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EPIDIOLEX has significant market potential due to the growing demand for alternative therapies and its effectiveness in treating rare seizure disorders like Lennox-Gastaut syndrome and Dravet syndrome. As awareness around the benefits of CBD in treating neurological conditions increases, EPIDIOLEX stands to benefit from expanding indications and market reach. LAS VEGAS, May 1, 2025 /PRNewswire/ -- DelveInsight's ' EPIDIOLEX Market Size, Forecast, and Market Insight Report' highlights the details around EPIDIOLEX, the first FDA-approved prescription cannabidiol (CBD) to treat seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome, or tuberous sclerosis complex (TSC) in patients 1 year of age or older. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of EPIDIOLEX. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Jazz Pharmaceuticals' EPIDIOLEX (cannabidiol) Overview EPIDIOLEX (formerly known as GWP42003-P and marketed as EPIDYOLEX in Europe) is the first plant-based, prescription cannabis-derived oral medication developed by GW Pharmaceuticals. It represents a new category of antiepileptic drugs with a unique mechanism of action. The active compound, cannabidiol (CBD), is a naturally occurring cannabinoid found in the Cannabis sativa L. plant. While the exact way EPIDIOLEX works to reduce seizures in humans remains unclear, it is distinct from traditional anticonvulsants. CBD does not act through CB1 receptors or by blocking voltage-gated sodium channels, which are typical pathways for other antiepileptic drugs. Instead, CBD is thought to work by influencing multiple internal pathways, potentially producing a combined anticonvulsant effect. These include enhancing neuronal inhibition (through synaptic and extrasynaptic GABA channels), affecting intracellular calcium levels (via TRPV channels, VDAC, and GPR55), and reducing inflammation, possibly via adenosine modulation. Importantly, CBD does not directly bind to or activate the CB1 or CB2 receptors at levels relevant to its seizure-reducing effects. Ongoing research is examining other possible mechanisms such as interactions with adenosine, glycine, GABAergic systems, and serotonin receptors. EPIDIOLEX has received regulatory approval in numerous countries, including the U.S. (FDA), Europe (European Commission), Great Britain (MHRA), Australia (Therapeutic Goods Administration), Switzerland (Swissmedic), Israel (Ministry of Health), and New Zealand (Medicines and Medical Devices Safety Authority). It is formulated as an oral solution containing highly purified CBD. In the U.S., it is approved to treat seizures linked to Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), and tuberous sclerosis complex (TSC) in patients aged one year and older. In the European Union, under the name Epidyolex, it is approved as an add-on treatment with clobazam for LGS and DS in patients two years and older, and for TSC-associated seizures as well. Both the FDA and EMA have granted Orphan Drug Designation (ODD) for EPIDIOLEX/EPIDYOLEX for these conditions. EPIDIOLEX/EPIDYOLEX net product sales rose by 15% to USD 972.4 million in 2024 and by 14% to USD 275.0 million in the fourth quarter of 2024, compared to the corresponding periods in 2023. Learn more about EPIDIOLEX/EPIDYOLEX projected market size for DEE @ EPIDIOLEX Market Potential Developmental and epileptic encephalopathies (DEE) encompass a group of severe neurological conditions marked by developmental delays and persistent