The pharmaceutical company, Kamada, has just submitted its proposed Phase III protocol for an inhaled alpha-1 antitrypsin therapy (inhaled AAT) to the U.S. Food and Drug Administration (FDA). This therapy is to be used for the treatment of alpha-1 antitrypsin deficiency (A1AD) disease.
Alpha-1 Antitrypsin Deficiency (A1AD) is an inherited condition that is characterized by low blood levels of the protein Alpha-1 Antitrysin (A1AT). This deficiency leads to lung and liver disease as well as skin problems in the affected individuals. To learn more about A1AD, click here!
The proposed Phase III clinical trial would treat 200-300 patients with alpha-1 antitrypsin deficiency disease with the inhaled alpha-1 antitrypsin therapy at an 80 mg dosage once daily over a span of two years.
At the end of these two years, Kamada is hoping that participants see an improvement in lung function and lung density, the primary and secondary endpoints of the study, respectively.
Kamada’s Vice President of Clinical Development, Dr. Naveh Tov, MD, PhD, explains that the purpose of this study reflects Kamada’s previous experience with inhaled AAT, specifically, a Phase II U.S. study that showed an increase in the level of AAT in the lungs with the proposed dosage of medication. In fact, both the primary and secondary endpoints in this study are based on successful results from previously conducted trials.
However, there has been some skepticism about this whole thing. The FDA is not completely certain about the safety of inhaled AAT in treating alpha-1 antitrypsin therapy. This is a huge problem that Kamada will have to address and resolve, or the U.S. FDA will not grant approval for the further clinical development of this therapy in the United States.
With this in mind, the FDA gave Kamada more detailed instruction for development of their synopsis and also asked that Kamada propose a fully completed (and safe) protocol to follow for subsequent phases before it will be eligible to advance with development and clinical studies in the U.S.
So, the polished complete study protocol was submitted to the U.S. FDA on July 18, 2017, and they are hoping for all the best.
Even so, the CEO of Kamada, Amir London, says that the company followed the FDA’s guidance and has designed an appropriate Phase 3 clinical trial that follows protocol.