Complement C5 Inhibitors Market: Competitive Landscape, Pipeline, and Market Analysis 2023

Complement component 5 is a part of the complement system in humans and is coded by the C5 gene. It is the initiator of the effector terminal phase of the complement system. It consists of 2 units- Alpha and Beta, which are linked by disulfide bonds. C5 convertase enzyme cleaves it into C5a and C5b. C5a is a potent inflammatory mediator with many functions like vasodilation, inducing contraction of smooth muscles, and increasing the permeability of small blood vessels. It can also stimulate histamine release from basophils leading to allergic and inflammatory responses. Therefore, the complement system is essential in controlling homeostasis and inflammation and providing defense from pathogens. Activation of C5 leads to inflammation and cell damage. Complement C5 Inhibitors are being tried for many diseases, but the primary use for which it is approved by the USFDA is in the treatment of Paroxysmal Nocturnal Haemoglobinuria (PNH). PNH is a rare and life-threatening disease of blood in which destruction of RBC takes place, blood clots form, and the function of bone marrow is impaired. Companies are trying to find new and novel molecules to prevent and cure these diseases. The development and launch of newer products by the market players will significantly enhance the revenue of the Complement C5 Inhibitors market over the forecast years. For instance, according to the National Organization for Rare Disorders, in 2023, the USA has a prevalence of PNH of about 0.5 – 1.5 per million people. 

Several of these inhibitors are on the market, while many are in ongoing clinical trials. 23 companies have various products in their pipelines, with 20 products in various stages of clinical trials. 2 products are in Phase 4, 11 products are in Phase 3, 4 products are in Phase 2, and 3 products are in Phase 1 of the clinical trials.

Key Developments of Complement C5 Inhibitors

• In June 2023, Soliris (eculizumab) by AstraZeneca was approved in China for the treatment of adults with Refractory Generalized Myasthenia Gravis.
• In August 2022, China accepted the application of Crovalimab and granted priority review for the PNH indication.
• In April 2022, Ultomiris (ravulizumab-cwvz) by Alexion Pharmaceuticals, Inc was approved by the FDA for the treatment of adults with Generalized Myasthenia Gravis.
• In June 2021, Ultomiris (ravulizumab-cwvz) by Alexion Pharmaceuticals, Inc was approved by the FDA for the treatment of children and adolescents with PNH
• In July 2021, Crovalimab was given the breakthrough Therapy designation in China for the disease PNH.
• In October 2019, Ultomiris (ravulizumab-cwvz) by Alexion Pharmaceuticals, Inc was approved by the FDA for the treatment of adult patients with Atypical Hemolytic Uremic Syndrome (Ahus).
• In December 2018, Ultomiris (ravulizumab-cwvz) by Alexion Pharmaceuticals, Inc was approved by the FDA for the treatment of adult patients with PNH.

Approved molecules

• Soliris (eculizumab)
• Ultomiris IV (ravulizumab-cwvz IV)

Drugs in Pipeline

• Gohibic (vilobelimab)
• pozelimab (REGN3918)
• zilucoplan (RA101495)
• cemdisiran (ALN-CC5)
• Coversin SC (nomacopan SC)
• Zimura (avacincaptad pegol)
• crovalimab (RG6107)
• tesidolumab (LFG316)
• Elizaria (eculizumab biosimilar)
• gefurulimab (ALXN1720)
• BCD-148 (eculizumab biosimilar)
• Bekemv (eculizumab biosimilar)
• CAN106
• SB12 (eculizumab biosimilar)
• Coversin topical (nomacopan topical)
• ISU 305 (eculizumab biosimilar)
• SOBI002
• ravulizumab/hyaluronidase (ALXN1810 SC)

Clinical Activity and Developments of Complement C5 Inhibitors

As of June 2023, 23 companies have approximately 20 products for 89 diseases. For these diseases, about 75 trials are being conducted by players globally. For instance,

• In February 2023, Alexion Pharmaceuticals, Inc. completed a Phase 3 clinical trial to have comparative knowledge of ALXN1210 (Ravulizumab) vs. Eculizumab in complement inhibitor treatment in adult participants with PNH.
• In March 2023, Novartis Pharmaceuticals completed a Phase 3 clinical trial to study the efficacy and safety of twice-daily oral LNP023 in adult PNH patients with Residual Anemia despite Anti-C5 Antibody treatment.
• Hoffmann-La Roche is conducting a Phase 3 clinical study to evaluate the safety, efficacy, and pharmacokinetics of Crovalimab vs Eculizumab in patients with PNH currently treated with Complement C5 Inhibitors.