NORTH AMERICA SICKLE CELL DISEASE MARKET ANALYSIS

The North America sickle cell disease market is estimated to be valued at US$ 1,036.5 million in 2023 and is expected to exhibit a CAGR of 16.2% during the forecast period (2023-2030).

Analysts’ Views on the North America Sickle Cell Disease Market:

Research on rare diseases is a never-ending topic among researchers, and it is continuously growing as investors show interest in funding new studies and advancements in treatment options. For instance, the prevalence of sickle cell disease is keeping on increasing due to factors such as lack of access to healthcare and genetic predisposition in certain populations. The North America market for the treatment for sickle cell disease is expected to grow significantly over the forecast period.

Figure 1. North America Sickle Cell Disease Market Share (%), By Drug Type, 2023

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North America Sickle Cell Disease Market– Drivers

• Granting designation by the U.S. Food and Drug Administration: The designation helps for faster approval by regulatory authorities, as it shows trust and the importance of the drug for sickle cell disease. It also provides eligibility for certain incentives and benefits. For instance, on April 27, 2023, Editas Medicine, Inc., a clinical stage genome editing company, announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to EDIT-301, an investigational gene editing medicine, for the treatment of sickle cell disease. The U.S. FDA previously granted Orphan Drug Designation to EDIT-301 for the treatment of beta thalassemia and Rare Pediatric Disease designation to EDIT-301 for the treatment of beta thalassemia and sickle cell disease.

Figure 2. North America Sickle Cell Disease Market Share (%), By Country, 2023

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North America Sickle Cell Disease Market – Country Analysis

Among country, the U.S. is estimated to hold a dominant position in the North America sickle cell disease market over the forecast period. The U.S. is estimated to hold 91.3% of the market share in 2023. The U.S. sickle cell disease market is expected to witness significant growth in the coming years, driven by the high prevalence of sickle cell disease. Therefore, key players are focusing on developing breakthrough drug for the treatment of sickle cell disease. For instance, on June 24, 2020, IMARA Inc., a clinical-stage biopharmaceutical company dedicated to developing and commercializing novel therapeutics to treat patients suffering from rare inherited genetic disorders of hemoglobin, announced that the U.S. Food and Drug Administration (FDA) had granted Orphan Drug Designation for IMR-687 for the treatment of patients with beta-thalassemia. The U.S. FDA previously granted Orphan Drug Designation for IMR-687 for the treatment of patients with sickle cell disease.

North America Sickle Cell Disease Market - Impact of Coronavirus (COVID-19) Pandemic

• Since the COVID-19 virus outbreak in December 2019, the disease has spread to over 100 countries across the globe, and the World Health Organization declared it a public health emergency on January 30, 2020.
• COVID-19 affected the economy in three main ways: by directly affecting the production and demand of drugs, by creating disruptions in distribution channels, and through its financial impact on firms and financial markets. Due to nationwide lockdowns, several countries, such as China, India, Saudi Arabia, the U.A.E., Egypt, and others, faced problems with the transportation of things from one place to another.
• However, the COVID-19 pandemic had a negative impact on the North America sickle cell disease market, owing to a reduction in bed and operating room capacity, which further led to considerable delays in treatment for sickle cell disease.

North America Sickle Cell Disease Market Segmentation:

The North America sickle cell disease market report is segmented into drug type, disease type, distribution channel.

• Based on disease type, the market is segmented into sickle cell anemia, sickle beta thalassemia, and sickle hemoglobin C disease. Out of which, sickle cell anemia is expected to hold a dominant position in the North America sickle cell disease market during the forecast period, and this is attributed to the increasing patient group in sickle cell disease.
• Based on drug type, the market is segmented into hydroxyurea, L-glutamine, crizanlizumab, pain-relievers, voxelotor, and others. Out of which, voxelotor is expected to dominate the market over the forecast period, and this is attributed to the higher efficacy in children aged 2 to 4 years with SCD.
• Based on distribution channel, the market is segmented into hospital pharmacies, retail pharmacies, and online pharmacies. Out of which, the hospital pharmacies are expected to dominate the market over the forecast period, and this is attributed to the easy availability of drugs for the treatment of SCD.
• Among all the segmentation, the disease type segment is expected to dominate the market over the forecast period, and this is attributed to the positive results of the clinical trials. On June 26, 2022, Agios Pharmaceuticals Inc., a public U.S.-based pharmaceutical company pioneering therapies for genetically defined diseases with a near-term focus on developing therapies for hemolytic anemias, announced that the Phase 2 portion of the global Phase 2/3 RISE UP study of mitapivat in sickle cell disease met its primary endpoint of hemoglobin response. The results support proceeding with the Phase 3 portion of the study.

