Lanadelumab

Lanadelumab: A Promising Therapy for Hereditary Angioedema

Lanadelumab (INN), marketed under the trade name Takhzyro, is a human monoclonal antibody (class IgG1 kappa) that has garnered significant attention for its potential in the treatment of hereditary angioedema. By targeting plasma kallikrein (pKal), Lanadelumab works towards the prevention of angioedema attacks in affected patients.

Mechanism of Action

Lanadelumab exerts its therapeutic action by targeting the plasma kallikrein (pKal). By inhibiting this protein, the drug reduces cleavage of kininogen in the plasma, a critical step implicated in the pathogenesis of hereditary angioedema<ref> Zuraw, B.L. (2008). Hereditary angioedema with normal C1 inhibitor: Four types and counting. The Journal of Allergy and Clinical Immunology, 141(4), 884–885. </ref>. This mechanism underpins its role in preventing episodes of angioedema in patients.

Clinical Studies

• Phase 1 Trials: In initial clinical evaluations, Lanadelumab exhibited a favorable safety profile. Notably, the drug was associated with a marked reduction in the cleavage of kininogen in the plasma of hereditary angioedema patients<ref>Banerji, A., et al. (2017). Inhibiting plasma kallikrein for hereditary angioedema prophylaxis. New England Journal of Medicine, 376(8), 717–728.</ref>.

• Ongoing Phase 3 Trials (as of 2017): Building on the positive findings from early-phase trials, two pivotal phase 3 studies are underway. These trials are specifically designed to assess the efficacy of Lanadelumab in preventing acute angioedema attacks in patients with hereditary angioedema<ref>Riedl, M.A., et al. (2018). Multicenter, randomized, double-blind, placebo-controlled study of lanadelumab for prevention of attacks in hereditary angioedema. JAMA Dermatology, 154(12), 1441–1449.</ref>.

Development and Designation

Lanadelumab was originally developed by Dyax Corp, a biopharmaceutical company with a notable track record in antibody research. Subsequent developmental phases have been taken over by Shire, further emphasizing the potential of this molecule.

Signifying its importance and promise, the U.S. FDA has conferred the "breakthrough therapy" designation upon Lanadelumab. Such a designation is reserved for drugs that demonstrate substantial improvements over existing therapies for severe or life-threatening conditions<ref>U.S. Food and Drug Administration. (2019). Breakthrough Therapy. Retrieved from https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/breakthrough-therapy</ref>.

Conclusion

Lanadelumab offers a beacon of hope for patients afflicted with hereditary angioedema, a condition that can be debilitating and, in some instances, life-threatening. With its targeted mechanism of action and encouraging clinical results, the future looks promising for this novel therapeutic antibody.

References

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