Emflaza

(Deflazacort)

Phase 3Small molecule

PPTC Therapeutics

Emflaza is one of 57 small molecule programs targeting Muscular Dystrophy. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

FDA Applications

NDA/BLA/ANDA regulatory filings

Pipeline

Status by Indication[3][1]

Indication

Muscular DystrophyActive, not recruiting

Duchenne Muscular DystrophyActive, not recruiting

Muscular DystrophyPhase 3

Active, not recruiting

Duchenne Muscular DystrophyPhase 3

Active, not recruiting

Competition

Competitive Landscape

125 competitors in Duchenne Muscular Dystrophy

#DrugCompanyPhaseTrials

Cialis

Eli Lilly

Phase 3

2 2

Rifadin

Sanofi

Phase 3

1 3

Nolvadex

AstraZeneca

Phase 3

1 4

Aldactone

Pfizer

Phase 3

1 5

Avicel

SSanthera Pharmaceuticals

Phase 3

6 6

Rayos

RRoxane Laboratories

Phase 3

5 7

Inspra

PPfizer's Upjohn has merged with Mylan to form Viatris Inc.

Phase 3

2 8

Duvyzat

Italfarmaco

Phase 3

2 9

Bystolic

VVitae Pharmaceuticals Inc., an Allergan affiliate

Phase 3

1 10

Creatine Monohydrate

MMassachusetts General Hospital

Phase 3

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Clinical

Trials by Indication[1]

Duchenne Muscular Dystrophy4 trials

Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy

Approved for marketing·PPTC Therapeutics··NCT02592941

A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy

Completed·PPTC Therapeutics·Phase 1·NCT02251600·24 enrolled

A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)

Withdrawn·PPTC Therapeutics·Phase 3·NCT03642145

Show 1 more trial

Finding the Optimum Regimen for Duchenne Muscular Dystrophy

Completed·UUniversity of Rochester·Phase 3·NCT01603407·196 enrolled

Corticosteroids1 trial

Study of the Excretion of Orally Administered Corticosteroids for the Improval of the Detection of Said Substances in Anti-doping Controls

Unknown status·PParc de Salut Mar·Phase 1·NCT04791345·50 enrolled

HIES1 trial

Deflazacort in Dysferlinopathies

Completed·LLudwig-Maximilians - University of Munich·Phase 2/3·NCT00527228·25 enrolled

Prednisolone1 trial

Deflazacort Vs. Prednisolone in Acute-stage ABPA

Active, not recruiting·PPost Graduate Institute of Medical Education and Research, Chandigarh·Phase 2/3·NCT04227483·150 enrolled

Renal Impairment1 trial

A Single Dose Evaluation of the Effects of Renal Impairment on Deflazacort Pharmacokinetics

Completed·PPTC Therapeutics·Phase 1·NCT02286622·16 enrolled

5 more indications

Muscular Dystrophy1 trial

A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)

Terminated·PPTC Therapeutics·Phase 3·NCT03783923·11 enrolled

Post Operative Pain1 trial

Evaluation of the Effects of Pre-operative Deflazacort on Post-operative Pain, Edema and Trismus

Unknown status·YYuzuncu Yil University·Phase 4·NCT04365088·35 enrolled

Single Dose1 trial

Evaluate Effects of Multiple Doses of Rifampin and Clarithromycin on the Single Dose Pharmacokinetics of Deflazacort

Completed·PPTC Therapeutics·Phase 1·NCT02286635·58 enrolled

Moderate Hepatic Impairment1 trial

A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort Pharmacokinetics

Completed·PPTC Therapeutics·Phase 1·NCT02286609·16 enrolled

Unspecified2 trials

An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort

Completed·PPTC Therapeutics·Phase 1·NCT02295748·24 enrolled

Food Effect and Bioavailability of Deflazacort Formulations in Healthy Volunteers

Completed·PPTC Therapeutics·Phase 1·NCT02485431·45 enrolled

Regulatory

FDA Approval Timeline[4]

Feb 9, 2017

EMFLAZA

NDA NDA208685

Feb 9, 2017

EMFLAZA

NDA NDA208684

Sources

References

[1]
ClinicalTrials.gov — 14 trial records for Deflazacort. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Deflazacort compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Deflazacort. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (NDA NDA208685). U.S. Food & Drug Administration.
[5]
SEC EDGAR — PTC Therapeutics company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 3[1]
Company: PPTC Therapeutics

Emflaza

Status by Indication[3][1]

Competitive Landscape

Trials by Indication[1]

Duchenne Muscular Dystrophy4 trials

Corticosteroids1 trial

HIES1 trial

Prednisolone1 trial

Renal Impairment1 trial

Muscular Dystrophy1 trial

Post Operative Pain1 trial

Single Dose1 trial

Moderate Hepatic Impairment1 trial

Unspecified2 trials

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

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