Alpha 1-antitrypsin is one of the first protein therapeutics introduced on the market – more than 30 years ago – and, to date, it is indicated only for the treatment of the severe forms of a genetic condition known as alpha-1 antitrypsin deficiency. The only approved preparations are derived from plasma, posing potential problems associated with its limited supply and high processing costs. Moreover, augmentation therapy with alpha 1-antitrypsin is still limited to intravenous infusions, a cumbersome regimen for patients. Here, we review the recent literature on its possible future developments, focusing on i) the recombinant alternatives to the plasma-derived protein, ii) novel formulations, and iii) novel administration routes. Regulatory issues and the still unclear noncanonical functions of alpha 1-antitrypsin – possibly associated with the glycosylation pattern found only in the plasma-derived protein – have hindered the introduction of new products. However, potentially new therapeutic indications other than the treatment of alpha-1 antitrypsin deficiency might open the way to new sources and new formulations.
Augmentation therapy with alpha 1-antitrypsin: present and future of production, formulation, and delivery / Bianchera, Annalisa; Alomari, Esra'A Ali Mohammad; Bruno, Stefano. - In: CURRENT MEDICINAL CHEMISTRY. - ISSN 1875-533X. - (In corso di stampa). [10.2174/0929867328666210525161942]
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