New and Emerging Therapies for Congenital Adrenal Hyperplasia (CAH)
Health ConditionsHormones & Metabolic Disorders

The Future of Congenital Adrenal Hyperplasia (CAH) Treatment: New and Emerging Therapies and Technologies

The Future of Congenital Adrenal Hyperplasia (CAH) Treatment: New and Emerging Therapies and Technologies
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Published on March 16, 2026
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Emerging treatments for classic congenital adrenal hyperplasia (CAH) may make it easier to manage the condition, which occurs when you don’t have enough of the enzyme you need to make the hormones cortisol and aldosterone, creating a hormonal imbalance.

Typically, CAH treatment includes glucocorticoids to replace that cortisol. But long-term steroid use can cause side effects that range from weight gain and high blood pressure to eye problems and reduced bone density.

 Also, getting the timing and dosage of glucocorticoids just right can feel like a full-time job.

Treatments and therapies in the pipeline attempt to alleviate these issues by reducing the medication dosage or improving its accuracy. And some researchers are even experimenting with gene-editing techniques that could fix hormone production, potentially representing a cure for CAH.

Steroid-Sparing Medications

New and emerging medications to treat CAH attempt to rebalance your hormones while reducing the amount of glucosteroids you take. The U.S. Food and Drug Administration (FDA) has approved one medication for use, while testing is underway on another.


Crinecerfont

The FDA approved crinecerfont (Crenessity) in 2024 to supplement glucocorticoid treatment in people with CAH who are age 4 and up. It is a CRF1 receptor antagonist that reduces the amount of the adrenocorticotropic hormone (ACTH) your pituitary gland makes.

A twice-daily pill, crinecerfont can help you gradually reduce glucocorticoids with your doctor’s help. The medication can help lower your need for glucocorticoids by as much as 27 percent.

“You want to take your time backing down over a matter of months, since lowering your dose too quickly can lead to withdrawal symptoms,” says Richard Auchus, MD, PhD, a professor of internal medicine in the division of metabolism, endocrinology, and diabetes at University of Michigan Health in Ann Arbor.

Atumelnant

Clinical trials are underway to test another once-daily medication called atumelnant. This experimental pill could also reduce your need for glucocorticoids.

Unlike crinecerfont, which works at the pituitary level, atumelnant blocks ACTH after the hormone is produced.

Phase 3 clinical trials are expected to wrap up by May 2027.

 The FDA has granted it an orphan drug designation for treating CAH, with potential full FDA approval to follow.

 “It looks very promising,” says Dr. Auchus.

Improved Steroid Delivery

Other new CAH treatments attempt to address the challenges of steroid timing, which can be tough to get right.

Cortisol levels normally fluctuate throughout the day.

 But traditional oral steroids don’t always align to those changes, producing cortisol peaks that are too high and leaving levels too low before your next dose, says Sasan Mirfakhraee, MD, an associate professor in the division of endocrinology at UT Southwestern Medical Center in Dallas.

“They don’t replicate the body’s natural rhythm,” he says.

Modified-Release Steroids

Only available in Europe, oral medications Efmody and Plenadren are modified-release forms of hydrocortisone. Compared with regular steroids, the body takes longer to break them down and to clear them from your system. This can allow for one or two daily doses, with a more gradual rise and drop in cortisol levels that more closely mimics what your body would do naturally.

There are no known plans for the FDA to approve either medication for use in the United States, Auchus says.

Steroid Pump Therapy

Continuous subcutaneous hydrocortisone infusion (CSHI), or steroid pumps, are another therapy that can mimic the ebbs and flows of cortisol. The FDA has not approved these devices, but doctors may prescribe them off-label.

These devices work like an insulin pump, delivering liquid hydrocortisone through a small tube that goes under your skin.

“The pump can be programmed to more closely match the body's natural rhythm, which generally allows for a lower total daily dose of steroid and improved symptoms such as energy and mood,” Dr. Mirfakhraee says.

