Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Label indication GILENYA is indicated for the treatment of relapsing forms of multiple sclerosis (MS), to include clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease, in patients 10 years of age and older. FDA label
Also known as Fingolimod, Gilenya®
FTY720 · 26 trials · 10 indications
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).
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).
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.
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
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.
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.
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.
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.
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.
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.
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).
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.
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.
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.
Brain Magnetic Resonance Imaging (MRI) was performed at Month 3 and Month 6. Gadolinium-enhancing lesions (active lesions) represent acute inflammatory activity only and dissipate within 2-8 weeks of appearance. MRI scans were analyzed by blinded readers at the central MRI Evaluation Center to ensure consistency. Any Gd-enhanced T1 weighted MRI data obtained less than 14 days after the steroid used to treat MS relapses is invalid and excluded.
Total number of post-baseline Gd-enhanced lesions is calculated as a sum of all Gd-enhanced lesions seen on post-baseline scans per visit. Real (not per slice) lesions are counted in this analysis.
Total number of post-baseline Gd-enhanced lesions is calculated as a sum of all Gd-enhanced lesions seen on post-baseline scans per visit. Real (not per slice) lesions are counted in this analysis.
Total number of post-baseline Gd-enhanced lesions is calculated as a sum of all Gd-enhanced lesions seen on post-baseline scans per visit. Real (not per slice) lesions are counted in this analysis.
Total number of post-baseline Gd-enhanced lesions is calculated as a sum of all Gd-enhanced lesions seen on post-baseline scans per visit. Real (not per slice) lesions are counted in this analysis. The last observation was the last observation available for each patient which ranged from 1 to 2801 days
| Arm | Type | Description |
|---|---|---|
| ofatumumab - 20 mg injection/ placebo | EXPERIMENTAL | Ofatumumab 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/ placebo | EXPERIMENTAL | Siponimod 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/ placebo | ACTIVE_COMPARATOR | Fingolimod capsule administered orally once daily at a dose of either 0.5 mg or 0.25 mg (depending on patient's body weight). |
| Fingolimod | EXPERIMENTAL | Fingolimod was administered orally once daily at a dose of either 0.5 mg or 0.25 mg (depending on patient's body weight) with the aim to achieve systemic exposure in range of that in adults at the licensed 0.5 mg dose. Participants in this arm during core continued into extension and received open-label treatment |
| Interferon beta-1a | ACTIVE_COMPARATOR | An intramuscular (IM) injection of Interferon beta-1a was administered once weekly during core phase. Participants switched to receive open-label fingolimod in extension phase |
| Fingolimod-Younger Cohort | EXPERIMENTAL | The 'younger cohort' refers to the new pediatric patients to be recruited in the extension phase who fulfill any single one or a combination of the following criteria: being ≤12 years of age, or weighing ≤40 kg, or being prepubertal (i.e. pubertal status of Tanner stage \<2) |
| Fingolimod (FTY720) | EXPERIMENTAL | Participants received Fingolimod 0.5 mg orally once daily. |
| Placebo | PLACEBO_COMPARATOR | Participants received matching placebo to Fingolimod orally once daily. |
| Fingolimod 0.5 mg/day | EXPERIMENTAL | Open-label fingolimod 0.5 mg, taken orally once daily |
| FTY720 | EXPERIMENTAL | - |
| Fingolimod 1.25 mg | EXPERIMENTAL | Patients 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 mg | EXPERIMENTAL | Patients continued the same dose to which they had been randomized in the Core study, fingolimod 0.5 mg/day, in this Extension study. |
| Placebo-fingolimod | EXPERIMENTAL | Patients 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 mg | EXPERIMENTAL | Patients randomized to placebo in the Core study were re randomized to fingolimod 1.25 mg/day in this Extension study. |
| Placebo-fingolimod 0.5 mg | EXPERIMENTAL | Patients randomized to placebo in the Core study were re randomized to fingolimod 0.5 mg/day in this Extension study. |
| Interferon β-1a 30 µg | ACTIVE_COMPARATOR | - |
| FTY720 5 mg + reduced-dose Neoral (RDN) + corticosteroids, | EXPERIMENTAL | - |
| FTY720 2.5 mg + full dose Neoral (FDN) + corticosteroids | EXPERIMENTAL | - |
| MMF 2 g + full-dose Neoral (FDN) + corticosteroids | EXPERIMENTAL | - |
