Miglustat API: Enzyme Inhibitor for Rare Diseases

Miglustat API is a potent active pharmaceutical ingredient (API) used primarily as an enzyme inhibitor for the treatment of Gaucher disease type 1 and Niemann-Pick disease type C (NP-C). At Vonage Pharma, we supply high-purity Miglustat API (CAS No: 72599-27-0) manufactured under stringent GMP conditions, ensuring consistent quality and compliance with global pharmaceutical standards.

What is Miglustat API?

Miglustat is a small molecule that functions as a competitive and reversible inhibitor of the enzyme glucosylceramide synthase. Specifically, this enzyme catalyzes the first step in the biosynthesis of glycosphingolipids, lipid molecules that accumulate abnormally in patients with Gaucher disease and Niemann-Pick disease. By inhibiting glucosylceramide synthase, Miglustat reduces the synthesis of glycosphingolipids, thereby decreasing their pathological accumulation in cells such as macrophages. This mechanism, known as substrate reduction therapy, helps alleviate symptoms and disease progression.

Therapeutic Applications

• Gaucher Disease Type 1: Miglustat is approved for patients who are unable to undergo enzyme replacement therapy. Furthermore, it helps reduce liver and spleen enlargement, improves hemoglobin levels, and increases platelet counts.

• Niemann-Pick Disease Type C: Miglustat is the first and only approved therapy for this rare neurodegenerative disorder. It has also been authorized for use in adult and pediatric patients in the European Union, Brazil, and South Korea to slow neurological symptom progression.

Key Features of Vonage Pharma’s Miglustat API

• High Purity and Quality: Produced in GMP-certified facilities, our Miglustat API meets rigorous quality control standards, including Certificate of Analysis (CoA) verification.

• Consistent Supply: We maintain reliable inventory levels to support your manufacturing and formulation needs.

• Regulatory Compliance: Our API complies with international regulatory requirements, including USDMF and CEP filings, facilitating smooth regulatory submissions.

• Flexible Packaging: Available in various packaging sizes to suit different production scales.

Why Choose Vonage Pharma for Miglustat API?

• Expertise in Specialty APIs: With years of experience in sourcing and supplying complex APIs, Vonage Pharma is a trusted partner for pharmaceutical manufacturers.

• Global Reach: We serve clients worldwide, ensuring timely delivery and competitive pricing.

• Technical Support: Our team provides comprehensive technical assistance and documentation to support your product development and regulatory processes.

Quality Assurance

Our Miglustat API undergoes thorough testing to ensure it meets all pharmacopeial specifications, including purity, identity, and residual solvents. We also adhere to current Good Manufacturing Practices (cGMP) and follow strict quality management systems to guarantee product consistency and safety.

Ordering and Inquiry

Vonage Pharma invites pharmaceutical companies, contract manufacturers, and research organizations to contact us for detailed product specifications, pricing, and availability. Our dedicated customer service team is ready to assist you with your Miglustat API requirements.

About Miglustat

Researchers initially developed Miglustat as an anti-HIV agent. Later, they discovered its role in treating lysosomal storage disorders due to its unique enzyme inhibition properties. It belongs to the glycoside class of compounds, known for their diverse therapeutic applications. Miglustat serves as a substrate reduction therapy agent and offers an oral treatment option. This provides convenience and improves patient compliance compared to intravenous enzyme replacement therapies.

Contact Vonage Pharma today to learn more about our Miglustat API and how we can support your pharmaceutical manufacturing needs with quality, reliability, and expertise.

FAQs About Miglustat API