HQ Team
September 26, 2024: Pfizer Inc.’s sickle cell therapy involving Oxbryta has been voluntarily withdrawn from global markets due to painful complications and death, according to a company statement.
Pfizer “is voluntarily withdrawing all lots of Oxbryta or voxelotor for the treatment of sickle cell disease at this time, in all markets where it is approved.” Since its first approval in 2019, Oxbryta has been marketed in more than 35 countries globally.
“Pfizer’s decision is based on the totality of clinical data that now indicates the overall benefit of Oxbryta no longer outweighs the risk in the approved sickle cell patient population.
“The data suggest an imbalance in vaso-occlusive crises and fatal events which require further assessment.”
Organ damage
A vaso-occlusive crisis is a painful episode that occurs when sickled red blood cells get stuck in blood vessels, blocking blood flow and causing organ damage.
The Manhattan, New York City-based company “is also discontinuing all active voxelotor clinical trials and expanded access programs worldwide.”
It has notified regulatory authorities about these decisions and is further reviewing the available data and investigating the findings, according to the statement.
“The safety and well-being of patients is of the utmost importance to Pfizer, and we believe this action is in the best interest of patients,” said Aida Habtezion, Chief Medical Officer and Head of Worldwide Medical and Safety at Pfizer.
“Our primary concern is for patients who suffer from sickle cell disease, which remains a very serious and difficult-to-treat disease with limited treatment options.
“We advise patients to contact their physicians to discuss alternative treatment while we continue to investigate the findings from our review of the data,” she said.
No revenue guidance impact
The company does not anticipate that this event will impact its full-year 2024 financial guidance. Pfizer expects to achieve full-year 2024 operational revenue growth of 8% to 10% ($58.5 to $61.5 billion),compared to 2023 revenues.
Pfizer acquired Oxbryta, as part of its $5.4 billion buyout of Global Blood Therapeutics in 2022. It reported $328 million in revenues from the therapy for the full year 2023.
Sickle cell disease is a lifelong, debilitating inherited blood disorder in which red blood cell sickling results in vascular inflammation and hemolytic anaemia.
Vascular inflammation, together with sickled red blood cells can lead to acute pain crises, vaso-occlusive crises, and progressive end-organ damage, including stroke.
Complications of the disease begin in early childhood and are associated with shortened life expectancy.
While rare, in developed markets 4.5 million people are living with the disease globally and more than 45 million people living with the sickle cell trait. It occurs particularly among those whose ancestors are from sub-Saharan Africa, though it also occurs in people of Hispanic, South Asian, Southern European and Middle Eastern ancestry.
