Biopharmaceutical company Kamada says data collected from dozens of patients over 13 months show excellent levels of tolerability and safety for its flagship congenital-emphysema product. AAT is undergoing decisive Phase III clinical trials.
The trials on the drug's safety and effectiveness are being carried out in European countries including Britain, Germany, the Netherlands, Denmark and Ireland. If all goes well, Kamada expects the treatment to be fast-tracked through the approval process due to its orphan-drug status.
This status is given in the United States to drugs for disorders affecting up to 200,000 people. It guarantees seven years of market exclusivity, during which no other drug will be approved for the same purpose.
Congenital emphysema is an incurable disease caused by a deficiency in the AAT protein. The ailment, sometimes fatal, is characterized by a chronic infection that lowers the lungs' functioning capacity by destroying tissue. Current treatments involve AAT infusions to prevent the disease from progressing.
Kamada's Phase III trials for delivering AAT through an inhaler will include 200 patients, about 80 of whom have already been enlisted. The study is expected to conclude in mid-2012.
When applied through an inhaler, the drug goes directly to the diseased tissue. An inhaler requires only 25% of the medicine used in infusions.
There are an estimated 100,000 potential patients in the United States and about the same number in Europe. But only about 5,000 people are under treatment in the United States and 1,000 in Europe. The U.S. market for the drug is worth about $400 million to $500 million with an annual growth rate of about 10%. The market in Europe is estimated at 500 million euros.
Glassia, an intravenous drug made by Kamada for congenital emphysema, was released in the United States in the third quarter of 2010 through a marketing agreement with Baxter International. The company expects Glassia sales to boost its revenues to $21 million for the fourth quarter of 2010, 23% more than its total sales in 2009, and to $35 million for all of 2010.