epilepsy. These disorders typically present early in life and are linked to significant cognitive deficits. According to DelveInsight's analysis, there were approximately 287K diagnosed prevalent cases of DEE across the 7MM in 2024. This number is projected to increase due to the rise in neurological disorders, including epilepsy, as well as environmental and maternal health factors contributing to a higher DEE prevalence throughout the forecast period (2025−2034). The US FDA has approved a range of medications for conditions like LGS, Dravet Syndrome, and CDKL5 Deficiency Disorder, including various drug classes such as sodium channel modulators, GABA receptor modulators, calcium channel blockers, receptor blockers, and others. Some of the available anti-epileptic drugs (AEDs) include FINTEPLA, EPIDIOLEX, TOPAMAX, BANZEL, LAMICTAL, FELBATOL, ONFI, KLONOPIN, DIACOMIT, SABRIL, and more. In 2024, the total market size for DEE in the 7MM was around USD 2.3 billion, and this figure is expected to grow by 2034, driven by wider adoption of approved therapies due to label expansions and the introduction of emerging treatments. Discover more about the DEE market in detail @ Developmental and Epileptic Encephalopathy Market Report Emerging Competitors of EPIDIOLEX Some of the drugs in the pipeline include EPX-100 (Harmony Biosciences), Bexicaserin (Lundbeck), Zorevunersen (Stoke Therapeutics/Biogen), Relutrigine (Praxis Precision Medicines), and others. In August 2024, the FDA removed a partial clinical hold as the company advances toward a Phase III registrational study of zorevunersen (STK-001) in children and adolescents with Dravet syndrome. In July 2024, Longboard Pharmaceuticals announced that the FDA had granted Breakthrough Therapy designation for its investigational drug bexicaserin for the treatment of seizures associated with DEEs for patients 2 years of age or older. To know more about the number of competing drugs in development, visit @ EPIDIOLEX Alternatives Key Milestones of EPIDIOLEX In August 2024, Jazz Pharmaceuticals reported top-line results from a Phase 3 open-label, single-arm study conducted in Japan, assessing the safety and effectiveness of its cannabidiol oral solution (marketed globally as EPIDIOLEX/EPIDYOLEX) as an add-on therapy for seizures linked to Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or tuberous sclerosis complex (TSC). While the trial did not achieve its primary efficacy goal—defined as a pre-specified percentage reduction in seizure frequency during the up-to-16-week treatment period compared to baseline in Japanese pediatric patients—numerical improvements were noted in both the primary and several secondary measures. The study did not identify any new safety concerns. In May 2021, Jazz Pharmaceuticals acquired EPIDIOLEX as part of the acquisition of GW Pharmaceuticals plc, or GW, which expanded its growing neuroscience business with a global, high-growth childhood-onset epilepsy franchise. In April 2021, EPIDYOLEX was approved for adjunctive therapy of seizures associated with TSC for patients 2 years of age and older in the EU. In July 2020, EPIDYOLEX was approved for the treatment of seizures associated with TSC in patients one year of age and older. FDA also approved the expansion of all existing indications, LGS and DS, to patients one year of age and older. In September 2019, the European Commission approved EPIDYOLEX for use as adjunctive therapy of seizures associated with LGS or DS, in conjunction with clobazam, for patients two years of age