Incyte and Syndax Announce U.S. Food and Drug Administration (FDA) Approval of Niktimvo™ (axatilimab-csfr) 9 mg and 22 mg Vial Sizes
Incyte and Syndax Pharmaceuticals announced FDA approval of Niktimvo (axatilimab-csfr) in 9 mg and 22 mg vial sizes, with U.S. launch expected in early February. Niktimvo is the first FDA-approved treatment targeting CSF-1R for chronic graft-versus-host disease (GVHD) in patients who failed at least two prior systemic therapies.
The approval was based on the AGAVE-201 trial results, where 75% of patients receiving 0.3 mg/kg every two weeks achieved response at six months. The approved dose is 0.3 mg/kg (max 35 mg) via intravenous infusion every two weeks for patients weighing at least 40 kg.
Notable adverse reactions occurred in 44% of patients, with 10% discontinuing treatment. The most common side effects included increased AST, infections, and decreased phosphate levels. The treatment has been added to NCCN Guidelines as a category 2A recommendation.
Incyte e Syndax Pharmaceuticals hanno annunciato l'approvazione da parte della FDA di Niktimvo (axatilimab-csfr) in flaconi da 9 mg e 22 mg, con un lancio previsto negli Stati Uniti all'inizio di febbraio. Niktimvo è il primo trattamento approvato dalla FDA che colpisce il CSF-1R per la malattia cronica da trapianto contro ospite (GVHD) in pazienti che hanno fallito almeno due precedenti terapie sistemiche.
L'approvazione si basa sui risultati dello studio AGAVE-201, dove il 75% dei pazienti che ricevevano 0.3 mg/kg ogni due settimane ha ottenuto una risposta a sei mesi. La dose approvata è di 0.3 mg/kg (massimo 35 mg) tramite infusione endovenosa ogni due settimane per pazienti che pesano almeno 40 kg.
Reazioni avverse significative si sono verificate nel 44% dei pazienti, con il 10% che ha interrotto il trattamento. Gli effetti collaterali più comuni includevano un aumento dell'AST, infezioni e livelli di fosfato ridotti. Il trattamento è stato aggiunto alle linee guida NCCN come raccomandazione categoria 2A.
Incyte y Syndax Pharmaceuticals anunciaron la aprobación de la FDA de Niktimvo (axatilimab-csfr) en presentaciones de 9 mg y 22 mg, con un lanzamiento en EE. UU. esperado para principios de febrero. Niktimvo es el primer tratamiento aprobado por la FDA que tiene como objetivo el CSF-1R para la enfermedad injerto contra huésped (GVHD) crónica en pacientes que no han tenido éxito con al menos dos terapias sistémicas previas.
La aprobación se basó en los resultados del ensayo AGAVE-201, donde el 75% de los pacientes que recibieron 0.3 mg/kg cada dos semanas alcanzaron respuesta a los seis meses. La dosis aprobada es de 0.3 mg/kg (máximo 35 mg) por infusión intravenosa cada dos semanas para pacientes que pesan al menos 40 kg.
Se produjeron reacciones adversas notables en el 44% de los pacientes, con un 10% interrumpiendo el tratamiento. Los efectos secundarios más comunes incluyeron aumento de AST, infecciones y niveles de fosfato disminuidos. El tratamiento se ha añadido a las guías NCCN como una recomendación de categoría 2A.
Incyte와 Syndax Pharmaceuticals는 Niktimvo (axatilimab-csfr)의 FDA 승인을 발표했으며, 9mg 및 22mg 바이알 크기로 제공됩니다. 미국 출시 예정일은 2월 초입니다. Niktimvo는 최소 두 가지 이전 체계 요법에 실패한 환자의 만성 이식 대비 숙주 질환(GVHD)에 대해 CSF-1R을 타겟으로 하는 FDA 승인 치료제입니다.
승인은 AGAVE-201 시험 결과를 기반으로 하며, 0.3 mg/kg을 2주마다 투여 받은 환자의 75%가 6개월 후에 반응을 보였습니다. 승인된 용량은 체중이 40kg 이상인 환자에게 2주마다 정맥 주사를 통해 0.3 mg/kg(최대 35 mg)입니다.
주목할만한 부작용은 환자의 44%에서 발생하였고, 10%는 치료를 중단했습니다. 가장 흔한 부작용으로는 AST 증가, 감염 및 인산염 수치 감소가 포함됩니다. 이 치료법은 NCCN 가이드라인에 카테고리 2A 권장 사항으로 추가되었습니다.
