Approval Probability
TA Base Rate
Adjusted LOA
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Also known as Difelikefalin Injection
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 developed by Fresenius Medical Care AG (ticker FMS) for pruritus in patients with end stage renal disease on hemodialysis. It is also referred to as Difelikefalin Injection. The program spans uremic pruritus and chronic kidney disease related itching, with clinical activity in China, Europe, the Middle East and the United Kingdom.
Difelikefalin is being studied to treat moderate-to-severe pruritus, including uremic pruritus, in adults and adolescents with chronic kidney disease who are on hemodialysis. Its trials enroll patients with end stage renal disease on hemodialysis and pruritus, a condition where itching is driven by kidney failure rather than a primary skin disorder.
Difelikefalin targets OPRK1, the kappa opioid receptor, and acts as an agonist at that receptor. By activating OPRK1, the drug is designed to modulate itch signaling. OPRK1 agonism is the mechanism underlying its development for pruritus in dialysis-dependent kidney disease patients.
Fresenius Medical Care AG, which trades under the ticker FMS, is the developer of Difelikefalin. The company is running the clinical program for the asset, including studies in Chinese hemodialysis patients and a multinational adolescent study. Difelikefalin is also known as Difelikefalin Injection.
Difelikefalin is in clinical development and is not described as approved. Its program includes a completed Phase 3 study in Chinese adult hemodialysis patients with moderate-to-severe pruritus, a completed Phase 1 pharmacokinetic study in Chinese adults on hemodialysis, and a recruiting Phase 2 study in adolescents. The overall development stage listed for the asset is Phase 1.
Difelikefalin has been studied in NCT05885737, a completed Phase 3 trial of 260 Chinese adult hemodialysis patients with moderate-to-severe pruritus, and NCT05885763, a completed Phase 1 pharmacokinetic study of 30 Chinese adults on hemodialysis. NCT06593392 is a recruiting Phase 2 safety and tolerability study in adolescents aged 12 and older on hemodialysis with moderate-to-severe pruritus.
Yes. Difelikefalin Injection is an alternative name for Difelikefalin, so searches for either term refer to the same asset from Fresenius Medical Care AG. The injectable form is the presentation used in the hemodialysis and pruritus clinical program, including the Phase 1, Phase 2 and Phase 3 studies.