and older. In June 2018, EPIDIOLEX was approved in the US for the treatment of seizures associated with two rare and severe forms of epilepsy, LGS and DS, in patients two years of age and older. EPIDIOLEX Launch Status EPIDIOLEX was launched on November 1, 2018, in the US market after FDA approval for the treatment of seizures associated with LGS or Dravet syndrome in patients two years of age and older. EPIDYOLEX was launched in 2019 in Germany and the UK market for use as adjunctive therapy for seizures associated with LGS or Dravet syndrome, in conjunction with clobazam, for patients two years of age and older (GW Pharmaceuticals, 2019b). In 2021, EPIDYOLEX was launched in Spain. In the first quarter of 2022, the company launched EPIDYOLEX for LGS, Dravet syndrome, and TSC in Ireland, for TSC in Scotland and Wales. In the third quarter of 2022, the company launched EPIDYOLEX for TSC in Italy and Switzerland. In the fourth quarter of 2022, the company successfully completed the pricing and reimbursement process and commercial launch of EPIDYOLEX in France. Discover how EPIDIOLEX/EPIDYOLEX is shaping the DEE treatment landscape @ EPIDIOLEX CBD EPIDIOLEX Market Dynamics EPIDIOLEX, the first FDA-approved cannabidiol (CBD)-based medication for the treatment of rare forms of epilepsy, has had a significant impact on the pharmaceutical market since its approval in 2018. The drug is primarily used to treat seizures associated with Dravet syndrome and Lennox-Gastaut syndrome, two rare and severe forms of epilepsy. The market dynamic for EPIDIOLEX is shaped by several factors, including increasing awareness of its therapeutic potential, the expanding medical cannabis industry, and the growing demand for alternative treatments to traditional anti-epileptic drugs. One of the main drivers of the EPIDIOLEX market is the rising acceptance of CBD-based therapies. As medical cannabis becomes more mainstream and research into its medicinal properties continues to grow, patients and healthcare providers are increasingly turning to EPIDIOLEX as a treatment option. The growing body of clinical evidence supporting its effectiveness and safety has reinforced this trend. Additionally, as the stigma around cannabis-derived medications continues to fade, it opens up more opportunities for EPIDIOLEX in both pediatric and adult patient populations. However, the market for EPIDIOLEX is not without its challenges. High treatment costs and insurance coverage issues can limit accessibility for some patients, especially in markets outside the United States. Despite its clinical efficacy, the price point for EPIDIOLEX remains a point of contention, as it may not be affordable for all families, especially when compared to other conventional anti-epileptic drugs. Furthermore, competition from other cannabinoid-based therapies, as well as new developments in the broader epilepsy drug market, could impact the drug's market share over time. Looking ahead, the market dynamics for EPIDIOLEX will likely continue to evolve as the global regulatory landscape changes and new clinical trials further establish its benefits in other neurological conditions. With increasing research into CBD's potential applications, there may be opportunities for EPIDIOLEX to expand its indications, providing a solid foundation for its continued growth. However, market growth will also depend on the ability to address pricing concerns, improve insurance coverage, and navigate potential competitive pressures from both traditional and emerging