Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Difelikefalin · 3 trials · 4 indications
On a daily basis, participants recorded the intensity of the worst itching they experienced over the past 24 hours using a numerical rating scale (NRS) scale from 0 to 10, where 0 represents "no itching" and 10 was "worst itching imaginable". A higher score indicated a more severe outcome. The weekly mean of the daily values of the daily 24-hour WI-NRS was calculated for the analysis. The least square (LS) means of change from baseline to Week 4 in the weekly mean of the daily 24-hour WI-NRS score was estimated using the mixed model repeated measures (MMRM) method. The MMRM model included use of prior anti-itch medication (yes/no), presence of specific medical conditions at baseline (yes/no), treatment, visit, and treatment-by-visit-interaction as fixed categorical effects and baseline WI-NRS score as fixed continuous effects.
To evaluate the safety of 0.5 μg/kg difelikefalin in HD adolescents (≥12 to \<18 years) with moderate-to-severe pruritus
Cmax = Maximum (peak) observed plasma concentration
Tmax = Time to reach maximum observed plasma concentration
AUC0-t = Area under the concentration-versus-time curve (AUC) from time zero to time "t"
AUCinf = AUC from time zero to infinity
AUCextrap(%) = percentage of AUCinf based on extrapolation
t½ = elimination half-life
Clearance = the volume of blood or plasma that can be freed of a specified constituent in a specified time by its excretion into the urine through the kidneys
Vz = volume of distribution
Cmax = Maximum (peak) observed plasma concentration
Tmax = Time to reach maximum observed plasma concentration
AUC0-t = Area under the concentration-versus-time curve (AUC) from time zero to time "t"
AUCinf = AUC from time zero to infinity
AUCextrap(%) = percentage of AUCinf based on extrapolation
t½ = elimination half-life
| Arm | Type | Description |
|---|---|---|
| 12-weeks double-blind period - Difelikefalin | EXPERIMENTAL | - |
| 12-weeks double-blind period - Placebo | PLACEBO_COMPARATOR | - |
| 14-weeks optional open-label period following the double-blind period - Difelikefalin | EXPERIMENTAL | - |
| Single arm | EXPERIMENTAL | - |
| 1-week, single arm, open label treatment phase | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| Difelikefalin Injection | DRUG | Participants receive Difelikefalin three times a week (0.5 micrograms/kg dry body weight). Difelikefalin is administered by intravenous bolus injection into the venous line of the dialysis circuit at the end of each dialysis. |
| Placebo Injection | DRUG | Participants receive Placebo three times a week (0.5 micrograms/kg dry body weight). Placebo is administered by intravenous bolus injection into the venous line of the dialysis circuit at the end of each dialysis. |
| Difelikefalin | DRUG | The study includes a screening period of up to 4 weeks (including a 7-day run-in period during the week prior to enrolment), a study treatment period of 12 weeks, and a safety follow-up visit at 7 (up to 10) days after EoT. Total study duration for a single participant is up to 17 to 18 weeks. |
Inclusion Criteria: * Subjects with chronic kidney disease (CKD) on HD 3 times weekly for ≥12 weeks prior to the informed consent procedure (including the date of informed consent) who can continue HD without changing its frequency or method. * If female, is not pregnant, or nursing. * If female: ...
Difelikefalin is an investigational small molecule being developed for pruritus, including uremic pruritus in patients with end stage renal disease on hemodialysis. It is also being studied in adolescents with moderate-to-severe pruritus associated with chronic kidney disease.
Difelikefalin is being developed by Fresenius Medical Care AG, a company listed on the New York Stock Exchange under the ticker symbol FMS.
Difelikefalin is in clinical development. It has completed a Phase 3 study in Chinese adults on hemodialysis with moderate-to-severe pruritus, completed a Phase 1 pharmacokinetic study, and is currently recruiting for a Phase 2 study in adolescents.
Difelikefalin has three clinical trials. NCT05885737 is a completed Phase 3 study in Chinese adults with uremic pruritus. NCT05885763 is a completed Phase 1 pharmacokinetic study in Chinese adults on hemodialysis. NCT06593392 is a recruiting Phase 2 study in adolescents with pruritus.
Difelikefalin is not FDA approved. It is an investigational drug currently in clinical development, with trials ranging from Phase 1 to Phase 3, and has not received marketing approval.
Difelikefalin is a small molecule therapeutic. Its specific molecular target has not been disclosed in the available clinical trial information.