Global Hemophilia A and B Market Projected to Reach $9.3 Billion by 2028

The hemophilia A and B market is expected to grow at a compound annual growth rate of 2.9% from $7.0 billion in 2018 to $9.3 billion in 2028 across the eight major markets (8MM), data and analytics company GlobalData announced in a Sept. 4, 2019 press release.

According to the company’s latest report, Hemophilia A and B: Global Drug Forecast and Market Analysis to 2028, the major drivers of growth during the forecast period will be the decreasing use of cheaper and less convenient plasma-derived products, the continued use of higher-cost long-acting factors, the uptake of alternative coagulation promoters (ACPs), and the introduction of gene therapies.

“ACPs and gene therapies will radically impact hemophilia A and B inhibitor and non-inhibitor segments and are expected to address major unmet needs in hemophilia. For ACPs, these include the development and management of inhibitors, particularly in hemophilia B patients, as well as convenient administration and improved prophylactic dosing schedule. Gene therapies are anticipated to provide long-term maintenance of the disease, with the potential to eradicate the need for prophylactic treatment and the risk of inhibitor development, providing a dramatic effect in the long term,” said Tajekesa Chapman, PhD, senior oncology and hematology analyst at GlobalData, in the press release.

GlobalData expects that the lack of long-term safety and efficacy data and the high cost of gene therapies targeting a small patient population, however, will pose a challenge to market access of these scientifically advanced hemophilia A and B therapies.

“A major barrier for market growth in hemophilia A and B will be the increased cost consciousness due to healthcare austerity measures being incorporated across the 8MM, which will limit premium pricing opportunities for pipeline agents in the hemophilia market and require new entrants, particularly ACPs and gene therapies, to consider the changing reimbursement landscape when determining pricing strategies for their drugs,” Chapman added in the press release.

The pricing strategy for Roche’s Hemlibra (emicizumab) over existing long-acting recombinant factor VIIIs (rFVIIIs) and bypassing agents enables the drug to exploit cross-segment efficacy to ultimately become a blockbuster in the hemophilia space, with expected sales of $1.5bn in 2028. Roche is, therefore, set to become the hemophilia market leader in 2028 with combined sales of $1.8bn from Hemlibra and gene therapies, upon the successful acquisition of Spark Therapeutics, according to GlobalData. Hemlibra was approved by FDA in November 2017 and had CHF 224 million (US$227 million) in 2018 sales.

“Developers are now focusing both on the identification of alternative modulators of the coagulation cascade and on innovative strategies that exploit new mechanisms of action, but companies are also moving toward the identification of a cure for the disease with the development of gene therapies. Acquisitions, licensing, and partnership agreements are becoming common in the hemophilia market, as some major players are actively on the lookout for ACPs and gene therapies,” Chapman stated in the press release.