The 2018 edition of Drugs to Watch, the annual industry forecast and analysis from Clarivate Analytics, anticipated the approval and market entry of Shire’s Takhzyro (lanadelumab). The drug was approved in the U.S. under accelerated review, with Orphan Drug, Fast Track and Breakthrough Therapy status, as expected in August 2018, for the prevention of hereditary angioedema (HAE) attacks in patients 12 years and older.1 Launch is anticipated later in 2018. A marketing authorization application submitted in the EU in February 2018 was accepted for accelerated review in March.3
Takhzyro is the only monoclonal antibody to inhibit plasma kallikrein and has been developed as a prophylaxis treatment to prevent attacks of HAE.”
Takhzyro is the only monoclonal antibody to inhibit plasma kallikrein and has been developed as a prophylaxis treatment to prevent attacks of HAE.1 HAE is a rare genetic disorder affecting around one in 50,000 individuals which is caused by mutations in the gene that codes for C1 esterase inhibitor. This results in excessive production of bradykinin, a vasodilatory mediator and enhancer of vascular permeability, which causes edema (swelling) in various parts of the body. By targeting kallikrein, Takhzyro prevents the production of bradykinin.2
Shire’s Cinryze (C1 esterase inhibitor [human]) is the dominant brand for prevention of angioedema attacks, but one of its main drawbacks is its intravenous administration.4 The first subcutaneous product to prevent attacks of angioedema was CSL Behring’s Haegarda, launched in mid-2017 and also a C1 esterase inhibitor.5 However, despite Haegarda’s first-mover advantage, Takhzyro is set to dominate the market for first-line prevention of angioedema attacks in patients with HAE and significantly increase the use of such prophylactic medications due to its superior efficacy and more convenient dosing.6
Despite Haegarda’s first-mover advantage, Takhzyro is set to dominate the market for first-line prevention of angioedema attacks in patients with HAE and significantly increase the use of such prophylactic medications due to its superior efficacy and more convenient dosing.”
In the phase III HELP study, Takhzyro significantly reduced the mean frequency of angiodema attacks in patients with HAE by 87% versus placebo.7 These findings compare favorably to the 84% and 54% reductions in attacks reported for Haegarda8 and Cinryze9, respectively.
While both Haegarda and Cinryze require twice-weekly dosing, Takhzyro is dosed once every two weeks with potential for once-monthly dosing.6,8,9 Takhzyro also has a smaller injection volume than Haegarda, allowing for more rapid injection, thereby giving it a competitive edge that will see blockbuster revenue.6,8
Sales of Cinryze are expected to decline from $603 million in 2018 to $285 million in 2022, according to Cortellis Consensus Sales Forecasts (source Thomson Reuters I/B/E/S). Meanwhile forecast sales for Takhzyro are anticipated to rise from $349 million in 2019 to $1.165 billion for 2022.
This article is part of an ongoing blog series profiling the 12 new, game-changing drugs predicted to achieve blockbuster status by 2022 in the 2018 edition of Drugs to Watch, the annual industry forecast and analysis from Clarivate Analytics. Read the full Drugs to Watch report here or follow the series for the latest updates.