YARGESA Drug Patent Profile

When do Yargesa patents expire, and when can generic versions of Yargesa launch?

Yargesa is a drug marketed by Edenbridge Pharms and is included in one NDA.

The generic ingredient in YARGESA is miglustat. There are two drug master file entries for this compound. Five suppliers are listed for this compound. Additional details are available on the miglustat profile page.

DrugPatentWatch^® Litigation and Generic Entry Outlook for Yargesa

A generic version of YARGESA was approved as miglustat by ANI PHARMS on April 17^th, 2018.

Summary for YARGESA

US Patents:	0
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
DailyMed Link:	YARGESA at DailyMed

Pharmacology for YARGESA

Drug Class	Glucosylceramide Synthase Inhibitor
Mechanism of Action	Glucosylceramide Synthase Inhibitors

US Patents and Regulatory Information for YARGESA

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Edenbridge Pharms	YARGESA	miglustat	CAPSULE;ORAL	209821-001	Aug 6, 2020	AB	RX	No	No

EU/EMA Drug Approvals for YARGESA

Company	Drugname	Inn	Product Number / Indication	Status	Generic	Biosimilar	Orphan	Marketing Authorisation	Marketing Refusal
Piramal Critical Care B.V.	Yargesa	miglustat	EMEA/H/C/004016 Yargesa is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease.Yargesa may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable.Yargesa is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type C disease.	Authorised	yes	no	no	2017-03-22
Janssen Cilag International NV	Zavesca	miglustat	EMEA/H/C/000435 Zavesca is indicated for the oral treatment of adult patients with mild to moderate type-1 Gaucher disease. Zavesca may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable.Zavesca is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type-C disease.	Authorised	no	no	no	2002-11-20	2009-06-16
Gen.Orph	Miglustat Gen.Orph	miglustat	EMEA/H/C/004366 Miglustat Gen.Orph is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease. Miglustat Gen.Orph may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable.Miglustat Gen.Orph is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type C disease.	Authorised	yes	no	no	2017-11-09
Dipharma Arzneimittel GmbH	Miglustat Dipharma	miglustat	EMEA/H/C/004904 Miglustat Dipharma is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease.Miglustat Dipharma may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable.Miglustat Dipharma is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type C disease.	Authorised	yes	no	no	2019-02-18
Amicus Therapeutics Europe Limited	Opfolda	miglustat	EMEA/H/C/005695 Opfolda (miglustat) is an enzyme stabiliser of cipaglucosidase alfa long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid α- glucosidase [GAA] deficiency).	Authorised	no	no	no	2023-06-26

YARGESA Market Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory for YARGESA (Miglustat)

Introduction to YARGESA

YARGESA, also known as miglustat, is a glucosylceramide synthase inhibitor used in the treatment of adult patients with Type 1 Gaucher disease, particularly those for whom enzyme replacement therapy is not a viable option. This rare genetic neurological condition results from the inadequate breakdown of the lipid glucosylceramide, leading to its accumulation in various organs and causing a range of symptoms including abdominal complaints, skeletal abnormalities, severe fatigue, and seizures[1].

Market Need and Patient Impact

Gaucher disease is a rare and debilitating condition that significantly affects the quality of life for those afflicted. The need for effective treatments is critical, and YARGESA fills this gap by offering an alternative to enzyme replacement therapy. Patients with Gaucher disease often experience a wide variety of symptoms, and treatments like YARGESA can substantially improve their lives[1].

Distribution and Partnership

Orsini Specialty Pharmacy has been selected by Edenbridge Pharmaceuticals as the preferred specialty pharmacy partner for the distribution of YARGESA. This partnership is significant due to Orsini's extensive experience with rare diseases, its comprehensive compliance programs, and its ability to provide home nursing services. These factors are crucial in ensuring that patients receive the necessary support and care to manage their therapies effectively[1].

Regulatory Approval

YARGESA has received regulatory approval from the European Medicines Agency (EMA) after demonstrating bioequivalence to the reference medicine, Zavesca. The EMA's Committee for Medicinal Products for Human Use (CHMP) concluded that YARGESA has comparable quality and benefits to Zavesca, recommending its approval for use in the EU[3].

Financial Implications

The financial trajectory of YARGESA is tied to its adoption and sales within the specialty pharmacy market. As a treatment for a rare disease, YARGESA's revenue is likely to be significant due to the high cost of specialty medications. The partnership with Orsini Specialty Pharmacy is expected to enhance its market reach and patient access, potentially leading to increased revenue for Edenbridge Pharmaceuticals.

