276. Achondroplasia Disease details / Clinical trials / Drug dev / DR info

Clinical trials : 51 /  Drugs : 34 - (DrugBank : 6) / Drug target genes : 4 - Drug target pathways : 26

Drugs and their primary sponsors and trial info
Active BMN 111: Daily subcutaneous injection of 15 ug/kg of BMN 111   
   BioMarin Pharmaceutical
      2019   Phase 2   NCT03989947   Australia;Japan;United Kingdom;United States;
BGJ398 (also known as BBP-831)   
   QED Therapeutics, Inc.
      2022   Phase 2   EUCTR2021-001855-15-ES   Australia;Canada;France;Spain;United Kingdom;United States;
      2020   Phase 2   EUCTR2019-002954-21-GB   Australia;Canada;France;Spain;United Kingdom;United States;
      2020   Phase 2   EUCTR2019-002954-21-FR   Australia;Canada;France;Spain;United Kingdom;United States;
BMN 111   
   BioMarin Pharmaceutical
      2018   Phase 2   NCT03583697   Australia;Japan;United Kingdom;United States;
      2017   Phase 3   NCT03424018   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States;
      2016   Phase 3   NCT03197766   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States;
      2016   Phase 2   NCT02724228   Australia;France;United Kingdom;United States;
      2014   Phase 2   NCT02055157   Australia;France;United Kingdom;United States;
      2012   Phase 1   NCT01590446   United States;
   BioMarin Pharmaceutical Inc.
      2020   Phase 2   EUCTR2020-001055-40-GB   Australia;United Kingdom;
      2019   Phase 3   EUCTR2017-002404-28-DE   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States;
      2018   Phase 3   EUCTR2017-002404-28-GB   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States;
      2018   Phase 3   EUCTR2017-002404-28-ES   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States;
      2018   Phase 3   EUCTR2015-003836-11-DE   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States;
      2018   Phase 2   EUCTR2016-003826-18-GB   Australia;Japan;United Kingdom;United States;
      2017   Phase 3   EUCTR2015-003836-11-GB   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States;
      2017   Phase 3   EUCTR2015-003836-11-ES   Australia;France;Germany;Japan;Spain;Turkey;United Kingdom;United States;
      2014   Phase 2   EUCTR2013-004137-32-GB   Australia;France;United Kingdom;United States;
      -   Phase 2   EUCTR2015-004004-30-GB   Australia;France;United Kingdom;United States;
      -   Phase 2   EUCTR2015-004004-30-FR   Australia;France;United Kingdom;United States;
      -   Phase 2   EUCTR2013-004137-32-FR   Australia;France;United Kingdom;United States;
C-TYPE NATRIURETIC PEPTIDE CONJUGATED TO MULTI-ARM POLYETHYLENE GLYCOL CARRIER THROUGH A CLEAVABLE LINKER   
   Ascendis Pharma Growth Disorders A/S
      2021   Phase 2   EUCTR2019-002754-22-PT   Australia;Austria;Canada;Denmark;Germany;Ireland;New Zealand;Portugal;Spain;Switzerland;United Kingdom;United States;
      2020   Phase 2   EUCTR2019-002754-22-DK   Australia;Austria;Canada;Denmark;Germany;Ireland;New Zealand;Portugal;Spain;Switzerland;United Kingdom;United States;
      2020   Phase 2   EUCTR2019-002754-22-DE   Australia;Austria;Canada;Denmark;Germany;Ireland;Italy;New Zealand;Portugal;Spain;Switzerland;United Kingdom;United States;
      2020   Phase 2   EUCTR2019-002754-22-AT   Australia;Austria;Canada;Denmark;Germany;Ireland;New Zealand;Portugal;Spain;Switzerland;United Kingdom;United States;
      2019   Phase 2   EUCTR2019-002754-22-IE   Australia;Austria;Canada;Denmark;Germany;Ireland;New Zealand;Portugal;Spain;Switzerland;United Kingdom;United States;
      -   Phase 2   EUCTR2019-002754-22-GB   Australia;Austria;Canada;Denmark;Germany;Ireland;Italy;New Zealand;Portugal;Spain;Switzerland;United Kingdom;United States;
Infigratinib   
   QED Therapeutics, Inc.
      2021   Phase 2   NCT05145010   Australia;United Kingdom;
      2020   Phase 2   NCT04265651   Australia;Canada;France;Spain;United Kingdom;United States;
Infigratinib phosphate   
   QED Therapeutics, Inc.
      2020   Phase 2   EUCTR2019-002954-21-GB   Australia;Canada;France;Spain;United Kingdom;United States;
      2020   Phase 2   EUCTR2019-002954-21-FR   Australia;Canada;France;Spain;United Kingdom;United States;
MODIFIED RHCNP   
   BioMarin Pharmaceutical Inc.
