- Two analyses of updated three-year follow-up data from the pivotal Phase 2 FIREFLY-1 study of OJEMDA® (tovorafenib) in BRAF-altered pediatric low-grade glioma will be featured in separate oral presentations
- Results from a survey of pediatric low-grade glioma caregivers unveiling challenges and opportunities to improve caregiver-physician communication will also be presented
BOSTON, June 26, 2026 /PRNewswire/ -- Servier today announced it will present new and updated data at the 22nd International Symposium on Pediatric Neuro-Oncology (ISPNO 2026) June 28 – July 1 in Sydney, Australia. Servier's presence at ISPNO 2026 emphasizes its advancing leadership in neuro-oncology research with the addition of OJEMDA® (tovorafenib) to its growing glioma portfolio following the recent acquisition of Day One Biopharmaceuticals.
"Our upcoming presentations at ISPNO 2026 help strengthen the robust body of evidence supporting the safety and efficacy of OJEMDA, further establishing it as a standard of care for children with relapsed/refractory pediatric low-grade glioma," said Elly Barry, MD, Chief Medical Officer, Day One Biopharmaceuticals, now part of Servier Group. "These latest analyses from the pivotal FIREFLY-1 study – as well as the results from our caregiver survey – underscore our fierce commitment to supporting children with pediatric low-grade glioma and their caregivers, and the continued development of OJEMDA as part of Servier's growing glioma portfolio."
Servier will present updated three-year follow up growth recovery data from the pivotal Phase 2 FIREFLY-1 study in an oral presentation on July 1. The results suggest that most children with BRAF-altered pediatric low-grade glioma (pLGG) who experienced reduced growth velocity while being treated with OJEMDA experienced growth recovery after discontinuing treatment.
A separate analysis of the three-year follow up FIREFLY-1 data evaluating the impact of prior therapy on OJEMDA activity in BRAF-altered pLGG will also be presented in an oral presentation on July 1. Clinical benefits were observed with OJEMDA across lines of therapy, with trends toward improved durability in MAPK inhibitor-naïve patients.
Servier will also share results from a survey of pLGG caregivers about communication with their child's diagnosing provider. Caregivers in the U.S. were invited by advocacy organizations and fellow caregivers to complete an online survey to understand caregivers' experience of provider communication about pLGG diagnosis, including what language was used, what information was communicated, and how the approach to communication affected understanding. Data were collected using a custom-developed survey comprised of primarily closed-ended questions – scaling, yes/no, and multiple choice. This study was funded by Day One Biopharmaceuticals, a Servier Group Company.
The survey results identified both effective and suboptimal aspects of caregiver-provider communication during pLGG diagnosis and early treatment decision-making, highlighting opportunities to improve communication, strengthen trust, and increase caregiver confidence and preparedness.
Media contact
Darby Malkin: darby.malkin@servier.com
About tovorafenib
Tovorafenib (known as OJEMDA® in the U.S.) is a Type II RAF kinase inhibitor of mutant BRAF V600, wild-type BRAF, and wild-type CRAF kinases. OJEMDA 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).
Tovorafenib was granted Breakthrough Therapy and Rare Pediatric Disease designations by the FDA for the treatment of patients with pLGG harboring an activating RAF alteration, and it was evaluated by the FDA under priority review. Tovorafenib has also received Orphan Drug designation from the FDA for the treatment of malignant glioma and from the European Commission for the treatment of glioma.
Please see full prescribing information including important safety information about OJEMDA at www.OJEMDA.com.
