Global Blood Therapeutics (GBT) has launched an early access program for voxelotor in Europe and other regions outside the U.S. to allow people with hemolytic anemia associated with sickle cell disease (SCD) to have access to treatment.
The early access program will be open to those 12 and older who have no alternative treatment options and are unable to access voxelotor through clinical trials.
“With no currently approved therapies outside the United States to treat hemolytic anemia in sickle cell disease, the unmet need in this devastating disease is profound,” Ted W. Love, MD, president and CEO of GBT, said in a press release.
“As part of our deep, long-term commitment to patient access worldwide, we are pleased to initiate this early access program for eligible patients who may benefit from voxelotor,” Love said.
Voxelotor is a first-in-class oral therapy designed to prevent hemoglobin molecules from sticking to each other, a process known as hemoglobin polymerization, which is the root cause of red blood cells’ sickling and destruction in SCD. Hemoglobin is a protein found in red blood cells that is responsible for carrying oxygen throughout the body.
By inhibiting this polymerization process, voxelotor is expected to reduce hemolytic anemia — a blood disorder in which red blood cells are destroyed faster than they can be replenished — and promote oxygen delivery to different tissues and organs in the body.
In the U.S., voxelotor has been approved under the brand name Oxbryta to treat SCD in children and adults 12 and older. Earlier this year, GBT also announced its plans to request the therapy’s approval in Europe for the same indication.
With the early access program in place, physicians in Europe and other regions now may request voxelotor be made available to treat eligible SCD patients before its approval.
GBT is implementing voxelotor’s early access program in partnership with Inceptua Group’s Medicines Access division, a leader in the design, strategy, and establishment of pre-approval access programs for unlicensed medicines.
Clinicians wishing to know more about voxelotor’s early access program may do so by reaching out to Inceptua at [email protected].
Patients living in one of the six countries making up the Gulf Cooperation Council (GCC) — Bahrain, Kuwait, Oman, Qatar, Saudi Arabia, and the United Arab Emirates — will not require the early access program to gain access to voxelotor.
That is because GBT previously entered an exclusive agreement with Biopharma-Middle East and Africa that allowed the company to distribute the medication while health authorities in these countries conducted regulatory reviews of voxelotor.
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