RegulationOrphan Drug (United States), Rare Pediatric Disease (United States), Orphan Drug (European Union), Priority Review (United States), Priority Review (China)

Structure/Sequence

Molecular FormulaC22H36O2

InChIKeyPGTVWKLGGCQMBR-FLBATMFCSA-N

CAS Registry38398-32-2

Clinical Trials associated with Ganaxolone

NCT05249556

/ Not yet recruitingPhase 3

Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone in the Treatment of Seizures Associated With Genetically Confirmed Cyclin-dependent Kinase-like 5 (CDKL5) Deficiency Disorder (CDD) in Pediatric Patients From 6 Months to Less Than 2 Years of Age.

This study will assess the efficacy, safety, and tolerability of ganaxolone (GNX) compared with placebo (PBO) as adjunctive therapy to the participant's standard anti-epileptic medication for the treatment of seizures in pediatric patients from 6 months to less than 2 years old with genetically confirmed CDD during a 12-week, DB phase. Pharmacokinetic (PK) assessments and population PK analyses will also be performed during this time. The DB phase will be followed by an optional long-term OL phase at which time all participants will receive GNX as an adjunct to their standard anti-seizure medication. Efficacy, safety and tolerability, and PK assessments will continue to be performed.

Start Date01 Mar 2027

Sponsor / Collaborator

Immedica Pharma AB

NCT07635862

/ Not yet recruitingPhase 2IIT

A Randomized Controlled Trial of Ganaxolone for Children With Autism Spectrum Disorder

The goal of this study is to conduct a randomized, placebo-controlled trial (RCT) of ganaxolone, a neuroactive steroid (NAS), in autistic children and adolescents aged 5 to 17 years old. Ganaxolone is approved and effective for treating seizures in children as young as 2 years old who have CDKL5 deficiency disorder (CDD), a neurogenetic condition associated with developmental delays, seizure disorder, hypotonia, visual impairments, and autistic features. The primary outcome of interest for this trial is irritability on the Aberrant Behavior Checklist (ABC) because it is a common symptom of emotion dysregulation in ASD that impacts quality of life, including mental health, independence, educational opportunities, and integration into the community. The secondary domains of interest for this trial are restricted and repetitive behaviors (RRB), specifically insistence on sameness (IS), a subdomain of RRB characterized by inflexibility and a strong preference for predictable routines and familiar environments. Secondary outcome measures include the IS subscale from the Dimensional Assessment of Repetitive Behaviors (DARB) and subscales of the Clinical Global Impressions Scale for irritability (CGI-IR) and IS (CGI-IS). For participants living within 150 miles of Stanford University, we require participants to attend site visits and attempt EEG and MRI procedures before and after the trial, though we are recruiting nationally and the study can be completed without site vists.

Start Date01 Jan 2027

Sponsor / Collaborator

Stanford University

[+1]

CTR20250141

/ Active, not recruitingPhase 4

一项关于加那索龙口服混悬剂治疗2岁及以上细胞周期蛋白依赖性激酶5（CDKL5）缺乏症（CDD）中国患者癫痫发作的安全性和有效性的多中心、前瞻性、观察性上市后研究

[Translation] A multicenter, prospective, observational post-marketing study to investigate the safety and efficacy of ganaxolone oral suspension for the treatment of seizures in Chinese patients aged 2 years and older with cyclin-dependent kinase 5 (CDKL5) deficiency (CDD)

主要目的评价加那索龙口服混悬剂用于治疗中国CDD患者癫痫发作的安全性次要目的评价加那索龙口服混悬剂用于治疗中国CDD患者癫痫发作的有效性

[Translation]

Primary objective To evaluate the safety of ganaxolone oral suspension in the treatment of epileptic seizures in Chinese CDD patients Secondary objective To evaluate the effectiveness of ganaxolone oral suspension in the treatment of epileptic seizures in Chinese CDD patients

Start Date22 Apr 2025

Sponsor / Collaborator

Altasciences Cdmo Philadelphia LLC

[+2]

100 Clinical Results associated with Ganaxolone

100 Translational Medicine associated with Ganaxolone

100 Patents (Medical) associated with Ganaxolone

242

Literatures (Medical) associated with Ganaxolone

01 Dec 2026·MOLECULAR BIOLOGY REPORTS

Molecular mechanisms of neurosteroids in temporal lobe epilepsy: current insights and therapeutic perspectives

