Alpha-1 antitrypsin deficiency (AATD) is a genetic illness, and may cause severe lung or liver disease. Symptoms may include trouble breathing and jaundiced, or yellow, skin The prevalence is nearly 0.025% in general population. Asia has comparatively less incidence of AATD as compared to Europe or North American population. North America and Europe are likely to be key markets.
Augmentation therapy is the only particular therapy for alpha-1 antitrypsin deficiency (AATD); during the therapy, preparations of alpha-1 antitrypsin protein isolated from pooled blood of healthy donors are administered by way of weekly intravenous infusion. This raises the blood levels of alpha-1 antitrypsin deficiency AATD to levels protective for the lung.
In 2022, Takeda and Arrowhead Pharmaceuticals Inc. announced that results from a Phase 2 clinical study (AROAAT-2002) of investigational fazirsiran (TAK-999/ARO-AAT) for the treatment of liver disease related with alpha-1 antitrypsin deficiency (AATD) were published in the New England Journal of Medicine (NEJM) and presented in an oral presentation at The International Liver Congress™ 2022 - The Annual Meeting of the European Association for the Study of the Liver (EASL). The results from the AROAAT-2002 study provided several lines of evidence that pre-existing liver damage in patients might be meaningfully improved following treatment with fazirsiran. Such developments are expected to encourage the global alpha-1 antitrypsin deficiency (AATD) treatment market.
In 2022, Inhibrx received FDA orphan-drug designation for INBRX-101 for the treatment of alpha-1 antitrypsin deficiency. Interim results reported in October 2021 from the Phase 1 study revealed a favourable safety and tolerability profile with no drug-related severe adverse events at doses up to and including 120 mg/kg single dose, and 80 mg/kg multi-dose administered intravenously, or IV. Data from the single ascending dose cohorts exhibited the potential to attain normal AAT levels with monthly dosing.
In 2021, the Food and Drug Administration (FDA) granted orphan drug designation to alvelestat (MPH-966) for the treatment of alpha-1 antitrypsin deficiency (AATD).
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