The FDA has granted Orphan Drug and Rare Pediatric Disease Designations to Grünenthal for tegocorat (GRM-01), a treatment for Duchenne muscular dystrophy (DMD).
The orally-available investigational compound is a non-steroidal selective glucocorticoid receptor agonist and modulator (SEGRAM). It could provide an alternative to the current standard of care — conventional glucocorticoids like prednisone that bind to the glucocorticoid receptor. The resulting impact on gene expression of these treatments leads to transrepression (which has anti-inflammatory side effects) and transactivation (which affects metabolism and growth).
The next steps for tegocorat are a phase 2 trial to evaluate its efficacy, safety, and tolerability. Grünenthal expects to commence the trial on patients with DMD in the U.S. and Europe later in 2026.
Earlier in the year, PTC Therapeutics withdrew its NDA for another oral drug to treat DMD. Translarna (ataluren) is a protein restoration therapy that enables synthesis of dystrophin. PTC made the decision to withdraw its NDA based on the fact that the FDA was unlikely to approve the submission, as it did not meet the agency’s threshold of evidence of effectiveness.