In this episode, we discuss gambling on clinical trials, the FDA approval of a new formulation of Sjögren’s disease treatment, and an approval in IgA nephropathy.
FDA Approves a New Liquid Formulation for Sjögren’s Disease
The FDA has approved a new oral solution of Evoxac, or cevimeline, from Cosette Pharmaceuticals, to treat dry mouth in adults with Sjögren’s disease. Until now, the drug was only available as a capsule.
Sjögren’s disease is an autoimmune disorder in which the immune system attacks secretory glands. The two hallmark symptoms are dry mouth and dry eyes, but patients can also have dryness of the skin, nose, and vagina. And it isn’t just about dryness.
Sjögren’s can affect other organs, including the kidneys, lungs, liver, pancreas, blood vessels, and even the brain. It also often occurs alongside other rheumatic diseases, such as rheumatoid arthritis or lupus. About 90 percent of patients are women.
Evoxac works by stimulating muscarinic receptors that in turn activate glands to produce saliva. One of the problems with dry mouth is that it makes it harder to swallow pills. So a liquid version makes a lot of sense for this patient population.
Cevimeline capsules were first approved in 2000 for Daiichi Sankyo. For the new liquid version, The manufacturer (Cosette) was able to rely on the existing safety and efficacy data from that earlier review of the capsule. So instead of repeating large clinical trials, the company mainly had to show that the new formulation behaves the way it should in the body.
Ultomiris Approved for IgA Nephropathy
The FDA has granted accelerated approval to Ultomiris, or ravulizumab, from AstraZeneca’s rare disease unit Alexion, to reduce proteinuria in adults with primary IgA nephropathy who are at risk of disease progression. It’s the first C5 complement inhibitor approved for this condition.
IgA nephropathy is a rare, inflammatory disease of the kidneys that results in accumulations of IgA clusters that block the kidney. Over time, that can lead to chronic kidney disease and eventually kidney failure.
Ultomiris blocks complement protein C5, which sits near the end of the complement cascade. In the Phase 3 I CAN trial, patients on Ultomiris had a 46.6 percent reduction in protein in the urine at week 34, compared with 5.6 percent on placebo.
The FDA granting accelerated approval for the drug based on the data but full approval will require the trial to continue and assess kidney function overall, measured by eGFR, over a 106 week period.
Ultomiris now has approvals across several conditions, including blood, kidney, and neurological diseases, all through inhibition of the complement protein C5.
That’s an important strategy in rare disease drug development. When the same biological pathway contributes to multiple conditions, a successful drug may have opportunities to expand into additional indications. Each expansion requires evidence that the treatment is appropriate for that particular disease, but the potential opportunity is much larger than a single indication.
In IgA nephropathy, Ultomiris will compete with treatments that use different mechanisms. The key questions are how well these therapies protect kidney function over time, how safely they do so, and how they compare in practice.
Placing Bets on Clinical Trials
Our final story looks at prediction markets, where people can bet on clinical trial outcomes and FDA approvals. The issue has sparked debate in both the New England Journal of Medicine and Science.
A clinical trial depends on a few basic principles. One is that nobody knows if the drug will work, or nobody knows who is getting which drug. That is why they’re double-blind and randomized. Focusing on blinding, these studies are designed to ensure patients’ and investigators’ expectations don’t influence the results. However, adding odds makers can undermine that. If patients go on a betting site and see that a trial has a 20% chance of success, they may decide not to enroll or drop out. If the odds are high, they may report feeling better than they really do.
These betting sites can also impact the sponsors of these clinical trials. For a large pharmaceutical company, one unfavorable signal may not have an impact but for a small biotech with one or two clinical programs, a single trial can determine the company’s future.
If a betting site signals that a trial is likely to fail, it could affect investor confidence, fundraising, and patient recruitment.
Prediction markets may offer a new way to assess clinical risk, but transparency alone isn’t enough. We need safeguards that protect patients, trial integrity, and smaller companies’ ability to develop treatments.
For rare disease biotech companies, the stakes are bigger than the bets.
References
FDA Approves New Formulation of Sjögren’s Disease Treatment
https://checkrare.com/fda-approves-new-formulation-of-sjorgens-disease-treatment/
Ultomiris Approved in the US in IgAN
https://www.astrazeneca.com/media-centre/press-releases/2026/ultomiris-approved-in-us-in-igan.html
FDA Grants Accelerated Approval to Ravulizumab for Proteinuria Reduction in IgA Nephropathy (Pharmacy Times)
Clinical Trial Prediction Markets — Information, at What Cost? N Engl J Med. 2026
https://www.nejm.org/doi/full/10.1056/NEJMp2610547
Ban Bets on Unfinished Clinical Trials. Science. 2026
https://www.science.org/doi/10.1126/science.ael8642
Betting on Biotech: Prediction Markets Set Sights on Clinical Trials (Fierce Biotech)
https://www.fiercebiotech.com/biotech/betting-biotech-prediction-markets-set-sights-clinical-trials