7 Sickle Cell Disease Therapies to Watch Following Pfizer’s OXBRYTA Withdrawal
The removal of Pfizer’s Oxbryta (osivelotor) from the market has significantly reshaped the sickle cell disease treatment landscape, opening opportunities for novel therapies to gain ground. With multiple ongoing clinical trials of sickle cell disease‐modifying treatments underway as of October 2024, the pipeline remains both dynamic and expanding. Below, we highlight seven pivotal therapies poised to influence the sickle cell disease drugs market.
1. Casgevy (exa-cel)
The highly anticipated Casgevy launch builds on encouraging results from Casgevy clinical trials. Its mechanism—CRISPR gene editing to correct hemoglobin mutations—positions it as a powerful sickle cell disease treatment with disease-modifying potential.
2. Etavopivat
Now in Phase 3 as of November 2024, including a London-based trial, etavopivat is an oral pyruvate kinase activator targeting red blood cell metabolism to reduce hemolysis. Backed by Novo Nordisk, it exemplifies new momentum in the expanding sickle cell disease drugs market.
3. Inclacumab
A key component of the Pfizer sickle cell pipeline, Inclacumab targets vaso-occlusive crises by working on the P-selectin pathway—a mechanism established by Adakveo. It’s expected to deliver improved efficacy and broader market reach in that segment.
4. Adakveo (crizanlizumab)
Already approved and well-established in the sickle cell disease drugs market, Adakveo continues to hold a strong position thanks to ongoing research and post-marketing data that reinforce its relevance even as newer therapies emerge.
5. PociRedir
Currently undergoing Phase 3 evaluation, PociRedir’s novel mechanism could position this agent as a cornerstone in the developing sickle cell disease pipeline, potentially reshaping future treatment paradigms.
6. Mitapivat
Once primarily used for pyruvate kinase deficiency, Mitapivat is now being assessed for its role in sickle cell therapy. The “Rise Up Mitapivat” trial, recruiting as of November 2024, may clarify its utility as an innovative sickle cell disease treatment.
7. Emerging Gene and Small Molecule Therapies
Beyond the therapies already mentioned, ongoing clinical trials of sickle cell disease modifying therapies (as of October 2024) include novel drugs, potential vaccines, and next-gen molecular approaches. Developments also span to possible FDA-approved treatments for sickle cell disease or vaccines in 2024, reinforcing a vibrant and evolving sickle cell disease drugs market on a global scale.
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