Limited Availability and Lack of Access are Unlikely to Hinder the Billion-Dollar Alpha-1 Antitrypsin Deficiency (AATD)

1. What are the roadblocks that are hindering the Alpha-1 Antitrypsin Deficiency (AATD) Treatment Market? Alpha-1 Antitrypsin Deficiency Treatment Landscape Public health has contributed significantly to the understanding and control of diseases in the twenty-first century. Sometimes monitoring and treating a serious ailment still depends on an early diagnosis. Alpha-1 antitrypsin deficiency (AATD), one of these uncommon genetic disorders, has seen improvements in terms of awareness and factors taken into account during treatment. Alpha 1 antitrypsin, a beneficial enzyme inhibitor, is depleted in AATD, a hereditary disorder that results in lung damage and functional loss. The other pathology causes the AAT protein to misfold (gain-of-function), which decreases the amount of AAT and increases liver proteolytic stress. Due to the fact that this condition is inherited, those who inherit two copies of the

2. defective gene are more likely to experience a variety of comorbidities. Approximately 80% of those who are impacted, according to published medical data, acquire lung disease. Alpha-1 antitrypsin augmentation therapy now dominates the market for treating alpha-1 antitrypsin deficiency. This is the process of obtaining normal AAT from blood plasma and injecting it. ARALAST (Takeda), GLASSIA (Kamada/Takeda), PROLASTIN/PROLASTIN-C (Grifols), ZEMAIRA (CSL Behring), and ALFALASTIN are some of the currently available alpha-1 antitrypsin augmentation therapies (LFB Biotechnologies). Despite the fact that these medications for alpha-1 antitrypsin insufficiency are expensive and do not have universal approval, the market for their therapy is worth billions of dollars. In the US and certain important European nations, Grifols is the leading competitor. Additionally, in 2021, Grifols got PMDA approval for its medicine LYNSPAD, which is used to treat alpha-1 antitrypsin insufficiency. Similar to this, the only alpha-1 antitrypsin therapy recognised in all of Europe is RESPREEZA (marketed as ZEMAIRA outside of the EU). RESPREEZA was granted a license in the UK in 2015, but a patient can only get the medication if the NHS agrees to cover the cost. NICE does not currently advise any specific alpha-1 antitrypsin deficiency treatment in the UK, with the goal of halting progression based on recommendations for related comorbidities. The future of the AATD market looks promising The alpha-1 antitrypsin deficiency treatment market's evolving landscape, despite the difficulties outlined above and the slow development, appears promising, with the new medicines having significant potential despite their limitations. The leader among them is Arrowhead's fazirsiran. The most sophisticated methods target AATD-related liver issues with RNAi. Off-label treatments for alpha-1 antitrypsin deficiency are now used to control the condition. Fazirsiran prevents severe AATD by reducing the generation of the Z-AAT protein. This also explains why the Japanese corporation Takeda is interested in these goods to establish its foothold in the market for expensive alpha-1 antitrypsin deficiency treatments. The aforementioned difficulties must be taken into consideration notwithstanding the encouraging alpha-1 antitrypsin insufficiency pipeline. The European Reference Network for Rare Lung Diseases (ERN-LUNG) and the European Alpha-1 Research Collaboration (EARCO) are two new patient-centered initiatives that have been launched in Europe to address these problems. Setting up quality control for AATD laboratories and a disease management programme are the two main goals of ERN-LUNG. At the same time, EARCO wants to establish a pan-European registry to comprehend natural history and support, manage, and advertise simple access to treatment options for alpha-1 antitrypsin deficiency. Given these initiatives, it is anticipated that the majority of issues related to the management and treatment of alpha-1 antitrypsin deficiency will be resolved soon.

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