Setmelanotide acetate - Generic Drug Details

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

Setmelanotide acetate is the generic ingredient in one branded drug marketed by Rhythm and is included in one NDA. There are three patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Setmelanotide acetate has ninety-seven patent family members in twenty-two countries.

Summary for setmelanotide acetate

International Patents:	97
US Patents:	3
Tradenames:	1
Applicants:	1
NDAs:	1
Raw Ingredient (Bulk) Api Vendors:	23
Patent Applications:	82
DailyMed Link:	setmelanotide acetate at DailyMed

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

Constraining patent/regulatory exclusivity:

11,129,869

Dosage:

SOLUTION;SUBCUTANEOUS

^*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.

Anatomical Therapeutic Chemical (ATC) Classes for setmelanotide acetate

A08AA	Centrally acting antiobesity products
A08A	ANTIOBESITY PREPARATIONS, EXCL. DIET PRODUCTS
A08	ANTIOBESITY PREPARATIONS, EXCL. DIET PRODUCTS
A	Alimentary tract and metabolism

US Patents and Regulatory Information for setmelanotide acetate

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Rhythm	IMCIVREE	setmelanotide acetate	SOLUTION;SUBCUTANEOUS	213793-001	Nov 25, 2020		RX	Yes	Yes
Rhythm	IMCIVREE	setmelanotide acetate	SOLUTION;SUBCUTANEOUS	213793-001	Nov 25, 2020		RX	Yes	Yes	9,458,195		Y	Y
Rhythm	IMCIVREE	setmelanotide acetate	SOLUTION;SUBCUTANEOUS	213793-001	Nov 25, 2020		RX	Yes	Yes
Rhythm	IMCIVREE	setmelanotide acetate	SOLUTION;SUBCUTANEOUS	213793-001	Nov 25, 2020		RX	Yes	Yes	11,129,869		Y
Rhythm	IMCIVREE	setmelanotide acetate	SOLUTION;SUBCUTANEOUS	213793-001	Nov 25, 2020		RX	Yes	Yes	8,039,435		Y	Y

International Patents for setmelanotide acetate

Country	Patent Number	Title	Estimated Expiration
Portugal	2970389
Australia	2006269261	Melanocortin receptor ligands
Denmark	2548568
South Korea	20100016120
Hong Kong	1258965

Supplementary Protection Certificates for setmelanotide acetate

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
2236151	122021000084	Germany		PRODUCT NAME: SETMELANOTID; REGISTRATION NO/DATE: EU/1/21/1564 20210716
2970389	301149	Netherlands		PRODUCT NAME: SETMELANOTIDE, DESGEWENST IN DE VORM VAN EEN FARMACEUTISCH AANVAARDBAAR ZOUT; REGISTRATION NO/DATE: EU/1/21/1564 20210719
2236151	52/2021	Austria		PRODUCT NAME: SETMELANOTID; REGISTRATION NO/DATE: EU/1/21/1564 (MITTEILUNG) 20210719
2970389	SPC/GB21/073	United Kingdom		PRODUCT NAME: SETMELANOTIDE AND PHARMACEUTICALLY ACCEPTABLE SALTS THEREOF; REGISTERED: UK EU/1/21/1564(FOR NI) 20210719; UK FURTHER MA ON IPSUM 20210719
2970389	CR 2021 00054	Denmark		PRODUCT NAME: SETMELANOTID ELLER ET FARMACEUTISK ACCEPTABELT SALT DERAF; REG. NO/DATE: EU/1/21/1564 20210719

Market Dynamics and Financial Trajectory for Setmelanotide Acetate

Introduction to Setmelanotide Acetate

Setmelanotide acetate, marketed as IMCIVREE, is a melanocortin-4 receptor (MC4R) agonist developed by Rhythm Pharmaceuticals. It is designed to treat rare genetic disorders of obesity, including Bardet-Biedl syndrome (BBS) and other conditions associated with hyperphagia and severe obesity.

Market Need and Target Population

Setmelanotide addresses a significant unmet medical need for patients with rare genetic obesity disorders. Hypothalamic obesity, for instance, affects approximately 5,000 to 10,000 individuals in the United States, with no approved therapies available until the introduction of setmelanotide[4].

Regulatory Approvals and Expansions

Current Approvals

IMCIVREE has received regulatory approvals in several countries, including the United States, for the treatment of patients with BBS and other rare genetic disorders of obesity. Recent approvals in Spain and Italy for reimbursement further expand its market reach[1].

Pending Approvals and Label Expansions

Rhythm Pharmaceuticals has submitted a supplementary New Drug Application (sNDA) to the FDA to expand the label to include patients as young as 2 years old in approved indications. This sNDA has been accepted for Priority Review with a PDUFA goal date of December 26, 2024[2][3].

Clinical Trials and Development

Phase 3 Trials

The company has completed enrollment in a pivotal Phase 3 trial evaluating setmelanotide in 120 patients with acquired hypothalamic obesity. This trial, which randomizes patients 2:1 to setmelanotide therapy or placebo, is expected to yield top-line data in the first half of 2025[1][2][4].

