (NYSE:PFE) and Spark Therapeutics (NASDAQ:ONCE) announced today that Pfizer initiated a Phase 3 open-label, multi-center, lead-in study (NCT03587116) to evaluate the efficacy and safety of current factor IX prophylaxis replacement therapy in the usual care setting. The factor IX prophylaxis efficacy data obtained in the lead-in study will serve as the within-subject control group for (Source: World Pharma News)
Source: World Pharma News - July 16, 2018 Category: Pharmaceuticals Tags: Featured Pfizer Business and Industry Source Type: news

Haemophilia A/sialorrhoea: Comparator therapies not implemented, added benefit not proven
(Institute for Quality and Efficiency in Health Care) In two early benefit assessments, IQWiG was unable to derive an added benefit from the data presented because current standards of care were not implemented in the studies. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - July 9, 2018 Category: International Medicine & Public Health Source Type: news

Takeda chief seeks to allay £46bn Shire bid concerns
Christophe Weber ‘very comfortable’ with durability of lucrative haemophilia franchise (Source: FT.com - Drugs and Healthcare)
Source: FT.com - Drugs and Healthcare - June 27, 2018 Category: Pharmaceuticals Source Type: news

ASH to collaborate on clinical practice guidelines on von Willebrand Disease
(American Society of Hematology) The American Society of Hematology (ASH) will collaborate with the International Society on Thrombosis and Haemostasis (ISTH), National Hemophilia Foundation (NHF), World Federation of Hemophilia (WFH), and the University of Kansas Medical Center to develop clinical practice guidelines on the diagnosis and management of von Willebrand Disease (VWD).   (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - June 21, 2018 Category: International Medicine & Public Health Source Type: news

FDA grants Priority Review to Roche ’s Hemlibra for people with haemophilia A without factor VIII inhibitors
Roche (SIX: RO, ROG; OTCQX: RHHBY) announced that the US Food and Drug Administration (FDA) has accepted the company's supplemental Biologics License Application (sBLA) and granted Priority Review for Hemlibra® (emicizumab-kxwh) for adults and children with haemophilia A without factor VIII inhibitors. The sBLA is based on data from the phase III HAVEN 3 study. (Source: World Pharma News)
Source: World Pharma News - June 7, 2018 Category: Pharmaceuticals Tags: Featured Roche Business and Industry Source Type: news

FDA grants Roche priority review for haemophilia treatment
The company's Hemlibra has nabbed a fast-track review for people with a certain form of haemophilia from by the U.S. Food and Drug Administration. (Source: PharmaManufacturing.com)
Source: PharmaManufacturing.com - June 5, 2018 Category: Pharmaceuticals Source Type: news

Roche gets priority review from FDA for Hemlibra
ZURICH (Reuters) - Roche has been given fast-track review by the U.S. Food and Drug Administration (FDA) for its Hemlibra treatment for people with a form of haemophilia, the Swiss drugmaker said on Tuesday. (Source: Reuters: Health)
Source: Reuters: Health - June 5, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

FDA grants Priority Review to Roche ’s Hemlibra for people with haemophilia A without factor VIII inhibitors
Roche today announced that the US Food and Drug Administration (FDA) has accepted the company ’s supplemental Biologics License Application (sBLA) and granted Priority Review for Hemlibra® (emicizumab-kxwh) for adults and children with haemophilia A without factor VIII inhibitors. (Source: Roche Media News)
Source: Roche Media News - June 5, 2018 Category: Pharmaceuticals Source Type: news

FDA grants Priority Review to Roche ’s Hemlibra for people with haemophilia A without factor VIII inhibitors
Roche today announced that the US Food and Drug Administration (FDA) has accepted the company ’s supplemental Biologics License Application (sBLA) and granted Priority Review for Hemlibra® (emicizumab-kxwh) for adults and children with haemophilia A without factor VIII inhibitors. (Source: Roche Investor Update)
Source: Roche Investor Update - June 5, 2018 Category: Pharmaceuticals Source Type: news

Spark, Pfizer report progress on gene therapy for hemophilia
Spark Therapeutcis and Pfizer have cleared a key hurdle in their effort to develop a gene therapy treatment for patient with hemophilia B. On Tuesday, the companies said all 15 hemophilia B patients in Philadelphia-based Spark’s ongoing phase-I/II clinical trial of its investigational gene therapy SPK-9001 had discontinued routine infusions of factor IX concentrates – and none of the 15 participants experienced serious adverse ev ents. Hemophilia B is a rare, genetic bleeding disorder found… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - May 22, 2018 Category: Biotechnology Authors: John George Source Type: news