Review

Author: Al-Qahtani, Zainah ; Al-Gareeb, Ali I ; Hussain, Nawar R ; Waheed, Huda J ; Al-Kuraishy, Hayder M ; Mustafa, Aya M ; El-Saber Batiha, Gaber ; Papadakis, Marios ; Albuhadily, Ali K ; Alexiu, Athanasios ; Alruwaili, Mubarak

Neurosteroids (NSs) are endogenous progesterone derivatives that act as natural allosteric modulators of GABA_A receptors, exerting distinct effects from conventional receptor agonists. They play critical roles in modulating neuronal excitability, cognitive function, and stress responses, and have shown potential in mitigating seizure severity and frequency, particularly in temporal lobe epilepsy (TLE). TLE is a common drug-resistant focal epilepsy characterized by complex pathophysiology involving hippocampal sclerosis, neuroinflammation, synaptic dysfunction, and alterations in GABA_A receptor subunits. Accumulating evidence indicates that NSs, including Allopregnanolone, brexanolone, and ganaxolone, may exert neuroprotective and anti-epileptogenic effects through both direct GABAergic modulation and indirect regulation of mTORC1, AMPK, and BDNF signaling pathways, as well as autophagy activation. Experimental studies demonstrate that NSs can prolong the latent period before epilepsy onset, reduce seizure recurrence, and potentially improve cognitive outcomes. However, clinical findings remain inconsistent, and prolonged NS exposure may lead to reduced receptor sensitivity. This review synthesizes current knowledge on the mechanistic roles of NSs in TLE, highlighting their potential as adjuvant therapies alongside antiepileptic drugs. Further translational studies are warranted to clarify optimal dosing strategies, identify patient subgroups most likely to benefit, and develop targeted NS analogues for refractory TLE management.

01 Jul 2026·JOURNAL OF BIOLOGICAL CHEMISTRY

Ganaxolone, an approved therapy for CDKL5-deficiency disorder, is an inhibitor of PTP1B

Article

Author: Utama, Raditya ; Tonks, Nicholas K ; Alves, Steven R ; Zubiete-Franco, Imanol ; Hu, Qingting

CDKL5-deficiency disorder or CDD, which results in intellectual disability, speech, and motor deficits, and seizures that can start as early as 6 weeks after birth, is caused by de novo mutations in the CDKL5 gene. In early 2022, the FDA approved ganaxolone (Commercial name: Ztalmy) for the treatment of seizures in CDD patients 2 years and older. Ganaxolone has been reported to act as a GABA_A receptor agonist that helps reduce neuronal excitability; however, based on its chemical structure, we hypothesized that ganaxolone may also act as a PTP1B inhibitor. We observed that ganaxalone was able to inhibit PTP1B activity both in an in vitro assay of enzyme activity and in different cell models, with similar potency to known inhibitors of PTP1B, including MSI-1436, which has a similar chemical structure. Additionally, inhibition of PTP1B in differentiating SH-SY5Y cells increased TRKB/BDNF signaling. This effect was prominent in CDKL5-KO cells, which have increased PTP1B expression. In these cells, inhibition of PTP1B not only brought BDNF signaling to levels similar to those of WT cells but also restored cellular morphology. Our results suggest that, like in Rett syndrome, targeting PTP1B may be a beneficial therapeutic strategy for CDD.

08 Jun 2026·Journal of Chemical Information and Modeling

Open-State Dynamics and Allosteric Modulation of the α1β3γ2 GABA _A Receptor Stabilized by L9′T/S Substitutions

Article

Author: Natesan, Senthil ; Borghese, Cecilia M. ; Goldschen-Ohm, Marcel P. ; Diyaolu, Ayobami

