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Fingolimod

Phase 3

Chronic Inflammatory Demyelinating Polyradiculoneuropathy | Small molecule | Neurology |Novartis AG|Last Updated: May 14, 2026

Target and mechanism

Molecular targetS1PR5, S1PR2, S1PR4, S1PR3, S1PR1
Target classAgonist
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment106

FDA Designations

No designations recorded

Clinical trial landscape

Fingolimod · 11 trials · 7 indications

Phase 3 9Phase 2 1Phase 1 1
NCT04926818Efficacy and Safety of Ofatumumab and Siponimod Compared to Fingolimod in Pediatric Patients With Multiple SclerosisMultiple Sclerosis (MS)
ACTIVE NOT_RECRUITING129 Analytics
NCT01625182Evaluate Efficacy and Safety of Fingolimod 0.5 mg Orally Once Daily Versus Placebo in Chronic Inflammatory Demyelinating Polyradiculoneuropathy Patients.Chronic Inflammatory Demyelinating Polyradiculoneuropathy
COMPLETED106 Analytics
NCT01497262Safety and Tolerability of Fingolimod in Patients With Relapsing-remitting Multiple SclerosisMultiple Sclerosis
COMPLETED162 Analytics
NCT01201356Long-term Safety and Tolerability of 0.5 mg Fingolimod in Patients With Relapsing Forms of Multiple SclerosisRelapsing Forms of Multiple Sclerosis
COMPLETED4,125 Analytics
NCT01199861Effect of Treatment With Fingolimod on the Immune Response Following Seasonal Flu Vaccination and Tetanus Booster Injection in Patients With Relapsing Multiple Sclerosis (MS)Relapsing Multiple Sclerosis
COMPLETED138 Analytics
NCT00662649Long-term Efficacy and Safety of Fingolimod (FTY720) in Patients With Relapsing-remitting Multiple SclerosisMultiple Sclerosis
COMPLETED920 Analytics
NCT00355134Efficacy and Safety of Fingolimod (FTY720) in Patients With Relapsing-remitting Multiple SclerosisMultiple Sclerosis
COMPLETED1,083 Analytics
NCT00340834Efficacy and Safety of Fingolimod in Patients With Relapsing-remitting Multiple Sclerosis With Optional Extension PhaseMultiple Sclerosis
COMPLETED1,292 Analytics
NCT00289978Efficacy and Safety of Fingolimod in Patients With Relapsing-remitting Multiple SclerosisRelapsing-remitting Multiple Sclerosis
COMPLETED1,272 Analytics
PHASE3ACTIVE NOT_RECRUITING
Efficacy and Safety of Ofatumumab and Siponimod Compared to Fingolimod in Pediatric Patients With Multiple Sclerosis
Multiple Sclerosis (MS)Unlock trial analytics
PHASE3COMPLETED
Evaluate Efficacy and Safety of Fingolimod 0.5 mg Orally Once Daily Versus Placebo in Chronic Inflammatory Demyelinating Polyradiculoneuropathy Patients.
Chronic Inflammatory Demyelinating PolyradiculoneuropathyUnlock trial analytics
PHASE3COMPLETED
Safety and Tolerability of Fingolimod in Patients With Relapsing-remitting Multiple Sclerosis
Multiple SclerosisUnlock trial analytics
PHASE3COMPLETED
Long-term Safety and Tolerability of 0.5 mg Fingolimod in Patients With Relapsing Forms of Multiple Sclerosis
Relapsing Forms of Multiple SclerosisUnlock trial analytics
PHASE3COMPLETED
Effect of Treatment With Fingolimod on the Immune Response Following Seasonal Flu Vaccination and Tetanus Booster Injection in Patients With Relapsing Multiple Sclerosis (MS)
Relapsing Multiple SclerosisUnlock trial analytics
PHASE3COMPLETED
Long-term Efficacy and Safety of Fingolimod (FTY720) in Patients With Relapsing-remitting Multiple Sclerosis
Multiple SclerosisUnlock trial analytics
PHASE3COMPLETED
Efficacy and Safety of Fingolimod (FTY720) in Patients With Relapsing-remitting Multiple Sclerosis
Multiple SclerosisUnlock trial analytics
PHASE3COMPLETED
Efficacy and Safety of Fingolimod in Patients With Relapsing-remitting Multiple Sclerosis With Optional Extension Phase
Multiple SclerosisUnlock trial analytics
PHASE3COMPLETED
Efficacy and Safety of Fingolimod in Patients With Relapsing-remitting Multiple Sclerosis
Relapsing-remitting Multiple SclerosisUnlock trial analytics

