New research conducted by Spherix Global Insights highlights opportunities for genetic therapies (Casgevy, Lyfgenia, Zynteglo) and emerging pipeline agents (including mitapivat, etavopivat, inclacumab and others).
Exton, PA – August 28, 2024 – Despite advancements in treatments, sickle cell disease and thalassemia continue to present significant challenges for patients and healthcare providers. These genetic disorders disrupt normal hemoglobin function, leading to severe health issues. Sickle cell disease causes red blood cells to become rigid and sickle-shaped, resulting in frequent pain and anemia, while thalassemia causes abnormal hemoglobin due to deficient globin chains.1 Current treatments, such as Oxbryta (Pfizer) Adakveo (Novartis), and Endari (Emmaus) for sickle cell disease, and Reblozyl (Bristol-Myers Squibb) for thalassemia, offer some relief but are not curative, leaving patients with ongoing difficulties and reduced life expectancy.
The landscape began to shift with the approval of the first gene therapy for transfusion-dependent beta thalassemia, Zynteglo, by Bluebird Bio in August 2022. This was followed by the approvals of Lyfgenia (Bluebird Bio) and Casgevy (Vertex), for sickle cell disease and transfusion-dependent beta thalassemia. Despite the enthusiasm for these therapies, their uptake has been hampered by logistical challenges, high costs, and complex coverage issues.
In light of these developments, Spherix Global Insights conducted research with 53 US hematologists, including eight follow-up in-depth interviews, as well as interviews with key opinion leaders (KOLs) to gauge their perspectives on current and emerging treatments. Market Dynamix™: Sickle Cell Disease and Thalassemia, released earlier this week to subscribed clients, reveals a strong consensus among hematologists on the pressing need for more effective treatments.
One expert highlighted the severity of the unmet need, stating, “The burden of sickle cell disease, both in the U.S. and globally, underscores the critical need for more effective treatments. The disease’s progressive nature makes this a significant challenge.” Another emphasized the urgency for thalassemia treatments, noting, “Without curative therapies, patients with thalassemia face a reduced life expectancy.”
Gene therapy offers a promising path to potentially cure these diseases, with hematologists estimating that up to one-third of their patients could be candidates for such treatments, with a slightly higher likelihood among thalassemia patients. While many believe in the potential of gene therapy, they also express concerns about long-term side effects, duration of response, and cost, which could limit broader accessibility.
Looking ahead, new therapies are on the horizon that could further transform patient care. A leading KOL remarked, “While gene therapy represents a significant advancement, there is still a need for non-curative options that can improve patients’ quality of life and alter the disease’s trajectory. Balancing curative and non-curative therapies will be essential.”
Physicians anticipate a strong future role for emerging agents like mitapivat (Agios), etavopivat (Novo Nordisk), inclacumab (Pfizer), and osivelotor (Pfizer), among others, and are prepared to adopt these therapies swiftly once approved.
Spherix Global Insights remains committed to tracking these evolving developments and providing valuable insights to guide healthcare providers in their decision-making processes.
Market Dynamix™ is an independent service providing analysis of markets anticipated to experience a paradigm shift within the next three to five years. Insights highlight market size, current treatment approaches, unmet needs, and expert opinions on the likely disruption introduced by pipeline agents.
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Spherix Global Insights Contacts
Tucker Hurtado, Hematology Franchise Launch Head
Tucker.Hurtado@spherixglobalinsights.com
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Source: 1 Hemoglobinopathy disorders. Memorial Sloan Kettering Cancer Center. (n.d.). https://www.mskcc.org/pediatrics/cancer-care/types/pediatric-blood-disorders/about-pediatric-blood-disorders/hemoglobinopathies
