#eloctate

[pulsesbm]

[robbiemorgot03]

[gmr2]

[maisuria]

How Roche’s HEMLIBRA will help in the Hemophilia Treatment Landscape?

Hemophilia A Treatment Landscape

Despite historical ups and downs,hemophilia A patient treatment has significantly improved over the past 40 years. The modern advancement of hemophilia therapies began with the revolutionary discovery of cryoprecipitate in 1964. Several years later, scientists discovered that plasma-derived factors are focused on FVIII products. This category saw the approval of a number of hemophilia A drugs that were easier to administer than cryoprecipitate or fresh-frozen plasma, such as FEIBA (Pfizer), MONOCLATEP (CSL Behring), and HEMOFIL-M. The development of recombinant therapy in the 1980s changed the situation for those who have hemophilia A.

There were only short half-life (SHL) recombinant medicines available prior to the approval of the first extended half-life (EHL) in the US (ELOCTATE; 2014). Other treatments avoid using the inhibitor-specific drugs utilized in hemophilia A. One of the most profitable bypass agents is Novonordisk's NOVOSEVEN, which holds the majority of the hemophilia A market share in comparison to Takeda's FEIBA and OBIZUR (not yet approved in Japan). Due to the adoption and use of EHL treatments, which reduced the frequency of injection to a deplorable level, the market for plasma-derived FVIII has already fallen and sales of SHL products have started to decline.

HEMLIBRA: Target patient population and strong uptake

The hemophilia A market products have remained quite fragmented because no one therapy has been able to capture a sizable portion of the market; however, the release of HEMLIBRA in 2017 changed this situation. With or without factor VIII inhibitors, HEMLIBRA, a bispecific factor IXa and factor X-directed antibody, is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with hemophilia A (congenital factor VIII deficiency), who are aged newborn and older. For hemophilia A patients taking inhibitors, HEMLIBRA was first approved in the US (November 2017), then in Europe (February 2018), and finally in Japan (March 2018).

Patients Switching to HEMLIBRA

For the non-inhibitor market, HEMLIBRA was introduced in 2018, and the hemophilia The significant uptake of HEMLIBRA over the past few years, where it crossed a billion in opportunity in 2019, makes it clear that a patient share has started to shift from EHL medicines to HEMLIBRA. HEMLIBRA had 17,000 patients receiving treatment worldwide as of the first quarter of 2022, according to the Global Pharma Survey 2022.

Factors contributing to Patient Switching to HEMLIBRA

Patients move to HEMLIBRA primarily on the advice of their doctors; at the moment, 50–60% of doctors view HEMLIBRA as the first-line hemophilia A treatment. Low administration frequency (as little as once per month) and longer reaction times are the key drivers of patient switching and doctor referrals.

Upcoming competition for HEMLIBRA

Fitusiran, a small interfering RNA treatment from Sanofi that is approved and delivered subcutaneously, is anticipated to hit the US market in 2025. A siRNA called fitusiran is made to reduce antithrombin levels in order to encourage enough thrombin production to stop bleeding. One of the main benefits of this therapy is that it can be administered once or twice a month to patients with hemophilia A or B. Despite this, we are still dubious about the drug's future due to safety concerns.

Additional hemophilia A therapies are expected to reach the 7MM in the following years and address the unmet needs of hemophilia A patients, including Valrox (Biomarin Pharmaceutical), BIVV001 (Sanofi), SPK-8011 (Roche/Spark Therapeutics), and giroctocogene fitelparvovec (Pfizer/Sangamo Therapeutics).

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Despite historical ups and downs,hemophilia A patient treatment has significantly improved over the past 40 years. The modern advancement of hemophilia therapies began with the revolutionary discovery of cryoprecipitate in 1964. 

Get to know more about the hemophilia A patient treatment landscape through our blog -https://www.delveinsight.com/blog/hemlibra-for-hemophilia-a-treatment?utm_source=blog&utm_medium=promotion&utm_campaign=gpr

[infusionventures]

[aishashopkeeper]

Eloct zapped Water this morning! Would anyone like her?

#neotag

[haydenlovesgotham-blog]

Hayden's Review on Gotham

Hayden: So I just saw 5xo5 of Gotham

My Friend: Did you like it?

Hayden: NO OF COURSE NOT ED GOT DRILLED IN HIS BRAIN AND ELOCTED!

my Friend: …

Hayden: want to watch 4x15?

My Friend: Hell Yeah!!!