therapies. Dive deeper to get more insight into EPIDIOLEX/EPIDYOLEX's strengths & weaknesses relative to competitors @ EPIDIOLEX Market Drug Report Table of Contents Related Reports Developmental and Epileptic Encephalopathies Market Developmental and Epileptic Encephalopathies Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key DEE companies, including vid Therapeutics, Stoke Therapeutics, Biogen, Harmony Biosciences, Lundbeck (Longboard Pharmaceuticals), Praxis Precision Medicines, SK Life Science, Cerecin Neurosciences, Encoded Therapeutics, Praxis Precision Medicines, among others. Developmental and Epileptic Encephalopathies Pipeline Developmental and Epileptic Encephalopathies Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key DEE companies, including Denovo Biopharma, Cantex Pharmaceuticals, CNS Pharmaceuticals, CANbridge Pharmaceuticals, Vaximm, Inovio Pharmaceuticals, Mustang Bio, Bullfrog AI Holdings, Cantex, Chimeric Therapeutics, Philogen, Boehringer Ingelheim, Photonamic GmbH, Berg Pharma, Beyond Bio, Genenta Science, Polaris Pharmaceuticals, Telix Pharmaceuticals, Shanghai Simnova Biotechnology, NEONC Technologies, among others. Dravet Syndrome Market Dravet Syndrome Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Dravet syndrome companies including Biocodex, GW Pharmaceuticals, Zogenix, Ovid Therapeutics, PTC Therapeutics, among others. Lennox-Gastaut Syndrome Market Lennox-Gastaut Syndrome Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Lennox-Gastaut syndrome companies, including GlaxoSmithKline, Meda Pharmaceuticals, Roche, Lundbeck, Greenwich Biosciences, Janssen Pharmaceuticals, Eisai, Zogenix, Takeda, Ovid Therapeutics, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

6个月大的乐乐（化名）从出生起就与众不同。别的宝宝酣睡时，他却会突然全身绷直、四肢抽搐，像被"电击"般惊跳起来。起初父母以为只是婴儿正常的惊跳反射，直到乐乐脸上出现几片树叶状的白色斑块，CT检查结果让所有人震惊——这个小小身躯里，大脑、心脏、肾脏竟然都长满了肿瘤！"就像身体里埋了无数个小炸弹。"乐乐的妈妈颤抖着描述。最可怕的是脑部检查发现的"烛泪征"——CT上那些如同融化蜡油般从脑室壁滴落的钙化结节。神经科医生立即安排了基因检测，结果显示TSC1基因存在致病突变，最终确诊为结节性硬化症（TSC）什么是结节性硬化结节性硬化(tuberous sclerosis complex，TSC)是一种常染色显性遗传皮肤神经综合征，最初由Recklinghausen于1862年首次描述，该病临床表现复杂多样，多以皮损、脑内结节、心脏横纹肌瘤、肺血管淋巴瘤、肾血管平滑肌瘤等全身多个器官出现错构瘤病变及神经精神症状为主。临床表现结节性硬化症（TSC）为多系统受累的遗传病，最常见表现为癫痫和面部血管纤维瘤。约80%～90%患儿有癫痫，TSC1突变起病早、多痉挛，TSC2突变起病晚、以局灶发作为主。部分患儿有智力减退、行为异常，MRI/CT可见皮质结节、室管膜下结节或巨细胞星形细胞瘤。皮肤表现见于90%以上患儿，包括面部血管纤维瘤、色素脱失斑、鲨革样斑和甲周纤维瘤，多于婴幼儿期出现，为重要诊断依据。心脏横纹肌瘤见于近半数患者，多在胎儿或出生早期发现，常随年龄缩小或消退。肾脏病变常见，主要为血管平滑肌脂肪瘤和肾囊肿，部分可致大出血和肾功能衰竭，是TSC死亡的重要原因。肺部受累多见于成年女性，表现为肺淋巴管平滑肌瘤病，偶见气胸、乳糜胸。眼部和骨骼亦可见错构瘤。诊断结节性硬化症（TSC）的诊断标准由国际结节性硬化症联盟于1992年制定，1998年和2012年两次修订，2012年版本首次将基因检测纳入标准。TSC可依临床表现或基因检测确诊。基因诊断标准为发现TSC1或TSC2基因突变即可确诊。临床诊断包括11项主要特征（如面部血管纤维瘤、甲周纤维瘤、色素脱失斑、脑皮质结构异常、心脏横纹肌瘤、肾血管平滑肌脂肪瘤等）及6项次要特征（如牙釉质小凹、口腔纤维瘤、斑驳皮肤等）。确诊标准：具TSC基因突变，或满足2项主要特征，或≥1项主要+≥2项次要特征。可能诊断：1项主要或≥2项次要特征。治疗结节性硬化症（TSC）目前尚无根治方法，治疗以控制症状为主。癫痫为常见症状，发作频率与智力倒退密切相关，早期控制癫痫尤为关键。常用抗癫痫药物包括丙戊酸、托吡酯、拉莫三嗪、左乙拉西坦等。若多药联合治疗无效，可加用mTOR抑制剂西罗莫司，已证实对TSC合并癫痫有效。如合并室管膜下巨细胞星形细胞瘤（SEGA）并引起脑积水等情况，应考虑手术治疗，但术后复发率和后遗症发生率较高。不适合手术者可用西罗莫司控制肿瘤生长。西罗莫司及其衍生物对SEGA、肾血管平滑肌脂肪瘤（AML）及肺淋巴管平滑肌瘤病（LAM）均有效。面部血管纤维瘤可行激光治疗，亦可尝试口服西罗莫司改善外观。新型mTOR ATP竞争性抑制剂已研发，抑制作用更强，但尚未进入临床应用。参考资料：1. 蔡燚，李汉忠，张玉石．结节性硬化症相关肾脏血管平滑肌脂肪瘤患者临床表型与基因型的关系[J]．中华泌尿外科杂志， 2016，37(12):912-915. DOI： 10.3760/cma.j.issn.1000-6702.2016.12.009 .2.孙丹，刘智胜，胡家胜，等．结节性硬化症患儿TSC1/TSC2突变基因型与表型的相关性分析[J]．中华实用儿科临床杂志， 2015，30(6):461-466. DOI： 10.3760/cma.j.issn.2095-428X.2015.06.016 .3.Kothare SV , Singh K , Chalifoux JR ，et al. Severity of manifestations in tuberous sclerosis complex in relation to genotype[J]．Epilepsia， 2014，55(7):1025-1029. DOI： 10.1111/epi.12680 .来源 | 梅斯医学编辑 | wanny神经系统罕见病交流群↓点击下方“阅读原文”，下载梅斯医学APP吧！