Pentameric GABA type A receptors (GABA_ARs) mediate fast inhibitory neurotransmission and are major targets of clinically important neuroactive drugs, yet the open state of heteropentameric receptors has remained difficult to define structurally and mechanistically. Here, we establish an experimentally supported, atomistic characterization that links pore hydration, ion-permeation energetics, conductance, and global conformational rearrangements in open-like α1β3γ2 GABA_AR ensembles and delineate how positive and negative allosteric modulators bias the gating landscape. We use in-silico mutagenesis and Gaussian-accelerated molecular dynamics to stabilize open-like ensembles of the α1β3γ2 receptor via hydrophilic substitutions at the conserved 9' hydrophobic gate (L9'T/L9'S). The mutants expand the pore at 9' and 20' (pore entry), increase hydration and water flux, and lower Cl^- permeation barriers at 9' and -2' (desensitization gate) from ∼ 19/∼9 kcal/mol in wild-type receptors to ∼ 1.3-1.6/∼3.0-3.4 kcal/mol, yielding ohmic conductance of ∼10-30 pS in computational electrophysiology. Conformationally, the mutants exhibit reduced global twist, outward M2 helix tilts, and orthosteric C-loop closure, consistent with activation-like signatures. On this open-like background, positive allosteric modulators (diazepam, ganaxolone) primarily tune the residual -2' constriction, whereas the orthosteric antagonist bicuculline drives a time-ordered, bottom-to-top closing sequence (-2' first, followed by 9'/20' and global twist) via an asymmetric collapse of the M2 bundle that approaches closed-state landmarks while sampling desensitized-like coupling of extracellular and transmembrane domains. Two-electrode voltage-clamp recordings confirm spontaneous activity in L9'T-containing receptors, supporting the mutant-stabilized open-like ensembles. Together, these results provide a coherent atomistic framework for gating and allosteric modulation in α1β3γ2 GABA_ARs and establish a tractable platform for state-selective structure-based design.

147

News (Medical) associated with Ganaxolone

25 Jun 2026

·www.immedica.com

Immedica announces marketing authorization approval in the UAE for Ztalmy® (ganaxolone) for CDKL5 deficiency disorder

Stockholm, Sweden, June 23, 2026 – Immedica today announced that the Emirates Drug Establishment (EDE) has granted Marketing Authorization (MA) approval for Ztalmy® (ganaxolone) for the treatment of seizures associated with CDKL5 deficiency disorder (CDD) in the United Arab Emirates (UAE). This marks an important milestone in making Ztalmy available to eligible patients in the UAE. “The approval of Ztalmy in the UAE is an important step in expanding access to treatment options for patients living with CDD,” said Ashraf Attia, General Manager MENA. “We are very pleased that eligible patients in the UAE will now have access to this important treatment option.” About ZTALMY® (ganaxolone) ZTALMY® is indicated specifically for seizures associated with cyclin-dependent kinase-like 5 deficiency disorder (CDD) in patients two years of age and older. ZTALMY® is approved in the United States, the European Union, United Kingdom, China, Kuwait and UAE. ZTALMY is a neuroactive steroid anticonvulsant, that acts as a positive allosteric modulator of GABAA receptors in the central nervous system. About CDKL5 Deficiency Disorder CDKL5 deficiency disorder (CDD) is a serious and rare genetic disorder that is caused by a pathogenic variant in the cyclin-dependent kinase-like 5 (CDKL5) gene, located on the X chromosome. CDD is characterized by early-onset, difficult-to-control seizures and severe neuro-developmental impairment. About Immedica Immedica is a pharmaceutical company, headquartered in Stockholm, Sweden, focused on the commercialization of medicines for rare diseases and specialty care products. Immedica’s capabilities cover marketing and sales, compliance, pharmacovigilance, quality assurance, regulatory, medical affairs and market access, as well as a global distribution network serving patients in more than 50 countries. Immedica is fully dedicated to helping those living with diseases which have a large unmet medical need. Immedica’s therapeutic areas are within RARE metabolic, RARE hematology & oncology, RARE neurology, RARE Endocrinology and specialty care. Immedica was founded in 2018 and employs today around 180 people across Europe, the Middle East and the United States. Immedica is backed by the investment firms KKR and Impilo. For more information visit www.immedica.com. Immedica contact: Linda Holmström Head of HR & Communication linda.holmstrom@immedica.comFormulärets överkant Formulärets nederkant We believe we have an important role to fill in society – bringing new, innovative treatments to patients with rare diseases. Focus areas are within RARE metabolic, RARE neurology, RARE hematology & oncology and specialty care. We operate products from phase 2 and all the way to mature products. ESG is an integrated part of our business model. Stay up to date with our latest news. Telephone: +46 (0) 8 533 39 500 E-mail: info@immedica.com

Drug Approval

20 Apr 2026

·www.biospace.com

Neurogene Appoints Christy Shafer as Chief Commercial Officer and Christine Mikail, J.D., to Board of Directors

