2022-09-23| Trials & Approvals

AstraZeneca’s Rare Disease Treatment Wins Third Approval in European Union

by Max Heirich
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The European Commission (EC) approved AstraZeneca’s Ultomiris (ravulizumab). The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended approval for the generalized myasthenia gravis based on the Phase 3 CHAMPION-MG trial results. 

Related Article: BridgeBio and Sentynl Therapeutics’ Nulibry Gets Greenlighted In Europe

Generalized Myasthenia Gravis and its Treatment

Generalized myasthenia gravis (gMG) is a rare chronic autoimmune, neuromuscular disease. Caused by an error in nerve impulses’ transmission to muscles, communication between the neurons and muscles is interrupted. Antibodies in patients with gMG block, alter, or destroy the receptors in neuromuscular junctions, preventing muscle contracting. As a result, affected patients experience weakness in their limbs, difficulty breathing, and various drooping facial features.

There are an estimated 36,000 to 60,000 cases in the United States, with women more commonly affected than men. Though there is no cure for the progressive disease, medications, and even surgery help manage it. Some treatments like Bausch Health Companies’ Mestinon (pyridostigmine) stimulate the neuromuscular receptors, while treatments like Mylan’s Deltasone (prednisone) suppress the overactive immune system. 

Similarly, Ultomiris is an immune system suppressant. The treatment inhibits the C5 protein in the terminal complement cascade. When left unchecked, this part of the immune system over-responds, forcing the body to attack its own cells. 

Having already gained approval for the treatment of gMG patients in the United States and Japan, the results of the CHAMPION-MG trial won the EC’s as well. 

The CHAMPION-MG trial and Another Approval

The CHAMPION-MG trial is a Phase 3 clinical trial evaluating the safety and efficacy of Ultomiris in adults with gMG. The one hundred and seventy-five trial patients were diagnosed with gMG for at least six months prior with a positive serologic test for anti-AChR antibodies. 

The results, published in NEJM Evidence, demonstrated Ultomiris’ to the placebo. On the Myasthenia Gravis–Activities of Daily Living scale, Ultomiris scored a -3.1 compared to the placebo group’s −1.4. In addition, Ultomiris scored a −2.8 on the Quantitative Myasthenia Gravis total scores compared to the other’s -0.8.

The results, presented to the EMA, won their recommendation of approval to the EC, who accepted it. 

On the approval, Marc Dunoyer, Chief Executive Officer of Alexion (AstraZeneca’s rare disease group), said, “This approval in Europe of the first and only long-acting C5 inhibitor is an important step towards realizing our vision of improving the lives of people living with gMG and increasing access to Ultomiris worldwide.”

Outside of its approvals in three countries, other regulatory bodies will continue evaluating AstraZeneca’s rare disease treatment.

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