About Pediatric Low-Grade Glioma
Pediatric low-grade gliomas (pLGG) are the most common brain tumor with an estimated US incidence of 1,100 and Europe incidence of 700 children per year who are eligible for front-line systemic therapy.i, ii BRAF is the gene most commonly altered in pLGG, which includes two primary types of BRAF alterations – a BRAF gene fusion and BRAF point mutation. These BRAF alterations account for >50% of pLGG cases worldwide and prior to the introduction of OJEMDA, there were no approved treatments for people with pLGG driven by BRAF fusions.i, iii
Pediatric low-grade gliomas can be chronic and relentless, with patients suffering profound side effects from both the tumor and the treatment, which may include chemotherapy and radiation. These side effects can impact their life over the long term, and may include motor deficiencies, vision loss, hormone deficiency and alterations in growth and development. Most children with pLGG will survive their cancer, but for the majority of those in whom a complete surgical resection is not possible, these tumors tend to recur frequently throughout childhood, necessitating multiple treatments. The cumulative toxicity of numerous therapies, along with the damage caused by multiple episodes of tumor progression, take a significant toll on the children and their families.
About Servier
Servier is an independent international pharmaceutical group governed by a foundation. With its governance model, the Group is committed to therapeutic progress to serve patients and integrates the patient voice at every stage of the medicine life cycle.
As a leading global player in cardiology and venous diseases, Servier aims to become a leading innovator in oncology and neurology. The Group intends to offer targeted therapeutic solutions, particularly in rare cancers and neurological diseases, and invests nearly 20% of its brand-name sales in R&D.
Headquartered in France, Servier relies on its more than 20,000 employees and a solid geographic presence with medicines distributed in more than 130 countries. In the 2024/25 financial year, the Group achieved revenues of €6.9 billion.
More information on the Group website: servier.com
Follow us on social media: LinkedIn, Facebook, X, Instagram
About Servier in Oncology
Servier is a global leader in oncology, governed by a non-profit foundation. Servier approaches innovation with a long-term vision, free of influence from fiduciary responsibilities.
Servier invests close to 20% of revenue from brand-name medicines in R&D each year. Servier aspires to advance more targeted therapies by identifying mutations and understanding how these mutations impact cancer and its progression. Servier believes we can serve more people by helping the right patients find the right treatment, at the right time.
Servier takes a One Innovation Engine approach to R&D and is actively seeking alliances, partnerships and acquisitions at various stages of the portfolio.
For more information about working with Servier to bring the promise of tomorrow to the patients it serves, visit Servier.us.
Important Safety Information
Before taking or giving OJEMDA, tell your healthcare provider about all of your or your child's medical conditions, including if you:
- have bleeding, skin, or liver problems
- are pregnant or plan to become pregnant. OJEMDA can harm your unborn baby.
Females who are able to become pregnant:
- You should use effective non-hormonal birth control (contraception) during treatment with OJEMDA and for 28 days after your last dose of OJEMDA.
Males with female partners who are able to become pregnant should use effective non-hormonal birth control (contraception) during treatment with OJEMDA and for 2 weeks after your last dose of OJEMDA.
- are breastfeeding or plan to breastfeed. Do not breastfeed during treatment and for 2 weeks after your last dose of OJEMDA.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
What should I avoid while taking OJEMDA?
Limit the amount of time you spend in sunlight. OJEMDA can make your skin sensitive to the sun (photosensitivity). Use sun protection measures, such as sunscreen, sunglasses and wear protective clothes that cover your skin during your treatment with OJEMDA.
What are the possible side effects of OJEMDA?
OJEMDA may cause serious side effects, including:
- bleeding problems (hemorrhage) are common and can also be serious. Tell your healthcare provider if you have any signs or symptoms of bleeding, including:
- headache, dizziness or feeling weak
- coughing up blood or blood clots
- vomiting blood or your vomit looks like "coffee grounds"
- red or black stools that look like tar
- skin reactions, including sensitivity to sunlight (photosensitivity). OJEMDA can cause skin reactions that can become severe. Tell your healthcare provider if you get new or worsening skin reactions, including:
- rash
- bumps or tiny papules
- acne
- peeling, redness, or irritation
- blisters
- liver problems. Your healthcare provider will do blood tests to check your liver function before and during treatment with OJEMDA. Tell your healthcare provider right away if you develop any of the following symptoms:
- yellowing of your skin or your eyes
- dark or brown (tea-colored) urine
- nausea or vomiting
- loss of appetite
- tiredness
- bruising
- bleeding
- pain in your upper right stomach area
- slowed growth in children. Growth will be checked routinely during treatment with OJEMDA.