#gotham edward nygma haydenradike

[sushi35848]

[newsupdate-1]

[industry365]

Hemophilia Treatment Market Is Expected To Reach New Growth Revenues During 2021-2028

Hemophilia A patients have begun to shift from short acting to EHL factors and from on demand therapies to prophylactics. The increasing prevalent population of Hemophilia A and the rise in demand of the prophylactics treatments in developed markets will have a great impact on this market.

According to the US Centers for Disease Control and Prevention, hemophilia A occurs in 1 in 5,000 live male births. Hemophilia A is about four times as common as hemophilia B.  The number of people with hemophilia in the United States is estimated to be about 20,000 individuals. Approximately 75% of people with hemophilia around the world still receive inadequate treatment or have no access to treatment.

As of January 2018, there are over 450 reported Clinical Trials for Hemophilia A with 50+ trials currently actively ongoing. Out of all the trials, 120+ studies are funded by the pharmaceutical companies alone. The major players with drugs in Phase III are Alnylam Pharmaceuticals, Sanofi (Genzyme), BioMarin Pharmaceutical, Hoffmann-La Roche, Sinocelltech Ltd., Jiangsu Chia-tai Tianqing Pharmaceutical Co.,Ltd, Octapharma, LFB USA, Inc., Novo Nordisk A/S, Pfizer and others.

Polaris Market Research expects that with the expected launch of the promising treatments, the market is expected to expand in Hemophilia A segment. The United States dominates the Hemophilia A treatment market followed by Europe 5 and Japan. The prophylaxis segments are predicted to be the fastest growing segment whereas the on-demand therapy segment is expected to contribute close to ~50% to the market during the forecast period. With the increase in the R&D investments and rising drug innovations in this field, the market is expected to significantly expand in the next five years.

Download sample: https://www.polarismarketresearch.com/industry-analysis/hemophilia-a-treatment-market/request-for-sample

RNA based therapeutics and curative gene therapies are rapidly progressing through clinical development and are seen as an attractive segment of this market.

Polaris Market Research has provided the forecasts of the Global Hemophilia A Market from 2016-2022. The major segments which has been investigated in the global market from 2016-2022 are:

Market Analysis by Hemophilia A treatment

Market Analysis by Treatments/Marketed Drugs

Market Analysis by G7 countries

The Marketed Therapies undertaken in forecast from 2016-2022 are:

Afstyla Sales Forecast-2016-2022

Eloctate Sales Forecast-2016-2022

Hemlibra Sales Forecast-2016-2022

Kogenate FS Sales Forecast-2016-2022

Kovaltry Sales Forecast-2016-2022

Nuwiq Sales Forecast-2016-2022

Obizur Sales Forecast-2016-2022

The Major Indications in Hemophilia A Therapy area:

Prophylaxis-Market Forecast 2016-2022

On-demand therapy-Market Forecast 2016-2022

Inhibitor therapy- Market Forecast 2016-2022

The Market Forecast of Hemophilia A Treatment by G7 Countries (2016-2022)

United States- Market Forecast (2016-2022)

Germany- Market Forecast (2016-2022)

France-Market Forecast (2016-2022)

Italy- Market Forecast (2016-2022)

Spain- Market Forecast (2016-2022)

United Kingdom- Market Forecast (2016-2022)

Japan- Market Forecast (2016-2022)

Leading Companies investigated in the Report are

CSL Behring

Shire

Bayer

Biogen

Genentech

Bioverativ

Alnylam Pharmaceuticals

Sanofi (Genzyme)

BioMarin Pharmaceutical

Hoffmann-La Roche

Sinocelltech Ltd.

Jiangsu Chia-tai Tianqing Pharmaceutical Co.,Ltd

Octapharma

LFB USA, Inc.

Novo Nordisk A/S

Pfizer

Get Discount offer: https://www.polarismarketresearch.com/industry-analysis/hemophilia-a-treatment-market/request-for-discount-pricing

[pulsesbm]

[udaycmi]

[blood-las]

Octopharma round table weekend

We made it back from Hoboken last night. I'm glad we went for sure. The boys are currently on ELOCTATE which we just started so we dont know how well itll work. In the search for better I wanted them on Nuvviq but we didnt have as much info on it as we do now. Hoping we can do a trial soon.

#haemophilia #hemophiliac #bleeding disorder #octopharma

[pharmaphorumuk]

Sanofi ups revenues forecasts as eczema drug Dupixent surges

Sanofi has upped its revenue forecasts for the year on the back of strong second quarter results from its new eczema and asthma injection Dupixent and its rare diseases and vaccines businesses.

The French company said that sales of Dupixent (dupilumab)  were up 168% to 496 million euros ($551 million) compared with the same quarter last year, with first half sales totalling 825 million euros ($917 million), up 175% compared with the previous half.

