On April 3, 2025, the FDA approved Uplizna® (inebilizumab-cdon) as the first and only approved treatment for Immunoglobulin G4-related disease (IgG4-RD), the condition behind Type 1 Autoimmune Pancreatitis (AIP).
This is a huge milestone. It’s the first time a treatment has been officially approved for this rare, immune-related cause of pancreatitis. For patients living with Type 1 AIP, this brings new hope—and for the broader pancreatitis community, it sets an important precedent for research and drug development.
What is IgG4-RD and Type 1 Autoimmune Pancreatitis?
IgG4-Related Disease (IgG4RD) is a chronic condition where the immune system attacks healthy tissue, causing inflammation and fibrosis (scarring) in multiple organs. When this immune activity targets the pancreas, it’s called Type 1 Autoimmune Pancreatitis.
While it can often be treated with steroids, long-term use of steroids carries serious side effects. Until now, there’s been no FDA-approved therapy that directly targets the disease itself.
What Makes This Approval So Important?
Uplizna was studied in a large clinical trial, the first randomized, double-blind, placebo-controlled trial ever conducted for IgG4-RD. And the results were positive:
- 87% reduction in the risk of flares
- More than half of patients achieved complete remission without needing steroids
- A ten-fold drop in the amount of steroids needed to control the disease
If you have autoimmune pancreatitis, you should talk to your doctor about whether this medicine is right for you.
But what makes this approval meaningful for people who don’t have autoimmune pancreatitis is how the researchers measured success in the trial.
What’s an Outcome in a Clinical Trial
In every clinical trial, researchers need to choose how they’ll measure whether a treatment works. These measurements are called outcomes or endpoints.
Outcomes help answer questions like: Did the treatment reduce symptoms? Did it improve quality of life? Was it safe to take?
Each trial has a primary outcome, which is the main thing researchers are trying to measure. There are also secondary outcomes, which give extra information to help understand the treatment’s full impact.
The Power of “Time to First Flare”
In the IgG4-RD clinical trial, the primary outcome was:
Time to first disease flare: how long a patient could go after receiving treatment before having a flare-up that needs care.
This is a clear, objective way to measure whether a treatment works. It’s especially helpful in conditions like Type 1 AIP, where symptoms can vary a lot between patients.
The study also looked at several secondary outcomes, including remission rates, flare frequency, and steroid use. These showed that the new therapy not only delayed flares but helped many patients stay flare-free without needing long-term steroids.
Why This Matters for All Types of Pancreatitis
Currently, researchers still don’t agree on the best way to measure success in clinical trials for chronic or recurrent pancreatitis. This slows down progress and makes it harder to get new treatments approved.
The Uplizna trial shows that using time to flare as an outcome is not only possible, it can lead to FDA approval. That sets a strong example for future studies in chronic pancreatitis and recurrent acute pancreatitis.
It’s a sign that clinical trials measuring things that are important to patients are feasible. This will help bring targeted therapies to more people, faster.
What This Means for Patients
If you’ve been diagnosed with Type 1 Autoimmune Pancreatitis, this new approval gives you and your doctor a reason to revisit your treatment options.
If you live with another type of pancreatitis, this breakthrough is still a win, it’s proof that progress is possible and that clinical research is moving in the right direction.
Learn More
Want to learn more about Type 1 AIP and how it fits into the bigger picture of autoimmune pancreatitis
About Mission: Cure
At Mission: Cure, we are dedicated to finding effective treatments and, ultimately, a cure for pancreatitis. Through patient education, research, and advocacy, we strive to improve the lives of those affected by this condition.
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