An upcoming influx of new therapies into the Alpha-1 Antitrypsin Deficiency (AATD) space in the next decade is expected to provide a significant boost to the treatment market for this inherited disorder that can greatly damage both lung and liver function, found GlobalData. According to the leading data and analytics company, the two major markets* will grow at an impressive compound annual growth rate (CAGR) of 11.2%.

GlobalData’s report ‘Alpha-1 Antitrypsin Deficiency (A1AD) Disease – Global Clinical Trials Review, H1, 2020’ reveals that the AATD market was worth $1.20 billion in 2021 in the 2MM* and will grow to $3.48 billion in 2031. The report also notes that this sales growth will be in line with the entrance of novel agents into the market. The AATD pipeline in the 2MM has five therapies in late-stage development: Kamada’s inhalable Alpha-1 proteinase inhibitor therapy, Mereo’s NE inhibitor alvelestat and pH pharma’s NE therapy PHP-303 and the two RNAi therapies Novo Nordisk’s belcesiran and Arrowhead’s ARO-AAT. The clinical data, regarding the NE and RNAi therapies, seem promising in terms of their efficacy, according to KOLs who were interviewed by GlobalData.

Akash Patel, Pharma Analyst at GlobalData comments: “The current AATD pipeline is very promising according to key opinion leaders (KOLs) interviewed by GlobalData, except for the possible addition of a second-generation inhalable alpha-1 proteinase inhibitor by Kamada Ltd., for which KOLs have expressed some scepticism regarding its efficacy due to inhalable method of administration likely hindering drug delivery to elastin tissue in the lungs specifically.

“GlobalData expects these novel therapies, in particular two neutrophil inhibitor therapies and two RNAi therapies, to have long-term efficacy and lead to prevention or reversal of lung and liver damage, which alone will have a huge impact on the lives of AATD patients.”

The uptake of therapies in the AATD pipeline by physicians will be driven by clinical data demonstrating efficacy and ease of administration as well as price. If ribonuclease interference (RNAi) therapy is able to demonstrate significant efficacy for treating liver fibrosis, particularly ARO-AAT launching in 2027, then it will obtain a large market share. Similarly, if neutrophil elastase (NE) inhibitor therapies can improve FEV1 and prevent the worsening of emphysema, particularly alvelestat launching in 2027, they will likely be adjunct therapy to A1-PIs and be another driver of market growth.

The AATD market is likely to grow significantly from the launch of novel RNAi and neutrophil inhibitor therapies in the 2MM. In addition, the treatment paradigm will change significantly during the forecast period with physicians adopting the neutrophil inhibitor therapies, alongside the existing A1-PI therapies. The RNAi therapies offer treatment for AATD-related hepatic failure for the first time, especially for pediatric cases where the main symptoms are liver fibrosis. These novel therapies will be the main drivers of market growth and offer novel therapeutic options for physicians and patients.