Trials Support Emicizumab as Standard Tx in Hemophilia A
(MedPage Today) -- $448,000 annual price would actually reduce per-patient costs (Source: MedPage Today Hematology/Oncology)
Source: MedPage Today Hematology/Oncology - May 21, 2018 Category: Hematology Source Type: news

Roche drug dramatically reduces bleeds in key hemophilia tests
LONDON (Reuters) - Roche's new hemophilia drug Hemlibra dramatically reduced bleeding in a broad population of hemophilia patients, results from two clinical trials showed on Monday, setting it up to take a dominant market position. (Source: Reuters: Health)
Source: Reuters: Health - May 21, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

Roche ’s Hemlibra reduced treated bleeds by 96 percent compared to no prophylaxis in phase III HAVEN 3 study in haemophilia A without factor VIII inhibitors
Roche today announced full results from the phase III HAVEN 3 study evaluating Hemlibra ® (emicizumab) prophylaxis administered every week or every two weeks in people with haemophilia A without factor VIII inhibitors and the phase III HAVEN 4 study evaluating Hemlibra prophylaxis administered every four weeks in people with haemophilia A with or without factor VIII inhibitors. (Source: Roche Media News)
Source: Roche Media News - May 21, 2018 Category: Pharmaceuticals Source Type: news

Roche ’s Hemlibra reduced treated bleeds by 96 percent compared to no prophylaxis in phase III HAVEN 3 study in haemophilia A without factor VIII inhibitors
Roche today announced full results from the phase III HAVEN 3 study evaluating Hemlibra ® (emicizumab) prophylaxis administered every week or every two weeks in people with haemophilia A without factor VIII inhibitors and the phase III HAVEN 4 study evaluating Hemlibra prophylaxis administered every four weeks in people with haemophilia A with or without factor VIII inhibitors. (Source: Roche Investor Update)
Source: Roche Investor Update - May 21, 2018 Category: Pharmaceuticals Source Type: news

Reminder: Invitation to Roche ’s Virtual Pipeline Event from WFH 2018 World Congress
We are pleased to invite investors and analysts to participate in a live audio webcast and conference call on Wednesday, 23 May 2018, from the World Federation of Hemophilia (WFH) 2018 World Congress (20-24 May 2018) in Glasgow, Scotland. (Source: Roche Investor Update)
Source: Roche Investor Update - May 18, 2018 Category: Pharmaceuticals Source Type: news

Roche to present new phase III data for Hemlibra in people with haemophilia A at the World Federation of Hemophilia 2018 World Congress
Roche today announced that phase III results for Hemlibra ® (emicizumab) will be presented for the first time during the World Federation of Hemophilia (WFH) 2018 World Congress from 20-24 May in Glasgow, Scotland. (Source: Roche Media News)
Source: Roche Media News - May 14, 2018 Category: Pharmaceuticals Source Type: news

Roche to present new phase III data for Hemlibra in people with haemophilia A at the World Federation of Hemophilia 2018 World Congress
Roche today announced that phase III results for Hemlibra ® (emicizumab) will be presented for the first time during the World Federation of Hemophilia (WFH) 2018 World Congress from 20-24 May in Glasgow, Scotland. (Source: Roche Investor Update)
Source: Roche Investor Update - May 14, 2018 Category: Pharmaceuticals Source Type: news

Roche to present new phase III data for Hemlibra in people with haemophilia A at the World Federation of Hemophilia 2018 World Congress
Roche today announced that phase III results for Hemlibra ® (emicizumab) will be presented for the first time during the World Federation of Hemophilia (WFH) 2018 World Congress from 20-24 May in Glasgow, Scotland. (Source: Roche Media News)
Source: Roche Media News - May 14, 2018 Category: Pharmaceuticals Source Type: news

Invitation to Roche ’s Virtual Pipeline Event from WFH 2018 World Congress
We are pleased to invite investors and analysts to participate in a live audio webcast and conference call on Wednesday, 23 May 2018, from the World Federation of Hemophilia (WFH) 2018 World Congress (20-24 May 2018) in Glasgow, Scotland. (Source: Roche Investor Update)
Source: Roche Investor Update - May 11, 2018 Category: Pharmaceuticals Source Type: news

Single injection treats hemophilia B for life, in proof-of-concept study
(Salk Institute) Salk researchers have demonstrated in mice that hemophilia B can be treated for life with one single injection containing disease-free liver cells that can produce their missing clotting factor. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - May 1, 2018 Category: International Medicine & Public Health Source Type: news

Blood scandal victims need more cash, says Haemophilia Wales
There are calls for Welsh infected blood scandal victims to get the same payments as those in Scotland. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - April 25, 2018 Category: Consumer Health News Source Type: news

