Hemophilia Clinical Landscape Market Report 2021-2027: Uptake of Gene Therapy will be Limited by High Upfront Costs and Uncertainty Over Long-term Effectiveness

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Jul 12, 2021, 13:30 ET

DUBLIN, July 12, 2021 /PRNewswire/ -- The "Disease Analysis: Hemophilia" report has been added to ResearchAndMarkets.com's offering.

Hemophilia is a rare, inherited X chromosome-linked bleeding disorder in which deficiencies in clotting factors prevent blood from clotting normally. Injuries therefore result in prolonged periods of bleeding. There are two types of hemophilia, A and B, with similar signs and symptoms but different genetic defects.

Latest Key Takeaways

The publisher estimates that in 2018, there were approximately 180,750 diagnosed prevalent cases of hemophilia A and 35,950 diagnosed prevalent cases of hemophilia B worldwide. These figures are forecast to increase to 193,730 cases and 38,570 cases, respectively, by 2027. There were approximately 82,550 diagnosed prevalent cases of von Willebrand disease (VWD) worldwide in 2018, which is forecast to increase to 86,150 cases by 2027.

Current treatments are largely focused on replacing factor VIII (fVIII) or factor IX (fIX), the deficiency of which causes hemophilia A or B, respectively. Recombinant fVIII and fIX are at the top of algorithms in the US and EU treatment guidelines. Until recently, full-length recombinant fVIII agents Advate and Kogenate, and the fIX agent BeneFIX, have dominated the hemophilia space and have held the largest portion of market share.

However, these drugs have been steadily losing market share to extended half-life recombinant factor products (EHLs) and Hemlibra, as physicians switch patients to newer-generation products with improved dosing frequencies. During 2018-20, sales of Advate more than halved (from $2,806m in 2018 to $1,213m in 2020) due to fierce competition primarily from Roche's Hemlibra, which was awarded a label expansion in the US and EU in the first quarter of 2019.

The therapeutic strategies for hemophilia were revolutionized after the introduction of replacement factors during the late 1990s and early 2000s, which were followed by EHLs over the past decade. Currently, the hemophilia market is undergoing a third revolution, with an anticipated shift towards alternative coagulation promoters and gene therapy.

While the 2017 launch of Roche's Hemlibra for the treatment of hemophilia A patients with fVIII inhibitors did not make a large impact on the hemophilia market, the extension of its US and EU labels in 2018 to include hemophilia A patients without inhibitors triggered a large increase in sales.

Hemlibra has captured market share from a host of replacement factors and EHLs and is set to achieve market leader status over the forecast period. Hemlibra is clinically more attractive than rival products because of its novel inhibitor-independent mechanism of action, its dosing schedule of once every two weeks, and its ability to be self-administered subcutaneously in a market dominated by intravenous agents.

There are a number of pipeline candidates in the hemophilia space which may make a significant impact in the market over the forecast period. Based on discussions with key opinion leaders (KOLs), the publisher expects that Roche's Hemlibra will further extend its lead in the hemophilia space, having captured market leader status from Takeda's Advate in 2019.

Gene therapies are expected to have initially muted uptake because of a high upfront cost for payers ($2m-$3m) and an initial reluctance from prescribers owing to uncertainty over their long-term efficacy and safety.

Key Topics Covered:

OVERVIEW

Latest key takeaways

DISEASE BACKGROUND

Definition
Patient segmentation
Etiology
Von Willebrand disease
Hemophilia symptoms
VWD symptoms
Hemophilia and VWD complications
Diagnosis
Treatment options

TREATMENT

EPIDEMIOLOGY

Prevalence methodology

MARKETED DRUGS

PIPELINE DRUGS

KEY REGULATORY EVENTS

Relief For UniQure Hemophilia Program Sends Ball To FTC's Court
BioMarin Ready To Talk To FDA About Gene Therapy CRL
BioMarin Pulls EU Hemophilia Gene Therapy Filing, But Is Not Giving Up
New Designations Set To Speed Hemlibra, CAR-T In China
Not Such A Sure Thing: FDA Knocks Back BioMarin's Roctavian

PROBABILITY OF SUCCESS

LICENSING AND ASSET ACQUISITION DEALS

Finance Watch: BioAtla, Virios And Scopus Launch Latest US IPOs
Atomwise, GC Pharma Team Up On Novel Hemophilia Therapies
uniQure Hemophilia B Deal With CSL Leaves M&A Fans Miffed

DRUG ASSESSMENT MODEL

Gene therapy for hemophilia A
Gene therapy for hemophilia B

CLINICAL TRIAL LANDSCAPE

Sponsors by status
Sponsors by phase
Recent events

MARKET DYNAMICS

FUTURE TRENDS

The hemophilia market will continue to expand over the forecast period
Hemlibra will continue to capture market share from replacement factors in the hemophilia A space
Pipeline alternative coagulation promoters will capture a limited amount of market share in the hemophilia B space
Uptake of gene therapy will be limited by high upfront costs and uncertainty over long-term effectiveness

CONSENSUS FORECASTS

RECENT EVENTS AND ANALYST OPINION

Roctavian for Hemophilia A (May 19, 2021)
Etranacogene Dezaparvovec for Hemophilia B (March 29, 2021)
Roctavian for Hemophilia A (January 10, 2021)
Etranacogene Dezaparvovec for Hemophilia B (December 21, 2020)
FLT180a for Hemophilia B (December 14, 2020)
Etranacogene Dezaparvovec for Hemophilia B (December 8, 2020)
Fitusiran for Hemophilia A and B (October 30, 2020)
Roctavian for Hemophilia A (August 18, 2020)
Concizumab for Hemophilia A and B (August 13, 2020)
Roctavian for Hemophilia A (June 17, 2020)
Concizumab for Hemophilia A and B (March 16, 2020)

KEY UPCOMING EVENTS

KEY OPINION LEADER INSIGHTS

UNMET NEEDS

BIBLIOGRAPHY

APPENDIX

For more information about this report visit https://www.researchandmarkets.com/r/yohssb

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