Incyte et Syndax Pharmaceuticals ont annoncé l'approbation par la FDA de Niktimvo (axatilimab-csfr) en flacons de 9 mg et 22 mg, avec un lancement prévu aux États-Unis début février. Niktimvo est le premier traitement approuvé par la FDA visant le CSF-1R pour la maladie chronique du greffon contre l'hôte (GVHD) chez des patients ayant échoué à au moins deux traitements systémiques précédents.
L'approbation repose sur les résultats de l'essai AGAVE-201, où 75 % des patients recevant 0,3 mg/kg toutes les deux semaines ont montré une réponse après six mois. La dose approuvée est de 0,3 mg/kg (max 35 mg) par perfusion intraveineuse toutes les deux semaines pour les patients pesant au moins 40 kg.
Des réactions indésirables notables sont survenues chez 44 % des patients, avec 10 % d'entre eux interrompant le traitement. Les effets secondaires les plus courants comprenaient une augmentation de l'AST, des infections et une diminution des niveaux de phosphate. Le traitement a été ajouté aux recommandations de catégorie 2A des lignes directrices NCCN.
Incyte und Syndax Pharmaceuticals haben die FDA-Zulassung von Niktimvo (axatilimab-csfr) in 9 mg und 22 mg Fläschchen bekannt gegeben, mit einem Marktstart in den USA, der für Anfang Februar erwartet wird. Niktimvo ist die erste von der FDA genehmigte Behandlung, die auf CSF-1R für die chronische Transplantat-gegen-Wirt-Krankheit (GVHD) bei Patienten abzielt, die mindestens zwei frühere systemische Therapien nicht erfolgreich abgeschlossen haben.
Die Zulassung basierte auf den Ergebnissen der AGAVE-201-Studie, in der 75 % der Patienten, die alle zwei Wochen 0,3 mg/kg erhielten, nach sechs Monaten eine Reaktion zeigten. Die genehmigte Dosis beträgt 0,3 mg/kg (max. 35 mg) durch intravenöse Infusion alle zwei Wochen für Patienten mit einem Gewicht von mindestens 40 kg.
Bemerkenswerte Nebenwirkungen waren bei 44 % der Patienten zu verzeichnen, wobei 10 % die Behandlung abbrachen. Die häufigsten Nebenwirkungen umfassten erhöhte AST-Werte, Infektionen und verringerte Phosphatspiegel. Die Behandlung wurde als Empfehlung der Kategorie 2A in die NCCN-Richtlinien aufgenommen.
- First-in-class FDA-approved treatment for chronic GVHD targeting CSF-1R
- High efficacy with 75% response rate in clinical trials
- Added to NCCN Guidelines as category 2A recommendation
- Co-commercialization agreement between Incyte and Syndax in the U.S.
- Serious adverse reactions in 44% of patients
- 10% treatment discontinuation rate due to adverse reactions
- 44% of patients required dose interruptions
- to patients weighing at least 40 kg
Insights
The FDA approval of Niktimvo represents a pivotal milestone for both Incyte and Syndax. The drug's unique mechanism targeting CSF-1R in chronic GVHD creates a significant market opportunity in an underserved therapeutic area. With 75% of patients achieving response at six months, the efficacy data is particularly compelling for a third-line therapy. The dual vial sizing strategy (9mg and 22mg) demonstrates commercial sophistication, optimizing for different patient weights while minimizing waste.
The early February launch timing, coupled with the existing NCCN Guidelines Category 2A recommendation, positions the drug favorably for rapid market adoption. The IncyteCARES support program addresses reimbursement barriers, which is important for specialty pharmaceuticals in this price range. The co-commercialization agreement with Incyte, particularly their established presence in GVHD, should accelerate market penetration.
The clinical profile of Niktimvo is particularly noteworthy. The 44% rate of serious adverse reactions, while significant, is manageable within the context of late-line GVHD therapy. The 10% discontinuation rate is relatively low for this patient population, suggesting favorable tolerability. The adverse event profile, primarily centered on infections and laboratory abnormalities, is monitorable and generally reversible.
For simpler understanding: Think of Niktimvo as a specialized tool that stops the body's repair cells (macrophages) from causing excessive scarring in transplant patients. It's like having a dimmer switch for inflammation, rather than completely shutting down the immune system like other treatments. The strong response rate means that for every 4 patients treated, 3 saw improvement - exceptional for patients who have already tried multiple other treatments.
The commercial implications for Syndax (market cap:
The inclusion in NCCN guidelines just two weeks after approval is particularly strategic, as it typically accelerates payer coverage decisions. The comprehensive patient support program through IncyteCARES addresses a critical commercial barrier in specialty pharmaceuticals. Based on the addressable market and positioning, this approval could be transformative for Syndax's revenue trajectory.