First approved in eczema in March 2017, Dupixent has since been approved in asthma, and this year for eczema in adolescents, and chronic rhinosinusitis and nasal polyps.

So far the drug has been launched in 28 countries, and 11 more launches in eczema and seven in asthma are planned over the course of the year.

Multiple sclerosis drug Aubagio (teriflunomide) saw sales increase almost 11% to $466 million, but that has been on the market much longer after first approval in the US as long ago as 2012.

The French company’s rare diseases and vaccines operations also helped to drive up sales, with sales of 810 million euros ($901 million) and 1,021 million euros ($1,136 million), up 23% and 25% respectively).

This helped to offset a decline in its diabetes franchise, as blockbuster Lantus (insulin glargine) continues to encounter tough competition after losing patent protection in the US, and newer branded drugs hit the market.

Diabetes drug sales decreased 7% to 1.290 billion euros ($1.434 billion) and Lantus sales were down 16% to $758 million.

There was also an on-off impairment charge of 1.8 billion euros ($2 billion) related to Eloctate for haemophilia after lower sales in the US and a revision of sales projections.

Net sales were just over 8.6 billion euros ($9.6 billion), increasing by 5.5% on a reported basis, and the company said it was predicting earnings per share growth of around 5% compared with its early target in the range of 3%-5%.

This is based on an assumption that sales of Dupixent will continue to grow as it launches in more countries.

The post Sanofi ups revenues forecasts as eczema drug Dupixent surges appeared first on Pharmaphorum.

from Pharmaphorum https://pharmaphorum.com/news/sanofi-ups-revenues-forecasts-as-eczema-drug-dupixent-surges/

[mrlongkgraves]

From frustration to freedom: My journey with hemophilia A

I was diagnosed with hemophilia A at 4 days old. My parents had no idea what it was, but they discovered that my mom was a carrier. They brought me to the Boston Hemophilia Center. It helped them to learn about hemophilia together with other families going through the same thing.

Early childhood with hemophilia A

As a kid, hemophilia really didn’t get in my way because of regular intravenous infusion of factor 8 clotting factor. I knew I had a disease that required clotting factor to prevent bleeding. My parents handled most of it; they took me to my appointments at the Boston Hemophilia Center and performed my daily factor infusions due to a hemophilia complication called an inhibitor. I later got a port-a-cath (central line) to make it easier to successfully infuse my frequently needed clotting factor. Daily or every other day infusions were very difficult on my veins. With a central line, I didn’t need to learn how to self-infuse early on.

I remember my great uncle, the only other family member we knew of who had hemophilia. When I’d visit him in the hospital, he was always in a wheelchair, watching “Meerkat Manor.” His hands and feet were bent and his body was crippled due to years of difficult to control hemophilia. It was scary and sad, and I just remember feeling so helpless. The last time I saw him, he gave me a baseball cap with a flying pig on it.

My great uncle died when I was only 7 years old. He didn’t have access to the treatments and the community that I do today.

Challenges with self-infusing

My port got infected when I was 13 years old and everything changed. To avoid additional infections, my best option was to remove the port and continue to receive daily clotting factor injections of a factor called Alphanate. Just a few months later, I started on a different factor, Eloctate, which required injections only every other day.

Because I didn’t have a lot of self-infusing experience, this transition was really hard. Every day, I’d come home from school and would have to go right to worrying about self-infusing. Some afternoons, I’d try three or four times, and I just couldn’t get it. I’d take a break, call my grandmother, take a warm shower, go back and try again. I’d end up calling a home nurse a couple of times a week to come out and infuse me.

I had good veins, but things just weren’t working. I was often bruised, sore and in pain. I bled two or three times a week. I missed 30 days of my sophomore year because of two major bleeds — one in my hip and one in my back — both which required hospital stays.

Those were probably the most difficult years of my life.

A clinical trial and a new life

When I was 16, my parents got a call from my doctor, Dr. Croteau, that there was a clinical trial for a new type of medicine for hemophilia A prophylaxis called emicizumab (Hemlibra), a medicine that’s injected once a week under the skin (subcutaneous) instead of in the vein. After years of frustration, I was finally on a good self-infusing streak, and I really didn’t want to try Hemlibra, but my parents insisted. I remember sitting on their bed that night, just crying and crying. I was scared and upset at the idea of trying something new.

Donovan and Dr. Stacy Croteau

But when I went in to meet Dr. Croteau, she really broke it down. She had this big packet with tons of data and took the time to explain everything to me. She told me stories and helped me understand why Hemlibra was worth trying. She gave me the assurance that everything would be OK.

