HAE Therapeutics in Asia-Pacific Markets to 2023 – Growth Driven by Ongoing Shift towards Plasma-Derivative Products and Expected Launch of Premium Therapies

Hereditary Angioedema (HAE) is a rare genetic disorder caused by a lack of a sufficient amount of a protein called C1 esterase inhibitor (C1-INH), which plays a role in immune system function, blood clotting, and bleeding. With insufficient C1-INH a peptide called bradykinin may be overproduced, which causes fluids to build up in body tissues (edema).

The onset of HAE may occur at any age but is most common during childhood or adolescence. The rarity of the disease, limited treatment options other than not-very-effective generic drugs, and the relatively high annual cost of therapy (ACoT) have made HAE treatment a highly lucrative market.

The approval of revolutionary plasma derivatives – including Cinryze and Berinert – for the treatment of HAE patients that are refractory to generic drugs triggered unparalleled growth in the market. Though the treatment options are limited to acute and prophylaxis patients, the HAE treatment patient pool is expected to increase over forecast period in APAC countries assessed in this report.

However, competition is intensifying as governments in Australia, South Korea and Japan are looking for more affordable treatment options due to the very high treatment costs. In 2018, two re-formulations of existing plasma derivative products will be launched – one each in Japan and Australia. Despite the superior efficacy of recently marketed therapies over traditional generic therapies, there are still unmet needs in the therapeutic landscape.

These include improved safety; the need to create products with more convenient and less invasive drug-delivery methods; and improved access to treatment. The difficulty of managing HAE is also compounded by poor patient adherence, a particular problem in countries where large segments of the population have limited access to healthcare. Most significantly, the HAE market lacks curative treatments, as current therapies aim to alleviate symptoms and reduce disease progression.

This major unmet need is not expected to be addressed directly by any of the pipeline agents. Additionally, any pipeline candidates that will be successfully approved and launched will compete for the same patient populations – although late-stage drugs are likely to address the need for a more convenient route of administration, as the pipeline contains potential kallikrein inhibitors that can be administered orally. Products with novel mechanisms of action and safer and more efficacious profiles will be welcome additions to the market.

Scope

– The current Asia-Pacific HAE market contains premium therapies such as Berinert, Cinryze, Ruconest and Firazyr.

– What are the competitive advantages of the existing premium therapies?

– There are 24 active pipeline molecules, and the late-stage investigational drug candidates feature improved administration routes compared with currently marketed products.

– Which classes of re-formulation drugs are most prominent in the pipeline?

– What is the potential for pipeline products to address unmet needs in the HAE market?

– Analysis of clinical trials since 2006 identified that the failure rates of HAE molecules were highest in Phase III, at 14.3%.

– How do failure rates vary by stage of development, molecule type, and molecular target?

– How do other factors, such as average trial duration and trial size, influence the costs and risks associated with product development?

– Over the 2016–2023 forecast period, the Asia-Pacific HAE therapeutics market is expected to grow at a CAGR of 9.6%, from $54.5m to over $103.5m.

– Which markets make the most significant contribution to the current market size?

– What are the epidemiology trends in these markets?

– Will new market entrants lead to substantial changes to annual therapy costs?

– How will different treatment usage patterns impact growth in the five assessed Asia-Pacific markets?

– The rising HAE prevalence population and the uptake of newer therapies will lead to significant market growth over the forecast period.

– Will the launch of emerging pipeline molecules threaten the commercial success of existing drugs?

– Licensing deals are the most common form of strategic alliance in the HAE therapeutics market, with deal values ranging from $2.7m to over $83m.

– How do deal frequency and value compare between target families and molecule types?

– What were the terms and conditions of key licensing deals?

Reasons to buy

- Understand the clinical context of HAE by considering epidemiology, symptoms, etiology and pathophysiology, diagnosis, prognosis, treatment guidelines and options, and orphan drug policies.

- Identify the therapeutic strategies, products, and companies that dominate the current marketed products landscape and recognize gaps and areas of unmet need.

- Identify key pipeline trends in terms of molecule type, administration route, molecular target, and novelty.

- Consider market opportunities and potential risks by examining trends in HAE clinical trial size, duration, and failure rate by stage of development, molecule type, and molecular target.

- Recognize the late-stage pipeline molecules that have demonstrated strong therapeutic potential in HAE by examining clinical trial data and multi-scenario product forecast projections.

- Compare treatment usage patterns, annual therapy costs, and market growth projections for India, China, Australia, Japan and South Korea.

- Discover trends in licensing and co-development deals concerning HAE products and identify the major strategic consolidations that have shaped the commercial landscape.

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