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OJEMDA (tovorafenib) logo: 100 mg tablets 25 mg/mL for oral suspension

For US healthcare professionals only

Efficacy

Efficacy Results

Results with OJEMDA

Major efficacy outcome: ORR using RAPNO-LGG criteria1

Overall response rate

- CR: 0% (n=0)

- PR: 36.8% (n=28)

- MR: 14.5% (n=11)

51%

95% CI: 40, 63 (n=39/76)

Secondary efficacy endpoint: CBR with SD ≥12 months1-3

CBR with SD ≥12 months

- ORR: 51.3% (n=39)

- SD ≥12 months: 5.2% (n=4)*

57%

(n=43/76)

  • Clinical benefit rate (CBR) measured by the proportion of patients with best overall response of CR, PR, MR, or SD lasting 12 months or more following initiation of tovorafenib. CBR was a prespecified secondary endpoint in FIREFLY-1

Descriptive analysis: CBR with SD at any length of time

82%

(n=62/76)

- ORR: 51.3% (n=39)

- SD of any length: 30.3% (n=23)*

  • Results of this analysis are descriptive in nature. No clinical or statistical conclusions can be drawn
  • Among patients with SD <12 months (n=19), 8 patients are continuing treatment, and 1 patient has entered a drug holiday at the time of data cutoff
  • Among patients with SD <12 months (n=19), 8 patients are continuing treatment, and 1 patient has entered a drug holiday at the time of data cutoff
*

SD is not a component of overall response rate and can reflect the natural history of disease rather than a direct therapeutic effect.

Patient Cases

See example patients who could be appropriate for OJEMDA

MEET EMILY

  • Emily was diagnosed 4 years ago with pLGG after a localized tumor in the optic pathway was suggested by MRI
  • Histology was consistent with pilocytic astrocytoma

Age: 6
Height: 3' 6"

Weight: 45 lb
Sex: F

Race/ethnicity: White/
Not Hispanic

GENOMIC TEST RESULTS

  • KIAA1549-BRAF fusion–positive

MEDICAL HISTORY

  • Diagnosed due to lethargy and decreased visual activity, including loss of sight (right eye)
  • Surgical resection was ruled out due to tumor’s location, but biopsy was recommended

PRIOR TREATMENT

  • Achieved partial response with chemotherapy (total treatment duration: 12 months)
  • Side effects of chemotherapy included anemia, grade 3 neutropenia, and neutropenic fever
  • Now shows signs of radiographic and clinical progression after 2 years in remission

WHY OJEMDA MAY BE RIGHT FOR PATIENTS LIKE EMILY

OJEMDA was studied in patients like Emily

LOCATION: 51% had an optic pathway glioma1

HISTOLOGY: 94% had tumors with astrocytic histology3

ALTERATION TYPE: 84% had a KIAA1549-BRAF fusion or rearrangement1

MEDIAN AGE: 8.5 years (range 2 to 21 years)1

Patients in the FIREFLY-1 trial saw clinically meaningful response rates1

Overall population (N=76):

  • ORR: 51% (95% CI: 40, 63)
  • Median TTR: 5.3 months (range 1.6-11.2 months)

Consider OJEMDA as your first choice for patients like Emily who progressed after chemotherapy.

BRAF=v-Raf murine sarcoma viral oncogene homolog B1; CBR=clinical benefit rate; CR=complete response; DOR=duration of response; MAPK=mitogen-activated protein kinase; MAPKi=mitogen-activated protein kinase inhibitor; MEK=mitogen-activated protein kinase kinase; MR=minor response; MRI=magnetic resonance imaging; NE=not estimable; ORR=overall response rate; pLGG=pediatric low-grade glioma; PR=partial response; RANO=Response Assessment in Neuro-Oncology; RANO-LGG=Response Assessment in Neuro-Oncology for Low-Grade Glioma; RAPNO=Response Assessment in Pediatric Neurology-Oncology; RAPNO-LGG=Response Assessment in Pediatric Neuro-Oncology for Low-Grade Glioma; R/R=relapsed/refractory; SD=stable disease; SPPD=sum of the products of the perpendicular diameters; TTR=time to response.

