Gaucher Disease Type
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2018-08-15Clinical Trial Update: A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy [Phase 2]
Phase 2 trial evaluating AT2101 (afegostat tartrate) in adults with Type 1 Gaucher disease already on enzyme replacement therapy has complet…
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CLINICAL_TRIALS trial_result · 2018-08-15Clinical Trial Update: A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients [Phase 2]
Long-term extension study update of AT2101 (Afegostat Tartrate) in patients with Type 1 Gaucher disease completed, with results posted in 20…
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CLINICAL_TRIALS trial_result · 2018-08-15Clinical Trial Update: A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease [Phase 2]
Phase 2 trial update for oral AT2101 (afegostat tartrate) in treatment-naive Gaucher disease patients completed with posted results.
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CLINICAL_TRIALS withdrawal · 2018-03-02Clinical Trial Update: The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease [Phase 4]
Phase 4 pediatric trial evaluating the effect of Velaglucerase alfa on skeletal development in Gaucher disease has been withdrawn.
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CLINICAL_TRIALS trial_result · 2017-07-12Clinical Trial Update: Phase III Study of ISU302 in Patients With Type 1 Gaucher Disease [Phase 3]
A Phase 3 trial evaluating ISU302 in Type 1 Gaucher disease has completed enrollment and posted results.
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CLINICAL_TRIALS withdrawal · 2016-08-23Clinical Trial Update: The Safety and Efficacy Study of ISU302 in Patient With Type I Gaucher Disease [Phase 3]
The Phase 3 safety and efficacy study of ISU302 for Gaucher disease type 1 has been withdrawn.
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CLINICAL_TRIALS trial_result · 2015-06-01Clinical Trial Update: Validating a New Severity Score System for Adults With Type 1 Gaucher Disease (GD1)
A completed clinical trial aimed at validating a new severity score system for adults with Gaucher disease type 1.
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CLINICAL_TRIALS withdrawal · 2015-05-08Clinical Trial Update: A Screening Study Evaluating Disease Status of Gaucher Type I Patients
A screening study evaluating disease status in Gaucher disease type 1 patients was withdrawn.
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CLINICAL_TRIALS trial_result · 2015-03-05Clinical Trial Update: A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease. [Phase 4]
A Phase 4 multicenter trial evaluating the efficacy of Cerezyme for skeletal disease in Gaucher disease type I has been completed.
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EMA approval · 2014-11-21Cerdelga recommended for approval in type 1 Gaucher disease
The EMA recommends approval of Cerdelga for type 1 Gaucher disease, indicating regulatory consideration for this indication.
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CLINICAL_TRIALS trial_result · 2014-11-19Clinical Trial Update: Comparison of BMD Measurement by DEXA to BeamMed Speed-of-Sound Measurement at Forearm in Patients With Gaucher Disease [NA]
This clinical trial update reports a completed study comparing bone mineral density measurements obtained by DEXA versus BeamMed speed-of-so…
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FDA approval · 2014-11-05FDA Approved: CERDELGA — Supplemental Application (Genzyme Corporation)
FDA approved a supplemental application for Cerdelga (eliglustat) from Genzyme Corporation.
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CLINICAL_TRIALS trial_result · 2014-09-04Clinical Trial Update: A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE) [Phase 3]
This Phase 3 ENCORE trial evaluated switching Gaucher disease type 1 patients who had achieved therapeutic goals on enzyme replacement thera…
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CLINICAL_TRIALS trial_result · 2014-09-03Clinical Trial Update: A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE) [Phase 3]
Phase 3 ENGAGE trial results for Eliglustat Tartrate in Gaucher disease type 1 were completed, with results posted in 2014.
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FDA approval · 2014-08-19FDA Approved: CERDELGA — Original Application (Genzyme Corporation)
FDA approved Cerdelga (eliglustat) from Genzyme Corporation as an oral substrate reduction therapy for adults with Gaucher disease type 1, m…
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FDA approval · 2014-02-10FDA Approved: ZAVESCA — Supplemental Application (Actelion Pharmaceuticals US, Inc.)
FDA approved a supplemental application for ZAVESCA (miglustat) from Actelion Pharmaceuticals US, Inc., typically indicating an update or ex…
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CLINICAL_TRIALS trial_result · 2014-01-28Clinical Trial Update: An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease [Phase 3]
Open-label extension study of GA-GCB enzyme replacement therapy in adults with Type 1 Gaucher disease; phase 3 completed with results posted…
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CLINICAL_TRIALS trial_result · 2013-02-15Clinical Trial Update: Clinical Trial of Ambroxol in Patients With Type I Gaucher Disease [Phase 1, Phase 2]
A Phase 1/2 clinical trial of Ambroxol in Gaucher disease type 1 has been suspended.
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CLINICAL_TRIALS trial_result · 2012-05-23Clinical Trial Update: Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease [Phase 3]
Miglustat was evaluated in a Phase 3 long-term study for efficacy, safety, and tolerability in patients with stable Gaucher disease type 1. …
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CLINICAL_TRIALS trial_result · 2011-01-04Clinical Trial Update: Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease [Phase 3]
Phase 3 trial comparing GA-GCB enzyme replacement therapy to imiglucerase in Type 1 Gaucher disease has completed with results posted.
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CLINICAL_TRIALS trial_result · 2010-09-10Clinical Trial Update: A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease [Phase 3]
Phase 3 trial of GA-GCB enzyme replacement therapy for Gaucher disease type 1 has completed with posted results.
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EMA guidance · 2009-08-14Supply shortage of Cerezyme – updated treatment recommendations required
EMA announces a supply shortage of Cerezyme and provides updated treatment recommendations to manage shortages.