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– Niktimvo is the first and only approved treatment for chronic GVHD that targets CSF-1R to reduce the drivers of inflammation and fibrosis –
– Pivotal data from the AGAVE-201 trial supporting FDA approval show treatment with Niktimvo resulted in durable responses across all organs studied and patient subgroups –
"We are thrilled to build on our strong commitment to the GVHD community with the
Niktimvo was approved by the FDA on August 14, 2024. The approval was based on positive data from the global AGAVE-201 trial, which were published in the New England Journal of Medicine in September 2024.1 The trial met the primary endpoint across all cohorts receiving Niktimvo with
"As the first and only FDA-approved anti-CSF-1R antibody targeting the drivers of inflammation and fibrosis in chronic GVHD, Niktimvo represents a major breakthrough for patient care," said Michael Metzger, Chief Executive Officer, Syndax. "Together with Incyte, we look forward to executing a robust commercial launch and advancing the treatment paradigm for patients with chronic GVHD who have progressed after at least two lines of systemic therapy."
Serious adverse reactions occurred in
The most common (≥
The approved dose of Niktimvo for adults and pediatric patients weighing at least 40 kg is 0.3 mg/kg, up to a maximum dose of 35 mg, as an intravenous infusion over 30 minutes every two weeks. Niktimvo will be available for healthcare providers to order through a network of specialty distributors in both 9 mg vial and 22 mg vial sizes to facilitate patient dosing.
Incyte and Syndax are committed to supporting patients and removing barriers to ensure access to Niktimvo. Eligible patients in the
On August 30, 2024, axatilimab-csfr (Niktimvo) was added to the latest NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) as a category 2A recommendation for the treatment of chronic GVHD after the failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg.2 Treatments are classified as category 2A when there is uniform NCCN consensus that the intervention is appropriate, based on lower-level evidence. The updated NCCN guidelines are available at www.nccn.org.
In the
About Chronic Graft-Versus-Host Disease (GVHD)
Chronic GVHD is a serious condition that can occur after an allogeneic stem cell transplant (the transfer of stem cells from a donor) in which the donated cells initiate an immune response and attack the transplant recipient's organs. Chronic GVHD is a leading cause of significant morbidity and mortality after an allogeneic stem cell transplant and is estimated to develop in approximately
About AGAVE-201
The global AGAVE-201 dose-ranging trial evaluated the efficacy, safety, and tolerability of axatilimab in 241 adult and pediatric patients with recurrent or refractory active chronic GVHD (GVHD) whose disease had progressed after two or more prior therapies. Patients were randomized to one of three treatment groups that investigated a distinct dose of axatilimab administered at 0.3 mg/kg every two weeks, 1.0 mg/kg every two weeks or 3.0 mg/kg every four weeks. The trial's primary endpoint was the proportion of patients in each dose group who achieved an objective response as defined by 2014 NIH Consensus Criteria for chronic GVHD by cycle 7 day 1. Secondary endpoints included duration of response, percent reduction in daily steroid dose, organ specific response rates and validated quality-of-life assessments using the Modified Lee Symptom Scale.
For more information about AGAVE-201, visit https://www.clinicaltrials.gov/study/NCT04710576.
About Niktimvo™ (axatilimab-csfr)
Niktimvo (axatilimab-csfr) is a first-in-class colony stimulating factor-1 receptor (CSF-1R)-blocking antibody approved for use in the
In 2016, Syndax licensed exclusive worldwide rights to develop and commercialize axatilimab from UCB. In September 2021, Syndax and Incyte entered into an exclusive worldwide co-development and co-commercialization license agreement for axatilimab in chronic GVHD and any future indications.
Axatilimab is being studied in frontline combination trials in chronic GVHD – a Phase 2 combination trial with ruxolitinib (NCT06388564) and a Phase 3 combination trial with steroids (NCT06585774) are underway. Axatilimab is also being studied in an ongoing Phase 2 trial in patients with idiopathic pulmonary fibrosis (NCT06132256).
Niktimvo is a trademark of Incyte.
All other trademarks are the property of their respective owners.
IMPORTANT SAFETY INFORMATION
WARNINGS AND PRECAUTIONS
Infusion-Related Reactions
Niktimvo™ (axatilimab-csfr) can cause infusion-related reactions. Infusion-related reactions, including hypersensitivity reactions, occurred in
Premedicate with an antihistamine and an antipyretic for patients who have previously experienced an infusion-related reaction to Niktimvo. Monitor patients for signs and symptoms of infusion-related reactions, including fever, chills, rash, flushing, dyspnea, and hypertension. Interrupt or slow the rate of infusion or permanently discontinue Niktimvo based on severity of the reaction.
Embryo-Fetal Toxicity
Based on its mechanism of action, Niktimvo may cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to the fetus. Advise females of reproductive potential to use effective contraception during treatment with Niktimvo and for 30 days after the last dose.
