Aachen (OTE) – Grünenthal announced today that its investigational
compound
tegacorat (GRM-01) received Orphan Drug and Rare Pediatric Disease
Designations from the US Food and Drug Administration (FDA) for the
treatment of Duchenne muscular dystrophy (DMD).
Tegacorat, a non-steroidal Selective Glucocorticoid Receptor
Agonist and Modulator (SEGRAM), is an orally available
investigational compound being developed to provide an alternative to
glucocorticoid-based treatments such as prednisone, the current
standard of care in DMD.
Conventional glucocorticoids bind to the glucocorticoid receptor
and influence gene expression. Among other mechanisms, this triggers
two patterns of activity: transrepression, mainly linked to anti-
inflammatory effects, and transactivation, predominantly associated
with metabolic and growth-related side effects. In contrast, SEGRAMs
are designed to influence receptor activity in a way that emphasises
anti-inflammatory pathways. While this has not yet been established
in clinical trials, it may allow for more efficacious dosing than
current standard treatments and potentially cause fewer side effects.
“With the current standard of care, people affected by DMD, their
caregivers and clinicians must constantly balance efficacy and the
burden of side effects as they pursue the essential goal of
preserving muscle function,” says Uli Brödl, Chief Scientific Officer
at Grünenthal. “We aim to address the unmet need for a long-term
therapy option with potent anti-inflammatory efficacy while reducing
dose- and duration dependent side effects. The Orphan Drug and Rare
Pediatric Disease designations are an important milestone in the
development of tegacorat.”
Grünenthal is currently preparing a Phase II trial to investigate
the efficacy, safety and tolerability of tegacorat in Duchenne
muscular dystrophy. The trial is expected to commence later in 2026
at centres in the US and Europe.
About Duchenne muscular dystrophy (DMD)
Duchenne muscular dystrophy is one of the most common recessive
genetic disorders, affecting around 1 in every 5,000 boys born.[1], [
2] Caused by mutations in the dystrophin gene that encodes for a
critical protein that protects muscles from damage, DMD causes
progressive muscle weakness throughout the body that eventually
impacts mobility, breathing and the heart.[2] DMD is incurable and
results in death, usually between 21 and 40 years of age.[3]
Today’s treatment options are neither curative nor preventive of
disease progression. Glucocorticoids serve as the standard of care,
as they can help slow the progression of muscle deterioration.
However, they come with significant side effects, including
cushingoid appearance, weight gain and behavioural changes.[4]
About Grünenthal Grünenthal is a global leader in pain management
and related diseases. As a science-based, fully integrated
pharmaceutical company, we have a long track record of bringing
innovative treatments and state-of-the-art technologies to patients
worldwide. Our purpose is to change lives for the better – and
innovation is our passion. We focus all our activities and efforts on
working towards our vision of a World Free of Pain. Grünenthal is
headquartered in Aachen, Germany, and has affiliates in 28 countries
across Europe, Latin America, and the U.S. Our products are available
in approx. 100 countries. In 2025, Grünenthal employed around 4,100
people and achieved revenues of Ꞓ1.8 billion.
More information: www.grunenthal.com and follow us on LinkedIn &
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[1] Crisafulli S, et al. Global epidemiology of Duchenne muscular
dystrophy: an updated systematic review and meta-analysis. Orphanet J
Rare Dis. 2020; 15(1):141.
[2] Bez Batti Angulski A, et al. Duchenne muscular dystrophy:
disease mechanism and therapeutic strategies. Front Physiol. 2023;
14:1183101.
[3] Landfeldt E, et al. Life expectancy at birth in Duchenne
muscular dystrophy: a systematic review and meta-analysis. Eur J
Epidemiol. 2020; 35(7):643-653. [Estimated mortality for those who
receive ventilatory support.]
[4] Fischer R, et al. A Mixed-Method Study Exploring Patient-
Experienced and Caregiver-Reported Benefits and Side Effects of
Corticosteroid Use in Duchenne Muscular Dystrophy. J Neuromuscul Dis.
2023;10(4):593-613.