Ms. Shafer, a proven commercial leader in rare neurological diseases, will lead Neurogene’s commercial strategy and launch readiness for NGN-401 gene therapy for Rett syndrome Ms. Mikail, President and CFO of Neurogene, adds strategic and operational leadership to the Board as Company advances key priorities NEW YORK--(BUSINESS WIRE)--Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced the appointment of Christy Shafer as Chief Commercial Officer and the appointment of Christine Mikail, J.D., to its Board of Directors. Ms. Shafer brings more than 20 years of industry experience building and leading high-performing commercial organizations and launching therapies for rare neurological diseases. Ms. Mikail brings to the Board deep expertise in strategic transactions, capital formation, and corporate development, with a track record of building and positioning companies for long-term value creation. “With the addition of Christy Shafer to Neurogene’s experienced executive team, we welcome a proven commercial leader with deep expertise in building organizations and executing rare neurological disease product launches to guide our commercial strategy and launch readiness for NGN-401, our gene therapy product for Rett syndrome,” stated Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. “Christine Mikail has deep strategic, financial, transactional, and operational expertise, along with a rigorous understanding of Neurogene’s business and long-term objectives. Her perspective and judgment will strengthen the Board as Neurogene continues to advance NGN-401 and prepares for its next phase of growth.” Since joining Neurogene, Ms. Mikail has played a central role in shaping the Company’s long‑term strategy, strengthening its financial foundation, and supporting advancement of its clinical and corporate objectives. “I am honored to join Neurogene’s Board of Directors at this important stage in the Company’s evolution,” said Ms. Mikail. “I look forward to contributing my experience and perspective to Neurogene’s long-term strategy as a member of the Board.” Christy Shafer most recently served as Senior Vice President and General Manager, North America, of Avidity Biosciences, a late-stage biotech developing RNA therapeutics for rare neuromuscular diseases that was acquired by Novartis. She was responsible for building and leading Avidity’s North America commercial organization in advance of its first U.S. product launches. Prior to Avidity, Ms. Shafer served as Chief Commercial Officer at Marinus Pharmaceuticals, where she built and led the commercial organization and launched Ztalmy®, the first approved therapy for seizures associated with CDKL5 deficiency disorder, which led to Marinus Pharmaceuticals’ acquisition by Immedica Pharma. Previously, Ms. Shafer held leadership roles of increasing responsibility at Alexion Pharmaceuticals, where she supported multiple neurology launches, including Soliris® for generalized myasthenia gravis and neuromyelitis optica spectrum disorder, as well as earlier commercial leadership positions at Pacira Pharmaceuticals and Sanofi Biosurgery (formerly Genzyme). She holds a Bachelor of Life Science degree in Cell and Molecular Biology and Genetics from the University of Maryland, College Park, and completed postbaccalaureate studies in immunology and pharmacology. “Neurogene’s differentiated approach to gene therapy represents a meaningful opportunity to transform outcomes for patients with devastating neurological diseases,” said Ms. Shafer. “I am thrilled to join this team at this inflection point and look forward to partnering across the organization to help deliver the capabilities, strategy, and infrastructure needed to support a successful launch and commercialization of NGN-401, if approved.” About Neurogene Neurogene (NASDAQ: NGNE) is a clinical-stage biotechnology company focused on developing life-changing genetic medicines for people and their families impacted by devastating neurological diseases. The Company is using a biology-first approach paired with optimized delivery to develop purpose-built genetic medicines, including programs powered by its novel and proprietary EXACT™ transgene regulation technology. Neurogene is advancing its lead gene therapy program, NGN-401, as a potential best-in-class, one-time treatment for Rett syndrome. For more information, visit neurogene.com or follow on LinkedIn . Cautionary Note Regarding Forward-Looking Statements Statements in this press release are made as of the date of this press release. Neurogene does not undertake any obligation to make any updates to these statements to reflect events that occur or circumstances that arise after the date of this press release, except as may be required under applicable U.S. securities law. Statements in this press release which are not historical in nature are intended to be, and hereby are identified as, forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current expectations and beliefs of the management of Neurogene, as well as assumptions made by, and information currently available to, management of Neurogene, including, but not limited to, statements regarding: the therapeutic potential and utility, efficacy and clinical benefits of NGN-401; the safety and tolerability pro NGN-401; the potential for NGN-401 to be a best-in-class gene therapy for Rett syndrome; the potential for future approval for commercialization of NGN-401 as a treatment for Rett syndrome; commercial launch readiness for NGN-401; and the potential for success of the Embolden registrational clinical trial of NGN-401 for Rett syndrome. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” “on track,” and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Forward-looking statements are based on current beliefs and assumptions that are subject to risks, uncertainties and assumptions that are difficult to predict with regard to timing, extent, likelihood, and degree of occurrence, which could cause actual results to differ materially from anticipated results and many of which are outside of Neurogene’s control. Such risks, uncertainties and assumptions include those identified under the heading "Risk Factors" included in Neurogene’s Annual Report on Form 10-K for the year ended December 31, 2025, filed with the Securities and Exchange Commission (“SEC”) on March 17, 2026, and other filings that the Company has made and may make with the SEC in the future. Nothing in this communication should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that the contemplated results of any such forward-looking statements will be achieved. Forward-looking statements in this communication speak only as of the day they are made and are qualified in their entirety by reference to the cautionary statements herein. Except as required by applicable law, Neurogene undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise. Contacts Company Contact: Mike Devine Executive Director, Corporate Communications michael.devine@neurogene.com Investor Contact: Lina Li Executive Director, Investor Relations lina.li@neurogene.com