| 1 | EXPERIMENTAL | - |
| 2 | PLACEBO_COMPARATOR | - |
| FTY720 0.5 mg | EXPERIMENTAL | FTY720 |
| FTY720 1.25 mg | EXPERIMENTAL | FTY720 |
| Fingolimod (FTY720) 1.25 mg/day | EXPERIMENTAL | Core study: patients received fingolimod 1.25 mg, once daily for 6 months. Extension: In dose -blind period and open label, fingolimod 1.25 mg once daily for 9-18 months (6 months to 24 months). Later, all patients converted to fingolimod 0.5 mg, once daily for rest of the study participation. |
| Placebo/Fingolimod (FTY720) | PLACEBO_COMPARATOR | Core study: patients received placebo, once daily for 6 months. Extension: In dose-blind period patients were re-randomized into either fingolimod 1.25 mg or 5.0 mg once per day for 6-15 months. In open-label period patients received fingolimod 1.25 mg once per day for 15 to 24 months. Later, all patients converted to fingolimod 0.5 mg, once daily for rest of the study participation. |
| Fingolimod (FTY720) 5.0 mg/day | EXPERIMENTAL | Core study: patients received fingolimod 5.0 mg, once daily for 6 months. Extension: In dose-blind period fingolimod 5.0 mg once daily for 6-15 months. For open-label phase 15 to 24 months 1.25mg once daily. Later, all patients converted to fingolimod 0.5 mg, once daily for rest of the study participation. |
| Name | Type | Description |
|---|---|---|
| Fingolimod | DRUG | Fingolimod capsule administered orally once daily at a dose of either 0.5 mg or 0.25 mg (depending on patient's body weight). |
| Ofatumumab | DRUG | Ofatumumab 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 | DRUG | Siponimod 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 placebo | OTHER | Fingolimod matching placebo capsule |
| Siponimod placebo | OTHER | Siponimod matching placebo tablet |
| Ofatumumab placebo | OTHER | Ofatumumab matching placebo autoinjector |
| Interferon beta-1a | DRUG | Administration once weekly via i.m. injections. |
| Placebo capsule | DRUG | Matching placebo capsule required for double-dummy masking to blind formulations. |
| Placebo i.m. injection | DRUG | Matching placebo i.m. injection required for double-dummy masking to blind formulations. |
| Placebo Comparator | DRUG | Matching placebo capsules |
| Placebo | DRUG | Matching placebo capsules for oral administration. |
| Seasonal influenza vaccine | BIOLOGICAL | Commercially available injectable influenza vaccine for the 2010/11 influenza season. |
| Tetanus toxoid vaccine | BIOLOGICAL | Commercially available tetanus toxoid vaccine booster injection. |
| FTY720 | DRUG | - |
| Fingolimod 0.5 mg | DRUG | Patients self-administered fingolimod 0.5 mg capsules orally once daily. |
| Fingolimod 1.25 mg | DRUG | Patients self-administered fingolimod 1.25 mg capsules orally once daily. |
| Interferon β-1a 30 µg | DRUG | Core: 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. |
| FTY720 5 mg + reduced-dose Neoral (RDN) + corticosteroids | DRUG | FTY720 5 mg + reduced-dose Neoral (RDN) + corticosteroids |
| FTY720 2.5 mg + full dose Neoral (FDN) + corticosteroids | DRUG | FTY720 2.5 mg + full dose Neoral (FDN) + corticosteroids |
| MMF 2 g + full-dose Neoral (FDN) + corticosteroids | DRUG | mycophenolate mofetil (MMF) + full-dose Neoral (FDN) + corticosteroids |
| FTY 720 | DRUG | - |
| fingolimod (FTY720) | DRUG | - |
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 ...
FTY720 is an investigational small molecule developed by Novartis AG. It has been studied in clinical trials for relapsing multiple sclerosis, multiple sclerosis, asthma, kidney transplantation, and renal transplantation. It is not described as an approved product and remains in clinical development, with Phase 2 studies among the trials listed.
FTY720 has been studied as a treatment for relapsing multiple sclerosis and multiple sclerosis. Trials include a Phase 3 study in relapsing MS with 2,417 patients (NCT01127750) and Phase 2 studies in Japan (NCT00537082) and Europe and Canada (NCT00333138). These studies are completed.
FTY720 is developed by Novartis AG, which trades under the ticker NVS. Novartis is the sponsor associated with the clinical trial program for FTY720 across multiple sclerosis, asthma, and transplantation indications.
FTY720 has been tested in Phase 2 and Phase 3 clinical trials, and the program is described as being at Phase 2. All six listed trials are completed, and none are currently active. FTY720 is investigational and is not described as an FDA-approved product.
Completed FTY720 trials include NCT01127750, a Phase 3 study in relapsing multiple sclerosis with 2,417 patients; NCT00785083, a Phase 2 asthma study with 36 patients; NCT00537082, a Phase 2 multiple sclerosis study in Japan with 171 patients; and NCT00333138, a Phase 2 multiple sclerosis study with 281 patients.
Yes. FTY720 is also written as FTY 720. In transplantation research it has additionally appeared as FTY720 5 mg plus reduced-dose Neoral and corticosteroids, and as FTY720 plus reduced-dose Neoral plus corticosteroids. These refer to the same agent studied under different regimen descriptions.