An artist illustration of a doctor holding lungs. The FDA granted orphan-drug status for KB408 in September for the treatment of serious lung disease caused by alpha-1 antitrypsin deficiency (AATD). Credit: mi-viri via Getty Images.

The US Food and Drug Administration has approved Krystal Biotech’s Investigational New Drug (IND) for a Phase I clinical trial of the investigational alpha-1 antitrypsin deficiency (AATD) treatment KB408.

Earlier this month, the FDA granted orphan-drug status for KB408 in AATD. The first patient in the planned Phase I trial is expected to receive treatment in Q1 2024. The Phase I trial, involving adult patients with AATD and the PI*ZZ genotype, will assess the safety, tolerability, and efficacy at three planned dose levels.

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AATD is a rare genetic disorder that occurs when there is insufficient production or impaired functionality of the alpha-1 antitrypsin (AAT) protein. Low AAT levels result in damage to lung tissue, leading to chronic obstructive pulmonary disorder (COPD).

KB408 is a gene therapy consisting of a modified herpes simplex virus 1 (HSV-1) derived vector, which delivers  two full-length copies of the SERPINA1 gene to enable AAT expression. The therapy is delivered via nebulisation.

Senior Vice President of Clinical Development at Krystal Biotech Hubert Chen said, “This IND acceptance represents an important milestone for us as we work to address a serious lung disease with limited treatment options and allows us to demonstrate the potential of our platform to deliver genes repeatedly to epithelial cells of the lung.”

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KB408 isn’t the first rare disease drug that Krystal Biotech has developed. In May, the FDA approved Vyjuvek, Krystal’s lead  drug used to treat dystrophic epidermolysis bullosa (DEB).

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According to GlobalData, the AATD market in the US and Germany is expected to see significant growth in the next few years, with its market size reaching $3.48bn in 2031.

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