Top Companies in the Sickle Cell Disease Treatment Market

The sickle cell disease treatment market was valued at USD 5.38 billion in 2023. The market is expected to grow at a CAGR of 16.9% during the forecast period of 2024-2032, with the values likely to reach USD 21.92 billion by 2032. The market is driven by the rising prevalence of sickle cell disease and advancements in therapies across the 8 major markets.

Treatment for sickle cell anemia involves addressing discomfort and organ damage caused by abnormal hemoglobin S, resulting in sickle-shaped and rigid red blood cells. Mutations in the hemoglobin subunit beta gene result in irregular production of red blood cells, leading to blockages and reduced oxygen delivery to organs. Managing sickle cell disease requires medication, blood transfusions, pain management, and possible treatments such as bone marrow transplants and gene therapies. Recent treatments such as novel therapeutics, blood transfusions, and hydroxyurea demonstrate potential for improved patient outcomes.

The rise in sickle cell disease cases is fueling the expansion of the market for sickle cell disease treatment. A rising need for new therapies and extended assistance, especially among the youth, is becoming increasingly apparent. Advancements in gene therapy and gene editing technology, like CRISPR-Cas9, are changing the way we treat illnesses by providing a possible permanent solution. Treatments like LentiGlobin and CRISPR are giving hope to patients, leading to an expected surge in market demand.

In December  2023, the FDA approved Casgevy and Lyfgenia as the first cell-based gene therapies for sickle cell disease in patients 12 years and older. Casgevy is also the first FDA-approved treatment using novel genome editing technology. The growth in the regulatory approvals of such innovative treatments is poised to elevate the market value in the coming years.

Let’s explore the profile of top companies leading the sickle cell disease treatment market in 2024.

Novartis AG

Novartis AG, a Swiss pharmaceutical company, specializes in the development of prescription and generic medications, along with eye care products. Their products address a range of illnesses including cancer, heart conditions, and respiratory ailments. In April  2024, the FDA approved Novartis' Adakveo as a treatment to lessen pain episodes in people with sickle cell disease. This drug aids in reducing the number of vaso-occlusive crises in individuals aged 16 and above. This makes them a leading player in the sickle cell treatment market.

Pfizer Inc.

Pfizer Inc. is a global pharmaceutical and biotechnology corporation headquartered in New York. It is widely acknowledged for creating drugs and vaccines for people and animals globally. In October  2022, the company completed the acquisition of Global Blood Therapeutics, Inc., a biotech firm focused on sickle cell disease, enhancing its dedication toward the treatment of this blood disorder.

Bluebird Bio, Inc.

Bluebird bio, Inc. is based in Massachusetts and is involved in developing gene therapies for genetic disorders. LYFGENIA (lovo-cel) was given approval by the FDA in December  2023 for individuals with sickle cell disease who are 12 years and older and have a history of vaso-occlusive events. LYFGENIA focuses on addressing the root cause of sickle cell disease through gene therapy.

Emmaus Life Sciences, Inc.

Emmaus Life Sciences, Inc. is a biopharmaceutical company that focuses on discovering, developing, and selling innovative treatments and therapies for rare and orphan diseases. In February  2024, the Health Department of Puerto Rico approved Endari® (L-glutamine oral powder) for the treatment of sickle cell disease. This milestone is an important part of Emmaus' objective to improve the well-being of sickle cell disease patients globally by providing therapy access in Puerto Rico.

Bristol Myers Squibb Company

Bristol-Myers Squibb, an American pharmaceutical company, focuses on researching, developing, and providing drugs for severe diseases. They are a leading player in the sickle cell disease treatment market. In 2016 , the company teamed up with the Angola Ministry of Health, Baylor College of Medicine, Texas Children’s Hospital, and Chevron to enhance healthcare for sickle cell patients in Angola and provide medication to children who were not able to access treatment.

Vertex Pharmaceuticals Incorporated

Vertex Pharmaceuticals is a Boston, Massachusetts-based biopharmaceutical company. It was among the first to embrace rational drug design. In December  2023, the company collaborated with CRISPR Therapeutics to secure FDA authorization for CASGEVY™, a cell therapy designed for sickle cell disease. This therapy is intended for individuals aged 12 and above who experience frequent episodes of vaso-occlusive crises.

Sangamo Therapeutics, Inc.

Sangamo Therapeutics, a biotech company based in Brisbane, California, focuses on cell and gene therapy for genetic diseases like haemophilia. In January  2022, it was disclosed that the sickle cell disease program SAR445136 was transferred from Sanofi back to Sangamo. Sanofi transferred its duties and privileges to the zinc finger nuclease gene-edited cell therapy candidate for sickle cell disease to the genomic medicines company.