Drugs / Deflazacort
Regulatory milestones approvals, filings & regulatory actions · 2 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Label expansion | US (FDA) | EMFLAZA | — | 2019-06-07 | fda.gov ↗ |
| Approved | US (FDA) | EMFLAZA | — | 2017-02-09 | fda.gov ↗ |
Trials 14
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 18 trials | ||||||
| Phase 1 | NCT04791345 | Feb 2021 → Feb 2022 | — | Parc de Salut Mar | Unknown | No outcome recorded |
| Phase 1 | NCT04543370 | Aug → Sep 2020 | — | Catabasis Pharmaceuticals | Completed | No outcome recorded |
| Phase 1 | NCT02485431 | Jun → Aug 2015 | — | PTC Therapeutics | Completed | No outcome recorded |
| Phase 1 | NCT02295748 | Dec 2014 → Aug 2017 | Duchenne muscular dystrophy | PTC Therapeutics | Completed | No outcome recorded |
| Phase 1 | NCT02286622 | Dec 2014 → Feb 2015 | impaired renal function disease | PTC Therapeutics | Completed | No outcome recorded |
| Phase 1 | NCT02286609 | Dec 2014 → Feb 2015 | — | PTC Therapeutics | Completed | No outcome recorded |
| Phase 1 | NCT02251600 | Dec 2014 → Oct 2015 | Duchenne muscular dystrophy | PTC Therapeutics | Completed | No outcome recorded |
| Phase 1 | NCT02286635 | Nov → Dec 2014 | — | PTC Therapeutics | Completed | No outcome recorded |
| Phase 2/31 trial | ||||||
| Phase 2/3 | NCT04227483 | Jan 2020 → Jul 2025 overdue | allergic bronchopulmonary aspergillosis | Post Graduate Institute of Medical Education and Research, Chandigarh | Active not recruiting | No outcome recorded |
| Phase 33 trials | ||||||
| Phase 3 | NCT03783923 | Oct 2019 → Jan 2021 | limb-girdle muscular dystrophy | PTC Therapeutics | Terminated | No outcome recorded Stop: Enrollment |
| Phase 3 | NCT03642145 | Oct 2018 → Jul 2021 | Duchenne muscular dystrophy | PTC Therapeutics | Withdrawn | No outcome recorded |
| Phase 3 | NCT01603407 | Jan 2013 → Nov 2019 | Duchenne muscular dystrophy | University of Rochester | Completed | No outcome recorded |
| Phase 41 trial | ||||||
| Phase 4 | NCT04365088 | Dec 2018 → Nov 2023 | impacted teeth, multiple | Yuzuncu Yil University | Unknown | No outcome recorded |
| Phase not stated1 trial | ||||||
| — | NCT02592941 | — | Duchenne muscular dystrophy | PTC Therapeutics | Approved for marketing | No outcome recorded |
| Comparator or background therapy5 trials | ||||||
| Phase 2/3 | NCT00527228 Comparator | Sep 2003 → Apr 2008 | Miyoshi myopathy, autosomal recessive limb-girdle muscular dystrophy type 2B | Ludwig-Maximilians - University of Munich | Completed | No outcome recorded |
| Phase 3 | NCT04632940 Background | Mar 2021 → Jun 2023 | Duchenne muscular dystrophy | Kyntra Bio | Terminated | Missed primary |
| Phase 3 | NCT04371666 Background | Aug 2020 → Feb 2023 | Duchenne muscular dystrophy | Kyntra Bio | Terminated | Missed primary |
| Phase 3 | NCT01282073 Comparator | Mar 2011 → May 2016 | membranous glomerulonephritis | Kyungpook National University Hospital | Completed | No outcome recorded |
| Phase 4 | NCT01817322 Background | Jun 2011 → Aug 2013 | chronic kidney disease | Samsung Medical Center | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 5
| Date | Issuer | Release |
|---|---|---|
| 2022-03-11 | PTC Therapeutics | Results PTC Therapeutics to Present New Data at the 2022 Muscular Dystrophy Association Clinical & Scientific Conference ptcbio.com ↗
These results have not been previously published and further advance the clinical understanding of steroid treatment in the continuum of DMD care. |
| 2021-06-24 | PTC Therapeutics | Results Results Add to Body of Evidence Confirming EMFLAZA®'s Benefit Over Prednisone ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) today presented pooled placebo data from multiple studies in which the results confirmed the clinical benefit of EMFLAZA® (deflazacort) over prednisone for the treatment of Duchenne muscular dystrophy (DMD). |
| 2021-03-15 | PTC Therapeutics | Results PTC Presents Results from a Real-World Study of Steroid Switching in the Treatment of Patients with Dystrophinopathies ptcbio.com ↗
During the 6-months average follow-up after switching, physician-reported outcomes were consistent with EMFLAZA addressing the primary reasons for switching. |
| 2019-06-07 | PTC Therapeutics | Regulatory PTC Therapeutics Receives FDA Approval for the Expansion of the EMFLAZA® (deflazacort) Labeling to Include Patients 2-5 Years of Age ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the U.S. Food and Drug Administration (FDA) approved the company's supplemental New Drug Application (sNDA) for Emflaza® (deflazacort) to expand its labeling to include patients with Duchenne muscular dystrophy who are between 2- and 5-years-old. |
| 2018-07-24 | PTC Therapeutics | Results PTC Therapeutics Announces Publication of Data Demonstrating the Clinical Differentiated Benefit of Deflazacort ptcbio.com ↗
The results demonstrated a clinically differentiated benefit of deflazacort over prednisone/prednisolone in slowing disease progression as measured using physical function endpoints and the time to delay loss of ambulation. |
All press releases naming this drug 8 releases
Evidence & citations 12 cited values
Every value below carries the sentence it was read from. 20 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Deflazacort | “Pamrevlumab 35 milligrams (mg)/kilogram (kg) intravenously (IV) every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks” NCT04371666 ↗18“Steroid: Methylprednisone 4mg tablet or Prednisolone 5mg tablet or Deflazacort 6mg tablet.” NCT01282073 ↗ “Systemic deflazacort or equivalent potency of corticosteroids administered orally” NCT04632940 ↗ |
| Known as | Azacortinol | ChEMBL registry synonym — accepted as the source's own label CHEMBL1201891 ↗ |
| Known as | Calcort | ChEMBL registry synonym — accepted as the source's own label CHEMBL1201891 ↗ |
| Known as | DL-458IT | ChEMBL registry synonym — accepted as the source's own label CHEMBL1201891 ↗ |
| Known as | Emflaza | ChEMBL registry synonym — accepted as the source's own label CHEMBL1201891 ↗ |
| Known as | L-5458 | ChEMBL registry synonym — accepted as the source's own label CHEMBL1201891 ↗ |
| Known as | MDL 458 | ChEMBL registry synonym — accepted as the source's own label CHEMBL1201891 ↗ |
| Known as | Zamene | ChEMBL registry synonym — accepted as the source's own label CHEMBL1201891 ↗ |
| Action | Activate | “Glucocorticoid receptor agonist” CHEMBL1201891 ↗ |
| Modality | Other / unclassified | “Deflazacort, a glucocorticoid with anti-inflammatory and immunosuppressive effects, is used in treating a variety of diseases.” NCT02286635 ↗ |
| Route | Oral | “by the mouth” NCT01603407 ↗ |
| Target | NR3C1 | “Glucocorticoid receptor agonist” CHEMBL1201891 ↗ |