Data presented at a large hematology conference in Europe indicate that a Phase III study of a drug in development for sickle cell disease reached its primary endpoint.
South San Francisco, California-based Global Blood Therapeutics said Friday that it had presented data from the Phase III HOPE trial of voxelotor, which it is testing among 274 patients aged 12 and older with SCD. The Phase III data, which were also published in the New England Journal of Medicine, showed that the drug produced an improvement in hemoglobin of 1 gram per deciliter of blood after 24 weeks of treatment, compared with placebo.
GBT said the updated results form the basis of a rolling submission of data to the Food and Drug Administration seeking accelerated approval for the drug, which the company anticipates completing in the second half of this year. HOPE randomized patients to receive the drug at 1,500mg, 900mg or placebo. The company previously presented results on 154 patients at the American Society of Hematology meeting in December of last year.
According to the results published in the NEJM, more than half of the 90 patients in the intent-to-treat and per-protocol analyses of the trial who received the drug at the 1,500mg dose had a hemoglobin response. By comparison, around one-third of the 92 patients in the 900mg arm had a hemoglobin response, while only single-digit percentages of the 76 in the placebo group did.
“These positive data from more than 270 patients enrolled in the HOPE study provide strong evidence that voxelotor, by significantly improving anemia and hemolysis, has the potential to be a disease-modifying treatment for SCD by preventing chronic organ damage and prolonging survival,” study investigator Dr. Jo Howard, a hematologist at King’s College London, said in a statement. “Given the observed ability of voxelotor to reduce anemia, hemolysis and red blood cell sickling, as well as a favorable safety profile, I am confident that voxelotor could potentially become a new standard of care for treating adolescents and adults with SCD.”
Current treatments for SCD include hydroxyurea, blood transfusions, antibiotics and pain relievers, according to the Mayo Clinic. In addition, bluebird bio announced Phase I/II data from a study of its gene therapy, Zynteglo (autologous CD34-positive cells encoding beta-A-T87Q gene), among SCD patients, at the EHA meeting.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
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