Velmanase alfa-tycv - Biologic Drug Details

Summary for velmanase alfa-tycv

Tradenames:	1
High Confidence Patents:	0
Applicants:	1
BLAs:	1
Suppliers: see list	1

Note on Biologic Patents

Matching patents to biologic drugs is far more complicated than for small-molecule drugs.

DrugPatentWatch employs three methods to identify biologic patents:

1. Brand-side disclosures in response to biosimilar applications

These patents were identified from disclosures by the brand-side company, in response to a potential biosimilar seeking to launch. They have a high certainty of blocking biosimilar entry. The expiration dates listed are not estimates — they're expiration dates as indicated by the brand-side company.

2. General brand-side disclosures

These patents were identified from searching drug labels and other general disclosures from the brand-side company. This list may exclude some of the patents which block biosimilar launch, and some of these patents listed may not actually block biosimilar launch. The expiration dates listed for these patents are estimates, based on the grant date of the patent.

3. Patents from broad patent text search

For completeness, these patents were identified by searching the patent literature for mentions of the branded or ingredient name of the drug. Some of these patents protect the original drug, whereas others may protect follow-on inventions or even inventions casually mentioning the drug. The expiration dates listed for these patents are estimates, based on the grant date of the patent.

1) High Certainty: US Patents for velmanase alfa-tycv Derived from Brand-Side Litigation

No patents found based on brand-side litigation

2) High Certainty: US Patents for velmanase alfa-tycv Derived from Company Disclosures

No patents found based on company disclosures

3) Low Certainty: US Patents for velmanase alfa-tycv Derived from Patent Text Search

No patents found based on company disclosures

Market Dynamics and Financial Trajectory for Velmanase Alfa-tycv

Introduction to Velmanase Alfa-tycv

Velmanase alfa-tycv, marketed under the name Lamzede, is a recombinant form of human alpha-mannosidase, an enzyme replacement therapy (ERT) specifically designed to treat non-central nervous system manifestations of alpha-mannosidosis, a rare genetic disorder[2][4].

Mechanism of Action and Clinical Significance

Velmanase alfa-tycv works by supplementing or replacing the deficient alpha-mannosidase enzyme in patients, thereby reducing the accumulation of mannose-rich oligosaccharides in the lysosome. This therapy has shown significant improvements in biochemical and functional measures in patients with alpha-mannosidosis, particularly when administered over long periods, such as up to 12 years[1][2].

Regulatory Approvals and Market Access

Velmanase alfa-tycv received marketing authorization from the European Commission in March 2018 and was approved by the FDA in February 2023. This approval has opened up market access in key regions, including the United States and Europe. Additionally, it is covered under the North Carolina Medicaid program as part of the Physician Administered Drug Program (PADP)[4][5].

Market Position within the Biological Drugs Sector

The biological drugs market, which includes therapeutic proteins like velmanase alfa-tycv, is experiencing robust growth. This sector was valued at USD 296.2 billion in 2023 and is projected to grow to USD 326.12 billion by 2030, with a compound annual growth rate (CAGR) of 10.1% during the forecast period. Therapeutic proteins, the sub-segment to which velmanase alfa-tycv belongs, are expected to generate the maximum revenue within this market[3].

Competitive Landscape

The competitive landscape of the biological drugs market is highly dynamic, with several major pharmaceutical companies investing heavily in research and development. Companies like AbbVie, with its monoclonal antibody adalimumab (Humira), and others are driving innovation and market growth. Chiesi Farmaceutici, the developer of velmanase alfa-tycv, is also a significant player, particularly in the rare disease segment[3].