Study Endpoints

Primary Endpoints

Annualized relapse rate (ARR) in target pediatric participants
Baseline up to 24 months

Frequency of relapses assessed by the annualized relapse rate (ARR). The ARR is defined as the average number of confirmed relapses per year (total number of confirmed relapses divided by the total days in the study multiplied by 365.25).

Time to First Confirmed Worsening on the Adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Scale
Month 12

Confirmed worsening in CIDP was measured by the adjusted INCAT Disability Scale. The adjusted INCAT disability scale measures arm disability and leg disability. For arm disability the scale ranges from 0 (no upper limb problems) to 5 (inability to use either arm for any purposeful movement). The leg disability scale ranges from 0 (walking not affected) to 5 (restricted to wheelchair, unable to stand and walk a few steps with help). The total adjusted INCAT disability score is calculated by the sum of the arm and leg disability scores where the total score ranges from 0 to 10. A confirmed worsening was defined as an increase by 1 or more points on the adjusted INCAT disability scale from the value at baseline.

Number of Participants With Adverse Events as a Measure of Safety and Tolerability
28 weeks

Any Adverse Event was defined as occurrence of any symptom regardless of intensity grade, Serious Adverse Event (SAEs) assessed as medical occurrences that result in death, are life threatening, require hospitalization or prolongation of hospitalization or result in persistent or significant disability/incapacity

Parts I and II: Number of Participants With Adverse Events, Serious Adverse Event, and Death
Baseline (Part I) to Month 6 Follow-up (Part II), up to 8 years

Analysis of absolute and relative frequencies for Adverse Event (AE), Serious Adverse Event (SAE) and Deaths by primary System Organ Class (SOC) to demonstrate that Fingolimod 0.5 mg/day is safe in patients with relapsing forms of Multiple Sclerosis (MS) through the monitoring of relevant clinical and laboratory safety parameters. Only descriptive analysis performed.

Immune Response 3 Weeks After Seasonal Influenza Vaccination
Week 6 (pre-vaccination) and 3 weeks after vaccination (Study week 9)

Percentage of participants who responded to treatment with the seasonal influenza vaccine 3 weeks after vaccination. Response was defined as patients fulfilling one of the following criteria for at least one of the three strains contained in the seasonal influenza vaccine: * Seroconversion: The pre-vaccination antibody titer measurement was \<1:10 and the post-vaccination measurement is ≥1:40. * Significant increase in antibody titer: The pre-vaccination antibody titer measurement was ≥1:10 and the increase in antibody titer from this to the post-vaccination measurement is ≥ 4-fold.

Annualized Aggregate Relapse Rate (ARR) During Months 0 to End of Study(Core [CFTY720D2301/NCT00289978] and Extension Study)
Months 0 to end of study (maximum up to 60 months)

ARR is defined as the number of confirmed relapses in a year. A relapse is defined as the appearance of a new or worsening of a previously stable or improving pre-existing neurological abnormality, separated by at least 30 days from onset of a preceding relapse. The abnormality must be present for at least 24 hours and occur in the absence of fever or infection. The annualized ARR for each treatment group was the mean of the annualized ARRs for all patients in the group, calculated as the total number of confirmed relapses divided by the total number of days on study multiplied by 365.25.

