Efficacy
2-year planned treatment period completed
Efficacy Results
Results with OJEMDA
Clinically meaningful tumor shrinkage for your patients1
Response rates per RAPNO-LGG criteria
53%
ORR
95% CI: 41, 64 (n=40/76)
Response rates
Minor response (MR): 14% (n=11)
Partial response (PR): 38% (n=29)
No complete responses were seen using RAPNO-LGG criteria
More than half of patients experienced tumor shrinkage with OJEMDA1
- The waterfall plot represents a prespecified exploratory analysis from the FIREFLY-1 trial. Depth of tumor shrinkage is derived from a descriptive analysis of data from this single-arm study2
- This is an exploratory analysis. Findings should be regarded as descriptive, exploratory, and interpreted with caution2
- The change in tumor volume shown in the waterfall plot represents the best change in SPPD of target lesions at any individual time point vs baseline. These changes do not necessarily reflect RAPNO response2
- Responses indicated on the plot were based on the RAPNO criteria and were confirmed by 2 consecutive MRI scans ≥28 days apart2
47% median reduction in tumor size from baseline in FIREFLY-1 (range: -98.1% to 106.3%)2
Time to Response & Duration of Response
OJEMDA: Rapid and durable tumor responses1,2
Tumor response was maintained for well over a year
57% (44/77) of patients completed 2 years of treatment with OJEMDA2
Additional information about tumor response
In the clinical trial, patients who had radiographic evidence of disease progression were allowed to continue tovorafenib treatment if, in the opinion of the investigator, the patient was deriving clinical benefit from continuing trial treatment.
- 13 patients were categorized as having progressive disease per RAPNO retrospectively and were allowed to continue treatment on tovorafenib2
- 6 out of those 13 patients were observed to have tumor shrinkage2
- This analysis is descriptive, exploratory, and should be interpreted with caution
Continue once-weekly dosing until disease progression or intolerable toxicity.
Response by prior MAPKi exposure1,2
| In exploratory analyses of prior therapies | Overall response rate |
|---|---|
No prior MAPK therapy (n=31) | 55% (95% CI: 36, 73) |
Prior MAPK therapy (n=45) | 51% (95% CI: 36, 66) |
This exploratory analysis was not statistically powered to evaluate these outcomes. Findings should be regarded as descriptive, exploratory, and interpreted with caution.
Response by prior line of therapy2
| Exploratory subgroup analysis by prior LoT | Overall response rate |
|---|---|
1 + 2 prior LoT (n=37) | 57% (95% CI: 40, 73) |
≥3 prior LoT (n=39) | 49% (95% CI: 32, 65) |
These exploratory analyses were not statistically powered to evaluate these outcomes. Findings should be regarded as descriptive, exploratory, and interpreted with caution.
About RAPNO-LGG
OJEMDA is FDA approved based on RAPNO-LGG criteria
FIREFLY-1 was the first pLGG study to use RAPNO-LGG for evaluating treatment response1-4
The RAPNO-LGG criteria were designed specifically for pediatric tumors, and they account for the unique heterogeneity of pLGG.
RAPNO criteria for assessment of tumor response in pLGG
| Type of response | Definition |
|---|---|
| Complete response | Complete disappearance of the target lesion and all areas of metastatic disease on MRI |
| Partial response | ≥50% reduction in the target lesion |
| Minor response | 25-49% reduction in the target lesion |
| Stable disease | <25% increase or <25% decrease in the target lesion |
| Progressive disease | ≥25% increase in target lesion, new lesion, visual progression, or clinical deterioration |
OJEMDA was generally well tolerated in
See safety information
Patient Cases
See why OJEMDA was appropriate for these patients from FIREFLY-12
These patient cases are from the FIREFLY-1 clinical trial. Each patient presented in these cases completed 2 years of treatment with OJEMDA. Data presented are the results seen in individual cases; individual results may vary.