I started the new drug at age 16. The team showed me the ropes of how to inject in this new way. My mom, my grandmother and the nurse were all there the first few times, but then I just figured it out and made it my own. It felt so good not to have to rely on anyone else. The trial was such a success that the medicine came to market sooner than anyone thought.

Focused on the future

I’ve had such good results with Hemlibra; it’s definitely the latest thing out there and the best medicine for me. I haven’t had a bleed all year, in fact, it’s been so long that I can’t even remember the last bleed. I don’t see myself ever changing treatments … unless of course gene therapy becomes available, because I know they’re working on that!

I’m now a senior in high school and will be going to college for mechanical engineering. Because of my absences over the years, I’ve had to work really hard to keep up in my honors courses and stay at the top of my class. People don’t always understand my disease, but I learned to advocate for myself at school. I can’t wait to graduate and see what else is out there in the world.

Learn more about the Boston Hemophilia Center at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center.

The post From frustration to freedom: My journey with hemophilia A appeared first on Thriving Blog.

from Thriving Blog https://on.bchil.org/2VNdIrz

[lizasandvik-blog]

Hemophilia Market Segmentation by Type, Applications, Key Vendors and Regions Report 2019-2026

[Feb, 2019] The global hemophilia market is expected to reach USD 16.9 billion by 2026. Increasing need for diagnosis of the target population in order to initiate prophylaxis treatment and supportive government programs is expected to be the vital impact rendering driver.

CDC's National Center on Birth Defects and Developmental Disabilities (NCBDDD) undertakes initiatives to improving the public understanding of blood disorders and associated complications. For instance, NCBDDD supported the 'The Red Tie Challenge' initiated by the National Hemophilia Foundation (NHF) in order to spread awareness about blessing disorders such as Von Willebrand disease, hemophilia and other rare bleeding disorders. Such initiatives are anticipated to boost the market growth

The market players are predicted to adopt strategies such as pipeline product development and geographic expansion in underdeveloped and developing regions, such as Middle Eastern and West African countries.

As of 2015, federal bodies such as the World Federation Hemophilia have vast information and treatment regulation in about 172 countries, of which 20 were added in 2015, including Nigeria, Togo, Mali, Zambia, Mauritania, and others.

Get Free Sample Copy of this Report at:  https://www.radiantinsights.com/research/hemophilia-market/request-sample

Competitive pricing strategy is a vital factor promoting market players' growth over the forecast period. For instance, Biogen launched Eloctate in 2014 in the U.S. at a lower price than the existing vastly used drug Advate.

Further key findings from the report suggest:

• Hemophilia B is expected to be the fastest growing hemophilia type over the forecast period, at a CAGR of 5.8%.

• The other segment, owing to spurt in the number of patients diagnosed with von Willebrand Disease (VWD), is also anticipated to witness lucrative growth over the forecast period.

• On-demand treatment dominated the segment over the forecast period and is expected to contribute more than 50.0% by 2026.

• On the basis of therapy, gene therapy is expected to be the fastest growing segment as a result of increasing R&D and innovations in the field. Furthermore, replacement therapy is expected to witness lucrative growth due to the launch of extended half-life versions of currently available drugs.

• Major market players include Biogen, Novo Nordisk, Baxalta, Octapharma, CSL Behring, Pfizer, Inc., and Bayer Healthcare. Most of the companies are actively involved in the awareness and treatment programs launched by regional governments and hemophilia associations.

Check out Full Report with TOC at:  https://www.radiantinsights.com/research/hemophilia-market

Table of Contents:

Chapter 1. Methodology and Scope1.1. Market segmentation & scope1.2. Information procurement1.2.1. Purchased database1.2.2. GVR's internal database1.2.3. Secondary sources & third-party perspectives1.2.4. Primary research1.3. Information analysis1.3.1. Data analysis models1.4. Market formulation & data visualization1.5. Data validation & publishing

Chapter 2. Executive Summary2.1. Market Outlook 2.2. Segment Outlook 2.3. Competitive Insights

Chapter 3. Hemophilia Market Variables, Trends & Scope3.1. Market Lineage outlook3.1.1. Parent market outlook3.1.2. Related/ancillary market outlook3.2. Penetration & Growth Prospect Mapping3.3. Product Pipeline Analysis, by Stage3.4. User perspective analysisContinued...

See our similar reports at:  https://www.radiantinsights.com/catalog/pharmaceutical

Contact Details:Michelle ThorasCorporate Sales Specialist, USARadiant Insights, Inc28 2nd Street, Suite 3036, San Francisco, CA 94105, United StatesPhone: 1-415-349-0054Toll Free: 1-888-202-9519Email: [email protected]

#Hemophilia Market Hemophilia Industry Hemophilia