Report Store | Pharma intelligence
  • My Account
    • 0Shopping Cart
  • $0.00
  • Disease analysis
    • Anemias
    • Bacterial infections
    • Bleeding disorders
    • Brain Cancer
    • Breast and gynecological cancer
    • Cardiovascular and Metabolic
    • Central Nervous System
    • Cerebrovascular diseases
    • Dermatology
    • Diabetes and diabetic complications
    • Gastroenterology
    • Gastrointestinal cancer
    • Head and Neck Cancer
    • Hypertension
    • Immunology and Inflammation
    • Infectious Diseases
    • Ischemic heart disease
    • Leukemia
    • Lung Cancer
    • Lymphoma
    • Oncology overview
    • Market Spotlight
    • Mens Health
    • Neurology
    • Oncology
    • Ophthalmology
    • Other Hematological Cancers
    • Other metabolic diseases
    • Other Solid Cancers
    • Pain
    • Psychiatry
    • Respiratory
    • Rheumatology
    • Skin cancer
    • Thrombosis
    • Urological cancer
    • Vaccines
    • Viral infections
    • Women’s Health
  • Strategy analysis
    • Bleeding disorders
    • Breast and gynecological cancer
    • Cardiovascular and Metabolic
    • Central Nervous System
    • Commercial Strategy
    • Dealmaking and R&D
    • Head and Neck Cancer
    • Hot Topics
    • Infectious Diseases
    • KOL Insight
    • Leukemia
    • Lung Cancer
    • Manufacturing
    • Market Access
    • Neurology
    • Oncology
    • Outsourcing
    • Pricing & Reimbursement
    • R&D
    • Regulatory & Therapeutic Area
    • Trends
    • Urological cancer
  • Company analysis
    • Big Pharma
    • Emerging Pharma
    • Hot Topics
    • Japan Pharma
    • M&A Analysis
    • Mid Pharma
  • Drug analysis
    • A
    • B-C
    • D-F
    • G-L
    • M-O
    • P-R
    • S-T
    • U-Z
  • MedTech analysis
    • Allergy
    • Autoimmune / Immunology
    • Cardiovascular
    • Dermatology
    • Endocrine
    • ENT / Dental
    • Gastroenterology
    • Hearing
    • Hematology
    • Infectious Diseases
    • Metabolic
    • Neurology
    • Obstetrics / Gynecology
    • Oncology
    • Ophthalmology
    • Orthopedics
    • Osteoporosis
    • Psychiatry
    • Renal
    • Respiratory
    • Rheumatology
    • Urology
  • Opinion & analysis
    • Business Development
    • Business Management
    • Clinical Trials
    • Digital Health
    • Cybersecurity
    • Compliance & Risk
    • Digital patient management
    • Digital policy & regulation
    • Finance & Pricing
    • Healthcare Innovation
    • ICT and Health
    • Leadership
    • Legislation Regulation & Policy
    • Market Access
    • Market Intelligence
    • Medical
    • Pharmaceutical Device
    • Pharmaceutical Research
    • Strategy & Innovation
    • Tech companies & Data
    • Tech & Pharma
  • Blogs
  • Search
  • Menu
You are here: Home > Blogs > Disease > New treatments – and maybe a cure – for hemophilia

New treatments – and maybe a cure – for hemophilia

April 3, 2019 | Disease

Melanie senior

Haemophilia is a rare, usually inherited, blood-clotting disorder. Patients with the most severe forms of the disease may bleed uncontrollably following even a simple scratch. Internal bleeds into the joints can cause irreversible damage; bleeding into the brain may be fatal. Thus these patients have little choice but to regularly administer replacement versions of the protein, or clotting factor, that they are missing. This two-to-three times a week treatment is life-saving for many.

Most patients have a version of the disease called hemophilia A, and need to take clotting factor VIII throughout their lives. Those with a rarer form, hemophilia B, need clotting factor IX. Several forms of both clotting factors are available – including Bayer’s factor VIII Kogenate (octocog alfa), or Pfizer’s BeneFix (nonacog alfa), a factor IX.

 

But replacing missing clotting factor does not work for about a third of patients with hemophilia A. These patients’ bodies reject the externally-administered protein as foreign, and develop antibodies against it. This group is typically given bypassing agents, which help blood clotting via a different mechanism. But these agents aren’t as effective as clotting factors, so patients are much more likely to suffer from bleeds and resulting complications – costing lives, and significant healthcare resources.

