Recent Updates
Recently added Catalysts

Deferasirox granule formulation

Phase 2

Transfusion-dependent Anemia | Small molecule | Hematology |Novartis AG|Last Updated: Sep 19, 2024

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
Premium
Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
Premium
Trial Design
RandomizedACTIVE_CONTROLLED
Total Trials1
Total Enrollment224
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02435212Study to Evaluate Treatment Compliance, Efficacy and Safety of an Improved Deferasirox Formulation (Granules) in Pediatric Patients (2-<18 Years Old) With Iron OverloadPHASE2 COMPLETED 224 — — Oct 21, 2015Jan 15, 2024Sep 19, 202443 United States, Belgium +15
Unlock Drug Trial Details
Study Endpoints
Primary Endpoints
Percentage of Overall Compliance Using Stick Pack or Tablet Counts in Iron Chelation Therapy (ICT)-naïve Participants During the Core Phase
24 weeks

Compliance was calculated as the ratio of total count consumed to total count prescribed of deferasirox granule stick packs or dispersible tablets, where total count consumed was derived from cumulative dispensed, returned and lost/wasted counts over 24 weeks of treatment and total count prescribed was derived from cumulative prescribed count over 24 weeks of treatment.

Change From Baseline in Serum Ferritin (SF) for Both Study Drug Formulations in ICT naïve Participants During the Core Phase
From Baseline to Week 25

The analysis included the comparison of means between the two treatment arms of change from baseline after 24 weeks of treatment in serum ferritin in pediatric ICT naïve participants with iron overload. The endpoint was assessed at Week 25 visit.

Secondary Endpoints
Percentage of Overall Compliance Using Stick Pack or Tablet Counts in ICT-naïve Participants During the Core Phase
48 weeks
Change From Baseline in Serum Ferritin (SF) for Both Study Drug Formulations in ICT naïve Participants During the Core Phase
From Baseline to 48 weeks
Change From Baseline in Serum Ferritin (SF) for Both Study Drug Formulations in Pre-treated Participants During the Core Phase
From Baseline to Week 25 and Week 48
Unlock Study Endpoints
Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
DFX DTACTIVE_COMPARATORParticipants will be administered deferasirox dispersible tablets orally once daily based on body weight for 48 weeks.
DFX GranuleEXPERIMENTALParticipants will be administered deferasirox granules orally once daily in the form of stick packs based on body weight for 48 weeks.
Interventions
NameTypeDescription
Deferasirox granule formulationDRUGDeferasirox granules will be provided as stick packs containing 90 mg, 180 mg and 360 mg granules for oral use and will be administered based on body weight.
Deferasirox DT formulationDRUGDeferasirox DT will be provided as 125 mg, 250 mg and 500 mg dispersible tablets for oral use and will be administered based on body weight.
Unlock Study Design Details
Eligibility Criteria
Age Range2 Years — 17 Years
SexALL
Healthy VolunteersNo
Study Sites43

Inclusion Criteria: * Written informed consent/assent before any study-specific procedures. Consent will be obtained from parent(s) or legal guardians. Investigators will also obtain assent of patients according to local guidelines. * Male and female children and adolescents aged ≥ 2 and \< 18 year...

Countries:United StatesBelgiumBulgariaEgyptFranceHungaryIndiaItalyLebanonMalaysiaOmanPanamaPhilippinesRussiaThailandTunisiaTurkey (Türkiye)
Unlock Eligibility Criteria