Ganaxolone - Generic Drug Details

What are the generic drug 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 eight patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Ganaxolone has forty-three patent family members in fifteen countries.

One supplier is listed for this compound.

Summary for ganaxolone

International Patents:	43
US Patents:	8
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	46
Clinical Trials:	24
Patent Applications:	937
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
Jed E. Rose	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	8,022,054		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	9,029,355		Y
Marinus	ZTALMY	ganaxolone	SUSPENSION;ORAL	215904-001	Jun 1, 2022		RX	Yes	Yes	8,367,651		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/005825 Ztalmy 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
World Intellectual Property Organization (WIPO)	2018031748
Canada	3032686	METHODES ET COMPOSITIONS POUR LE TRAITEMENT DE TROUBLES EPILEPTIQUES (USE OF GANAXOLONE FOR TREATING STATUS EPILEPTICUS IN A SUBJECT)
Spain	2812250
European Patent Office	3586845	COMPOSITIONS POUR LE TRAITEMENT DU TREMBLEMENT ESSENTIEL (COMPOSITIONS FOR TREATMENT OF ESSENTIAL TREMOR)

Ganaxolone Market Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory for Ganaxolone

Introduction

Ganaxolone, developed by Marinus Pharmaceuticals, is a promising therapeutic agent that has been making significant strides in the treatment of various seizure disorders and other neurological conditions. Here, we delve into the market dynamics and financial trajectory of ganaxolone, highlighting its current status, growth prospects, and the challenges it faces.

Mechanism of Action and Clinical Uses

Ganaxolone is a positive allosteric modulator of GABA_A receptors, which distinguishes it from traditional benzodiazepines. It is being developed in multiple formulations, including oral suspension, capsules, and intravenous forms, to cater to both adult and pediatric patient populations in acute and chronic care settings[4].

Market Potential and Growth

The market potential for ganaxolone is substantial, particularly in the treatment of rare and refractory seizure disorders. For instance, the U.S. market opportunity for ganaxolone in treating conditions like tuberous sclerosis complex (TSC) and refractory status epilepticus (RSE) is estimated to be over $3 billion[1].

Commercial Performance of ZTALMY (Ganaxolone Oral Suspension CV)

ZTALMY, the oral suspension formulation of ganaxolone, has shown impressive commercial growth. In Q3 2024, it generated net product revenue of $8.5 million, representing a 56% growth compared to Q3 2023. The full-year 2024 revenue guidance for ZTALMY has been narrowed to $33 to $34 million[2].

User Adoption and Market Acceptance

The uptake of ZTALMY has been steady, with over 120 unique prescribers and approximately 140 patients active on therapy as of Q3 2023. The median age of patients is around 9 years, with continued use in some young adults. Positive payer acceptance has also been a significant factor, with over 225 million covered lives[1].

Clinical Trials and Regulatory Milestones

Marinus Pharmaceuticals has been actively involved in various clinical trials for ganaxolone. The Phase 3 TrustTSC trial results were reported, and the company is scheduled to meet with the FDA in Q4 2024 to discuss a potential path forward for IV ganaxolone in refractory status epilepticus. However, further ganaxolone clinical development has been suspended following a trial setback[2][5].

Financial Health and Cost Reduction Measures

Despite the revenue growth, Marinus Pharmaceuticals faces significant financial challenges. The company has initiated cost reduction measures, including a workforce reduction of approximately 45%, to extend its cash runway into Q2 2025. As of September 30, 2024, Marinus had $42.2 million in cash and cash equivalents[2].

Strategic Alternatives and Market Valuation

Marinus has commenced a process to explore strategic alternatives, engaging Barclays as an advisor. This move is aimed at maximizing value for stockholders. The company's market capitalization stands at $93.09 million, reflecting the market's current valuation post the clinical trial setback[5].

Competitive Landscape and Market Forecast

The market for ganaxolone is competitive, with other emerging therapies targeting similar conditions. The forecasted sales data from 2020 to 2030 indicate a potential market size in the millions, but the launch of late-stage emerging therapies will significantly impact the market dynamics[4].

SWOT Analysis

• Strengths: Strong commercial performance of ZTALMY, positive payer acceptance, and a robust market potential.
• Weaknesses: Clinical trial setbacks, significant cash burn rate, and the need for cost reduction measures.
• Opportunities: Potential launches in new indications like tuberous sclerosis complex and the exploration of strategic alternatives.
• Threats: Competitive market landscape, regulatory uncertainties, and the impact of trial setbacks on investor confidence[4].

Quotes from Industry Experts

"Marinus continues to support the commercial growth of ZTALMY; further ganaxolone clinical development has been suspended," highlights the company's current focus on optimizing existing products while navigating clinical challenges[2].

Illustrative Statistics

• Q3 2024 net product revenue for ZTALMY: $8.5 million, a 56% growth versus Q3 2023[2].
• Full-year 2024 revenue guidance for ZTALMY: $33 to $34 million[2].
• Cash and cash equivalents as of September 30, 2024: $42.2 million[2].
• Market capitalization: $93.09 million[5].

Highlight

"Ganaxolone exhibits anti-seizure and anti-anxiety activity via its effects on synaptic and extra-synaptic GABAA receptors," which differentiates it from traditional benzodiazepines and underscores its therapeutic potential[4].

Key Takeaways

• Ganaxolone has shown significant commercial growth, particularly with ZTALMY.
• The drug faces challenges due to clinical trial setbacks and financial constraints.
• Marinus Pharmaceuticals is exploring strategic alternatives to maximize value.
• The market potential for ganaxolone remains robust, especially in rare and refractory seizure disorders.

FAQs

1. What is ganaxolone and how does it work? Ganaxolone is a positive allosteric modulator of GABA_A receptors, used in the treatment of seizure disorders and other neurological conditions. It works by enhancing the activity of GABA_A receptors, which are involved in inhibitory neurotransmission.

2. What are the current commercial performance and growth prospects of ZTALMY? ZTALMY has shown a 56% growth in Q3 2024 compared to Q3 2023, with a full-year 2024 revenue guidance of $33 to $34 million. The drug has seen steady uptake with over 120 unique prescribers and approximately 140 patients active on therapy.

3. What are the challenges faced by Marinus Pharmaceuticals in the development of ganaxolone? Marinus Pharmaceuticals has faced clinical trial setbacks, leading to the suspension of further ganaxolone clinical development. The company is also experiencing significant financial challenges, including a high cash burn rate.

4. What strategic alternatives is Marinus Pharmaceuticals exploring? Marinus has commenced a process to explore strategic alternatives, engaging Barclays as an advisor, to maximize value for its stockholders.

5. What is the market potential for ganaxolone in the coming years? The market potential for ganaxolone is substantial, particularly in the treatment of rare and refractory seizure disorders, with an estimated U.S. market opportunity of over $3 billion.

Cited Sources:

1. Marinus Pharmaceuticals Investor Day Full Deck FINAL V3[1]
2. Marinus Pharmaceuticals Provides Business Update and Reports Third Quarter 2024 Financial Results[2]
3. Ovid Therapeutics Reports Business Update, Fourth Quarter and Full-Year 2021 Financial Results[3]
4. Ganaxolone - Emerging Insight and Market Forecast - 2030[4]
5. Marinus halts ganaxolone development after trial setback[5]