Key Market Analysis of Sickle Cell Disease, Fanconi Anemia, and Diamond-Blackfan Anemia in the Inherited Types of Anemia Therapeutic Space

Anemia poses a significant public health challenge, primarily impacting the health of young children, pregnant and postpartum women, as well as menstruating adolescent girls and adult women. On a global scale, approximately 40% of children aged 6 to 59 months, 37% of expectant mothers, and 30% of women between the ages of 15 and 49 suffer from anemia.

LAS VEGAS, Nov. 20, 2023 /PRNewswire/ — Inherited types of anemia encompass a group of genetic disorders characterized by a deficiency in the production, structure, or function of hemoglobin—the protein responsible for carrying oxygen in our red blood cells. These conditions are typically passed down from one generation to the next and can manifest in various ways, each with its unique genetic mutation. While the specific genetic mutations and clinical symptoms vary among these disorders, they all share the common feature of leading to a reduced ability of the blood to transport oxygen effectively, resulting in fatigue, weakness, and other health complications. Management and treatment of inherited anemias often require lifelong medical care and may include blood transfusions, medication, and, in some cases, bone marrow transplantation.

DelveInsight has recently released a series of epidemiology-based market reports focusing on inherited types of anemia including Sickle Cell Disease, Fanconi Anemia, and Diamond-Blackfan Anemia. These reports include a comprehensive understanding of current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032  segmented into 7MM [the United States, the EU-4 (Italy, Spain, France, and Germany), the United Kingdom, and Japan].

Additionally, the reports feature an examination of prominent companies working with their lead candidates in different stages of clinical development. Let’s deep dive into the assessment of these inherited types of anemia markets individually.

Sickle Cell Disease Market

Sickle cell disease (SCD) is a group of inherited lifelong conditions that impact the structure of hemoglobin, a crucial protein in red blood cells. It is characterized by a chronic hemolytic disorder, where hemoglobin molecules tend to clump together and distort the shape of red blood cells, forming a sickle or crescent shape (Hb S). This leads to distinct vaso-occlusive events and accelerated destruction of red blood cells. SCD encompasses several different types, each determined by the specific genes inherited from one’s parents. The most prevalent SCD types include HbSS, commonly known as sickle cell anemia and the most severe form of the disease; HbSC, a milder form of SCD; and HbS beta-thalassemia, another less severe variation of the disease. As per DelveInsight analysis, the total prevalent cases of sickle cell disease in the US were ~105K in 2022, projected to increase during the forecast period (2023–2032).

The primary objectives in managing SCD involve alleviating pain, reducing infection risks, and effectively addressing complications. These goals focus on symptom control and complication management. Treatment initiation hinges on the prompt recognition of pain in SCD patients, as recurrent, severe pain episodes are characteristic of the disease. Currently, treatment strategies encompass both pharmacologic and nonpharmacologic avenues. Pharmacologic interventions, such as hydroxyurea, ENDARI (L-glutamine), ADAKVEO (crizanlizumab), and OXBRYTA (voxelotor), have received approval. Pain management options span opioids, nonsteroidal anti-inflammatory drugs (NSAIDs), acetaminophen, corticosteroids, and more. Acute vaso-occlusive crises are typically addressed with a combination of opioids and NSAIDs. Nonpharmacologic therapies offer complementary approaches, including cognitive behavioral therapy, biofeedback, relaxation techniques, acupuncture, and hypnosis.

Learn more about the FDA-approved drugs for sickle cell disease @ Drugs for Sickle Cell Disease Treatment

The existing treatment paradigms for sickle cell disease create opportunities for emerging therapies to fill crucial gaps. These novel treatments aim to deliver holistic cures, reduce treatment-related side effects, and offer enhanced dosing options. As the patient population continues to grow, the approval of these emerging drugs is expected to drive market expansion. Therefore, investing in this therapeutic sector is likely to yield substantial returns. As per DelveInsight analysis, in 2022, the United States accounted for ~USD 700 million, which is expected to increase by the end of 2032.