Time to First Confirmed Relapse up to End of Study: Kaplan-Meier Estimate of Percentage of Patients Relapse-free
Core baseline to end of study (maximum up to 60 months)

A relapse was confirmed when it was accompanied by an increase of at least half a step (0.5) on the Expanded Disability Status Scale (EDSS) or an increase of 1 point on two different Functional Systems (FS) of the EDSS or 2 points on one of the FS (excluding Bowel/Bladder or Cerebral FS). Kaplan-Meier estimates of the percentage of relapse-free patients at end of study and and 95% confidence intervals (CIs) were presented for the treatment groups.

Annualized Aggregate Relapse Rate (ARR) During Months 0-24 (Core Study) and Months 24-48 (Extension Study)
Months 0-24 (core study) and Months 24-48 (extension study)

ARR is defined as the number of confirmed relapses in a year. A relapse is defined as the appearance of a new or worsening of a previously stable or improving pre-existing neurological abnormality, separated by at least 30 days from onset of a preceding relapse. The abnormality must be present for at least 24 hours and occur in the absence of fever or infection. The annualized ARR for each treatment group was the mean of the annualized ARRs for all patients in the group, calculated as the total number of confirmed relapses divided by the total number of days on study multiplied by 365.25.

Change (Expressed as Ratio) in the Annualized Aggregate Relapse Rate (ARR) From Months 0-24 (Core Study) to Months 24-48 (Extension Study)
Months 0-24 (core study) and Months 24-48 (extension study)

ARR is defined as the number of confirmed relapses in a year. A relapse is defined as the appearance of a new or worsening of a previously stable or improving pre-existing neurological abnormality, separated by at least 30 days from onset of a preceding relapse. The abnormality must be present for at least 24 hours and occur in the absence of fever or infection. The annualized ARR for each treatment group was the mean of the annualized ARRs for all patients in the group, calculated as the total number of confirmed relapses divided by the total number of days on study multiplied by 365.25.

Aggregate Annualized Relapse Rate (ARR) Estimate up to Month 24
24 months

ARR is the average number of relapses in a year calculated by negative binomial regression as the sum of confirmed relapses of all patients in the group divided by the sum of the number of days on study of all patients in the group and multiplied by 365.25. A relapse was defined as the appearance of a new neurological abnormality or worsening of previously stable or improving pre-existing neurological abnormality, separated by at least 30 days from onset of a preceding clinical demyelinating event. The abnormality must be present for at least 24 hours and occur in the absence of fever (\<37.5C) or known infection. A relapse must be confirmed by the Independent Evaluating Physician (examining neurologist). ARR estimates were calculated from a negative binomial regression model adjusted for treatment, pooled center, number of relapses in the previous 2 years prior to enrollment, and Baseline expanded disability status scale (EDSS).

Estimated Annualized Aggregate Relapse Rate (ARR) in the Core Phase of the Study
Baseline to Month 12

The ARR is defined as the number of confirmed relapses in a year. A relapse is defined as the appearance of a new or worsening of a previously stable or improving pre existing neurological abnormality, separated by at least 30 days from onset of a preceding relapse. The abnormality must be present for at least 24 hours and occur in the absence of fever or infection. The annualized ARR for each treatment group was calculated using negative binomial regression adjusted by treatment, country, number of relapses in the previous 2 years, and the baseline Expanded Disability Status Scale score.

Estimated Annualized Aggregate Relapse Rate (ARR)
Baseline to end of study (Month 24)

The ARR is defined as the number of confirmed relapses in a year. A relapse is defined as the appearance of a new or worsening of a previously stable or improving pre-existing neurological abnormality, separated by at least 30 days from onset of a preceding relapse. The abnormality must be present for at least 24 hours and occur in the absence of fever or infection. The annualized ARR for each treatment group was the mean of the annualized ARRs for all patients in the group calculated as the total number of confirmed relapses divided by the total number of days on study, multiplied by 365.25.

