SICKLE CELL DISEASE TREATMENT MARKET SIZE AND SHARE ANALYSIS - GROWTH TRENDS AND FORECASTS (2025 - 2032)

Market Concentration and Competitive Landscape

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Key Developments:

• In September 2024, Pfizer Inc., a pharmaceutical company, announced that it is voluntarily withdrawing all lots of OXBRYTA (voxelotor) for sickle cell disease treatment from worldwide markets and discontinuing all related clinical trials and expanded access programs. This decision follows clinical data suggesting an imbalance in vaso-occlusive crises and fatal events, which requires further assessment. Pfizer Inc. emphasized that patient safety is its priority and advised patients to consult with their physicians for alternative treatments while the company continues its review.
• In December 2023, Vertex Pharmaceuticals, a biotechnology company, and CRISPR Therapeutics, a gene editing company, announced that the U.S. Food and Drug Administration (FDA) approved CASGEVY (exa-cel), a CRISPR/Cas9 genome-edited cell therapy for treating sickle cell disease (SCD) in patients aged 12 and older with recurrent vaso-occlusive crises (VOCs). This approval offers approximately 16,000 patients the potential for a one-time transformative therapy that could eliminate severe VOCs and hospitalizations. CASGEVY is the first CRISPR-based gene-editing therapy approved in the U.S. and marks a significant milestone in SCD treatment.
• In February 2022, Global Blood Therapeutics Inc., a biopharmaceutical company, received marketing authorization for Oxbryta, a treatment for hemolytic anemia due to sickle cell disease (SCD) in adults and pediatric patients aged 12 years and older, either as a monotherapy or in combination with hydroxycarbamide

Top Strategies Followed by Global Sickle Cell Disease Treatment Market Players

• Established Players - Leading pharmaceutical companies are heavily investing in research and development to innovate high-performance treatments for sickle cell disease (SCD)
  • For instance, in December 2022, bluebird bio, Inc., a biotechnology company, received the U.S. FDA approval for Zynteglo, a gene therapy for beta-thalassemia, indicating significant advancements in related hemoglobinopathies
• Mid-Level Players - Mid-sized companies are focusing on delivering quality, budget-friendly treatments targeting price-sensitive consumers. Emmaus Medical, Inc. offers Endari, an U.S. FDA-approved treatment for SCD, providing a more affordable option for patients. Partnerships aimed at boosting technology, production capabilities, and market presence are common.
  • For instance, in April 2023, Editas Medicine, Inc. received Orphan Drug Designation from the U.S. FDA for its EDIT-301, a gene-editing therapy for sickle cell disease, highlighting collaborative efforts in advancing treatment options
• Small-Scale Players - Small players are targeting niche areas or specific gene mutations to carve out a unique identity. They are focusing research on personalized therapies tailored to the needs of certain patient subsets. This helps them attract funding and partnerships while remaining competitive against larger companies.

Emerging Startups - Sickle Cell Disease Treatment Industry Ecosystem

• Innovative Technologies - Startups are employing technologies like nanotechnology, 3D bioprinting, and gene therapy to develop transformative treatment approaches. Companies like BioNano Genomics are using optical genome mapping to improve diagnostics. SickleTouch is developing a smartphone-based diagnostic tool using computer vision.
• Sustainable Solutions - Some startups are introducing eco-friendly approaches. Biolyfe is producing biodegradable pills from algae to reduce plastic waste. NuGenerex is focusing on regenerative medicine using adult stem cells to provide an alternative to blood transfusions.
• Market Contribution - Startups like Fulcrum Therapeutics are addressing unique market needs by focusing on specific pathways involved in SCD, offering targeted therapeutic options. These startups often engage in partnerships with established companies or academic institutions to drive technological and sustainable innovation, enhancing their market presence and contributing to the overall advancement of SCD treatments.