Real-World Data and Early-Access Programs

Real-world data from an early-access program in France showed significant BMI reductions in adult patients with acquired hypothalamic obesity. Eight patients achieved a mean BMI reduction of 12.8% at three months, and five patients achieved a mean BMI reduction of 21.3% after six months on therapy[2][3].

Financial Performance

Revenue Growth

In the fourth quarter of 2023, Rhythm Pharmaceuticals reported net revenue from global sales of IMCIVREE of $24.2 million. This figure increased to $33.3 million in the third quarter of 2024, driven primarily by sales for BBS. The revenue growth is notable, with a 14% increase on a sequential basis from the second quarter of 2024[1][2].

Geographic Revenue Distribution

In the third quarter of 2024, $23.3 million (70% of product revenue) was generated in the United States, and $10.0 million (30% of product revenue) was generated outside the United States. This indicates a strong presence in both domestic and international markets[2].

Costs and Expenses

The company's costs and expenses include significant investments in research and development, as well as selling, general, and administrative expenses. For the third quarter of 2024, research and development expenses were $37.9 million, and selling, general, and administrative expenses were $35.4 million[2].

Cash Position and Funding

As of September 30, 2024, Rhythm Pharmaceuticals had approximately $298.4 million in cash, cash equivalents, and short-term investments. This financial position is expected to support planned operations into 2026. Additionally, the company secured $150 million in convertible preferred stock financing in the first quarter of 2024[3][5].

Market Impact and Competitive Landscape

Setmelanotide's approval and ongoing clinical trials position it as a leader in the treatment of rare genetic obesity disorders. The lack of approved therapies for conditions like hypothalamic obesity gives IMCIVREE a significant market advantage.

Competitive Advantage

The drug's ability to achieve rapid and sustained weight loss, as demonstrated in Phase 2 and long-term extension trials, underscores its competitive advantage. For instance, 89% of patients in a Phase 2 trial achieved a BMI decrease greater than 5% at 16 weeks on setmelanotide therapy[4].

Future Outlook

Upcoming Milestones

• Top-line data readout for the Phase 3 trial in hypothalamic obesity: Expected in the first half of 2025.
• sNDA approval for pediatric patients: Anticipated by December 26, 2024.
• Initiation of Phase 1 trial for RM-718: Planned for the first half of 2024.
• Announcement of DAYBREAK Stage 2 data: Expected in the second half of 2024[1][2][5].

Global Expansion

Rhythm Pharmaceuticals is also planning to initiate dosing of the first patients in Japan in the second quarter of 2024 as part of its Phase 3 development plan for setmelanotide in hypothalamic obesity[5].

Key Takeaways

• Strong Revenue Growth: IMCIVREE has shown significant revenue growth, driven by sales in the U.S. and internationally.
• Clinical Success: Real-world data and clinical trials demonstrate the efficacy of setmelanotide in treating rare genetic obesity disorders.
• Regulatory Advancements: Pending approvals and label expansions are set to further broaden the drug's market reach.
• Financial Stability: The company's cash position and funding ensure support for planned operations into 2026.
• Market Leadership: Setmelanotide's unique position in treating rare genetic obesity disorders solidifies its market advantage.

FAQs

Q: What is setmelanotide acetate used for?

A: Setmelanotide acetate, marketed as IMCIVREE, is used to treat rare genetic disorders of obesity, including Bardet-Biedl syndrome (BBS) and other conditions associated with hyperphagia and severe obesity.

Q: What are the key clinical trials for setmelanotide?

A: The key clinical trials include a Phase 3 trial evaluating setmelanotide in 120 patients with acquired hypothalamic obesity, with top-line data expected in the first half of 2025.

Q: What is the current revenue performance of IMCIVREE?

A: In the third quarter of 2024, IMCIVREE generated $33.3 million in net revenue from global sales, with a 14% increase on a sequential basis from the second quarter of 2024.

Q: What are the upcoming regulatory milestones for setmelanotide?

A: The upcoming milestones include the potential approval of an sNDA to expand the label to include patients as young as 2 years old by December 26, 2024.

Q: How does setmelanotide impact the treatment of hypothalamic obesity?

A: Setmelanotide has shown significant potential to transform the care of individuals living with hypothalamic obesity, achieving rapid and sustained weight loss in clinical trials.

Sources

1. Rhythm Pharmaceuticals Reports Fourth Quarter 2023 Financial Results and Business Update. Rhythm Pharmaceuticals.
2. Rhythm Pharmaceuticals Reports Third Quarter 2024 Financial Results and Business Update. Rhythm Pharmaceuticals.
3. Rhythm Pharmaceuticals Reports Third Quarter 2024 Financial Results and Business Update. BioSpace.
4. Rhythm Pharmaceuticals Presents Data from Phase 2 and Long-term Extension Trials Evaluating Setmelanotide for the Treatment of Hypothalamic Obesity. Rhythm Pharmaceuticals.
5. Rhythm Pharmaceuticals Reports First Quarter 2024 Financial Results and Business Update. BioSpace.