Kenya:One in 10,000 Kenyans Has Haemophilia, Says Report
[Nation] Kenya joined other countries in marking the World Haemophilia Day on Tuesday. (Source: AllAfrica News: Health and Medicine)
Source: AllAfrica News: Health and Medicine - April 18, 2018 Category: African Health Source Type: news

Mauritius:Signing of Medical Twinning With South Africa in the Field of Hemophilia
[Government of Mauritius] The Jawaharlal Nehru Hospital and the Charlotte Maxeke Johannesburg Academic Hospital of South Africa signed yesterday a medical twinning in the field of hemophilia at the Victoria Hospital in Candos. (Source: AllAfrica News: Health and Medicine)
Source: AllAfrica News: Health and Medicine - April 17, 2018 Category: African Health Source Type: news

FDA grants Breakthrough Therapy Designation for Roche's Hemlibra in haemophilia A without inhibitors
Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to Hemlibra® (emicizumab-kxwh) for people with haemophilia A without factor VIII inhibitors. Breakthrough Therapy Designation is designed to accelerate the development and review of medicines intended to treat a serious condition with preliminary evidence that indicates they may demonstrate a substantial improvement over existing therapies. (Source: World Pharma News)
Source: World Pharma News - April 17, 2018 Category: Pharmaceuticals Tags: Featured Roche Business and Industry Source Type: news

Britain ’s use of contaminated blood was no ‘tragedy’ – it was a scandal | Simon Hattenstone
The latest inquiry must discover how thousands of haemophiliacs contracted HIV or hepatitis C from blood products, and why it was covered up for so longIn two weeks ’ time Sir Brian Langstaff will take up his post as chair of thepublic inquiry into contaminated blood and contaminated blood products. Today, World Haemophilia Day, is the perfect occasion to remind Langstaff what the thousands of haemophiliac victims need from this inquiry if they are to get justice.In the 1970s and 1980s more than 4,600 haemophiliacs contracted HIV or hepatitis C after being infected by contaminated blood-clotting products. Much of the...
Source: Guardian Unlimited Science - April 17, 2018 Category: Science Authors: Simon Hattenstone Tags: Health Society Hepatitis B Hepatitis C Aids and HIV NHS Health policy Politics UK news Science Medical research Source Type: news

Roche's Hemlibra gets speedy FDA review for wider use
ZURICH (Reuters) - Roche's hemophilia A drug Hemlibra will get an accelerated review by U.S. regulators for use in a new group of patients, a key part of the Swiss drug company's plans to muscle in on turf dominated by rivals including Shire. (Source: Reuters: Health)
Source: Reuters: Health - April 17, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

FDA grants Breakthrough Therapy Designation for Roche ’s Hemlibra in haemophilia A without inhibitors
Roche today announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to Hemlibra ® (emicizumab-kxwh) for people with haemophilia A without factor VIII inhibitors. (Source: Roche Media News)
Source: Roche Media News - April 17, 2018 Category: Pharmaceuticals Source Type: news

FDA grants Breakthrough Therapy Designation for Roche ’s Hemlibra in haemophilia A without inhibitors
Roche today announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to Hemlibra ® (emicizumab-kxwh) for people with haemophilia A without factor VIII inhibitors. (Source: Roche Investor Update)
Source: Roche Investor Update - April 17, 2018 Category: Pharmaceuticals Source Type: news

FDA grants Breakthrough Therapy Designation for Roche ’s Hemlibra in haemophilia A without inhibitors
Roche today announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to Hemlibra ® (emicizumab-kxwh) for people with haemophilia A without factor VIII inhibitors. (Source: Roche Media News)
Source: Roche Media News - April 17, 2018 Category: Pharmaceuticals Source Type: news

FDA Grants Breakthrough Therapy Designation For Genentech ’s Hemlibra (emicizumab-kxwh) in Hemophilia A Without Inhibitors
South San Francisco, CA -- April 16, 2018 -- Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to Hemlibra... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - April 16, 2018 Category: Drugs & Pharmacology Source Type: news

Patient Support Program Triumphs
A patient support program offering around the clock assistance to glioblastoma multiforme (GBM) patients undergoing an innovative therapy has been named the Most Valuable Patient Initiative award at eyeforpharma ’s annual awards ceremony in Philadelphia.   Reflecting on the win, Novocure said: “We are humbled to have helped so many patients and to be recognised for it by our peers who have made careers out of trying to help patients in the most need. The eyeforpharma awards are helping pharma to focus on its core constituent — the patient.”  In naming the PSP as the winner of the 2018...
Source: EyeForPharma - April 10, 2018 Category: Pharmaceuticals Authors: Adam Chapman Source Type: news

