While much of the biotech sector is usually hated, BioMarin Pharmaceutical has been carving its own path inside space, with six drugs on the market in which treat highly uncommon conditions.
One of the drugs in its pipeline is usually a “very exciting product” in which would likely treat hemophilia A, a blood disorder in which affects approximately 0,000 people, Chairman as well as CEO Jean-Jacques Bienaimé told Cramer in a Thursday interview. Click here for the full video.
“We are developing the first gene therapy for hemophilia A,” the CEO said. “We’re trying to replace two to three injections intravenously every week by one injection, potentially, inside lifetime of the patient.”
While BioMarin hasn’t obtained data tracking the treatment over a given patient’s lifetime, tests on of hemophilia-afflicted animals have shown in which the disorder is usually curbed for their entire lifetimes of about 10 years, Bienaimé said.
“We’re going to give an update next week at the earth Hemophilia Federation meeting with two years of data” on human patients, the CEO told Cramer. “This kind of is usually such novel therapy in which some patients are going to jump on This kind of, yet some patients are going to want to see several years of data before they get treated. So the market is usually not going to disappear after three years. This kind of will take a while to penetrate.”