Emflaza

(Deflazacort)

Phase 3Small molecule

PPTC Therapeutics

Indications

Total Trials

Active Trials

Competitors

FDA Applications

NDA/BLA/ANDA regulatory filings

Pipeline

Status by Indication[3][1]

Indication

PrednisoloneActive, not recruiting

PrednisolonePhase 3

Active, not recruiting

Clinical

Trials by Indication[1]

Duchenne Muscular Dystrophy4 trials

Finding the Optimum Regimen for Duchenne Muscular Dystrophy

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

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

Show 1 more trial

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

Withdrawn·PPTC Therapeutics·Phase 3·NCT03642145

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

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

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

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

5 more indications

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

Renal Impairment1 trial

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

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

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

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