Executive ChangeGene TherapyAcquisition

01 Apr 2026

·www.immedica.com

Immedica announces first marketing authorization approval in the MENA region for Ztalmy® (ganaxolone) for CDKL5 deficiency disorder

Stockholm, Sweden, April 1, 2026 – Immedica today announced that the Kuwait Ministry of Health (MOH) has granted Marketing Authorization (MA) approval for Ztalmy® (ganaxolone) for the treatment of seizures associated with CDKL5 deficiency disorder (CDD) in Kuwait. This marks the first approval for Ztalmy in the Middle East and North Africa (MENA) region. “This is an important milestone for Immedica and for the expansion of Ztalmy in the MENA region,” said Ashraf Attia, General Manager MENA. “We are particularly pleased that patients in Kuwait will now be able to benefit from this important treatment option”. This approval underscores Immedica’s commitment to improving access to innovative therapies for patients with rare diseases across underserved regions. About Ztalmy® (ganaxolone) Ztalmy® is indicated specifically for seizures associated with cyclin-dependent kinase-like 5 deficiency disorder (CDD) in patients two years of age and older. Ztalmy® is approved in the United States, the European Union, United Kingdom and China. Ztalmy, a neuroactive steroid anticonvulsant that acts as a positive allosteric modulator of GABAA receptors in the central nervous system, About CDKL5 Deficiency Disorder CDKL5 deficiency disorder (CDD) is a serious and rare genetic disorder that is caused by a pathogenic variant in the cyclin-dependent kinase-like 5 (CDKL5) gene, located on the X chromosome. CDD is characterized by early-onset, difficult-to-control seizures and severe neuro-developmental impairment. About Immedica Immedica is a pharmaceutical company, headquartered in Stockholm, Sweden, focused on the commercialization of medicines for rare diseases and specialty care products. Immedica’s capabilities cover marketing and sales, compliance, pharmacovigilance, quality assurance, regulatory, medical affairs and market access, as well as a global distribution network serving patients in more than 50 countries. Immedica is fully dedicated to helping those living with diseases which have a large unmet medical need. Immedica’s therapeutic areas are within RARE metabolic, RARE hematology & oncology, RARE neurology, RARE Endocrinology and specialty care. Immedica was founded in 2018 and employs today around 180 people across Europe, the Middle East and the United States. Immedica is backed by the investment firms KKR and Impilo. For more information visit www.immedica.com. Immedica contact: Linda Holmström Head of HR & Communication linda.holmstrom@immedica.comFormulärets överkant Formulärets nederkant [JS1]Suggestion We believe we have an important role to fill in society – bringing new, innovative treatments to patients with rare diseases. Focus areas are within RARE metabolic, RARE neurology, RARE hematology & oncology and specialty care. We operate products from phase 2 and all the way to mature products. ESG is an integrated part of our business model. Stay up to date with our latest news. Telephone: +46 (0) 8 533 39 500 E-mail: info@immedica.com

Drug Approval

100 Deals associated with Ganaxolone

External Link

KEGG	Wiki	ATC	Drug Bank
D04300	Ganaxolone	-	DB05087

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Seizures	European Union	Immedica Pharma US, Inc.	26 Jul 2023
Seizures	Iceland	Immedica Pharma US, Inc.	26 Jul 2023
Seizures	Liechtenstein	Immedica Pharma US, Inc.	26 Jul 2023
Seizures	Norway	Immedica Pharma US, Inc.	26 Jul 2023
CDKL5 Deficiency Disorder	United States	Immedica Pharma AB	18 Mar 2022

