(Reuters) – U.S. biotech company Global Blood Therapeutics Inc said on Wednesday its experimental drug met the main goal of a late-stage trial in patients with sickle cell disease, a potentially life-shortening inherited blood disorder.
A statistically significant number of patients on the treatment, voxelotor, showed a rise in levels of hemoglobin – a protein in red blood cells that carries oxygen throughout the body – compared to a placebo.
The data was from the first phase of the late-stage trial, and the company said it was in talks with the U.S. Food and Drug Administration on potential accelerated approval of the drug based on the main goal.
Sickle cell disease affects about 100,000 Americans and in 1 in 13 black or African-American babies are born with the trait, the Centers for Disease Control and Prevention estimates.
Mandatory screenings in the United States help diagnose newborns at birth and patients are currently treated with hydroxyurea, an old cancer drug, and antibiotics like penicillin to prevent infections.
The only potential cure at present is a bone marrow transplant but the procedure presents a risk of infection, infertility and death.
The company said it plans to present results from the study at a medical meeting later this year.
Treatments of the disease focus on managing symptoms of the disease rather than curing it.
Global Blood has designed voxelotor as a once-a-day pill for life.
“It is very much like HIV. If you take the medicine, the virus is not going to hurt you. Our goal here is that if you take your medicine, you’re not going to die of the disease,” Chief Executive Officer Ted Love told Reuters on a phone call ahead of the results.
Patients are frequently hospitalized and Love estimates that about $200,000 is spent a year per patient.
“We’re spending a lot of money. These people are suffering a great deal and they’re still dying in their forties,” Love said.
“So if we can introduce an option that extends their survival and may be eats away at a lot of that cost – that’s extraordinary. I think that will help get the drug reimbursed.”
The company’s shares were marginally down in volatile premarket trading.
Reporting by Manas Mishra and Tamara Mathias in Bengaluru; Editing by Maju Samuel