NDAORALSUSPENSIONPriority 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]. Approved for duchenne muscular dystrophy. First approved in 2017.
Drug data last refreshed 20h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
EMFLAZA (deflazacort) is an oral corticosteroid suspension approved for Duchenne Muscular Dystrophy (DMD), a rare progressive genetic disorder affecting skeletal muscle. It works as a corticosteroid hormone receptor agonist to reduce inflammation and slow muscle deterioration. The product targets a specialized pediatric population with a severe, life-limiting condition.
Peak1mo to LOE
EMFLAZA is at peak lifecycle with established market presence but faces imminent patent expiration, suggesting consolidation of commercial teams and transition planning underway.
Mechanism of Action
Corticosteroid Hormone Receptor Agonists
Pharmacologic Class:
Corticosteroid
Indications (1)
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
An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort
Started Dec 2014
24 enrolled
Duchenne Muscular Dystrophy
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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.