RARE Daily

GBT Initiates Early Access Program for SCD Patients in Europe

December 2, 2020

Rare Daily Staff

Global Blood Therapeutics said it has initiated an early access program for its drug voxelotor in Europe and other regions outside the United States for the treatment of hemolytic anemia in sickle cell disease patients ages 12 years and older.

Voxelotor is approved in the United States under the trade name Oxbryta for the treatment of SCD in patients ages 12 years and older. GBT previously announced it plans to seek regulatory approval to treat hemolytic anemia in SCD in patients ages 12 years and older in Europe. An early access program is a mechanism to make medicines available pre-approval upon request by a physician for appropriate patients with no alternative treatment option.

“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,” said Ted Love, president and CEO of GBT. “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.”

A first-in-class oral, once-daily therapy, voxelotor directly inhibits hemoglobin polymerization, the root cause of the sickling and destruction of red blood cells in SCD. The sickling process causes hemolytic anemia (low hemoglobin due to red blood cell destruction), which impairs adequate oxygen delivery to the tissues and organs in the body.

Through the voxelotor early access program, physicians in countries with an early access regulatory and legal pathway may be able to request voxelotor for eligible SCD patients who do not have access to the medicine as part of a clinical trial.

Inceptua Group’s Medicines Access division will implement the early access program for GBT. Inceptua has expertise in the strategy, design, and operational implementation of pre-approval access programs that make pharmaceutical products in clinical development available to patients as appropriate.

For the six Middle Eastern countries in the Gulf Cooperation Council, GBT previously announced an exclusive distributorship that references the U.S. approval of Oxbryta to allow for access to the medicine while health authorities conduct their regulatory reviews, thereby obviating the need for this new early access program.

Photo: Ted Love, president and CEO of Global Blood Therapeutics

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