      2020   Phase 2   EUCTR2020-001055-40-GB   Australia;United Kingdom;
      2019   Phase 3   EUCTR2017-002404-28-DE   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States;
      2019   Phase 2   EUCTR2018-004364-66-GB   Australia;Japan;United Kingdom;United States;
      2018   Phase 3   EUCTR2017-002404-28-GB   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States;
      2018   Phase 3   EUCTR2017-002404-28-ES   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States;
      2018   Phase 3   EUCTR2015-003836-11-DE   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States;
      2018   Phase 2   EUCTR2016-003826-18-GB   Australia;Japan;United Kingdom;United States;
      2017   Phase 3   EUCTR2015-003836-11-GB   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States;
      2017   Phase 3   EUCTR2015-003836-11-ES   Australia;France;Germany;Japan;Spain;Turkey;United Kingdom;United States;
      2014   Phase 2   EUCTR2013-004137-32-GB   Australia;France;United Kingdom;United States;
      -   Phase 2   EUCTR2015-004004-30-GB   Australia;France;United Kingdom;United States;
      -   Phase 2   EUCTR2015-004004-30-FR   Australia;France;United Kingdom;United States;
      -   Phase 2   EUCTR2013-004137-32-FR   Australia;France;United Kingdom;United States;
Modified recombinant human C-type natriuretic peptide   
   BioMarin Pharmaceutical Inc.
      2020   Phase 2   EUCTR2020-001055-40-GB   Australia;United Kingdom;
      2019   Phase 3   EUCTR2017-002404-28-DE   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States;
      2018   Phase 3   EUCTR2017-002404-28-GB   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States;
      2018   Phase 3   EUCTR2017-002404-28-ES   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States;
      2018   Phase 3   EUCTR2015-003836-11-DE   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States;
      2018   Phase 2   EUCTR2016-003826-18-GB   Australia;Japan;United Kingdom;United States;
      2017   Phase 3   EUCTR2015-003836-11-GB   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States;
      2017   Phase 3   EUCTR2015-003836-11-ES   Australia;France;Germany;Japan;Spain;Turkey;United Kingdom;United States;
      2014   Phase 2   EUCTR2013-004137-32-GB   Australia;France;United Kingdom;United States;
      -   Phase 2   EUCTR2015-004004-30-GB   Australia;France;United Kingdom;United States;
      -   Phase 2   EUCTR2015-004004-30-FR   Australia;France;United Kingdom;United States;
      -   Phase 2   EUCTR2013-004137-32-FR   Australia;France;United Kingdom;United States;
   BioMarin Pharmaceutical Inc.(ICCC:EPS International Holdings Co. Ltd.)
      2018   Phase 3   JPRN-JapicCTI-184167   Japan, North America, Europe, Oceania;
Modified recombinant human c-type   
   BioMarin Pharmaceutical Inc.
      2019   Phase 2   EUCTR2018-004364-66-GB   Australia;Japan;United Kingdom;United States;
Natriuretic peptide   
   BioMarin Pharmaceutical Inc.
      2019   Phase 2   EUCTR2018-004364-66-GB   Australia;Japan;United Kingdom;United States;
Normal Saline   
   BioMarin Pharmaceutical
      2012   Phase 1   NCT01590446   United States;
PF-07256472   
   PFIZER INC
      2021   Phase 2   EUCTR2020-001189-13-IT   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United Kingdom;United States;
   Pfizer Inc.
      2021   Phase 2   EUCTR2021-003149-39-ES   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States;
      2021   Phase 2   EUCTR2020-001189-13-PT   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United Kingdom;United States;
      -   Phase 2   EUCTR2021-003149-39-BE   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States;
   Pfizer Inc., 235 East 42nd Street, New York, NY 10017
      2020   Phase 2   EUCTR2020-001189-13-DK   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States;
      -   Phase 2   EUCTR2020-001189-13-BE   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States;
Placebo   
   BioMarin Pharmaceutical Inc.(ICCC:EPS International Holdings Co. Ltd.)
      2018   Phase 3   JPRN-JapicCTI-184167   Japan, North America, Europe, Oceania;
Recifercept   
   PFIZER INC
      2021   Phase 2   EUCTR2020-001189-13-IT   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United Kingdom;United States;
   Pfizer
      2021   Phase 2   NCT05116046   -
      2020   Phase 2   NCT04638153   Australia;Belgium;Denmark;Italy;Portugal;Spain;United States;
Recifercept (proposed INN)   
   Pfizer Inc.
