NDAORALSUSPENSIONPriority Review
Approved
Feb 2017
Lifecycle
Peak
Competitive Pressure
30/100
Clinical Trials
9
Data completeness
4/8 — Partial
Priority Review
EMFLAZA (deflazacort) by PTC Therapeutics is corticosteroid hormone receptor agonists [moa]. First approved in 2017.
Drug data last refreshed 6h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
EMFLAZA (deflazacort) is an oral corticosteroid suspension approved in 2017 for duchenne muscular dystrophy (DMD) and other corticosteroid-responsive conditions. It works as a corticosteroid hormone receptor agonist to reduce inflammation and slow disease progression. Deflazacort is a prodrug that converts to an active metabolite with potentially improved tolerability compared to prednisone.
PeakPost-LOE
EMFLAZA is in peak lifecycle with moderate competitive pressure (30%), indicating stable commercial operations but potential resource reallocation as LOE approaches in 2026.
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.