Parents can face anguish when their child is ill. But when the illness is rare, they must also struggle beyond their initial shock and learn about a disease they hardly knew existed.
This is what Kim and Scott Martin faced when their infant son was diagnosed with hemophilia B. Seeking answers, the family consulted with specialists and cast a wide net to see if there was anyone who could help them cope and learn to take care of their son with this rare bleeding disorder. Luckily, the Martins found John and Joyce Taylor, whose son also had been diagnosed with hemophilia B and confronted similar problems.
Creating a Company
When the two families met, they determined there had to be a way they could help their sons other than with traditional therapies. Together, they formed the Laguna Niguel-based company Inspiration Biopharmaceuticals in 2004. Since then the company has grown to 38 full-time employees and one part-timer.
According to the World Federation of Hemophilia, only about 25 percent of the estimated 400,000 people with hemophilia worldwide receive adequate treatment.
Since the treatment of hemophilia in much of the world is limited, many people have trouble obtaining adequate access to medication. The high price and the low supply of the treatment make it very hard to help people who are struggling every day with the disease.
“We want to open up to the global market, and one of the things that is very important to us is to improve access to care,” said Gordon Busenbark, chief financial officer of Inspiration Biopharmaceuticals.
What Is Hemophilia?
Hemophilia is a disorder that can lead to uncontrolled internal bleeding that usually starts in the joints. These bleeds cause severe pain, joint damage and disability after frequent occurrences. There are two types of hemophilia. Hemophilia A affects one out of 5,000 male births and is caused by an absence of factor VIII, a naturally occurring protein that is essential to blood clotting.
Hemophilia B is rarer, affecting one out of 30,000 male births, and is caused by a deficiency of factor IX, another protein that is part of the clotting system.
Inspiration Biopharmaceuticals is working on two potential products to help treat both types of hemophilia. IB1001 is an intravenous factor IX product for the treatment and prevention of bleeding for people with hemophilia B. OBI-1 is designed to help those who have developed an immune reaction against their factor VIII. Both IB1001 and OBI-1 are in the last stage of clinical testing and will be submitted for approval from the Food and Drug Administration, according to Busenbark.
"This is the first time we know of that families affected by a disease have actually developed a product that is nearing the market," Busenbark said.
Why Laguna Niguel
Laguna Niguel is the home to Inspiration Biopharmaceuticals because of the many resources it has to offer, he said. Many individuals who helped to create the company live in the area.
"The company is located in Laguna Niguel because of its convenient location in a biotech hub," Busenbark said.
The hemophilia community has been advocating efforts worldwide to help people deal with this disorder. National Hemophilia Foundation chapters are set up in various locations around the country.
The local National Hemophilia Foundation chapter is in Hollywood. Visit the website at hemophilia.org/NHFWeb/MainPgs/MainNHF.aspx?menuid=51&contentid=245&rptname=chapter.