Avalglucosidase alfa-ngpt - Biologic Drug Details

Summary for avalglucosidase alfa-ngpt

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High Confidence Patents:	0
Applicants:	1
BLAs:	1
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Pharmacology for avalglucosidase alfa-ngpt

Established Pharmacologic Class	Hydrolytic Lysosomal Glycogen-specific Enzyme
Chemical Structure	alpha-Glucosidases

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1) High Certainty: US Patents for avalglucosidase alfa-ngpt Derived from Brand-Side Litigation

No patents found based on brand-side litigation

2) High Certainty: US Patents for avalglucosidase alfa-ngpt Derived from Company Disclosures

No patents found based on company disclosures

3) Low Certainty: US Patents for avalglucosidase alfa-ngpt Derived from Patent Text Search

No patents found based on company disclosures

Market Dynamics and Financial Trajectory for Avalglucosidase Alfa-ngpt

Introduction to Avalglucosidase Alfa-ngpt

Avalglucosidase alfa-ngpt, marketed as Nexviazyme, is a next-generation recombinant human acid alpha-glucosidase (GAA) enzyme replacement therapy (ERT) designed for the treatment of late-onset Pompe disease (LOPD) and infantile-onset Pompe disease (IOPD). Here, we delve into the market dynamics and financial trajectory of this biologic drug.

Market Size and Growth

The global Pompe disease market, within which avalglucosidase alfa-ngpt operates, has been expanding steadily. In 2021, the global Pompe disease market size was estimated at USD 1,386.09 million and is projected to reach USD 1,971.13 million by 2030, growing at a CAGR of 3.9%[4].

Approval and Clinical Trials

The approval of avalglucosidase alfa-ngpt was based on the results of the Phase 3 COMET trial, a 49-week randomized, double-blind, and multinational noninferiority study. This trial compared avalglucosidase alfa-ngpt with alglucosidase alfa in treatment-naïve patients with LOPD. The primary endpoint was the effect on respiratory muscle function, measured by upright forced vital capacity (FVC) % predicted. Avalglucosidase alfa-ngpt demonstrated noninferiority to alglucosidase alfa and showed greater improvements in several secondary endpoints, including functional endurance and muscle strength[3].

Market Segmentation

The Pompe disease market is segmented based on disease type, with LOPD and IOPD being the primary segments. Avalglucosidase alfa-ngpt is predominantly used for LOPD, which accounts for the larger share of the market. Geographically, Europe and North America are significant markets, but the Asia Pacific region is expected to grow at the fastest CAGR due to increasing awareness and funding for rare disease treatments[4].

Cost-Effectiveness and Pricing

Avalglucosidase alfa-ngpt is priced at $1,597 per 100 mg vial, which is 5% less expensive per mg compared to alglucosidase alfa ($840 per 50 mg vial). A cost-minimization analysis showed that the average annual cost of avalglucosidase alfa-ngpt ($518,539 per patient) results in a savings of $27,292 per patient per year compared to alglucosidase alfa. This cost savings is significant, especially considering the identical recommended dosing for both products[5].

Budget Impact Analysis

The sponsor's budget impact analysis suggests substantial savings for health systems. The analysis indicates that the yearly incremental savings associated with the reimbursement of avalglucosidase alfa-ngpt could be $746,548 in Year 1, $1,046,458 in Year 2, and $1,285,095 in Year 3, totaling $3,078,101 over three years. When including dispensing fees and markups, the cumulative savings could reach $3,081,382 over the same period[5].

Regulatory and Approval Landscape

Avalglucosidase alfa-ngpt has received favorable opinions from regulatory bodies such as the European Medicines Agency (EMA) and has been approved in several countries, including the United Kingdom, European Union, and the United States for patients with LOPD aged 1 year and older[2][3].

Patient Experience and Home Infusions

Clinical studies have shown that avalglucosidase alfa-ngpt is well tolerated and results in clinically meaningful improvements in respiratory function, ambulation, and functional endurance. Home infusions of avalglucosidase alfa-ngpt have been found to be clinically effective, preferred by patients, and cost-saving compared to infusions in outpatient medical facilities or hospitals[2].

Competitive Landscape

The market for Pompe disease treatments is evolving with new therapeutic options. For instance, the Marketing Authorization Applications (MAA) for AT-GAA, an investigational two-component medication developed by Amicus Therapeutics, have been verified by the EMA. However, avalglucosidase alfa-ngpt remains a leading treatment due to its improved cellular uptake and glycogen clearance compared to its predecessor, alglucosidase alfa[4].

Future Outlook

The future outlook for avalglucosidase alfa-ngpt is promising, driven by increasing awareness of Pompe disease, growing demand for effective treatments, and ongoing clinical studies. The expiration of the patent for alglucosidase alfa in 2021 may lead to the emergence of biosimilars, which could impact the market dynamics. However, the current cost savings and clinical benefits of avalglucosidase alfa-ngpt position it favorably in the market[5].

Key Takeaways

• Market Growth: The global Pompe disease market is expected to grow at a CAGR of 3.9% from 2021 to 2030.
• Clinical Efficacy: Avalglucosidase alfa-ngpt has demonstrated noninferiority to alglucosidase alfa and shown greater improvements in several clinical endpoints.
• Cost-Effectiveness: Avalglucosidase alfa-ngpt offers significant cost savings compared to alglucosidase alfa.
• Regulatory Approval: Approved in several countries for the treatment of LOPD.
• Patient Experience: Well tolerated and preferred by patients, especially for home infusions.

FAQs

1. What is avalglucosidase alfa-ngpt used for?

   • Avalglucosidase alfa-ngpt is used for the treatment of late-onset Pompe disease (LOPD) and infantile-onset Pompe disease (IOPD).

2. How does avalglucosidase alfa-ngpt compare to alglucosidase alfa?

   • Avalglucosidase alfa-ngpt has a 15-fold increase in mannose-6-phosphate content, leading to improved cellular uptake and glycogen clearance compared to alglucosidase alfa.

3. What are the cost savings of avalglucosidase alfa-ngpt?

   • Avalglucosidase alfa-ngpt results in a savings of $27,292 per patient per year compared to alglucosidase alfa.

4. Is avalglucosidase alfa-ngpt approved for home infusions?

   • Yes, avalglucosidase alfa-ngpt has been found to be clinically effective and preferred by patients for home infusions.

5. What is the future outlook for avalglucosidase alfa-ngpt in the market?

   • The future outlook is promising due to increasing awareness of Pompe disease and the ongoing demand for effective treatments, despite potential competition from biosimilars.

Sources

1. Grand View Research - Alpha-glucosidase Inhibitors Market Size, Share Report 2030
2. EUR - Pure - Home infusion experience in patients with Pompe disease receiving avalglucosidase alfa
3. MyHealthToolkit - Nexviazyme™ (avalglucosidase alfa-ngpt) Injection - CAM 359
4. Emergen Research - Pompe Disease Market Trend, Forecast [2021-2030]
5. NCBI - Pharmacoeconomic Review - Avalglucosidase Alfa (Nexviazyme)