7 Sickle Cell Disease Therapies to Keep an Eye on Post-Pfizer’s OXBRYTA Withdrawal

The recent withdrawal of Pfizer’s Oxbryta (voxelotor) has significantly altered the landscape of sickle cell disease treatment, creating an opportunity for newer therapies to advance in the sickle cell disease drugs market. The field remains highly dynamic, with numerous ongoing clinical trials of sickle cell disease modifying agents as of October 2024, signaling robust innovation and expansion in treatment options.

Among the most anticipated developments is the Casgevy launch, following promising results from its clinical trials. Casgevy (exa-cel) employs CRISPR-based gene editing to correct hemoglobin mutations in hematopoietic stem cells, offering a potential disease-modifying therapy with long-lasting effects. Its approval and subsequent rollout represent a significant milestone in sickle cell care, although accessibility challenges and treatment logistics remain factors to monitor.

Etavopivat, another emerging therapy, is progressing through Phase III studies, including recruitment in London in 2024, with results expected in November 2024. This oral pyruvate kinase activator, supported by Novo Nordisk, targets red blood cell metabolism to reduce hemolysis, reflecting growing interest in metabolic-based approaches to sickle cell disease treatment.

Inclacumab, part of the Pfizer sickle cell pipeline, focuses on mitigating vaso-occlusive crises by blocking P-selectin, building on the mechanism established by Adakveo (crizanlizumab). Although some late-stage trials faced recruitment challenges, ongoing studies continue to evaluate its efficacy and potential market impact.

Adakveo, already approved, remains a key contributor to the sickle cell disease drugs market. Its long-standing use in preventing vaso-occlusive events, coupled with post-marketing data, maintains its relevance as newer therapies, such as Casgevy and mitapivat, enter the treatment landscape.

PociRedir, currently in Phase III development, is garnering attention for its novel mechanism of action. While full clinical results are pending, it has the potential to become a cornerstone in assessing new therapies within the sickle cell disease drugs market.

Mitapivat, initially approved for pyruvate kinase deficiency, is now being evaluated for sickle cell disease in the global RISE UP study, with patient recruitment completed in November 2024. This oral therapy aims to enhance red cell energy metabolism, reduce hemolysis, and improve hemoglobin levels, highlighting its potential impact in the expanding therapeutic landscape.

Beyond these highlighted treatments, the landscape continues to evolve with ongoing clinical trials of sickle cell disease modifying agents, including novel drugs, vaccines, and other innovative approaches. The broader field remains highly dynamic, with attention also on potential FDA-approved treatments for sickle cell disease in 2024 and emerging global advancements in patient care.

As the sickle cell disease drugs market continues to grow, biopharmaceutical companies are actively exploring new mechanisms, clinical indications, and strategies to address unmet needs, aiming to answer the central question for patients and clinicians alike: What therapies can meaningfully improve outcomes in sickle cell disease treatment today and in the years ahead?