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Hemophilia Disease Forecast and Market Analysis to 2035

  • ID: 3797432
  • Report
  • July 2020
  • Region: Global
  • 86 Pages
  • Datamonitor Healthcare
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Disease Overview

Hemophilia is a relatively rare hereditary genetic disorder characterized by an inability to produce a clot capable of stopping bleeding. Patients without hemophilia who develop inhibitors against clotting factor VIII or factor IX are diagnosed as having acquired hemophilia A or B, respectively. Another disease associated with blood coagulation dysfunction is von Willebrand disease, which is caused by a deficiency in von Willebrand factor. Although von Willebrand disease is more common than hemophilia, it is relatively less severe.ilia, it is relatively less severe.

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 or IX, the deficiency of which causes hemophilia A or B, respectively. Recombinant factor VIII and IX are at the top of algorithms in the US and EU treatment guidelines. Until now, full-length recombinant factor VIII agents Advate and Kogenate, and the factor IX agent BeneFIX, have dominated the hemophilia space and hold 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. In 2019, sales of Advate almost halved 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 factor VIII 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 sharp uptake 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 fortnightly dosing schedule, 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.

In the hemophilia B space, pipeline alternative coagulation promoters such as the siRNA agent fitusiran, and the tissue factor pathway inhibitors (TFPIs) concizumab and marstacimab, may struggle to gain uptake due to safety concerns. These drugs meet the need for an effective therapy for hemophilia B patients with inhibitors, provide a more convenient subcutaneous route of administration, and, in the case of fitusiran, provide an impressive once-monthly dosing regimen. However, whether these drugs will come to market is still unclear given substantial safety concerns. While development of fitusiran has continued after a preventable fatality in Phase II trials, continued development of Novo Nordisk’s concizumab may be unlikely after three thrombotic adverse events in Phase III trials, representing another setback for the TFPI class. Concerning safety events have not yet been observed in a single Phase I/II study of marstacimab, but the safety of the class as a whole is now in doubt. Given these safety concerns and a lack of physician familiarity, it seems unlikely these drugs will rival recombinant factor IX market leaders BeneFIX and Alprolix for the treatment of hemophilia B patients without inhibitors.

Roctavian (valoctocogene roxaparvovec) is the most advanced gene therapy in the hemophilia A space, and assuming EU and US approvals in Q3 2020, BioMarin is expected to launch the product at a price of $2m–$3m per patient. The extremely high upfront cost of Roctavian will be a significant deterrent to uptake, and we expect its initial use to be limited to severe hemophilia patients on chronic prophylaxis therapy. While gene therapy meets the high unmet need of a long-term therapy, with a projected eight-year interval between doses, Roctavian may face initial payer resistance due to the lack of long-term safety and efficacy data as the follow-up period in current studies has been limited to three years.

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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

  • Gene Therapies: US FDA Sticks With Bleeding Rate For Hemophilia Approval Endpoint
  • Orphan Exclusivity For Gene Therapies Hinges On Two Big Factors

PROBABILITY OF SUCCESS

LICENSING AND ASSET ACQUISITION DEALS

  • uniQure Hemophilia B Deal With CSL Leaves M&A Fans Miffed
  • Gene Therapy Companies Among Top M&A Targets in 2020
  • Roche/Spark Deal Clears FTC In A Sigh Of Relief For Pharma Dealmakers
  • Novo Nordisk, Bluebird Targeting ‘Lifelong’ Gene Therapies

CLINICAL TRIAL LANDSCAPE

  • Sponsors by status
  • Sponsors by phase
  • Recent events

DRUG ASSESSMENT MODEL

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
  • Hemophilia A
  • Hemophilia B

CONSENSUS FORECASTS

RECENT EVENTS AND ANALYST OPINION

  • Roctavian for Hemophilia A (June 17, 2020)
  • Marzeptacog alfa for Hemophilia A and B (July 7, 2019)
  • Roctavian for Hemophilia A (May 28, 2019)
  • SB-525 for Hemophilia A (April 2, 2019)
  • SPK-8011 for Hemophilia A (February 25, 2019)

KEY UPCOMING EVENTS

KEY OPINION LEADER INSIGHTS

UNMET NEEDS

  • Physician survey

BIBLIOGRAPHY

  • Prescription information

APPENDIX

LIST OF FIGURES
Figure 1: Hemophilia etiology
Figure 2: Trends in diagnosed prevalent cases of hemophilia A, 2018–27
Figure 3: Trends in diagnosed prevalent cases of hemophilia B, 2018–27
Figure 4: Trends in diagnosed cases of Von Willebrand disease, 2018–27
Figure 5: Overview of pipeline drugs for hemophilia in the US
Figure 6: Pipeline drugs for hemophilia, by company
Figure 7: Pipeline drugs for hemophilia, by drug type
Figure 8: Pipeline drugs for hemophilia, by classification
Figure 9: Probability of success in the hemophilia pipeline
Figure 10: Clinical trials in hemophilia
Figure 11: Top 10 drugs for clinical trials in hemophilia
Figure 12: Top 10 companies for clinical trials in hemophilia
Figure 13: Trial locations in hemophilia
Figure 14: Hemophilia trials status
Figure 15: Hemophilia trials sponsors, by phase
Figure 16: The publisher's drug assessment summary for hemophilia
Figure 17: Market dynamics in hemophilia (1 of 2)
Figure 18: Market dynamics in hemophilia (2 of 2)
Figure 19: Future trends in hemophilia
Figure 20: Future treatment of hemophilia A – physician survey results
Figure 21: Future treatment of hemophilia B – physician survey results
Figure 22: Roctavian for Hemophilia A (June 17, 2020) – Phase I/II PoC
Figure 23: Marzeptacog alfa for Hemophilia A and B (July 7, 2019) – Phase II/III – MAA-201
Figure 24: Key upcoming events in hemophilia
Figure 25: Unmet needs in hemophilia, physician survey results

LIST OF TABLES
Table 1: Drug classes for hemophilia A, B, and VWD
Table 2: MASAC recommendations for hemophilia A, B, and VWD
Table 3: Gender distributions of diagnosed prevalent cases of hemophilia A, hemophilia B, and Von Willebrand disease
Table 4: Diagnosed prevalent cases of hemophilia A, 2018–27
Table 5: Diagnosed prevalent cases of hemophilia B, 2018–27
Table 6: Diagnosed prevalent cases of Von Willebrand disease, 2018–27
Table 7: Marketed drugs for hemophilia A
Table 8: Marketed drugs for hemophilia B
Table 9: Marketed drugs for hemophilia A and B
Table 10: Marketed drugs for Von Willebrand disease
Table 11: Pipeline drugs for hemophilia A
Table 12: Pipeline drugs for hemophilia B
Table 13: Pipeline drugs for both hemophilia A and B
Table 14: Pipeline drugs for Von Willebrand disease
Table 15: Historical global sales, by drug ($m), 2015–19
Table 16: Forecasted global sales, by drug ($m), 2020–24
Table 17: Roctavian for Hemophilia A (June 17, 2020)
Table 18: Marzeptacog alfa for Hemophilia A and B (July 7, 2019)
Table 19: Roctavian for Hemophilia A (May 28, 2019)
Table 20: SB-525 for Hemophilia A (April 2, 2019)
Table 21: SPK-8011 for Hemophilia A (February 25, 2019)

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