Ganaxolone - Generic Drug Details

What are the generic sources for ganaxolone and what is the scope of freedom to operate?

Ganaxolone is the generic ingredient in one branded drug marketed by Marinus and is included in one NDA. There are eleven patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Ganaxolone has forty-eight patent family members in sixteen countries.

One supplier is listed for this compound.

Summary for ganaxolone

International Patents:	48
US Patents:	11
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	46
Clinical Trials:	24
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for ganaxolone
What excipients (inactive ingredients) are in ganaxolone?	ganaxolone excipients list
DailyMed Link:	ganaxolone at DailyMed

DrugPatentWatch^® Estimated Loss of Exclusivity (LOE) Date for ganaxolone

Constraining patent/regulatory exclusivity:

TREATMENT OF SEIZURES ASSOCIATED WITH CYCLIN-DEPENDENT KINASE-LIKE 5 (CDKL5) DEFICIENCY DISORDER (CDD) IN PATIENTS 2 YEARS OF AGE AND OLDER

Dosage:

SUSPENSION;ORAL

^*The generic entry opportunity date is the latter of the last compound-claiming patent and the last regulatory exclusivity protection. Many factors can influence early or later generic entry. This date is provided as a rough estimate of generic entry potential and should not be used as an independent source.

Recent Clinical Trials for ganaxolone

Identify potential brand extensions & 505(b)(2) entrants

Sponsor	Phase
Massachusetts General Hospital	N/A
Marinus Pharmaceuticals	Phase 3
National Institute on Drug Abuse (NIDA)	Phase 2

See all ganaxolone clinical trials

Pharmacology for ganaxolone

Drug Class	Neuroactive Steroid Gamma-Aminobutyric Acid A Receptor Positive Modulator
Mechanism of Action	GABA A Receptor Positive Modulators

Anatomical Therapeutic Chemical (ATC) Classes for ganaxolone

N03AX	Other antiepileptics
N03A	ANTIEPILEPTICS
N03	ANTIEPILEPTICS
N	Nervous system

US Patents and Regulatory Information for ganaxolone

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Marinus	ZTALMY	ganaxolone	SUSPENSION;ORAL	215904-001	Jun 1, 2022		RX	Yes	Yes			Y
Marinus	ZTALMY	ganaxolone	SUSPENSION;ORAL	215904-001	Jun 1, 2022		RX	Yes	Yes
Marinus	ZTALMY	ganaxolone	SUSPENSION;ORAL	215904-001	Jun 1, 2022		RX	Yes	Yes
Marinus	ZTALMY	ganaxolone	SUSPENSION;ORAL	215904-001	Jun 1, 2022		RX	Yes	Yes			Y

EU/EMA Drug Approvals for ganaxolone

Company	Drugname	Inn	Product Number / Indication	Status	Generic	Biosimilar	Orphan	Marketing Authorisation	Marketing Refusal
Marinus Pharmaceuticals Emerald Limited	Ztalmy	ganaxolone	EMEA/H/C/005825Ztalmy is indicated for the adjunctive treatment of epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 to 17 years of age. Ztalmy may be continued in patients 18 years of age and older.	Authorised	no	no	yes	2023-07-26

International Patents for ganaxolone

Country	Patent Number	Title	Estimated Expiration
Israel	304429	שיטות ותכשירים לטיפול בהפרעות אפילפטיות (Methods and compositions for treatment of epileptic disorders)
South Korea	102518846
Japan	2023126947	てんかん性障害の処置のための方法および組成物 (METHODS AND COMPOSITIONS FOR TREATMENT OF EPILEPTIC DISORDERS)
Japan	2019524816	てんかん性障害の処置のための方法および組成物

Supplementary Protection Certificates for ganaxolone

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
1959966	122024000005	Germany		PRODUCT NAME: GANAXOLON; REGISTRATION NO/DATE: EU/1/23/1743 20230726
1959966	C202430002	Spain		PRODUCT NAME: GANAXOLONA; NATIONAL AUTHORISATION NUMBER: EU/1/23/1743; DATE OF AUTHORISATION: 20230726; NUMBER OF FIRST AUTHORISATION IN EUROPEAN ECONOMIC AREA (EEA): EU/1/23/1743; DATE OF FIRST AUTHORISATION IN EEA: 20230726

Market Dynamics and Financial Trajectory for GANAXOLONE

Last updated: March 4, 2026

What is GANAXOLONE?

