All FDA-approved EMA-approved PMDA-approved
Target Card Uniprot Example: P23975

ganaxolone 🐶 Veterinary Use | Indications/Contra | FAERs-F | FAERs-M | Orange Bk | PK/Tox | Related Drugs | BioActivity |

Stem definition	Drug id	CAS RN
steroids not used as glucocorticosteroids	5508	38398-32-2

Description:

Molecule	Description
Molfile Inchi Smiles Synonyms: ganaxolone ztalmy GNX CCD 1042	The precise mechanism by which ganaxolone exerts its therapeutic effects in the treatment of seizures associated with CDD is unknown, but its anticonvulsant effects are thought to result from positive allosteric modulation of the gamma-aminobutyric acid type A (GABAA) receptor in the CNS. Molecular weight: Formula: CLOGP: 5.03 LIPINSKI: 0 HAC: 2 HDO: 1 TPSA: ALOGS: -5.67 ROTB: 1

Drug dosage:

None

ADMET properties:

None

Predicted pharmacokinetic/toxicological properties (PK-AZ):

None

Approvals:

Date	Agency	Company	Orphan
March 18, 2022	FDA	MARINUS PHARMA INC
July 26, 2023	EMA	Marinus Pharmaceuticals Emerald Limited

FDA Adverse Event Reporting System (Female)

MedDRA adverse event term	Likelihood ratio	Likelihood ratio threshold	Patients taking drug having adverse event	Patients taking drug not having adverse event	Patients not taking drug having adverse event	Patients not taking drug not having adverse event
Product dose omission issue	77.12	71.57	20	39	291667	90336721

FDA Adverse Event Reporting System (Male)

None

FDA Adverse Event Reporting System (Geriatric)

None

FDA Adverse Event Reporting System (Pediatric)

None

Pharmacologic Action:

Source	Code	Description
ATC	N03AX27	NERVOUS SYSTEM ANTIEPILEPTICS ANTIEPILEPTICS Other antiepileptics
MeSH PA	D000081227	Neurosteroids
MeSH PA	D018377	Neurotransmitter Agents
MeSH PA	D018682	GABA Agents
MeSH PA	D018757	GABA Modulators
FDA MoA	N0000193997	GABA A Receptor Positive Modulators
FDA EPC	N0000194006	Neuroactive Steroid Gamma-Aminobutyric Acid A Receptor Positive Modulator
CHEBI has role	CHEBI:35469	antidepressant
CHEBI has role	CHEBI:35623	anticonvulsant
CHEBI has role	CHEBI:53784	antispasmodic drug

Related Drugs by ATC class:

N03AX Other antiepileptics 18 drugs

beclamidebrivaracetamcannabidiolcarisbamatecenobamatefelbamatefenfluraminelacosamidelamotriginelevetiracetamperampanelphenacemide show all 18 →

Drug Use | Suggest Off label Use Form| |View source of the data|

Disease	Relation	SNOMED_ID	DOID
Seizures associated with CDKL5 deficiency disorders	indication	773230003

🐶 Veterinary Drug Use

None

🐶 Veterinary products

None

Acid dissociation constants calculated using MoKa v3.0.0

None

Orange Book patent data (new drug applications)

