The haemophilia A and B market is set to grow from $6.93 billion in 2018 to $9.29 billion in 2028 at a modest compound annual growth rate (CAGR) of 3%.
The most anticipated drug classes for the treatment of haemophilia A and B are alternative coagulation promoters (ACPs), which are therapies with alternative targets in the coagulation cascade, and gene therapies, which produce steady levels of FVIII or FIX to reduce or eliminate the need for intravenous infusions of plasma-derived or recombinant FVIII and FIX replacement factors.
Replacement factors are the current standard-of-care, representing 97% of the total sales of the haemophilia market in 2018. However, their market share is expected to decline to 70% by 2028. This change will be driven by the introduction of ACPs and gene therapies, which promise to target significant areas of unmet need by reducing the risk of developing neutralising antibodies or inhibitors against replacement therapies, providing more effective treatments for patients with inhibitors, providing more convenient administration routes and less frequent dosing, and, in the case of gene therapies, providing a potential cure for the disease.
Key opinion leaders interviewed by GlobalData in the eight major markets (8MM: the US, France, Germany, Italy, Spain, the UK, Japan, and China) are highly optimistic about these therapies and agree that they will drive change in the treatment landscapes for haemophilia A and B.
GlobalData’s research has identified ACPs such as Roche’s recently approved Hemlibra (emicizumab) for haemophilia A patients with and without inhibitors and Alnylam’s late-stage fitusiran for haemophilia A and B patients with and without inhibitors, which is expected to launch in 2021. GlobalData anticipates that the total sales of these two therapies will increase from $0.23 billion in 2018 to $1.67 billion in 2028 at a significant CAGR of 22.2%, making up 18% of total haemophilia drug revenues in 2028 in the 8MM.
BioMarin’s ValRox (valoctocogene roxaparvovec; BMN-270) and Spark Therapeutics’ SPK-8011 for haemophilia A, and uniQure’s AMT-061 and Pfizer/Spark Therapeutics’ SPK-9001 (fidanacogene elaparvovec) for haemophilia B, are leading the clinical development race for gene therapies. ValRox is expected to launch as early as 2020.
GlobalData anticipates the total revenues from these four therapies to be approximately $1.15 billion in 2028, which will make up 12% of total haemophilia drug revenues in the 8MM. As these high-cost, single-dosage treatments enter the haemophilia market over the forecast period, competition for market share seems inevitable. Gene therapies are positioned for a limited patient population, such as severe adult haemophiliacs, previously treated patients, and patients with no history of inhibitors, and questions have already been raised about their limited long-term safety and efficacy data. Therefore, first-to-market advantage will drive commercial success.
The figure below represents annual haemophilia A and B revenues by therapeutic classes in 2018 and 2028.
GlobalData (2019). Hemophilia A and B – Epidemiology Forecast to 2028, to be published.
GlobalData (2019). Hemophilia A and B – Global Drug Forecast and Market Analysis to 2028, to be published.