3-year-old male, tumor located in deep midline structure, BRAF fusion
Initial diagnosis
Initially presented at age 2 and underwent sub-total tumor resection
Prior treatment history
- Patient received 2 prior lines of therapy
- 1L was a combination of anti-VEGF therapy and a vinca alkaloid for 7 months, responded before experiencing progression
- 2L platinum agent was given for ~5 months, with a best response of progressive disease
- Radiographic progression occurred in deep midline structures 3 weeks after stopping 2L treatment
- At time of radiographic progression, patient also experienced clinical symptoms, ie, left hemiplegia, which limited physical activities
Patient enrolled in FIREFLY-1 with OJEMDA one month after disease progression
- Dose: OJEMDA 420 mg/m2 (total 300 mg)
- Patient completed 2 years of treatment with OJEMDA
- Patient entered posttreatment observation period after completion of 26 treatment cycles
Efficacy and safety data for this patient are as of May 10, 2024; follow-up is currently ongoing.
Results
May 10, 2024 data cutoff from FIREFLY-1
- Partial response per RAPNO-LGG (-69.8% best change SPPD)
- PR was defined as a ≥50% reduction in the target lesion by blinded independent central review based on RAPNO-LGG criteria4
Scan images
Best overall response was defined as the best response (CR, PR, MR, stable disease, PD, and not evaluable) recorded from the start of study treatment until the start of any subsequent anticancer therapy or disease progression/recurrence or death.2
Adverse events experienced by the patient2
| Management strategy | ||
|---|---|---|
| Adverse event | Highest grade experienced | Management taken |
| Reduction in growth velocity | Grade 3 |
|
| Viral upper respiratory tract infection | Grade 3 |
|
| Otitis media | Grade 3 |
|
| Otitis externa | Grade 3 | |
| Sepsis | Grade 3 | |
| Device-related infection | Grade 3 | |
Additional AEs experienced that did not require dose modifications:
Grade 1 drug eruption, anemia, vomiting, lymphocyte count decreased, skin exfoliation, blood phosphokinase increased, white blood cell count decreased, pain, blood lactate dehydrogenase increased, hair color changes, viral infection, vomiting, pyrexia, aspartate aminotransferase increased, alopecia, COVID-19, upper respiratory tract infection, hypophosphatemia, enterovirus infection, rhinovirus infection, fatigue, adenovirus infection, platelet count decreased, diarrhea, parainfluenza virus infection, lymphocyte count decreased, ear pain, hypocalcemia, hypophosphatemia, hypoalbuminemia, hypokalemia.
Grade 2 anemia, diarrhea, enterocolitis infection, hyperkalemia, hypoalbuminemia.
Adverse events did not require a dose reduction, and the patient completed the full course of treatment.
Results representative of this patient’s experience. Individual results may vary.
Changes in height on and off OJEMDA treatment1,2
OJEMDA can cause reductions in growth velocity. Growth velocity improved after interruption of treatment with OJEMDA. Routinely monitor patient growth during treatment with OJEMDA.
Growth velocity improved after treatment completion with OJEMDA1
1L=first-line; 2L=second-line; AE=adverse event; BRAF=v-Raf murine sarcoma viral oncogene homolog B1; CI=confidence interval; CR=complete response; DOR=duration of response; FDA=Food and Drug Administration; LoT=line of therapy; MAPK=mitogen-activated protein kinase; MAPKi=mitogen-activated protein kinase inhibitor; MR=minor response; MRI=magnetic resonance imaging; NR=not reported; ORR=overall response rate; PD=progressive disease; pLGG=pediatric low-grade glioma; PR=partial response; R/R=relapsed/refractory; RAPNO=Response Assessment in Pediatric Neuro-Oncology; RAPNO-LGG=Response Assessment in Pediatric Neuro-Oncology for Low-Grade Glioma; SPPD=sum of the products of the perpendicular diameters; TTR=time to response; VEGF=vascular endothelial growth factor.
OJEMDA has a generally well-tolerated safety profile
See the safety profile