CSHI can improve well-being and quality of life in people with CAH.

 But it comes with a major risk: If a steroid pump fails without you noticing, it can interrupt how hydrocortisone is administered, which could lead to life-threatening complications, such as adrenal crisis.

Mirfakhraee says that more research is needed to determine whether the benefits of CSHI outweigh its risks, compared with other treatment types.

Easier Hormone Sampling

Blood tests are the go-to for tracking your hormones and seeing whether you need an adjustment to your steroid dosage to treat CAH. But people with CAH generally only take a new blood test every once in a while: Your doctor may order lab work every few months, depending on your age and other factors.

 Home-monitoring options could make it easier to keep track of your levels and help your care team determine if treatment changes are necessary.

Cortisol Sensors

Researchers are looking at small wearable devices that can sample your cortisol levels over the course of a day as you go about your normal activities.

 Devices such as the U-RHYTHM cortisol sensor measure your hormones by collecting tiny amounts of interstitial fluid, found in the spaces surrounding your body’s cells, from your abdomen every 20 minutes. It then sends the data to your doctor, though not in real time.

“If your control isn’t at goal, it could help figure out what’s going wrong,” Auchus says.

Wearable cortisol sensors in development can give patients immediate feedback on their hormone levels.

 Auchus says that these could help people with CAH see how their cortisol level changes when they take steroids with food, without food, or at different dosages.

Home Hormone Sampling

In addition to measuring cortisol in blood, doctors can measure it in your sweat, saliva, and hair.

 In fact, endocrinologists in countries such as the Netherlands already routinely use saliva sample kits instead of blood draws to monitor hormone levels in people with CAH, Auchus says. “It’s not a CAH treatment in itself,” he says, “but it’s a way to monitor and make adjustments to a patient’s treatment as needed.”
Researchers and doctors are looking at ways to allow you to complete these tests at home, such as by sampling saliva throughout the day.

The technology for hormone sampling already exists, Auchus says, but there isn’t yet a system in place to make the tests available at home for people with CAH.

Gene Therapy

Researchers are looking at ways to repair the defective gene that produces the enzymes that make cortisol and aldosterone. This gene therapy would allow people with CAH to produce their own hormones without medication, effectively curing the condition, says Lara Graves, MBBS, PhD, a pediatric endocrinologist who studies gene therapy for CAH at the Children’s Hospital at Westmead in Australia.

This research is a work in progress, however, with studies looking at multiple types of gene therapy in mice.

 The treatment will need years of human studies and trials before approval.

“I don’t think any of the current therapies in preclinical development are good enough yet,” Dr. Graves says. “But the technological space is rapidly evolving, so it may be sooner than we think.”

The Takeaway

  • Steroid-sparing treatments, including experimental medications and the newly approved crinecerfont, may allow people with CAH to use smaller doses of steroids, reducing the impact of steroid side effects..
  • Changes to corticosteroid delivery, from timed release to steroid pumps, could align steroid treatment to when you need it most. Ask your doctor about these, as the FDA has not approved numerous treatments.
  • Wearable cortisol sensors and home hormone-sampling kits could look at your hormone levels closer to real time to help your care team determine the correct dosage and times.
  • In theory, gene therapy could essentially cure CAH, but experiments with the treatment are in early stages.

Resources We Trust

EDITORIAL SOURCES
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Resources
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Anna L. Goldman, MD

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Anna L. Goldman, MD, is a board-certified endocrinologist. She teaches first year medical students at Harvard Medical School and practices general endocrinology in Boston.

Dr. Goldman attended college at Wesleyan University and then completed her residency at Icahn School of Medicine at Mount Sinai Hospital in New York City, where she was also a chief resident. She moved to Boston to do her fellowship in endocrinology at Brigham and Women's Hospital. She joined the faculty after graduation and served as the associate program director for the fellowship program for a number of years.

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