Two weeks bring two new treatments for sickle cell
On Nov. 15, the FDA approved
Adakveo from Novartis, the first treatment created specifically for sickle cell disease. Right on its heels was the FDA’s Nov. 25 approval of
Voxelotor from Global Blood Therapeutics.
“Adakveo is the first targeted therapy approved for sickle cell disease, specifically inhibiting selectin, a substance that contributes to cells sticking together and leads to vaso-occlusive crisis,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research. “Vaso-occlusive crisis can be extremely painful and is a frequent reason for emergency department visits and hospitalization for patients with sickle cell disease.”
Voxelotor directly inhibits sickle hemoglobin polymerization, the root cause of SCD. The once a day tablet for children and adults 12 and over is expected to be available through GBT’s specialty pharmacy partner network within two weeks.
“When we started our journey with the SCD community more than eight years ago, we set out to transform the way this devastating, lifelong disease is treated,” said Ted W. Love, M.D., president and chief executive officer of GBT. “We are proud to bring this breakthrough therapy to the SCD community. Uniquely developed from inception to treat SCD, Oxbryta embodies GBT’s commitment to develop and deliver innovative medicines for patients with overlooked, life-limiting chronic diseases. We are grateful to the patients, caregivers, clinical trial investigators, healthcare providers and advocates who have worked alongside us to develop this first-in-class therapy.”
“Thanks to the Orphan Drug Act, there are numerous promising clinical trials underway. We are also encouraged by the National Institutes of Health’s Cure Sickle Cell Initiative striving for a genetic cure in the next five to 10 years,” said Dr. Wanda Whitten-Shurney. “Our next challenge is to make sure the medication is accessible to the patients who so desperately need it. Individuals with sickle cell disease are living longer, but we are also focused on improving their quality of life.”