Percentage of Patients Free of Gd-enhanced T1 Weighted Magnetic Resonance Imaging (MRI) Lesions
Months 6, 9, 12, 18, 24, 36, and 48

To ensure consistency, MRI scans were evaluated centrally at the Institute of Neurotherapeutics in Kyoto, Japan. After checking the scans for completeness and quality, all scans were analyzed by blinded readers (experienced neurologists). The number of Gd-enhanced T1 weighted MRI lesions were counted and recorded. Lesions expanding through several slices were counted as only 1 lesion.

PK profile comparison between healthy volunteers and severe renal impaired patients, 3 weeks

Secondary Endpoints

Annualized relapse rate (ARR) as compared to historical interferon β-1a data
Baseline up to 24 months
Annualized T2 lesion rate
Baseline up to 24 months
Neurofilament light chain (NfL) concentrations
Day 1, Months 3,6,12,18,24
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
ofatumumab - 20 mg injection/ placeboEXPERIMENTALOfatumumab as a solution for injection in an autoinjector containing 20 mg ofatumumab (50 mg/mL, 0.4 mL content) for subcutaneous administration. A loading dose at Day1, Day 7 and Day 14 and then injections every 4 weeks/ 6 weeks (depending on patient's body weight).
siponimod - 0.5 mg, 1 mg or 2 mg/ placeboEXPERIMENTALSiponimod tablet administered orally once daily. Titration period, Day 1 to Day 6, first dose is either 0.1 mg or 0.25 mg up to daily dose of either 0.5 mg, 1 mg or 2 mg (depending on CYP2C9 genotype and body weight).
fingolimod - 0.5 mg or 0.25 mg/ placeboACTIVE_COMPARATORFingolimod capsule administered orally once daily at a dose of either 0.5 mg or 0.25 mg (depending on patient's body weight).
Fingolimod (FTY720)EXPERIMENTALParticipants received Fingolimod 0.5 mg orally once daily.
PlaceboPLACEBO_COMPARATORParticipants received matching placebo to Fingolimod orally once daily.
FingolimodEXPERIMENTALOpen-label fingolimod 0.5 mg, taken orally once daily for 4 months
Fingolimod 0.5 mg/dayEXPERIMENTALOpen-label fingolimod 0.5 mg, taken orally once daily
Fingolimod 1.25 mgEXPERIMENTALPatients continued the same dose to which they had been randomized in the Core study (CFTY720D2301/NCT00289978), fingolimod 1.25 mg/day, in this Extension study.
Fingolimod 0.5 mgEXPERIMENTALPatients continued the same dose to which they had been randomized in the Core study, fingolimod 0.5 mg/day, in this Extension study.
Placebo-fingolimodEXPERIMENTALPatients randomized to placebo in the Core study were re randomized to fingolimod (either 0.5 or 1.25 mg/day) in this Extension study.
Placebo-fingolimod 1.25 mgEXPERIMENTALPatients randomized to placebo in the Core study were re randomized to fingolimod 1.25 mg/day in this Extension study.
Placebo-fingolimod 0.5 mgEXPERIMENTALPatients randomized to placebo in the Core study were re randomized to fingolimod 0.5 mg/day in this Extension study.
Interferon β-1a 30 µgACTIVE_COMPARATOR -
1EXPERIMENTAL -

