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

 

The withdrawal of Pfizer’s Oxbryta (voxelotor) has reshaped the sickle cell disease treatment landscape, creating new opportunities for therapies in development to step forward. With multiple ongoing clinical trials of sickle cell disease modifying therapies active as of October 2024, the pipeline is broad, dynamic, and rapidly evolving. Seven notable therapies are emerging as key drivers in the sickle cell disease drugs market.

The much-anticipated Casgevy launch marks a historic milestone, following successful clinical trials demonstrating its promise. Casgevy, also known as exagamglogene autotemcel, is a CRISPR-based gene editing therapy designed to correct mutations in hemoglobin, offering the potential to fundamentally alter the course of sickle cell disease. Its approval has positioned it as the first gene-editing therapy available for this condition, and its role as a disease-modifying treatment could be transformative.

Etavopivat, an oral pyruvate kinase activator backed by Novo Nordisk, is steadily gaining attention. With a phase 3 trial in London and another expected to complete in November 2024, this candidate targets red blood cell metabolism to reduce hemolysis. If successful, it may serve as a convenient oral option within the expanding treatment landscape.

Inclacumab, a therapy within the Pfizer sickle cell pipeline, is aimed at reducing vaso-occlusive crises by blocking P-selectin, a mechanism similar to that of Adakveo but intended to offer improved efficacy and durability. By addressing the hallmark complication of vaso-occlusion, it may strengthen Pfizer’s presence in the market following the withdrawal of Oxbryta.

Adakveo, or crizanlizumab, is already approved and remains a significant player in the sickle cell disease drugs market. Supported by post-marketing studies and real-world evidence, it continues to be relevant, though its position may face challenges as newer, potentially more effective therapies come to the forefront.

PociRedir is currently undergoing phase 3 evaluation for sickle cell disease and introduces a novel mechanism of action. If trial outcomes prove successful, PociRedir could establish itself as a cornerstone option within the evolving pipeline of therapies and strengthen the breadth of available treatments.

Mitapivat, traditionally recognized in the management of pyruvate kinase deficiency, is expanding its development into sickle cell disease. The ongoing Rise Up Mitapivat phase 3 trial, actively recruiting through November 2024, will determine whether its role can be extended into this therapeutic area, potentially offering patients another oral treatment alternative.

Beyond these candidates, a number of gene-based and small molecule therapies are under investigation in ongoing clinical trials of sickle cell disease modifying treatments. These include innovative drug candidates, potential vaccines, and approaches aimed at long-term disease control. Growing interest also surrounds the possibility of FDA-approved treatments for sickle cell disease 2024 and even a potential FDA-approved vaccine for sickle cell disease 2024, signaling global momentum in sickle cell disease treatment innovation.

As the sickle cell disease drugs market continues to evolve, pharmaceutical companies and researchers are racing to address the critical question at the center of patient care: what new therapies can provide durable, effective, and accessible options for those living with sickle cell disease?

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