Beyond Gene Therapy: Pyruvate Kinase Activators (Agios, Novo Nordisk) Offer a New Route to Address Hemoglobin, VOCs, and Transfusion Burden in Sickle Cell Disease
Physician perspectives point to strong interest in metabolic modulation as a novel pathway in sickle cell disease, particularly for patients who cannot currently access gene therapy for clinical or logistical reasons, according to Spherix Global Insights.
EXTON, PA, Aug. 06, 2025 (GLOBE NEWSWIRE) -- According to the latest findings from Spherix Global Insights’ syndicated Market Dynamix™ service, the US sickle cell disease (SCD) market is approaching a pivotal juncture, with gene therapies and novel oral agents, including pyruvate kinase (PK) activators, garnering heightened enthusiasm among treating hematologists. While the promise of innovation is substantial, real-world barriers in access, adherence, and patient support continue to temper expectations.
SCD is marked by chronic hemolytic anemia and recurrent vaso-occlusive crises (VOCs), driven in part by the reduced lifespan and impaired function of red blood cells. By targeting red blood cell energy metabolism, PK activators such as mitapivat (Agios) and etavopivat (Novo Nordisk) aim to enhance ATP production and reduce 2,3-DPG levels, thereby improving red blood cell survival, oxygen affinity, and resilience against sickling. This metabolic modulation, physicians note, offers a fundamentally different – and potentially complementary – pathway to existing disease-modifying agents.
Surveyed hematologists see these agents as particularly promising for their ability to address multiple clinical challenges simultaneously: raising hemoglobin, reducing transfusion dependence, and decreasing VOC frequency. On average, physicians report that roughly 40% of their SCD patients could be appropriate candidates for PK activator therapy, with potential utility in both transfusion-dependent and non-transfusion-dependent populations. Oral administration, favorable safety profiles, and the opportunity for earlier intervention further enhance their appeal.
Still, while enthusiasm runs high, physicians stress the importance of long-term efficacy and safety data, particularly regarding sustained hemoglobin improvement and meaningful VOC reduction. Many anticipate that, if approved, PK activators will be incorporated into combination approaches or used to bridge patients to curative options such as gene therapy.
As the treatment paradigm evolves, hematologists continue to seek therapies that can be integrated into real-world care with minimal burden, offering both efficacy and convenience. The growing interest in PK activation reflects a broader shift toward diversifying mechanisms of action to better address the multifaceted nature of SCD.
Market Dynamix™ is an independent, data-driven service focused on understanding the evolving dynamics of specialty markets poised for disruption. Leveraging quantitative and qualitative research, the service evaluates current treatment approaches, unmet needs, and likely impact of pipeline agents over a three-to-five-year horizon.
About Spherix Global Insights
Spherix is a leading independent market intelligence and advisory firm that delivers commercial value to the global life sciences industry, across the brand lifecycle.
The seasoned team of Spherix experts provides an unbiased and holistic view of the landscape within rapidly evolving specialty markets, including dermatology, gastroenterology, rheumatology, nephrology, neurology, ophthalmology, and hematology. Spherix clients stay ahead of the curve with the perspective of the extensive Spherix Physician Community.
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NOTICE: All company, brand or product names in this press release are trademarks of their respective holders. The findings and opinions expressed within are based on Spherix Global Insight’s analysis and do not imply a relationship with or endorsement of the companies or brands mentioned in this press release.
Sarah Hendry, Hematology Franchise Head Spherix Global Insights 4848794284 sarah.hendry@spherixglobalinsights.com
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