VAMOROLONE - Generic Drug Details

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

Vamorolone is the generic ingredient in one branded drug marketed by Catalyst Pharms and is included in one NDA. There are six patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Vamorolone has forty-six patent family members in twenty-two countries.

One supplier is listed for this compound.

Summary for VAMOROLONE

International Patents:	46
US Patents:	6
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	37
Clinical Trials:	7
Patent Applications:	201
What excipients (inactive ingredients) are in VAMOROLONE?	VAMOROLONE excipients list
DailyMed Link:	VAMOROLONE at DailyMed

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

Constraining patent/regulatory exclusivity:

TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (DMD) 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 VAMOROLONE

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

Sponsor	Phase
Santhera Pharmaceuticals	Phase 2
ReveraGen BioPharma, Inc.	Phase 1/Phase 2
Newcastle University	Phase 2

See all VAMOROLONE clinical trials

Anatomical Therapeutic Chemical (ATC) Classes for VAMOROLONE

H02AB	Glucocorticoids
H02A	CORTICOSTEROIDS FOR SYSTEMIC USE, PLAIN
H02	CORTICOSTEROIDS FOR SYSTEMIC USE
H	Systemic hormonal preparations, excluding sex hormones and insulins

US Patents and Regulatory Information for VAMOROLONE

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Catalyst Pharms	AGAMREE	vamorolone	SUSPENSION;ORAL	215239-001	Oct 26, 2023		RX	Yes	Yes			Y	Y
Catalyst Pharms	AGAMREE	vamorolone	SUSPENSION;ORAL	215239-001	Oct 26, 2023		RX	Yes	Yes
Catalyst Pharms	AGAMREE	vamorolone	SUSPENSION;ORAL	215239-001	Oct 26, 2023		RX	Yes	Yes			Y
Catalyst Pharms	AGAMREE	vamorolone	SUSPENSION;ORAL	215239-001	Oct 26, 2023		RX	Yes	Yes
Catalyst Pharms	AGAMREE	vamorolone	SUSPENSION;ORAL	215239-001	Oct 26, 2023		RX	Yes	Yes			Y

International Patents for VAMOROLONE

Country	Patent Number	Title	Estimated Expiration
Canada	2725008	MODULATEURS STEROIDIENS NON HORMONAUX DE NF-KB POUR LE TRAITEMENT D'UNE MALADIE (NON-HORMONAL STEROID MODULATORS OF NF-KB FOR TREATMENT OF DISEASE)
Japan	2011521964
World Intellectual Property Organization (WIPO)	2017004205
South Korea	20110020868	NON-HORMONAL STEROID MODULATORS OF NF-κB FOR TREATMENT OF DISEASE
Denmark	2444092

Supplementary Protection Certificates for VAMOROLONE

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
2805720	CA 2024 00018	Denmark		PRODUCT NAME: VAMOROLONE; REG. NO/DATE: EU/1/23/1776 20231215
2805720	301273	Netherlands		PRODUCT NAME: VAMOROLONE; REGISTRATION NO/DATE: EU/1/23/1776 20231215

VAMOROLONE Market Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory for Vamorolone

Introduction to Vamorolone

Vamorolone is an investigational drug developed for the treatment of Duchenne Muscular Dystrophy (DMD), a severe and progressive genetic disorder. The drug is a dissociative anti-inflammatory steroid, designed to offer the anti-inflammatory benefits of traditional corticosteroids while minimizing their adverse effects.

Market Overview for DMD Treatments

The market for DMD treatments is poised for significant growth due to increasing research and healthcare spending globally. The emergence of new therapies, including vamorolone, is expected to expand the market size, allowing drug manufacturers to penetrate deeper into the market[1].

Regulatory Milestones and Development Activities

Vamorolone has made substantial progress in its regulatory journey. Santhera Pharmaceuticals, in collaboration with ReveraGen BioPharma, has submitted regulatory filings for vamorolone in the U.S., EU, and UK. These filings include a New Drug Application (NDA) in the U.S. and a Marketing Authorization Application (MAA) in the EU, with potential approvals anticipated in Q4 2023 and launches planned for Q1 2024[2][5].

Clinical Trials and Efficacy

The VISION-DMD study, a pivotal clinical trial, has shown positive topline results, demonstrating vamorolone's sustained efficacy and good safety and tolerability over 48 weeks. These results support the potential benefits of vamorolone in overcoming the challenges associated with long-term use of corticosteroids in DMD patients[3].

Market Competition

Vamorolone faces competition from other emerging products for DMD. However, its unique profile as a dissociative anti-inflammatory steroid positions it favorably in the market. The launch of late-stage emerging therapies in the near future will significantly impact the market dynamics, but vamorolone's early mover advantage and strong clinical data are expected to give it a competitive edge[1].