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View a well-tolerated safety profile

See the safety profile

References

  • 1. OJEMDA™ [Package Insert]. Brisbane, CA: Day One Biopharmaceuticals, Inc.; 2024.
  • 2. Kilburn LB, Khuong-Quang DA, Hansford JR, et al. The type II RAF inhibitor tovorafenib in relapsed/refractory pediatric low-grade glioma: the phase 2 FIREFLY-1 trial. Nat Med. 2024;30(207-217). doi:10.1038/s41591-023-02668-y
  • 3. Data on file. Day One Biopharmaceuticals, Inc.
  • 4. ClinicalTrials.gov identifier: NCT02684058. Published December 13, 2023. Accessed February 20, 2024. https://clinicaltrials.gov/study/NCT02684058
  • 5. Fangusaro J, Witt O, Driever PH, et al. Response assessment in paediatric low-grade glioma: recommendations from the Response Assessment in Pediatric Neuro-Oncology (RAPNO) working group. Lancet Oncol. 2020;21(6):e305-e316. doi:10.1016/S1470-2045(20)30064-4

IMPORTANT SAFETY INFORMATION and INDICATION

Warnings and Precautions

Hemorrhage

Hemorrhage, including major hemorrhage defined as symptomatic bleeding in a critical area or organ, can occur with OJEMDA. Advise patients and caregivers of the risk of hemorrhage during treatment with OJEMDA. Monitor for signs and symptoms of hemorrhage and evaluate as clinically indicated. Withhold and resume at reduced dose upon improvement, or permanently discontinue based on severity.

Skin Toxicity Including Photosensitivity

OJEMDA can cause rash, including maculopapular rash and photosensitivity. Monitor for new or worsening skin reactions. Consider dermatologic consultation and initiate supportive care as clinically indicated. Withhold, reduce the dose, or permanently discontinue OJEMDA based on severity of adverse reaction.

Photosensitivity

Advise patients to use precautionary measures against ultraviolet exposure such as use of sunscreen, sunglasses, and/or protective clothing during treatment with OJEMDA. Withhold, reduce the dose, or permanently discontinue OJEMDA based on severity of adverse reaction.

Hepatotoxicity

OJEMDA can cause hepatotoxicity. Monitor liver function tests, including ALT, AST and bilirubin, before initiation of OJEMDA, one month after initiation and then every three months thereafter and as clinically indicated. Withhold and resume at the same or reduced dose upon improvement, or permanently discontinue OJEMDA based on the severity.

Effect on Growth

OJEMDA can cause reductions in growth velocity. Growth velocity recovered after interruption of treatment with OJEMDA. Routinely monitor patient growth during treatment with OJEMDA.

Embryo-Fetal Toxicity

Based on findings from animal studies and its mechanism of action, OJEMDA may cause fetal harm when administered to a pregnant woman. Advise pregnant women and females of reproductive potential of the potential risk to a fetus.

Advise females of reproductive potential to use effective nonhormonal contraception during treatment with OJEMDA and for 28 days after the last dose, since OJEMDA can render some hormonal contraceptives ineffective. Advise male patients with female partners of reproductive potential to use effective nonhormonal contraception during treatment with OJEMDA and for 2 weeks after the last dose.

NF1 Associated Tumors

Based on nonclinical data in NF1 models without BRAF alterations, tovorafenib may promote tumor growth in patients with NF1 tumors. Confirm evidence of a BRAF alteration prior to initiation of treatment with OJEMDA.

Adverse Reactions

The most common adverse reactions (≥30%) were rash, hair color changes, fatigue, viral infection, vomiting, headache, hemorrhage, pyrexia, dry skin, constipation, nausea, dermatitis acneiform, and upper respiratory tract infection.

Please see full Prescribing Information.

Indication

OJEMDATM (tovorafenib) is indicated for the treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation.

This indication is approved under accelerated approval based on response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).