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Last Updated: February 11, 2025

Asfotase alfa - Biologic Drug Details


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Summary for asfotase alfa
Tradenames:1
High Confidence Patents:0
Applicants:1
BLAs:1
Suppliers: see list1
Recent Clinical Trials: See clinical trials for asfotase alfa
Recent Clinical Trials for asfotase alfa

Identify potential brand extensions & biosimilar entrants

SponsorPhase
Alexion Pharmaceuticals, Inc.Phase 4
Alexion Pharmaceuticals, Inc.Phase 3
Alexion PharmaceuticalsPhase 4

See all asfotase alfa clinical trials

Pharmacology for asfotase alfa
Established Pharmacologic ClassTissue-nonspecific Alkaline Phosphatase
Chemical StructureAlkaline Phosphatase
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
  2. 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.

  3. General brand-side disclosures
  4. 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.

  5. Patents from broad patent text search
  6. 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 asfotase alfa Derived from Brand-Side Litigation

No patents found based on brand-side litigation

2) High Certainty: US Patents for asfotase alfa Derived from Company Disclosures

No patents found based on company disclosures

3) Low Certainty: US Patents for asfotase alfa Derived from Patent Text Search

No patents found based on company disclosures

Market Dynamics and Financial Trajectory for the Biologic Drug: Asfotase Alfa

Introduction

Asfotase alfa, marketed under the brand name Strensiq, is a human recombinant targeted alkaline phosphatase enzyme replacement therapy designed to treat hypophosphatasia (HPP), a rare and life-threatening genetic metabolic disorder. Here, we delve into the market dynamics and financial trajectory of this critical biologic drug.

Market Need and Patient Population

Hypophosphatasia is an ultra-rare disease characterized by skeletal deformity, severe muscle weakness, and progressive damage to vital organs. The lack of approved treatments prior to asfotase alfa made it a highly unmet medical need, particularly for patients with pediatric-onset and adult forms of the disease[2].

Regulatory Approvals and Designations

Asfotase alfa was awarded orphan drug designation in the U.S. and EU in 2008 and received Fast Track status in the U.S. in 2009. These designations highlight the drug's potential to address a significant unmet medical need and expedite its development and review process[2].

Clinical Efficacy and Outcomes

Clinical trials and real-world studies have demonstrated the efficacy of asfotase alfa in reducing tissue non-specific alkaline phosphatase (TNSALP) substrates, such as inorganic pyrophosphate (PPi) and pyridoxal 5' phosphate (PLP), which are key markers of the disease. Patients treated with asfotase alfa showed significant improvements in functional capacity, as measured by the six-minute walk test (6MWT), and in health-related quality of life (HRQoL)[1][4].

Market Acquisition and Development

In 2011, Alexion Pharmaceuticals acquired Enobia Pharma Corp., gaining full worldwide development and commercial rights to asfotase alfa. This acquisition was a strategic move to leverage Alexion's expertise in developing and commercializing therapies for rare and ultra-rare diseases[2].

Financial Performance and Revenue Growth

The acquisition and subsequent approval of asfotase alfa contributed significantly to Alexion's financial performance. In 2016, Alexion reported total revenues of $3.084 billion, representing an 18% revenue growth and 22% volume growth from 2015. The launch of Strensiq in the U.S., Germany, and Japan was a key factor in this growth[3].

Cost-Effectiveness and Access

Despite its high cost, asfotase alfa has been shown to improve mobility, reduce fractures, and enhance mental health in patients with HPP. However, cost-effectiveness remains a concern. Alexion has implemented patient access schemes and discounts to make the drug more accessible. For instance, the company increased the discount offered in its patient access scheme to address concerns from health technology assessment bodies like NICE[5].

Real-World Impact and Patient Outcomes

Real-world cohort studies have reinforced the clinical trial data, showing sustained improvements in physical functioning and HRQoL among adult patients with pediatric-onset HPP. These studies underscore the long-term efficacy and safety of asfotase alfa in routine clinical practice[4].

Global Expansion and Market Reach

Alexion has been expanding patient access to Strensiq in additional European countries, enhancing disease education efforts to identify and serve more patients. The drug's approval in multiple countries has helped in transforming the lives of patients with HPP worldwide[3].

Challenges and Risks

While asfotase alfa has shown promising results, there are risks associated with its development and commercialization. These include regulatory uncertainties, potential delays in manufacturing and commercial infrastructure, and the risk that third-party payors may not reimburse the drug at acceptable rates[1].

Financial Projections and Future Outlook

Given the drug's efficacy and the growing recognition of its benefits, asfotase alfa is expected to continue contributing significantly to Alexion's revenue. The company's focus on expanding patient access and enhancing disease education will likely drive further growth. However, ongoing monitoring of cost-effectiveness and reimbursement strategies will be crucial for sustaining market traction[3].

Key Takeaways

  • Clinical Efficacy: Asfotase alfa significantly reduces TNSALP substrates and improves functional capacity and HRQoL in patients with HPP.
  • Market Acquisition: Alexion's acquisition of Enobia Pharma Corp. was pivotal in bringing asfotase alfa to market.
  • Financial Performance: The drug has contributed to Alexion's revenue growth, with total revenues reaching $3.084 billion in 2016.
  • Cost-Effectiveness: Despite high costs, patient access schemes and discounts have been implemented to improve accessibility.
  • Global Expansion: Efforts to expand patient access and enhance disease education are ongoing.

FAQs

What is asfotase alfa used for?

Asfotase alfa is used to treat hypophosphatasia (HPP), a rare genetic metabolic disorder characterized by skeletal deformity, muscle weakness, and organ damage.

What are the key clinical benefits of asfotase alfa?

Asfotase alfa reduces TNSALP substrates, improves functional capacity as measured by the 6MWT, and enhances HRQoL in patients with HPP.

Who acquired the rights to asfotase alfa?

Alexion Pharmaceuticals acquired Enobia Pharma Corp. in 2011, gaining full worldwide development and commercial rights to asfotase alfa.

How has asfotase alfa impacted Alexion's financial performance?

The launch of asfotase alfa contributed to Alexion's revenue growth, with total revenues reaching $3.084 billion in 2016, representing an 18% increase from 2015.

What challenges does asfotase alfa face in terms of market access?

Despite its clinical benefits, asfotase alfa faces challenges related to cost-effectiveness and reimbursement. Alexion has implemented patient access schemes and discounts to address these concerns.

How is asfotase alfa administered?

Asfotase alfa is administered via subcutaneous injections, with patients typically receiving doses of 2.1 mg/kg/week or 3.5 mg/kg/week[1].

Sources

  1. Alexion Pharmaceuticals. Asfotase Alfa Significantly Decreased TNSALP Substrates and Improved Functional Capacity in Adolescents and Adults with Hypophosphatasia (HPP).
  2. Alexion Pharmaceuticals. Alexion to Acquire Enobia Pharma Corp. and First Potential Treatment for Ultra-Rare Life-Threatening Genetic Metabolic Disease.
  3. Alexion Pharmaceuticals. Annual Reports.
  4. Oxford Academic. Improvement in quality of life after asfotase alfa treatment in adults with hypophosphatasia.
  5. NICE. Asfotase alfa for treating paediatric-onset hypophosphatasia.

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