The most common side effects of OJEMDA include:
- rash
- hair color changes
- tiredness
- viral infection
- vomiting
- headache
- fever
- dry skin
- constipation
- nausea
- acne
- upper respiratory tract infection
OJEMDA may cause fertility problems in males and females, which may affect your ability to have children.
These are not all the possible side effects of OJEMDA. Call your healthcare provider for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088. You may also report side effects to Day One Biopharmaceuticals at 1-877-204-2820.
Please see full Patient Information, including Instructions for Use for more information.
Disclosures
This release contains general information about the Servier Group and its entities (hereinafter "Servier and its Affiliates") and is intended for informational purposes only. The information is thought to be reliable; however, Servier and its Affiliates make no representation as to the accuracy or completeness of the information contained herein or otherwise provided and accept no responsibility or liability, in contract, in tort, in negligence, or otherwise, should the information be found to be inaccurate or incomplete in any respect.
Servier and its Affiliates are not acting as an advisor to the recipient of this information, and the ultimate decision to proceed with any transaction rests solely with the recipient of this information. Therefore, prior to entering into any proposed transaction, the recipient of this information should determine, without reliance upon Servier or its Affiliates, the economic risks and merits, as well as the legal, tax, and accounting characterizations and consequences, of the transaction and that it is able to assume these risks.
This statement also contains forward-looking statements that are subject to varying levels of uncertainty and risk. Investigational new drugs and indications are subject to further scientific and medical review and regulatory approval. They are not approved for use by the FDA.
Any reliance placed on this document is done entirely at the risk of the person placing such reliance. The information contained in this document is neither an offer to sell nor the solicitation of an offer to enter into a transaction.
The content of this document is a summary only, is not complete, and does not include all material information about Servier and its Affiliates, including potential conflicts of interest.
To the maximum extent permitted by applicable laws and regulations, Servier and its Affiliates disclaim all representations, warranties, conditions and guarantees, whether express, implied, statutory or of other kind, nor does it accept any duty to any person, in connection with this document. Without prejudice to the generality of the foregoing, Servier and its Affiliates do not warrant or represent that the information or opinions contained in this document is accurate or complete.
To the maximum extent permitted by applicable laws and regulations, Servier and its Affiliates shall not be liable for any loss, damage or expense whatsoever, whether direct or indirect, howsoever arising, whether in contract, tort (including negligence), strict liability or otherwise, for direct, indirect, incidental, consequential, punitive or special damages arising out of or in connection with this document, including (without limitation) any course of action taken on the basis of the same. The estimates, strategies, and views expressed in this document are based upon past or current data and information and are subject to change without notice.
References
[i] Ryall S, et al. Acta Neuropathol Commun. 2020;8(1):30.
[ii] Estimates of annual incidence and prevalence for addressable patient population in E.U. 4 + U.K. are based on Ipsen calculations from publicly available data (Eurostat, <25yo population; Global Burden of Disease 2019; Desandes et al. Incidence and survival of children with central nervous system primitive tumors in the French National Registry of Childhood Solid Tumors. Neuro Oncol. 2014 Jul;16(7):975-83. doi: 10.1093/neuonc/not309; Qaddoumi et al. Outcome and prognostic features in pediatric gliomas: a review of 6212 cases from the Surveillance, Epidemiology, and End Results database. Cancer. 2009 Dec 15;115(24):5761-70. doi: 10.1002/cncr.24663)
[iii] Traunwieser T, et al. Neurooncol Adv. 2020;2(1):vdaa094.
View original content to download multimedia:https://www.prnewswire.com/news-releases/servier-presentations-at-ispno-2026-highlight-neuro-oncology-leadership-and-expanding-glioma-portfolio-302811874.html
SOURCE Servier Pharmaceuticals
