HQ Team
September 25, 2024: Amgen Inc.’s experimental drug to treat patients suffering from a rare muscle-weakening disease, myasthenia gravis, has shown positive results in an end-stage trial and the California-based company has started talks with regulators for its approval.
Inebilizumab or Uplizna was tested in 238 patients with the chronic, rare autoimmune immune disorder. Its symptoms include weakness in voluntary muscles, especially those that control the eyes, mouth, throat, and limbs.
Myasthenia gravis is an autoimmune disease, which means that the body’s defence system mistakenly attacks healthy cells or proteins needed for normal functioning
“Clinically meaningful and statistically significant MG-ADL score improvement” was achieved after 26 weeks with two doses of Uplinza compared to a placebo, said Jay Bradner, MD, Executive VP, Research & Development, Amgen, during a presentation to the investors.
MG-ADL score
The patients who received the drug showed an improvement of 4.2 points, or 1.9 when adjusted for placebo, on a commonly used scale for measuring disease impact on daily activities. The Myasthenia Gravis Activities of Daily Living (MG-ADL) score is a measure of the severity of myasthenia gravis symptoms.
The MG-ADL score ranges from 0 to 24, with higher scores indicating more severe symptoms.
The neuromuscular disorder is classified into two main types — AChR+ and MuSK+.
The trial “achieved clinically meaningful and statistically significant benefit in a change in MG-ADL score for both AcHR+ and MuSK+ cohorts,” Bradner said during the presentation, according to the slides posted on the company’s website. “Assessment of additional efficacy, durability of response and safety” are ongoing and no “new safety signals were identified.”
The investigational drug is effective in generalized myasthenia gravis with “patient-centred dosing and has the potential to reduce the use of steroids.”
New therapies
The US drug regulator, the Food and Drug Administration, had given its nod for Argenx SE’s Vyvgart Hytrulo as a treatment option for myasthenia gravis last year.
Researchers continue to gain a better understanding of myasthenia gravis, its causes, and the structure and function of the neuromuscular junction. Technological advances have led to more timely and accurate diagnosis of myasthenia gravis and new and enhanced therapies have improved treatment options.
Currently, researchers are working on developing better medications, identifying new ways to diagnose and treat individuals and improving treatment options, according to the National Institute of Neurological Disorders and Stroke.