      2021   Phase 2   EUCTR2021-003149-39-ES   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States;
      2021   Phase 2   EUCTR2020-001189-13-PT   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United Kingdom;United States;
      -   Phase 2   EUCTR2021-003149-39-BE   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States;
   Pfizer Inc., 235 East 42nd Street, New York, NY 10017
      2020   Phase 2   EUCTR2020-001189-13-DK   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States;
      -   Phase 2   EUCTR2020-001189-13-BE   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States;
Somatropin   
   Novo Nordisk A/S
      2012   -   NCT01435629   Japan;
      1997   -   NCT01516229   Japan;
TA-46   
   Pfizer Inc.
      2021   Phase 2   EUCTR2021-003149-39-ES   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States;
      2021   Phase 2   EUCTR2020-001189-13-PT   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United Kingdom;United States;
      -   Phase 2   EUCTR2021-003149-39-BE   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States;
   Pfizer Inc., 235 East 42nd Street, New York, NY 10017
      2020   Phase 2   EUCTR2020-001189-13-DK   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States;
      -   Phase 2   EUCTR2020-001189-13-BE   Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain;United States;
TBD   
   BioMarin Pharmaceutical Inc.
      -   Phase 2   EUCTR2013-004137-32-FR   Australia;France;United Kingdom;United States;
TransCon CNP   
   Ascendis Pharma A/S
      2022   Phase 2   NCT05246033   China;
      2020   Phase 2   NCT04085523   Australia;Austria;Denmark;Germany;Ireland;New Zealand;United States;
TransCon CNP 3.9 mg CNP-38/vial   
   Ascendis Pharma Growth Disorders A/S
      2021   Phase 2   EUCTR2019-002754-22-PT   Australia;Austria;Canada;Denmark;Germany;Ireland;New Zealand;Portugal;Spain;Switzerland;United Kingdom;United States;
      2020   Phase 2   EUCTR2019-002754-22-DK   Australia;Austria;Canada;Denmark;Germany;Ireland;New Zealand;Portugal;Spain;Switzerland;United Kingdom;United States;
      2020   Phase 2   EUCTR2019-002754-22-DE   Australia;Austria;Canada;Denmark;Germany;Ireland;Italy;New Zealand;Portugal;Spain;Switzerland;United Kingdom;United States;
      2020   Phase 2   EUCTR2019-002754-22-AT   Australia;Austria;Canada;Denmark;Germany;Ireland;New Zealand;Portugal;Spain;Switzerland;United Kingdom;United States;
      2019   Phase 2   EUCTR2019-002754-22-IE   Australia;Austria;Canada;Denmark;Germany;Ireland;New Zealand;Portugal;Spain;Switzerland;United Kingdom;United States;
      -   Phase 2   EUCTR2019-002754-22-GB   Australia;Austria;Canada;Denmark;Germany;Ireland;Italy;New Zealand;Portugal;Spain;Switzerland;United Kingdom;United States;
Vorsoritide   
   BioMarin Pharmaceutical Inc.
      2017   Phase 3   EUCTR2015-003836-11-ES   Australia;France;Germany;Japan;Spain;Turkey;United Kingdom;United States;
      2017   Phase 1   EUCTR2017-000701-21-DE   Australia;France;Germany;Japan;Spain;Turkey;United Kingdom;United States;
Vosoritide   
   BioMarin Pharmaceutical
      2020   Phase 2   NCT04554940   Australia;United Kingdom;
   BioMarin Pharmaceutical Inc.
      2020   Phase 2   EUCTR2020-001055-40-GB   Australia;United Kingdom;
      2019   Phase 3   EUCTR2017-002404-28-DE   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States;
      2019   Phase 2   EUCTR2018-004364-66-GB   Australia;Japan;United Kingdom;United States;
      2018   Phase 3   EUCTR2017-002404-28-GB   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States;
      2018   Phase 3   EUCTR2017-002404-28-ES   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States;
      2018   Phase 3   EUCTR2015-003836-11-DE   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States;
      2018   Phase 2   EUCTR2016-003826-18-GB   Australia;Japan;United Kingdom;United States;
      2017   Phase 3   EUCTR2015-003836-11-GB   Australia;Germany;Japan;Spain;Turkey;United Kingdom;United States;
      2014   Phase 2   EUCTR2013-004137-32-GB   Australia;France;United Kingdom;United States;
      -   Phase 2   EUCTR2015-004004-30-GB   Australia;France;United Kingdom;United States;
      -   Phase 2   EUCTR2015-004004-30-FR   Australia;France;United Kingdom;United States;
   BioMarin Pharmaceutical Inc.(ICCC:EPS International Holdings Co. Ltd.)
      2018   Phase 3   JPRN-JapicCTI-184167   Japan, North America, Europe, Oceania;