NDAORALTABLETPriority Review
Approved
Feb 2017
Lifecycle
Peak
Competitive Pressure
30/100
Clinical Trials
9
Data completeness
5/8 — Partial
Priority Review
EMFLAZA (deflazacort) by PTC Therapeutics is corticosteroid hormone receptor agonists [moa]. First approved in 2017.
Drug data last refreshed 3h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
EMFLAZA (deflazacort) is an oral corticosteroid that acts as a hormone receptor agonist, approved in 2017 for treating Duchenne muscular dystrophy and other inflammatory conditions. It modulates immune and inflammatory responses through glucocorticoid receptor activation. The drug is currently in peak commercial lifecycle with modest but stable utilization.
$15MPart D
PeakStable
Post-LOEEMFLAZA is in peak lifecycle with modest Part D penetration (1,181 claims in 2023), suggesting a stable but niche market requiring focused commercial and medical affairs execution.
Mechanism of Action
Corticosteroid Hormone Receptor Agonists
Pharmacologic Class:
Corticosteroid
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (9)
A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
Started Oct 2019
11 enrolled
Limb-Girdle Muscular Dystrophy
A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)
Started Oct 2018
0Duchenne Muscular Dystrophy
Food Effect and Bioavailability of Deflazacort Formulations in Healthy Volunteers
Started Jun 2015
45 enrolled
Healthy Volunteers
An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort
Started Dec 2014
24 enrolled
Duchenne Muscular Dystrophy
A Single Dose Evaluation of the Effects of Renal Impairment on Deflazacort Pharmacokinetics
Started Dec 2014
16 enrolled
Renal Impairment
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
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.