The European Medicines Agency (EMA) has decided not to approve Kamada Ltd.'s (Nasdaq: KMDA); TASE: KMDA) inhalation product for the treatment of Alpha-1 Antitrypsin Deficiency (AATD - hereditary emphysema). The EMA did not actually refuse to approve it, but the company has already received signals from the Agency that the current submission was inadequate, and that more data would be required in order to approve the drug. Kamada therefore decided to withdraw its request, collect the data in the framework of a trial it was planning in the US, and submit all the results to the US Food and Drug Administration (FDA) and the EMA simultaneously. The US trial will begin in 2018, and is expected to take a number of years.
Kamada's share price tumbled 23.44% on the Tel Aviv Stock Exchange today.
The news is a blow to Kamada's image, because the company had insisted to the market that the product could be approved, even though it had not met the primary endpoint of the clinical trial conducted, which ended in 2014. Then-Kamada CEO David Tsur maintained that the trial results were good, despite having failed to meet the primary endpoint (the time that passed before the first attack of the disease). Another analysis showed that most of the secondary endpoints were also not achieved, but Tsur maintained that the product was suitable for approval, because it had met one of the secondary endpoints - improvement of lung function. Tsur argued that understanding of the disease had improved in the four years between the beginning and the end of the trial, and that everyone now knew that lung function, in which the company had attained significant results, was actually the important statistic.
Current Kamada CEO Amir London, who took up his position in 2015, one year after the failure of the trial, seconded what Tsur had said, and announced that in a meeting with the EMA, the latter had told the company, "We will not hold it against you" that Kamada had not met the trial's primary endpoint, but would evaluate the clinical results in their entirety. London has since quoted this sentence several times, interpreting it to mean that the fact that the company had met a secondary endpoint was likely to suffice for approval of the product. This assessment, however, proved over-optimistic.
"Globes": What does the EMA actually want to see now?
London: "We met the endpoint for improvement of lung function, with statistical and clinical significance, but the fact that this was a retrospective analysis of the data detracted from its force, and the Agency wanted to see us meet this endpoint in a trial specially designed for the purpose. It also wanted to see additional data, including the effect on lung tissue in the effect on attacks, the endpoint that we didn't meet."
Is there a risk that this will again be the primary endpoint?
"Not really. Attacks will not be the primary endpoint, because we understand the disease much better now. Lung function is the important variable."
London notes that Kamada was in any case preparing for another trial, in which lung function is the primary endpoint, for the purpose of obtaining FDA approval. This trial will begin in 2018. The company is now hoping that this trial will be enough to answer all of the EMA's questions.
Will you have to invest more money or time in the US trial than you thought?
"No, we believe that our plans will not change, and that we will allocate the same resources for the trial that we were planning in any case, and we will also continue our other clinical plans as before."
Published by Globes [online], Israel Business News - www.globes-online.com - on June 22, 2017
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