While much of the biotech sector is hated, BioMarin Pharmaceutical has been carving its own path in the space, with six drugs on the market that treat highly uncommon conditions.
One of the drugs in its pipeline is a “very exciting product” that would treat hemophilia A, a blood disorder that affects approximately 120,000 people, Chairman and 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, in the 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 that the disorder is curbed for their entire lifetimes of about 10 years, Bienaimé said.
“We’re going to give an update next week at the World Hemophilia Federation meeting with two years of data” on human patients, the CEO told Cramer. “This is such novel therapy that some patients are going to jump on it, but some patients are going to want to see several years of data before they get treated. So the market is not going to disappear after three years. It will take a while to penetrate.”