Sickle Cell Disease Pipeline Therapies and Key Companies

• Exa-cel: Vertex Pharmaceuticals/CRISPR Therapeutics
• Lovo-cel: Bluebird Bio
• GBT-601: Global Blood Therapeutics/Pfizer
• Mitapivat: Agios Pharmaceuticals
• EDIT-301: Editas Medicine
• Inclacumab: Global Blood Therapeutics/Pfizer
• Etavopivat: Forma Therapeutics/Novo Nordisk
• NDec: Novo Nordisk
• Rifaximin: Bausch Health

Dive deeper for rich insights into the Sickle Cell Disease Clinical Trials

Fanconi Anemia Market

Fanconi anemia, an rare genetic disorder, disrupts the bone marrow’s ability to generate an adequate supply of blood cells, encompassing red blood cells, white blood cells, and platelets. Typically, this condition is identified prior to the age of 15. In the majority of cases, Fanconi anemia follows an autosomal recessive inheritance pattern, resulting in progressive bone marrow dysfunction, congenital malformations, and an elevated risk of developing solid and hematological tumors at an earlier age compared to the general population. Assessments as per DelveInsight’s analysts showed that the total 7MM diagnosed prevalent Fanconi anemia cases in 2021 were close to 6.7K out of which the highest cases were observed in the United States. EU4 and the UK countries accounted for around 2.8K cases in 2021.

Fanconi anemia treatment is tailored to the specific symptoms exhibited by each individual, often requiring a coordinated effort from a multidisciplinary team of medical professionals. This team may consist of pediatricians, surgeons, cardiologists, nephrologists, urologists, gastroenterologists, audiologists, otolaryngologists, ophthalmologists, and other healthcare providers who collaborate to develop a holistic treatment strategy for the affected person.

There is currently no robust development in the Fanconi anemia pipeline, mainly comprising only two therapies. However, DelveInsight analysts predicted that the Fanconi anemia will increase from USD 45 million in 2021 at a decent CAGR by 2032 owing to the better understanding of Fanconi anemia, growing awareness, improved management, and rising research and development activities.

Fanconi Anemia Pipeline Therapies and Key Companies

• RP-L102 (lentiviral gene therapy): Rocket Pharmaceuticals
• FP-045: Foresee Pharmaceuticals

For a comprehensive view of the Fanconi anemia market, check out the Fanconi Anemia Market Assessment

Diamond-Blackfan Anemia Market

Diamond-Blackfan anemia (DBA) is a rare and severe inherited blood disorder characterized by a profound shortage of red blood cells, which are responsible for carrying oxygen throughout the body. This condition typically manifests in early childhood and can lead to symptoms such as fatigue, pale skin, and an increased risk of infections. Diamond-Blackfan anemia is a genetic disorder caused by mutations in certain genes that affect the production of red blood cells in the bone marrow. Although it is a rare condition, its prevalence is estimated to be around 5 to 7 cases per million live births, making it one of the less common types of anemia. Due to its rarity and the lifelong challenges it presents, individuals affected by Diamond-Blackfan anemia often require specialized medical care and support to manage their condition and improve their quality of life.

Treatment for Diamond-Blackfan anemia typically revolves around managing the symptoms and complications associated with this rare inherited disorder. The primary focus is on addressing the anemia, which is often severe and characterized by a shortage of red blood cells. Most individuals with DBA require regular blood transfusions to maintain an adequate hemoglobin level and alleviate symptoms like fatigue and pallor. Additionally, corticosteroid medications, such as prednisone, are often prescribed as they can stimulate red blood cell production in some patients. In some cases, hematopoietic stem cell transplantation (HSCT) may be considered, especially when other treatments are ineffective or when there are specific genetic mutations involved. Treatment plans are usually tailored to the individual’s unique circumstances and can evolve over time as new therapies and research developments emerge. Close monitoring and ongoing medical care are essential components of managing Diamond-Blackfan anemia.

DelveInsight estimated that the Diamond-Blackfan anemia market is anticipated to show positive growth in the coming years. This is because the increasing awareness of rare diseases and advancements in genetic research have spurred interest in Diamond-Blackfan anemia. This has led to a growing number of research initiatives and collaborations aimed at developing novel therapies, including gene therapies and small molecules, to address the underlying causes of Diamond-Blackfan anemia. Furthermore, regulatory incentives such as orphan drug designation and fast-track approval pathways have incentivized investment in the Diamond-Blackfan anemia market.

For a deeper understanding of the Diamond-Blackfan anemia market landscape, explore the Diamond-Blackfan Anemia Market Outlook

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SOURCE DelveInsight Business Research, LLP