Ultomiris (ravulizumab), developed by Alexion Pharmaceuticals, has expanded the spectrum of indications by adding children (age 1 month and up) and adolescents to the treatment of paroxysmal nocturnal hemoglobinuria.

The regulatory approval, issued by the U.S. Food and Drug Administration (FDA) in June 2021, drew on interim results from the phase 3 clinical trial NCT03406507 (non-randomized, open-label, multicenter, international) that evaluated the safety and efficacy of ravulizumab among pediatric patients (n=13) with paroxysmal nocturnal hemoglobinuria who were previously untreated or receiving Soliris (eculizumab).

After 26 weeks of therapy, administration of Ultomiris relieved 60% and 100% of patients who had not previously been treated and had previously received Soliris, respectively, from dependence on transfusions. Among subjects in the first category, Ultomiris reduced lactate dehydrogenase (LDH) levels, which indicate intravascular hemolysis, and relieved symptoms of fatigue in a clinically significant way. Stabilization of hemoglobin levels was fair for 60% and 75% of patients. In any case, further evaluation of the therapeutic efficacy of ravulizumab in the pediatric population needs more long-term follow-up of patients.

Ravulizumab is a humanized monoclonal antibody that specifically and with high affinity binds to the component 5 (C5) of the complement system and inhibits its cleavage to C5a and C5b fragments, which prevents formation of the membrane attack complex (MAC), thereby inhibiting pathological immune attacks.

Ultomiris, which debuted in December 2018 for therapy of paroxysmal nocturnal hemoglobinuria (PNH) in adults and then, in October 2019, connected children and adults with atypical hemolytic uremic syndrome (aHUS), is an improved version of eculizumab. Whereas maintenance therapy with Soliris requires intravenous infusions every two weeks, Ulltomiris requires infusions every eight weeks. The clinical efficacy of both drugs is comparable.

In December 2020, AstraZeneca set out to acquire Alexion for $39.4 billion. The takeover should strengthen AstraZeneca’s position in the orphan diseases sector. The deal is expected to close in the third quarter of 2021.

Alexion Pharmaceuticals.

AstraZeneca + Alexion = Rescue of Both Companies

Alexion Pharmaceuticals, which owns Soliris, one of the most expensive drugs on Earth, has agreed to sell AstraZeneca for $39 billion.

In May 2021, the FDA approved Empaveli (pegcetacoplan), a new drug indicated to treat paroxysmal nocturnal hemoglobinuria in adults. The therapeutic efficacy of Empaveli is by all accounts superior to that of Soliris. Pegcetacoplan (APL-2), backed by Apellis Pharmaceuticals, is a synthetic cyclic peptide, conjugated to polyethylene glycol, that binds to complement component 3 (C3) and its C3b fragment. C3 inhibition is thought to block all pathways of complement activation: the classical, lectin, and alternative pathways. In addition, it is blocking C3 that suppresses all three principle immune responses: opsonization, inflammation, and MAC formation.


Empaveli: Powerful New Drug for Paroxysmal Nocturnal Hemoglobinuria

Pegcetacoplan is more effective in treating paroxysmal nocturnal hemoglobinuria compared to Soliris.



Ultomiris (ravulizumab). Prescribing information. U.S. [PDF]

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