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

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Prescribing Information

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Study Design

FIREFLY-1 Study Design

OJEMDA: Studied in FIREFLY-1, the largest clinical trial to date in children with BRAF-altered R/R pLGG1,2

137 patients received OJEMDA in the pivotal FIREFLY-1 trial conducted in collaboration with the Pacific Pediatric Neuro-Oncology Consortium3

FIREFLY-1 is a global, multicenter, open-label, single-arm clinical trial.3

Key inclusion criteria3

  • 6 months to 25 years old
  • BRAF-altered R/R pLGG
  • At least 1 prior systemic therapy with documented radiographic progression

Key exclusion criteria4

  • Known or suspected diagnosis of NF1

Patients received OJEMDA ≈420 mg/m2 orally once weekly (range: 290 mg/m2 to 476 mg/m2, 0.76-1.25 times the approved recommended dosage) according to BSA with a maximum dosage of 600 mg until disease progression or unacceptable toxicity.1

Patients who had radiographic disease progression were permitted to continue on treatment with OJEMDA at the investigator’s discretion.5

  • Arm 1: Primary efficacy analysis (n=76)*
  • Arm 1 + Arm 2: Primary safety analysis (n=137)†

Trial endpoints1,5

Major efficacy outcome1‡

  • ORR (RAPNO-LGG criteria)§

Select secondary outcomes1

  • Time to response
  • Duration of response

Safety outcome5

  • Safety and tolerability
  • *77 patients were enrolled in Arm 1; 76 patients were considered evaluable for response by RAPNO-LGG criteria.1
  • †Arm 2 is an extension arm, which provided treatment access for patients with BRAF-altered pLGG after the registrational arm closure.5
  • ‡The primary endpoint was the ORR according to RANO-HGG criteria.5
  • §ORR was defined as the proportion of patients with CR, PR, or MR by independent review based on RAPNO-LGG criteria.1

2-year Update

Patients from the efficacy arm have completed 2 years of treatment with OJEMDA in FIREFLY-15

  • The trial includes a 2-year treatment period (26 cycles of treatment) and a 3-year posttreatment observation period
  • Patients from both arms of the trial entered the 3-year posttreatment observation period after completing 26 cycles of treatment

Treatment exposure duration

  • The median duration of the OJEMDA primary treatment exposure in the efficacy arm was 24 months (range: 0.7 to 32.5 months) based on the May 10, 2024 data cutoff5

In FIREFLY-1, OJEMDA delivered clinically meaningful tumor shrinkage1

See the results

Baseline Characteristics

Baseline characteristics from FIREFLY-1, reflective of patients seen in clinical practice7

OJEMDA was studied in a population where almost half of patients received 1 or 2 prior lines of therapy3

Baseline characteristics (N=76)1,3

Age (years)

Median (range)

8.5 (2-21)

Sex

Male

53%

Female

47%

BRAF alterations

BRAF fusion

74%

V600E mutation

16%

Other (including BRAF duplication or rearrangement)

11%

Number of prior
systemic regimens

Median (range)

3 (1-9)

1

22%

2

27%

≥3

51%

Prior MAPK-targeted therapy

No

41%

Yes

59%

Common tumor locations1

Cerebral hemisphere

0%

Optic pathway

0%

Cerebellum

0%

Brain
stem

0%

Deep midline structures

0%

BRAF=v-Raf murine sarcoma viral oncogene homolog B1; BSA=body surface area; CR=complete response; FDA=Food and Drug Administration; MAPK=mitogen-activated protein kinase; MR=minor response; NF1=neurofibromatosis type 1; ORR=overall response rate; pLGG=pediatric low-grade glioma; PR=partial response; RAF=rapidly accelerated fibrosarcoma; RANO-HGG=Response Assessment in Neuro-Oncology for High-Grade Glioma; RAPNO-LGG=Response Assessment in Pediatric Neuro-Oncology for Low-Grade Glioma; R/R=relapsed/refractory.

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IMPORTANT SAFETY INFORMATION
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 improved 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.

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).

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References