EMFLAZA (deflazacort) by PTC Therapeutics is corticosteroid hormone receptor agonists [moa]. Approved for duchenne muscular dystrophy. First approved in 2017.
Drug data last refreshed Yesterday · AI intelligence enriched 3w ago
EMFLAZA (deflazacort) is an oral corticosteroid tablet approved by the FDA in February 2017 for treating Duchenne muscular dystrophy (DMD) in patients 2 years of age and older. It functions as a corticosteroid hormone receptor agonist, working through glucocorticoid receptor activation to reduce inflammation and slow muscle degeneration. EMFLAZA represents a disease-modifying therapeutic option in a rare pediatric neuromuscular disorder with limited treatment alternatives.
Corticosteroid Hormone Receptor Agonists
Corticosteroid
A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)
Food Effect and Bioavailability of Deflazacort Formulations in Healthy Volunteers
A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort Pharmacokinetics
A Single Dose Evaluation of the Effects of Renal Impairment on Deflazacort Pharmacokinetics
Worked on EMFLAZA at PTC Therapeutics? Share your interview experience or compensation data (+7 days Pro)
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.
Upgrade to Pro to access patent cliff timelines and LOE dates and other premium pharma intelligence.
Upgrade to Pro — $25/moUpgrade to Pro to access Medicare Part D spending data and other premium pharma intelligence.
Upgrade to Pro — $25/moEMFLAZA supports 21 currently open job positions, primarily in brand management, medical science liaison, and field sales roles focused on pediatric neurology and rare disease specialists. Success in this role requires deep knowledge of DMD pathophysiology, pediatric dosing considerations, and the small, specialized population of prescribers (pediatric neurologists and neuromuscular specialists). Candidates benefit from experience in orphan drug commercialization, managed care navigation, and patient advocacy engagement.