Interventions

NameTypeDescription
FingolimodDRUGFingolimod capsule administered orally once daily at a dose of either 0.5 mg or 0.25 mg (depending on patient's body weight).
OfatumumabDRUGOfatumumab as a solution for injection in an autoinjector containing 20 mg ofatumumab (50 mg/mL, 0.4 mL content) for subcutaneous administration. A loading dose at Day1, Day 7 and Day 14 and then injections every 4 weeks/ 6 weeks (depending on patient's body weight).
SiponimodDRUGSiponimod tablet administered orally once daily. Titration period, Day 1 to Day 6, first dose is either 0.1 mg or 0.25 mg up to daily dose of either 0.5 mg, 1 mg or 2 mg (depending on CYP2C9 genotype and body weight).
Fingolimod placeboOTHERFingolimod matching placebo capsule
Siponimod placeboOTHERSiponimod matching placebo tablet
Ofatumumab placeboOTHEROfatumumab matching placebo autoinjector
Placebo ComparatorDRUGMatching placebo capsules
PlaceboDRUGMatching placebo capsules for oral administration.
Seasonal influenza vaccineBIOLOGICALCommercially available injectable influenza vaccine for the 2010/11 influenza season.
Tetanus toxoid vaccineBIOLOGICALCommercially available tetanus toxoid vaccine booster injection.
Fingolimod 0.5 mgDRUGPatients self-administered fingolimod 0.5 mg capsules orally once daily.
Fingolimod 1.25 mgDRUGPatients self-administered fingolimod 1.25 mg capsules orally once daily.
Interferon β-1a 30 µgDRUGCore: Patients self-administered interferon β-1a 30 μg in an intramuscular (im) injection once weekly. In addition, they self-administered a fingolimod placebo capsule orally once daily. Extension: Patients self-administered either fingolimod 1.25 mg or 0.5 mg capsules orally once daily until switched to 0.5 mg capsules upon study protocol amendment.
fingolimod (FTY720)DRUG -
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Eligibility Criteria

Age Range10 Years to 17 Years
SexALL
Healthy VolunteersNo
Study Sites51

Inclusion Criteria: 1. Between 10 to \<18 years of age (i.e., have not yet had their 18th birthday) at randomization 2. Diagnosis of multiple sclerosis 3. EDSS score of 0 to 5.5, inclusive 4. At least one MS relapse/attack during the previous year or two MS relapses in the previous two years prior ...

Countries:United StatesArgentinaAustraliaAustriaBelgiumBrazilCanadaChileCroatiaEstoniaFranceGermanyGuatemalaIndiaIsraelItalyLatviaMexicoPolandPortugalSerbiaSlovakiaSpainTaiwanTurkey (Türkiye)CzechiaGreeceJapanNetherlandsUnited KingdomColombiaJordanMalaysiaPanamaPeruDenmarkEgyptFinlandHungaryIrelandNorwayRomaniaRussiaSouth AfricaSouth KoreaSwedenSwitzerlandLithuania
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Frequently asked questions about Fingolimod

What is Fingolimod used for?

Fingolimod is used for chronic inflammatory demyelinating polyradiculoneuropathy and multiple sclerosis, including relapsing forms of multiple sclerosis and relapsing-remitting multiple sclerosis. It is also studied in patients with renal insufficiency. Fingolimod is a small molecule being developed by Novartis AG for neurological conditions.

How does Fingolimod work?

Fingolimod is a small molecule that modulates sphingosine 1-phosphate receptors, which are involved in lymphocyte trafficking. By preventing lymphocytes from leaving lymph nodes, it reduces the number of circulating immune cells that can attack the central nervous system, thereby decreasing inflammation and demyelination in multiple sclerosis.

Who makes Fingolimod?

Fingolimod is developed by Novartis AG, a multinational pharmaceutical company listed on the New York Stock Exchange under the ticker symbol NVS. Novartis is conducting clinical trials to evaluate the drug's efficacy and safety in multiple sclerosis and other neurological conditions.

What phase is Fingolimod in?

Fingolimod is in Phase 3 clinical development for multiple sclerosis and related conditions. It has completed multiple Phase 3 trials, including studies in relapsing-remitting multiple sclerosis. The drug is investigational and has not been confirmed as approved based on the available data.

What clinical trials is Fingolimod in?

Fingolimod has been studied in several clinical trials, including NCT00289978, NCT00355134, and NCT00662649, all Phase 3 trials in relapsing-remitting multiple sclerosis. NCT00670449 is a Phase 2 extension study in relapsing multiple sclerosis. These trials have enrolled over 3,400 patients across multiple countries.

Is Fingolimod the same as FTY720?

Yes, Fingolimod is also known as FTY720. Clinical trial records, such as NCT00355134 and NCT00662649, refer to the drug as Fingolimod (FTY720), confirming that these names identify the same investigational compound being developed by Novartis.