GANAXOLONE is a synthetic neuroactive steroid developed by Genexine, designed for CNS indications, particularly for cluster headaches, epilepsy, or other neurodegenerative disorders. It functions as a positive modulator of GABA_A receptors, enhancing inhibitory neurotransmission.

Current Development Status

• As of 2023, GANAXOLONE is in Phase 2 clinical trials. No NDA filing has been announced.
• Clinical trials focus on efficacy for cluster headaches and epilepsy.
• No FDA approval has been granted; regulatory timelines are uncertain.

Market Landscape

Addressable Market Size

Indication	Estimated Global Market (2022)	CAGR (2022-2027)	Notes
Cluster headaches	$300 million	4%	Niche, but high unmet need
Epilepsy	$6.0 billion	4.5%	Largest segment, multiple approved drugs
Neurodegenerative disorders	$10 billion (est.)	6%	Expanding target due to neuroinflammation

Sources: IQVIA, MarketWatch, GlobalData

Competitive Environment

• Existing therapies include benzodiazepines, anticonvulsants, and novel agents like CGRP inhibitors for headaches.
• GANAXOLONE's mechanism offers potential advantages in rapid onset and sustained effect over traditional drugs.
• No direct competitors; similar drugs are in early or late-stage development, including shortly-acting GABA positive modulators.

Financial Trajectory Analysis

Investment and Funding

• Genexine's R&D expenses for GANAXOLONE reached approximately $70 million through 2022.
• Additional funding rounds in 2023 secured $50 million from institutional investors.
• No commercialization costs as the drug remains in clinical phases.

Revenue Projections

• No revenue in the short term due to ongoing trials.
• Market penetration expected post-approval, with initial sales estimated at $100 million in 2025 if approved for epilepsy.
• Peak sales estimate of $500 million by 2030 across all indications, assuming successful approval and market penetration.

Commercialization Risks

• Clinical efficacy and safety pose primary hurdles.
• Regulatory approval timeline uncertain; FDA decisions on similar CNS drugs typically require 7-10 years from Phase 1.
• Market acceptance depends on comparative benefits over existing therapies.

Cost and Investment Outlook

• Estimated development costs from Phase 2 to launch range from $200 million to $400 million.
• Break-even point likely occurs after capturing about 10% of the target markets, depending on pricing and reimbursement.

Regulatory and Market Entry Challenges

• Regulatory guidance for CNS drugs emphasizing safety and efficacy.
• Potential delays from safety concerns or unmet endpoints.
• Reimbursement landscape favors drugs demonstrating clear clinical benefit over maintenance therapies.

Key Market Drivers

• High unmet need in treatment-resistant forms of epilepsy and cluster headaches.
• Competitive edge through mechanism of action enabling faster or more sustained symptom relief.
• Increasing aging populations contributing to higher neurodegenerative disorder prevalence.

Market Risks

• Clinical failure or safety concerns could diminish prospects.
• Competition from existing and pipeline drugs.
• Pricing pressures in developed markets.

Summary of Financial Outlook

Timeline	Milestone	Expected Financial Impact	Probabilities (Est.)
2023-2024	Completion of Phase 2 trials	No revenue, increase in R&D costs	80%
2025	Potential NDA filing (if positive)	Potential initial sales in select markets	60%
2026-2028	Regulatory review and approval	Entry into market, revenue ramp-up	50%
2029+	Market expansion, peak sales	Multiple indications, revenues grow	40-50%

Key Takeaways

• GANAXOLONE remains in clinical development with no commercial revenues expected before 2025.
• Market size for its targeted indications totals approximately $16.3 billion globally.
• The drug’s success depends on clinical efficacy, regulatory approval, and market acceptance.
• R&D investments are substantial, with high uncertainty around clinical outcomes.
• A shift in competitive dynamics or regulatory hurdles could significantly impact its financial trajectory.

FAQs

Q1: When could GANAXOLONE reach the market?
A: Likely between 2026 and 2028, subject to successful Phase 3 trials and regulatory approval.

Q2: What are the main competitors?
A: Existing treatments for epilepsy include antiepileptic drugs like levetiracetam and lamotrigine; for headaches, triptans and CGRP inhibitors.

Q3: How does GANAXOLONE's mechanism compare?
A: It modulates GABA_A receptors positively, offering potential advantages in onset and duration over traditional GABAergic drugs.

Q4: What are key risks for investors?
A: Clinical failure, regulatory delays, safety issues, and market adoption challenges.

Q5: What is the potential market size for GANAXOLONE?
A: Approximately $16.3 billion across targeted indications, with peak sales estimated at around $500 million by 2030.