Financial Trajectory

The financial trajectory for velmanase alfa-tycv is influenced by several factors:

• Revenue Potential: Given the rare nature of alpha-mannosidosis and the lack of alternative treatments, velmanase alfa-tycv has a significant revenue potential. The drug is priced at a premium, with each unit costing approximately $432 for Medicaid reimbursement in North Carolina[5].
• Market Expansion: With approvals in key markets, the drug is poised for expanded market reach. The orphan drug designation for alpha-mannosidosis also provides incentives such as extended patent protection and tax credits, which can enhance profitability[4].
• Research and Development Costs: The development of velmanase alfa-tycv involved substantial investment in research and clinical trials. However, the long-term data presented at the 20th Annual WORLD Symposium™ Research Meeting underscores the drug's efficacy and safety, potentially reducing future R&D costs and increasing its market viability[1].

Reimbursement and Pricing

The reimbursement landscape for velmanase alfa-tycv is favorable, especially in regions with well-developed healthcare systems. In the United States, for example, Medicaid coverage and the use of HCPCS code J3590 for billing ensure that the drug is accessible to patients who need it. The pricing strategy, while high, is consistent with other biologic therapies for rare diseases[5].

Adverse Effects and Safety Profile

While velmanase alfa-tycv has a generally favorable safety profile, it does come with some risks, including infusion-associated reactions and hypersensitivity reactions, which can be severe. The FDA label includes a black box warning for hypersensitivity reactions, including anaphylaxis[4].

Long-Term Efficacy and Patient Outcomes

Long-term data from up to 12 years of treatment with velmanase alfa-tycv has shown sustained efficacy in reducing oligosaccharide levels and improving biochemical and functional measures in patients with alpha-mannosidosis. This long-term efficacy is crucial for maintaining patient health and justifying the continued use and reimbursement of the drug[1].

Impact on Rare Disease Treatment

Velmanase alfa-tycv represents a significant advancement in the treatment of alpha-mannosidosis, a condition that was previously without an effective therapeutic option. The drug's approval and market availability highlight the growing focus on rare diseases within the pharmaceutical industry and the potential for biologic therapies to transform patient care in these areas[1][4].

Key Takeaways

• Market Growth: Velmanase alfa-tycv is part of a rapidly growing biological drugs market.
• Regulatory Approvals: Approved in the EU and US, with Medicaid coverage in North Carolina.
• Financial Potential: Significant revenue potential due to its premium pricing and orphan drug designation.
• Long-Term Efficacy: Demonstrated sustained efficacy over up to 12 years of treatment.
• Safety Profile: Generally favorable but includes risks such as hypersensitivity reactions.

Frequently Asked Questions (FAQs)

Q1: What is velmanase alfa-tycv used for? Velmanase alfa-tycv is used for the treatment of non-central nervous system manifestations of alpha-mannosidosis, a rare genetic disorder.

Q2: How is velmanase alfa-tycv administered? It is administered via weekly intravenous infusion at a dose of 1 mg/kg of actual body weight.

Q3: What are the common adverse reactions associated with velmanase alfa-tycv? Common adverse reactions include infusion-associated reactions, nasopharyngitis, headache, diarrhea, fatigue, nausea, back pain, pain in extremity, and sinusitis.

Q4: Is velmanase alfa-tycv covered by Medicaid? Yes, it is covered under the North Carolina Medicaid program as part of the Physician Administered Drug Program (PADP).

Q5: What is the orphan drug status of velmanase alfa-tycv? Velmanase alfa-tycv has been granted orphan drug status for the treatment of alpha-mannosidosis, which provides incentives such as extended patent protection and tax credits.

Cited Sources

1. Chiesi Global Rare Diseases Presents Long-Term Data on Treatment with Velmanase Alfa in Alpha-Mannosidosis - Chiesi Global Rare Diseases.
2. Velmanase alfa: Uses, Interactions, Mechanism of Action - DrugBank.
3. Biological Drugs Market: Global Industry Analysis - Maximize Market Research.
4. Velmanase alfa - Chiesi Farmaceutici - AdisInsight - Springer.
5. Velmanase Alfa-tycv for Injection, for Intravenous Use (Lamzede) - NC Medicaid.

Last updated: 2024-12-22