Formulation strength	Trade name	Applicant	Application number	Approval date	Type	Dose form	Route	Patent number	Patent expiration date	Patent use
50MG/ML	ZTALMY	IMMEDICA PHARMA	N215904	June 1, 2022	RX	SUSPENSION	ORAL	8618087	Nov. 28, 2026	TREATMENT OF SEIZURES ASSOCIATED WITH CYCLIN-DEPENDENT KINASE-LIKE 5 (CDKL5) DEFICIENCY DISORDER (CDD) IN PATIENTS 2 YEARS OF AGE AND OLDER
50MG/ML	ZTALMY	IMMEDICA PHARMA	N215904	June 1, 2022	RX	SUSPENSION	ORAL	9056116	Nov. 28, 2026	TREATMENT OF SEIZURES ASSOCIATED WITH CYCLIN-DEPENDENT KINASE-LIKE 5 (CDKL5) DEFICIENCY DISORDER (CDD) IN PATIENTS 2 YEARS OF AGE AND OLDER
50MG/ML	ZTALMY	IMMEDICA PHARMA	N215904	June 1, 2022	RX	SUSPENSION	ORAL	10603308	Aug. 10, 2037	TREATMENT OF SEIZURES ASSOCIATED WITH CYCLIN-DEPENDENT KINASE-LIKE 5 (CDKL5) DEFICIENCY DISORDER (CDD) IN PATIENTS 2 YEARS OF AGE AND OLDER
50MG/ML	ZTALMY	IMMEDICA PHARMA	N215904	June 1, 2022	RX	SUSPENSION	ORAL	12144801	Aug. 10, 2037	TREATMENT OF SEIZURES ASSOCIATED WITH CYCLIN-DEPENDENT KINASE-LIKE-5 (CDLK5) DEFICIENCY DISORDER (CDD) IN PATIENTS 2 YEARS OF AGE AND OLDER
50MG/ML	ZTALMY	IMMEDICA PHARMA	N215904	June 1, 2022	RX	SUSPENSION	ORAL	12115169	Sept. 30, 2042	TREATMENT OF SEIZURES ASSOCIATED WITH CYCLIN-DEPENDENT KINASE-LIKE 5 (CDKL5) DEFICIENCY DISORDER (CDD) IN PATIENTS 2 YEARS OF AGE AND OLDER
50MG/ML	ZTALMY	IMMEDICA PHARMA	N215904	June 1, 2022	RX	SUSPENSION	ORAL	12268696	Sept. 30, 2042	TREATMENT OF SEIZURES ASSOCIATED WITH CYCLIN-DEPENDENT KINASE-LIKE 5 (CDKL5) DEFICIENCY DISORDER (CDD) IN PATIENTS 2 YEARS OF AGE AND OLDER

Orange Book exclusivity data (new drug applications)

Formulation strength	Trade name	Applicant	Application number	Approval date	Type	Dose form	Route	Exclusivity date	Description
50MG/ML	ZTALMY	IMMEDICA PHARMA	N215904	June 1, 2022	RX	SUSPENSION	ORAL	June 1, 2027	NEW CHEMICAL ENTITY
50MG/ML	ZTALMY	IMMEDICA PHARMA	N215904	June 1, 2022	RX	SUSPENSION	ORAL	Oct. 16, 2028	REVISED DOSING TITRATION REGIMEN
50MG/ML	ZTALMY	IMMEDICA PHARMA	N215904	June 1, 2022	RX	SUSPENSION	ORAL	June 1, 2029	TREATMENT OF SEIZURES ASSOCIATED WITH CYCLIN-DEPENDENT KINASE-LIKE 5 (CDKL5) DEFICIENCY DISORDER (CDD) IN PATIENTS 2 YEARS OF AGE AND OLDER

Bioactivity Summary:

Target	Class	Pharos	UniProt	Action	Type	Activity value (-log[M])	Mechanism action	Bioact source	MoA source
GABA A receptor alpha-1/beta-1/gamma-2	Ion channel	P14867 P18505 P18507	GBRA1_HUMAN GBRB1_HUMAN GBRG2_HUMAN	MODULATOR	EC50	6.67		SCIENTIFIC LITERATURE
GABA A receptor alpha-3/beta-1/gamma-2	Ion channel	P18505 P18507 P34903	GBRB1_HUMAN GBRG2_HUMAN GBRA3_HUMAN	MODULATOR	EC50	6.91		SCIENTIFIC LITERATURE
GABA A receptor alpha-2/beta-1/gamma-2	Ion channel	P18505 P18507 P47869	GBRB1_HUMAN GBRG2_HUMAN GBRA2_HUMAN	MODULATOR	EC50	7.03		SCIENTIFIC LITERATURE

External reference:

ID	Source
CHEBI:177658	CHEBI
CHEMBL1568698	ChEMBL_ID
C105051	MESH_SUPPLEMENTAL_RECORD_UI
11942	IUPHAR_LIGAND_ID
DB05087	DRUGBANK_ID
4041529	VANDF
C0537150	UMLSCUI
D04300	KEGG_DRUG
7476	INN_ID
6918305	PUBCHEM_CID
357899	MMSL
40556	MMSL
d09866	MMSL
018994	NDDF
98WI44OHIQ	UNII
2604689	RXNORM

Pharmaceutical products:

Product	Category	Ingredients	NDC	Form	Quantity	Route	Marketing	Label
ZTALMY	HUMAN PRESCRIPTION DRUG LABEL	1	81583-100	SUSPENSION	50 mg	ORAL	NDA	35 sections
ZTALMY	HUMAN PRESCRIPTION DRUG LABEL	1	81583-100	SUSPENSION	50 mg	ORAL	NDA	35 sections