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 21h ago · AI intelligence enriched 3d ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
EMFLAZA (deflazacort) is an oral corticosteroid tablet approved in 2017 for duchenne muscular dystrophy and other corticosteroid-responsive conditions. It works as a corticosteroid hormone receptor agonist to reduce inflammation and immune-mediated muscle degeneration. Deflazacort has a favorable tolerability profile compared to traditional corticosteroids like prednisone.
$0.015BPart D
PeakPost-LOEPeak-stage product with modest Part D penetration (1,181 claims in 2023) presents stable commercial infrastructure but limited growth runway given competitive pressure of 30%.
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
A Single Dose Evaluation of the Effects of Renal Impairment on Deflazacort Pharmacokinetics
Started Dec 2014
16 enrolled
Renal Impairment
A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort Pharmacokinetics
Started Dec 2014
16 enrolled
Hepatic 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.