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Epilepsy	Phase 3	United States	Immedica Pharma US, Inc.	17 May 2022
Epilepsy	Phase 3	United States	Tenacia Biotechnology (Shanghai) Co. Ltd.	17 May 2022
Epilepsy	Phase 3	Australia	Immedica Pharma US, Inc.	17 May 2022
Epilepsy	Phase 3	Australia	Tenacia Biotechnology (Shanghai) Co. Ltd.	17 May 2022
Epilepsy	Phase 3	Belgium	Tenacia Biotechnology (Shanghai) Co. Ltd.	17 May 2022
Epilepsy	Phase 3	Belgium	Immedica Pharma US, Inc.	17 May 2022
Epilepsy	Phase 3	Canada	Immedica Pharma US, Inc.	17 May 2022
Epilepsy	Phase 3	Canada	Tenacia Biotechnology (Shanghai) Co. Ltd.	17 May 2022
Epilepsy	Phase 3	France	Tenacia Biotechnology (Shanghai) Co. Ltd.	17 May 2022
Epilepsy	Phase 3	France	Immedica Pharma US, Inc.	17 May 2022

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05604170 (CTgov)	Phase 3	Tuberous Sclerosis	117	Ganaxolone	vxkpdiaflt = noandplwjf tmibxuprjb (pzjusyuwcs, hptonrecsx - lubrndeusf) View more	-	01 Oct 2025
NCT05323734 (TrustTSC, CTgov)	Phase 3	Tuberous Sclerosis	129	Ganaxolone (Ganaxolone)	vchtsmuuqu(aqylvxhtij) = gqrvtgetuk ffjjbcqwlu (dexiavfhtm, 60.430) View more	-	11 Jul 2025
				placebo (Matching Placebo)	vchtsmuuqu(aqylvxhtij) = xluetfhfsx ffjjbcqwlu (dexiavfhtm, 77.374) View more
NCT04391569 (RAISE, CTgov)	Phase 3	Status Epilepticus	100	Ganaxolone (Ganaxolone)	fbegwuwvva = mybylwmtsq tbnrjzepng (kzuoqpfnac, zvsawupaig - varthfirlw) View more	-	29 May 2025
				Placebo+ganaxolone (Placebo)	fbegwuwvva = nyouhrnzfl tbnrjzepng (kzuoqpfnac, bxpyipiefu - uawiqfstms) View more
NCT05323734 (TrustTSC, Biospace) Manual	Phase 3	Tuberous Sclerosis Third line	129	Ganaxolone	mjebscqyro(xuqomgwnch) = yspvydafll svlydsoveg (bvvycugydb ) Not Met	Negative	24 Oct 2024
				Placebo	mjebscqyro(xuqomgwnch) = eendmfnzkj svlydsoveg (bvvycugydb ) Not Met
NCT04391569 (RAISE, Biospace) Manual	Phase 3	Epilepsy	96	Ganaxolone	stxziyemqj(ufefjdnhqa) = qatbkpvdhb jhbdjftwry (yymzogyzti ) Met View more	Positive	17 Jun 2024
				Placebo	stxziyemqj(ufefjdnhqa) = gyoditcpuh jhbdjftwry (yymzogyzti ) Met View more
NCT03572933 (MARIGOLD Study, AAN2024)	Phase 3	CDKL5 Deficiency Disorder	101	Valproate	hdobchdekw(nfnpbuqkop) = TEAEs and SAEs were similar in ganaxolone-treated patients across all concomitant ASM subgroups. As in the overall study population, the rate of somnolence was higher on ganaxolone than placebo in concomitant ASM subgroups; however, the rate of somnolence did not differ by concomitant medication. jgujxoitwe (whxsiprqjo ) View more	Positive	09 Apr 2024
				Levetiracetam
CNS2023	Not Applicable	CDKL5 Deficiency Disorder	101	Ganaxolone	guvqpolfbj(gzqqhhpzde) = There was one death reported, which was deemed unrelated to study treatment cqlsezzofn (dplzljiwvs ) View more	Positive	01 Oct 2023
IEC2023	Not Applicable	CDKL5 Deficiency Disorder	101	Ganaxolone	xiuzlgedrs(idwagfxzlf) = 1 hnvmaqlxxu (vjhcjjssxe ) View more	Positive	04 Sep 2023
				Placebo
IEC2023	Phase 1	-	36	IV ganaxolone 10mg	rhxhqgipbn(eqbhtxrpgt) = No deaths were reported tzqdlppjvj (nwwcibljnb ) View more	-	04 Sep 2023
				IV ganaxolone 30mg
NCT00442104 (CTgov)	Phase 2	Spasms, Infantile	54	Ganaxolone	evklazakrc = byllfmbokv cawfoicoak (fdmhyivkzu, cdqrcdkjsd - afntnpwyzy) View more	-	02 Jun 2023

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