FDA approves inebilizumab, now Uplizna, to treat adults with gMG
Infusion therapy already approved in US for 2 other immune disorders
- The U.S. Food and Drug Administration has approved inebilizumab, under the brand name Uplizna, to treat adults with generalized myasthenia gravis.
- The infusion therapy targets B-cells in gMG patients with AChR or MuSK antibodies.
- Patients receive infusions every six months, which may help lessen treatment burden.
The U.S. Food and Drug Administration (FDA) has approved inebilizumab as a treatment for generalized myasthenia gravis (gMG).
The newly approved therapy, which developer Amgen will market under the brand name Uplizna, is specifically indicated for adults with gMG who are positive for antibodies targeting the acetylcholine receptor (AChR) or muscle-specific kinase (MuSK). These are the two most common types of MG-causing antibodies.
In a company press release announcing the approval, Amgen stated that “Uplizna offers gMG patients deep and durable symptom control and twice-yearly dosing.” Patients first receive two loading doses of the infusion therapy, which is already approved in the U.S. as a treatment for two other immune system disorders.
“This approval marks a significant advancement for people living with gMG,” said Jay Bradner, MD, Amgen’s executive vice president of research and development. “Uplizna is conveniently dosed twice a year and delivers durable efficacy, helping people manage debilitating symptoms that can compromise daily function — including trouble breathing, speaking and seeing.”
The FDA’s approval was met with enthusiasm by the myasthenia gravis (MG) community, with one nonprofit advocacy organization calling it a decisive moment.
Treatment with Uplizna offers patients 6 months between doses
“Managing a rare and chronic illness can mean facing unpredictable relapsing symptoms and demanding treatment schedules,” said Samantha Masterson, president and CEO of the Myasthenia Gravis Foundation of America. “This approval marks an important milestone, offering durable efficacy and a dosing schedule that provides people living with generalized myasthenia gravis six months of treatment-free time between maintenance doses.”
An autoimmune disease, MG is driven by self-reactive antibodies that interfere with the communication between muscle and nerve cells, leading to symptoms such as muscle weakness and fatigue.
Uplizna is an antibody-based therapy designed to reduce disease activity by depleting or lowering the number of B-cells in the body. These are the immune cells that are mainly responsible for producing antibodies, including the self-reactive ones that drive MG. The therapy works by binding to CD19, a protein found on the surface of B-cells, and triggering an immune mechanism that leads to their death.
“By selectively targeting CD19-positive B cells, Uplizna offers a new approach to treatment that addresses a biological root cause of disease,” Bradner said.
The therapy is administered intravenously, or via infusions into the bloodstream. The first two infusions are given two weeks apart, and subsequent treatment is administered every six months. The treatment had previously been FDA-approved for other B-cell-driven diseases, including neuromyelitis optica spectrum disorder and IgG4-related disease.
Uplizna’s approval for gMG was based on data from a Phase 3 clinical trial called MINT (NCT04524273). The study enrolled 238 adults with gMG who were positive for antibodies targeting AChR or MuSK. Each participant was randomly assigned to receive either Uplizna or a placebo.
Top-line results from MINT showed that after 26 weeks, or approximately six months, Uplizna outperformed the placebo in its ability to reduce (improve) scores on the MG Activities of Daily Living (MG-ADL). That patient-rated assessment that evaluates the degree to which MG is causing issues in a person’s day-to-day life. Specifically, MG-ADL scores dropped by more than four points from the study’s start to week 26 in patients treated with Uplizna, while those on the placebo saw their scores decrease by about two points.
Uplizna offers a new approach to treatment that addresses a biological root cause of [gMG].
Additional exploratory analyses revealed that anti-AChR-positive patients continued to experience benefits after one year of therapy, with MG-ADL scores dropping by an average of 2.8 points compared with those receiving the placebo.
“Uplizna showed strong efficacy at 26 weeks in both [AChR-positive] and [MuSK-positive] patients, with [AChR-positive] patients continuing to improve through 52 weeks in MINT,” said Richard J. Nowak, MD, global principal investigator and director of the MG Clinic at Yale University in Connecticut.
According to Nowak, “this approval brings a new first-in-class approach to gMG, expanding treatment options for clinicians and patients.”
Headache, infusion-related reactions are most common side effects in gMG
According to its prescribing information, the most common side effects of Uplizna in gMG patients are headache and infusion-related reactions.
Amgen is offering a support program, called Amgen By Your Side, that can help individuals prescribed Uplizna to access educational and financial resources, as well as other patient support services.
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