Roche/Chugai’s Hemlibra (emicizumab), approved in the US in 2017 and in the EU in early 2018, offered treatment-resistant hemophilia A patients another life-line. Hemlibra activates the blood clotting system differently again, by binding together two other coagulation proteins, Factors IX and X. Hemlibra has since been approved for all severe hemophilia A patients, not only those with Factor VIII inhibitors. Trials show that the treatment can significantly reduce bleeding in both groups of patients, considerably expanding the market for the drug. Hemlibra, given as a subcutaneous injection, also offers more flexible dosing options than Factor VIII – including a once-a-month prophylactic regime.

 

Hemlibra has drawbacks, though: regulators slapped a black-box warning – the most severe kind – on the drug for thrombosis, unwanted and potentially dangerous blood clots. Appropriate dosing and monitoring are vital.

 

Gene-therapy has the potential to provide an even safer, more convenient option for hemophilia patients. By introducing a working version of the defective F8 gene, it may offer a one-time treatment that reduces or even removes the need for regular injections or infusions, allowing patients to lead close to normal lives. BioMarin has a Phase III hemophilia A gene therapy candidate; Spark Therapeutics (recently acquired by Roche) has a Phase III hemophilia B program with partner Pfizer, and a hemophilia A program on the cusp of Phase III.

 

These programs are not yet proven. Nor indeed is the longer-term efficacy and safety of gene therapy as a class.

 

Yet if they are approved, these potentially more effective, and more convenient treatments will come at a cost. Payers are already likely to balk at Hemlibra’s sub-$500,000 per-year price if carried over to all hemophilia A patients, let alone the likely up-front cost of gene therapy. Competition may help: Alynlam/Sanofi’s Phase III fitusiran lowers levels of anti-thrombin, a protein that inactivates coagulation enzymes. It might serve patients with both Hemophilia A and B, both with or without inhibitors.

 

Fitusiran will not come cheap either, though – it is among the first of a new class of RNAi drugs. Still, this potential new treatment era in hemophilia is unlikely to be a re-play of Sovaldi in Hepatitis C, which caused massive short-term cost increases and threatened many payers’ sustainability. With more than one option for hemophilia patients that can’t use replacement clotting factors, payers will have some leverage.

Related Content

Hemophilia Disease Forecast Market Analysis to2035
Hemophilia - Pricing Reimbursement and Access

FacebookTweetPin

Related posts:

Coronavirus: A catalyst for digital health?
Disease

Coronavirus: A catalyst for digital health?

  • March, 20 2020
  • 1573

Every cloud has a silver lining. Coronavirus will be the same. One of its silver linings may be faster uptake of digital health tools and gadgets, as individuals are compelled to remain at home amid one of the century’s most concerning pandemics.   

More Options for Ovarian cancer
Disease

More Options for Ovarian cancer

  • March, 17 2020
  • 1035

One in 78 women will suffer from ovarian cancer during their lifetime. The condition accounts for more deaths than any other cancer of the reproductive system – and ranks fifth in overall cancer deaths among women.  The good news is that the range

The Big 2020 Challenge
Disease

The Big 2020 Challenge

  • January, 16 2020
  • 2150

Over the last three decades, the biotechnology industry has transformed our medicines cabinet, bringing new, effective treatments for a range of unmet needs, from rare genetic conditions, to stubborn cancers and debilitating chronic diseases. Yet several

Related Content
Read more
  • Home
  • Terms and Conditions
  • Refund policy
  • Privacy Policy
  • Cookie Policy
  • Contact Us
  • About Us
  • Discounts
  • Market Spotlight – Reports
  • twitter
  • linkedin
  • Sitemap
Datamonitor Healthcare is part of Pharma intelligence Datamonitor Healthcare is a trading division of Datamonitor Limited, a company registered in England and Wales with company number 2306113 whose registered office is 5 Howick Place, London, SW1P 1WG. VAT GB365462636. Datamonitor Limited is part of Informa PLC.
Sniffing out Parkinson’s Disease Cannabis-based Epidiolex helps some epilepsy sufferers

Sign up to the Pharma Intelligence Report Store Newsletter to get the latest blogs, news, reports and discounts!

Pharma Intelligence is part of the Business Intelligence Division of Informa PLC
  • Informa PLC
  • ABOUT US
  • INVESTOR RELATIONS
  • TALENT
This site is operated by a business or businesses owned by Informa PLC and all copyright resides with them. Informa PLC's registered office is 5 Howick Place, London SW1P 1WG. Registered in England and Wales. Number 8860726
informa
Call Back
Scroll to top