North America Sickle Cell Disease Market - Cross Sectional Analysis:

• Among disease type, sickle cell anemia segment is expected to be dominant during the forecast period in the U.S. due to the key players focusing on organic strategies, such as getting designations from the U.S., which is expected to drive segment growth over the forecast period. For instance, in March 2022, Nicox SA, an international ophthalmology company, and Fera Pharmaceuticals, a privately-held, U.S.-based specialty pharmaceutical company, announced that the U.S. Food and Drug Administration (FDA) had granted Orphan Drug Designation for naproxcinod for the treatment of sickle cell disease, which affects an estimated 100,000 Americans. Naproxcinod is a nitric oxide (NO)-donating naproxen combining the cyclooxygenase (COX) inhibitory activity of naproxen with that of nitric oxide developed by Nicox and exclusively licensed to Fera in the U.S.

North America Sickle Cell Disease Market: Key Developments

• In October 2022, Pfizer Inc., a U.S.-based multinational pharmaceutical and biotechnology corporation, announced the completion of its acquisition of Global Blood Therapeutics, Inc. (GBT), a biopharmaceutical company dedicated to the discovery, development, and delivery of life-changing treatments that provide hope to underserved patient communities starting with Sickle Cell Disease (SCD). The acquisition reinforces Pfizer’s commitment to SCD, building on a 30-year legacy in the rare hematology space.
• In July 2022, Global Blood Therapeutics, Inc. announced that the Medicines and Healthcare Products Regulatory Agency (MHRA) has granted Great Britain marketing authorization for Oxbryta (voxelotor) for the treatment of hemolytic anemia due to Sickle Cell Disease (SCD) in adult and pediatric patients 12 years of age and older as monotherapy or in combination with hydroxycarbamide (hydroxyurea). Voxelotor, an oral treatment taken once daily, is the first medicine authorized in Great Britain that directly inhibits sickle hemoglobin (HbS) polymerization, the molecular basis of sickling, and the destruction of red blood cells in SCD.
• In May 2021, Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., an international research-focused healthcare Group (Chiesi Group), announced that the U.S. Food and Drug Administration (FDA) has approved FERRIPROX (deferiprone) for the treatment of transfusional iron overload due to Sickle Cell Disease (SCD) or other anemias in adult and pediatric patients 3 years of age and older. This U.S. FDA approval expands the use of FERRIPROX for patients with SCD or other anemias as well as patients with thalassemia, regardless of prior iron chelation exposure.

North America Sickle Cell Disease Market: Key Trends

• A new technology platform is the key for market expansion: The advancement in the technology platform for the treatment of sickle cell disease is the key to market expansion over the forecast period. It is expected to improve the quality of life of patients and potentially provide a cure for the disease. This technological advancement has the potential to revolutionize the way sickle cell disease is managed, offering more effective and personalized treatment options. Additionally, it may also reduce the economic burden associated with long-term disease management, leading to improved healthcare outcomes for patients worldwide. For instance, on May 19, 2023, Tessera Therapeutics, a biotechnology company pioneering a new approach in genetic medicine known as Gene Writing, announced that the RNA Gene Writing platform demonstrated continued advances in rewriting to correct pathogenic mutations responsible for phenylketonuria (PKU), alpha-1 antitrypsin deficiency (AATD), and Sickle Cell Disease (SCD) including proof of concept in non-human primates (NHPs) for PKU.

North America Sickle Cell Disease Market: Restraint

• High cost of treatment: CRISPR gene editing has made it possible to cure sickle cell disease, which affects millions worldwide, but most people with the condition won't be able to afford the cost of the treatment. Due to CRISPR gene editing, millions of individuals may now be cured of sickle cell disease, a terrible congenital blood illness, but the cost of therapy is so costly that not many people will likely receive it in the near future. The high cost of treatment is a significant barrier to widespread accessibility and availability. This creates a stark inequality in healthcare, as those who can afford the treatment will have the opportunity to live a healthier life, while those who cannot will continue to suffer from the debilitating effects of sickle cell disease. The development of more affordable options or financial assistance programs is crucial in order to ensure equal access to this life-saving treatment for all individuals affected by sickle cell disease. As a result, the companies operating in this market are now focusing on the development of cost-effective and reliable treatment methods for patients with sickle cell disease.

North America Sickle Cell Disease Market - Key Players

The major players operating in the North America sickle cell disease market include Novartis International AG, Bristol-Myers Squibb Company, Global Blood Therapeutics, Inc., Pfizer Inc., Emmaus Life Sciences, Inc., ADDMEDICA, Sangamo Therapeutics, Inc., Acceleron Pharma, Inc., Agios Pharmaceuticals, Inc., Chiesi Farmaceutici S.p.A., Vertex Pharmaceuticals, Inc., IMARA, Inc., bluebird bio, Inc., CRISPR Therapeutics, Scribe Therapeutics Inc., Fulcrum Therapeutics, Inc., HPC International, Editas Medicine, Inc., Nicox SA, Fera Pharmaceuticals, and Tessera Therapeutics.