Helping Patients Learn Healthy Habits
What if we told you we could improve health outcomes for more than 50% of patients and save the US healthcare system up to $289bn a year?That ’s the averagecost of nonadherence to prescribed medications, or in layman ’s terms, not taking your meds. Fixing this epidemic could not only save billions of dollars but, with around 125,000 deaths a year in the US due to failure to follow doctor’s orders, it could save thousands of lives too.Better lifestyle decisions can solve many of our personal health problems; eating healthily, regular exercise, taking your pills, are all positive behaviors that contribute t...
Source: EyeForPharma - April 5, 2018 Category: Pharmaceuticals Authors: Steve Peretz and Meg Donchak Source Type: news

Roche shares drop after deaths of patients taking Hemlibra
ZURICH (Reuters) - Swiss drugmaker Roche's shares dropped on Wednesday after it told U.S.-based haemophilia advocacy groups that five patients treated with its medicine Hemlibra had died, while maintaining that the therapy was not the cause of the deaths. (Source: Reuters: Health)
Source: Reuters: Health - March 28, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

Roche shares drop after reports of deaths of patients taking Hemlibra
ZURICH (Reuters) - Roche shares dropped on Wednesday after U.S.-based National Hemophilia Foundation said Roche's Genentech unit informed it of five deaths of patients treated with its drug Hemlibra. (Source: Reuters: Health)
Source: Reuters: Health - March 28, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

Tokyo court rejects Shire claim against Roche hemophilia drug
ZURICH (Reuters) - A Tokyo court has ruled that Shire's claim against Swiss drugmaker Roche's new Hemlibra hemophilia medicine should be dismissed, Roche's Japanese subsidiary Chugai said on Wednesday, helping to clear up legal uncertainty over the prospective blockbuster medicine. (Source: Reuters: Health)
Source: Reuters: Health - March 28, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

Nanostructures created by UCLA scientists could make gene therapies safer, faster and more affordable
UCLA scientists have developed a new method that utilizes microscopic splinter-like structures called “nanospears” for the targeted delivery of biomolecules such as genes straight to patient cells. These magnetically guided nanostructures could enable gene therapies that are safer, faster and more cost-effective.The research waspublished in the journal ACS Nano by senior author Paul Weiss, UC Presidential Chair and distinguished professor of chemistry and biochemistry, materials science and engineering, and member of theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.Gene the...
Source: UCLA Newsroom: Health Sciences - March 15, 2018 Category: Universities & Medical Training Source Type: news

Chance and Prepared Minds Lead from Lab to New Drug Development
A ""failed"" experiment in the lab in 1992 ultimately led to the development of new drugs to treat hemophilia, that were approved by the FDA in 2014. (Source: NIDDK News)
Source: NIDDK News - March 15, 2018 Category: Endocrinology Source Type: news

Sanofi Completes Acquisition of Bioverativ Inc.
Bioverativ Inc. is a fully owned subsidiary of Sanofi Paris (France) - March 8, 2018 -- (Healthcare Sales & Marketing Network) -- Sanofi announced today the successful completion of its acquisition of Bioverativ Inc. ("Bioverativ") for $105 pe... Biopharmaceuticals, Mergers & Acquisitions Sanofi, Bioverativ, hemophilia (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - March 8, 2018 Category: Pharmaceuticals Source Type: news

Britain's contaminated blood scandal: ‘I need them to admit they killed our son’
In the 1970s and 80s, 4,689 British haemophiliacs were treated with contaminated blood products. So far, more than half of them have died. The government knew there were risks involved. The patients didn ’t. Will they ever get justice?It has been called the biggest treatment disaster in the history of the NHS, and a “horrific human tragedy”. But Su Gorman, whose husband has endured years of ill health as a result of haemophilia treatment, does not believe this adequately describes Britain’s contaminated blood scandal. As far as she is concerned, it is simply a crime.In the 1970s and 80s, 4,689 haemo...
Source: Guardian Unlimited Science - March 3, 2018 Category: Science Authors: Simon Hattenstone Tags: Health Society & wellbeing Life and style Medical research Science NHS Hepatitis C Aids and HIV Health policy Source Type: news

ICER finds emicizumab offers improvements for haemophilia A
(Source: PharmacoEconomics and Outcomes News)
Source: PharmacoEconomics and Outcomes News - March 1, 2018 Category: Drugs & Pharmacology Source Type: news

Roche says hemophilia drug Hemlibra wins EU approval
ZURICH (Reuters) - Swiss group Roche said on Tuesday the European Commission has approved its drug Hemlibra for people with hemophilia A who have developed resistance to standard treatments. (Source: Reuters: Health)
Source: Reuters: Health - February 27, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

European Commission approves Roche's Hemlibra for people with haemophilia A with inhibitors
Roche today announced that the European Commission has approved 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 - February 27, 2018 Category: Pharmaceuticals Source Type: news