Revenue Projections and Market Performance

While specific revenue projections for YARGESA are not publicly available, the overall market for rare disease treatments is growing. Specialty pharmacies like Orsini play a crucial role in this growth by ensuring that patients have access to these treatments. The success of other rare disease treatments can provide insights into the potential financial performance of YARGESA. For instance, Blueprint Medicines reported significant revenue growth for its product AYVAKIT, which is used for different indications but also targets a niche market[2].

Operational Complexity and Supply Chain

The pharmaceutical industry, including the segment for rare disease treatments, faces several operational challenges such as rising costs, supply chain pressures, and the need for innovative solutions. These challenges can impact the financial trajectory of YARGESA by affecting production costs, distribution efficiency, and overall profitability. Companies must navigate these complexities to maintain profitability and ensure consistent supply to patients[4].

Competitive Landscape

The market for rare disease treatments is highly competitive, with several pharmaceutical companies investing heavily in research and development. Servier, for example, has seen significant growth in its generics revenue, which can indicate a broader trend in the pharmaceutical industry towards specialized and generic treatments[5].

Patient Access and Compliance Programs

Orsini Specialty Pharmacy's role in providing comprehensive compliance programs is crucial for ensuring patient access to YARGESA. These programs help patients and providers navigate the complexities of obtaining coverage and managing therapies, which can directly impact the financial success of the drug by ensuring consistent and compliant use[1].

Future Trends and Implications

The future of pharmaceutical operations, including those for rare disease treatments like YARGESA, is likely to be shaped by trends such as digital acceleration, new modalities like cell and gene therapy, and increasing ESG expectations. These trends may require pharmaceutical companies to adapt their operations, invest in new technologies, and reskill their workforce to remain competitive and profitable[4].

Key Takeaways

• Market Need: YARGESA addresses a critical need for patients with Type 1 Gaucher disease who cannot undergo enzyme replacement therapy.
• Distribution Partnership: The partnership with Orsini Specialty Pharmacy is key to ensuring patient access and comprehensive care.
• Regulatory Approval: YARGESA has been approved by the EMA, demonstrating bioequivalence to the reference medicine Zavesca.
• Financial Trajectory: The financial success of YARGESA will depend on its market adoption, patient access programs, and the ability to navigate operational complexities.
• Operational Challenges: The pharmaceutical industry faces rising costs, supply chain pressures, and the need for innovative solutions, all of which can impact YARGESA's financial performance.

FAQs

Q: What is YARGESA used for?

YARGESA (miglustat) is used for the treatment of adult patients with Type 1 Gaucher disease, particularly those for whom enzyme replacement therapy is not a therapeutic option.

Q: Who is the preferred specialty pharmacy partner for YARGESA?

Orsini Specialty Pharmacy has been selected by Edenbridge Pharmaceuticals as the preferred specialty pharmacy partner for the distribution of YARGESA.

Q: What regulatory approval has YARGESA received?

YARGESA has received approval from the European Medicines Agency (EMA) after demonstrating bioequivalence to the reference medicine Zavesca.

Q: What are the key challenges facing the pharmaceutical industry that could impact YARGESA?

The pharmaceutical industry faces challenges such as rising costs, supply chain pressures, and the need for innovative solutions, all of which can impact the financial performance and operational efficiency of YARGESA.

Q: How does Orsini Specialty Pharmacy support patients with YARGESA?

Orsini Specialty Pharmacy provides comprehensive compliance programs, home nursing services, and other support mechanisms to ensure that patients have access to and can manage their YARGESA therapies effectively.

Cited Sources:

1. Orsini Specialty Pharmacy Chosen to Dispense YARGESA (miglustat)
   • PR Newswire, April 18, 2024
2. Blueprint Medicines Reports Second Quarter 2024 Results and Raises AYVAKIT®/AYVAKYT® (avapritinib) Full Year Revenue Guidance
   • Blueprint Medicines, August 1, 2024
3. Yargesa | European Medicines Agency (EMA)
   • European Medicines Agency
4. Emerging from disruption: The future of pharma operations strategy
   • McKinsey, October 10, 2022
5. Servier confirms its 2025 trajectory to achieve its 2030 ambition
   • Servier, February 7, 2024