GBT Initiates Early Access Program to Make Voxelotor Available in Brazil

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Note: This story has been updated July 19, 2022, to correct that voxelotor is approved in the U.S. to treat sickle cell in patients ages 4 and older.

Global Blood Therapeutics, known as GBT, has initiated an expanded access program to make voxelotor, its daily oral therapy targeting the root cause of sickle cell disease (SCD), available to adults and children ages 12 and older in Brazil.

ANVISA, the Brazilian health regulatory agency, has given the go-ahead to the expanded access program and patients are already being enrolled at clinical sites around the country.

“There are up to 100,000 people estimated to have sickle cell disease in Brazil, yet there are no currently approved treatments that directly address the underlying cause of the disease,” Kim Smith-Whitley, MD, executive vice president and head of research and development at GBT, said in a press release.

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“As part of our deep, ongoing commitment to patient access worldwide, we are pleased to initiate this expanded access program for eligible patients in Brazil who may benefit from voxelotor, which we believe can potentially modify the course of this debilitating disease,” Smith-Whitley said.

SCD is caused by mutations that lead to the production of an abnormal form of hemoglobin — the protein that red blood cells use to transport oxygen through the bloodstream. This abnormal hemoglobin sticks to itself and forms clumps — a process known as polymerization — causing red blood cells to take on the sickle-like shape that gives the disease its name and leading ultimately to their destruction (hemolysis) and anemia.

Voxelotor is a first-in-class oral therapy designed to block hemoglobin polymerization, thereby eliminating the root cause of SCD. The therapy is approved to treat sickle cell in patients ages 4 and older in the U.S. and patients 12 and up in the EU, where it is marketed under the brand name Oxbryta. However, ANVISA has not approved the therapy for marketing in Brazil.

The expanded access program, or EAP, aims to make medications available prior to approval for patients with no other treatment options who are expected to benefit from them. The EAP was authorized based on criteria that included the seriousness of SCD and the lack of alternative treatment options.

Under the program, voxelotor will be provided free of charge for as long as treatment is required. The program is being administered by the Inceptua Group.