Licensing and Partnerships

Santhera has secured strategic partnerships to enhance the global reach of vamorolone. The company has granted an exclusive North American license to Catalyst Pharmaceuticals, which expects to launch vamorolone in the U.S. early in Q1 2024, subject to regulatory approval. Additionally, Santhera has out-licensed vamorolone for the Greater China Region to Sperogenix[2][4].

Financial Performance and Funding

Santhera's financial performance has been influenced significantly by the development and regulatory activities surrounding vamorolone. In the first half of 2023, Santhera reported revenue from contracts with customers of CHF 3.9 million, with operating expenses of CHF 22.5 million, resulting in an operating loss of CHF 20.3 million. However, the company has secured financing through licensing deals, including a USD 90 million upfront payment from Catalyst Pharmaceuticals, which extends Santhera's cash reach into 2025[2][5].

Revenue Projections

Santhera estimates the peak sales potential for vamorolone in DMD to be in excess of USD 500 million in the U.S. and the largest five European countries combined. This projection underscores the significant market potential for vamorolone, driven by its efficacy and the growing demand for effective DMD treatments[3].

Operational Expenses and Cost Structure

The development expenses for vamorolone have been substantial, reflecting the costs associated with clinical trials, regulatory services, and market readiness preparations. In 2022, development expenses amounted to CHF 30.5 million, with additional costs related to intangible impairment and market preparations. However, these expenses are expected to yield long-term benefits as vamorolone approaches commercialization[5].

Strategic Initiatives and Market Readiness

Santhera has been actively preparing for the commercial launch of vamorolone. The company has expanded its U.S. operations, concluded out-licensing agreements, and implemented measures to secure funding and strengthen its capital structure. These initiatives are crucial for ensuring a successful market entry and maximizing the drug's market potential[2][5].

Financial Outlook

The financial outlook for Santhera, driven by vamorolone, is promising. With secured financing and a solid cash position, the company is well-positioned to navigate the upcoming regulatory decisions and commercialization efforts. The potential approvals and launches in 2024 are expected to significantly improve Santhera's financial performance, transitioning the company from a development phase to a commercial phase[2].

Key Takeaways

• Regulatory Progress: Vamorolone has advanced significantly in its regulatory journey, with potential approvals in Q4 2023 and launches in Q1 2024.
• Clinical Efficacy: Positive clinical trial results have demonstrated vamorolone's efficacy and safety, positioning it favorably in the DMD treatment market.
• Market Potential: Estimated peak sales of over USD 500 million in key markets highlight the significant commercial potential of vamorolone.
• Financial Stability: Secured financing and strategic partnerships ensure Santhera's financial stability as it approaches commercialization.
• Competitive Edge: Vamorolone's unique profile and early mover advantage give it a competitive edge in the DMD treatment market.

FAQs

What is vamorolone and how does it differ from traditional corticosteroids?

Vamorolone is a dissociative anti-inflammatory steroid designed to offer the anti-inflammatory benefits of traditional corticosteroids while minimizing their adverse effects.

What are the key regulatory milestones for vamorolone?

Vamorolone has submitted regulatory filings in the U.S., EU, and UK, with potential approvals anticipated in Q4 2023 and launches planned for Q1 2024.

Who are the key partners involved in the development and commercialization of vamorolone?

Santhera Pharmaceuticals is developing vamorolone in collaboration with ReveraGen BioPharma. Santhera has also partnered with Catalyst Pharmaceuticals for North American licensing and Sperogenix for the Greater China Region.

What is the estimated market potential for vamorolone in DMD?

Santhera estimates the peak sales potential for vamorolone in DMD to be in excess of USD 500 million in the U.S. and the largest five European countries combined.

How has Santhera secured financing for the launch preparations of vamorolone?

Santhera has secured financing through licensing deals, including a USD 90 million upfront payment from Catalyst Pharmaceuticals, which extends the company's cash reach into 2025.

Sources

1. ResearchAndMarkets.com, "Vamorolone - Emerging Insight and Market Forecast - 2030".
2. BioSpace, "Santhera Announces Half-Year 2023 Financial Results and Provides Corporate Update".
3. Santhera Pharmaceuticals, "Santhera and ReveraGen Announce Positive Topline Results with Vamorolone after Completion of the VISION-DMD Study".
4. Catalyst Pharmaceuticals, "Catalyst Pharmaceuticals Acquires Exclusive North American License For Vamorolone for Duchenne Muscular Dystrophy from Santhera Pharmaceuticals".
5. Santhera Pharmaceuticals, "Santhera Announces Preliminary Unaudited 2022 Annual Results Ahead of Full Report Publication by End of May and Provides Corporate Update".