ADVERSE REACTIONS
Serious adverse reactions occurred in
The most common (≥
Clinically relevant adverse reactions in <
- Eye disorders: periorbital edema
- Skin and subcutaneous skin disorders: pruritus
- Vascular disorders: hypertension
Immunogenicity: Anti-Drug Antibody–Associated Adverse Reactions
Across treatment arms in patients with cGVHD who received Niktimvo in clinical trials, among the patients who developed anti-drug antibodies (ADAs), hypersensitivity reactions occurred in
USE IN SPECIFIC POPULATIONS
Lactation
Because of the potential for serious adverse reactions in a breastfed child, advise women not to breastfeed during treatment and for 30 days after the last dose of Niktimvo.
Females and Males of Reproductive Potential
Pregnancy Testing
Verify pregnancy status in females of reproductive potential prior to initiating Niktimvo.
Contraception
Females
Advise females of reproductive potential to use effective contraception during treatment with Niktimvo and for 30 days after the last dose of Niktimvo.
DOSAGE AND ADMINISTRATION
Dosage Modifications for Adverse Reactions
Monitor aspartate aminotransferase (AST), alanine aminotransferase (ALT), alkaline phosphatase (ALP), creatine phosphokinase (CPK), amylase, and lipase prior to the start of Niktimvo therapy, every 2 weeks for the first month, and every 1 to 2 months thereafter until abnormalities are resolved. See Table 1 in the Prescribing Information for more recommendations.
Please see the full Prescribing Information for Niktimvo.
About Incyte
A global biopharmaceutical company on a mission to Solve On., Incyte follows the science to find solutions for patients with unmet medical needs. Through the discovery, development and commercialization of proprietary therapeutics, Incyte has established a portfolio of first-in-class medicines for patients and a strong pipeline of products in Oncology and Inflammation & Autoimmunity. Headquartered in
For additional information on Incyte, please visit Incyte.com or follow us on social media: LinkedIn, X, Instagram, Facebook, YouTube.
About Syndax
Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company developing an innovative pipeline of cancer therapies. Highlights of the Company's pipeline include Revuforj® (revumenib), an FDA-approved menin inhibitor, and Niktimvo™ (axatilimab-csfr), an FDA-approved monoclonal antibody that blocks the colony stimulating factor 1 (CSF-1) receptor. Fueled by our commitment to reimagining cancer care, Syndax is working to unlock the full potential of its pipeline and is conducting several clinical trials across the continuum of treatment. For more information, please visit www.syndax.com/ or follow the Company on X (formerly Twitter) and LinkedIn.
Incyte Forward-Looking Statements
Except for the historical information set forth herein, the matters set forth in this press release, including statements regarding whether Niktimvo might provide a successful treatment option for patients with chronic GVHD: expectations regarding the launch of Niktimvo; and the potential for axatilimab to treat additional conditions, contain predictions, estimates and other forward-looking statements.
These forward-looking statements are based on Incyte's current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the
Syndax Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "may," "will," "expect," "plan," "anticipate," "estimate," "intend," "believe" and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. These forward-looking statements are based on Syndax's expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties. Actual results may differ materially from these forward-looking statements. Forward-looking statements contained in this press release include, but are not limited to, statements about the progress, timing, clinical development and scope of clinical trials, the reporting of clinical data for Syndax's product candidates, the acceptance of Syndax and its partners' products in the marketplace, sales, marketing, manufacturing and distribution requirements, and the potential use of our product candidates to treat various cancer indications and fibrotic diseases. Many factors may cause differences between current expectations and actual results, including: unexpected safety or efficacy data observed during preclinical or clinical trials; clinical trial site activation or enrollment rates that are lower than expected; changes to Niktimvo's commercial availability; changes in expected or existing competition; changes in the regulatory environment; failure of Syndax's collaborators to support or advance collaborations or product candidates; and unexpected litigation or other disputes. Other factors that may cause Syndax's actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in Syndax's filings with the
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1 Wolff D, et al. Axatilimab in Recurrent or Refractory Chronic Graft-versus-Host Disease. N Engl J Med 2024;391:1002-14. DOI: 10.1056/NEJMoa2401537.
2 NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Hematopoietic Cell Transplantation (HCT). Version 2.2024 – August 30, 2024. NCCN makes no warranties of any kind whatsoever regarding their content, use or application and disclaims any responsibility for their application or use in any way.
3 Data on file.
4 Bachier, CR. et al. ASH annual meeting 2019; abstract #2109 Epidemiology and Real-World Treatment of Chronic Graft-Versus-Host Disease Post Allogeneic Hematopoietic Cell Transplantation: A
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SOURCE Syndax Pharmaceuticals; Incyte
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