Pegvaliase-pqpz - Biologic Drug Details

Summary for pegvaliase-pqpz

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

Pharmacology for pegvaliase-pqpz

Established Pharmacologic Class	Phenylalanine Metabolizing Enzyme
Chemical Structure	Phenylalanine Ammonia-Lyase

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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

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1) High Certainty: US Patents for pegvaliase-pqpz Derived from Brand-Side Litigation

No patents found based on brand-side litigation

2) High Certainty: US Patents for pegvaliase-pqpz Derived from Company Disclosures

No patents found based on company disclosures

3) Low Certainty: US Patents for pegvaliase-pqpz Derived from Patent Text Search

No patents found based on company disclosures

Market Dynamics and Financial Trajectory for Pegvaliase-pqpz (Palynziq)

Introduction to Pegvaliase-pqpz (Palynziq)

Pegvaliase-pqpz, marketed as Palynziq, is a groundbreaking biologic drug developed by BioMarin Pharmaceutical Inc. for the treatment of phenylketonuria (PKU), a rare genetic disease. This enzyme substitution therapy is designed to reduce blood phenylalanine (Phe) concentrations in adult patients with PKU who have uncontrolled blood Phe levels despite existing management strategies.

FDA Approval and Regulatory Milestones

Palynziq received standard approval from the U.S. Food and Drug Administration (FDA) in May 2018 for the treatment of adult PKU patients with uncontrolled blood Phe concentrations greater than 600 micromol/L on existing management[1][4][5].

In October 2020, the FDA approved a supplemental Biologics License Application (sBLA) to increase the maximum allowable dose of Palynziq from 40 mg to 60 mg, based on data from the Phase 3 PRISM studies. This expansion included longer-term efficacy data and further supported the drug's well-characterized safety profile[2][4].

Clinical Efficacy and Safety Profile

Clinical trials, including the PRISM studies, have demonstrated the efficacy of Palynziq in reducing blood Phe levels. In the PRISM-1 study, Palynziq reduced mean blood Phe concentrations from 1232.7 micromol/L at baseline to 564.5 micromol/L at 12 months and 311.4 micromol/L at 24 months. A significant reduction of 20% or greater was achieved by 71.8% of the participants, and reductions to 360 micromol/L or less were achieved by 44% at 12 months and 60.7% at 24 months[5].

The long-term safety data, extending over six years, have consistently supported the safety profile of Palynziq, with no significant new safety concerns identified[2][4].

Market Impact and Patient Access

Palynziq is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the Palynziq REMS, which ensures careful monitoring and management of potential side effects[1].

The drug has significantly improved the lives of PKU patients by offering a treatment option that can control Phe levels more effectively than traditional dietary restrictions alone. BioMarin has been committed to bringing this therapy to patients outside the U.S., with the European Medicines Agency accepting the Marketing Authorization Application for Palynziq in March 2018[1].

Financial Considerations and Cost-Effectiveness

The financial trajectory of Palynziq is influenced by several factors, including its high development and production costs, as well as its pricing strategy. The cost per patient for the titration year ranges from $130,205 to $260,615, and for maintenance years, it ranges from $147,825 to $443,475, depending on the dose and titration speed[3].

Cost-utility analyses have shown mixed results regarding the cost-effectiveness of Palynziq. When compared to medical nutrition therapy (MNT) alone, the incremental cost-effectiveness ratio (ICER) is high, requiring significant price reductions to be considered cost-effective. However, when compared to sapropterin plus MNT, Palynziq has been found to be more effective and less costly, making it a dominant option in this context[3].

Patient Adherence and Economic Impact

Patient adherence to Palynziq is crucial for its effectiveness. Studies have assumed an adherence rate of 81% for Palynziq, which is higher than the adherence rate for MNT alone (20%) but slightly lower than that for sapropterin (92%)[3].

The economic impact of Palynziq extends beyond direct treatment costs. It also includes the reduction in costs associated with dietary supplementation and the improvement in quality of life for patients, which can lead to better employment and social outcomes[3].

Market Competition and Future Developments

BioMarin is a pioneer in PKU treatments and has developed multiple therapies for this condition. Besides Palynziq, the company has another approved therapy and is currently developing an investigational gene therapy, BMN 307, which could potentially become the third approved treatment for PKU[2][4].

The market for PKU treatments is niche but growing, driven by the need for more effective and sustainable treatment options. Palynziq's unique mechanism of action as an enzyme substitution therapy sets it apart from other treatments, making it a significant player in this market.

Global Reach and Expansion

BioMarin's commitment to the global PKU community is evident in its efforts to bring Palynziq to patients outside the U.S. The acceptance of the Marketing Authorization Application by the European Medicines Agency is a key step in this expansion[1].

Key Takeaways

• FDA Approval: Palynziq received FDA approval in May 2018 and a label expansion in October 2020 to increase the maximum dose to 60 mg.
• Clinical Efficacy: Significant reductions in blood Phe levels have been demonstrated in clinical trials, with long-term safety data supporting its use.
• Market Impact: Palynziq has improved the lives of PKU patients by offering a more effective treatment option beyond dietary restrictions.
• Financial Considerations: High costs are associated with Palynziq, but it can be cost-effective compared to certain other treatments.
• Global Expansion: BioMarin is working to make Palynziq available globally, with ongoing efforts in Europe.

FAQs

What is Palynziq used for?

Palynziq (pegvaliase-pqpz) is used to reduce blood phenylalanine (Phe) concentrations in adult patients with phenylketonuria (PKU) who have uncontrolled blood Phe levels despite existing management.

How is Palynziq administered?

Palynziq is administered via subcutaneous injection, with a gradual dose escalation to achieve the optimal maintenance dose, which can be up to 60 mg daily.

What are the clinical trial results for Palynziq?

Clinical trials have shown that Palynziq significantly reduces blood Phe levels, with a 20% or greater reduction achieved by 71.8% of participants and reductions to 360 micromol/L or less achieved by 44% at 12 months and 60.7% at 24 months.

Is Palynziq cost-effective?

The cost-effectiveness of Palynziq varies depending on the comparison. It is more effective and less costly than sapropterin plus MNT but requires significant price reductions to be considered cost-effective compared to MNT alone.

What are the long-term safety data for Palynziq?

Long-term safety data extending over six years have consistently supported the well-characterized safety profile of Palynziq, with no significant new safety concerns identified.

Is Palynziq available globally?

While initially approved in the U.S., BioMarin is working to make Palynziq available globally, with the European Medicines Agency having accepted the Marketing Authorization Application.

Sources

1. BioMarin Receives Standard Approval for Palynziq™ (pegvaliase-pqpz) Injection - PR Newswire
2. BioMarin, Pioneer in Rare Disease Treatments for Phenylketonuria (PKU), Receives FDA Approval of Label Expansion to Allow Maximum Dose of 60 mg for Palynziq® (pegvaliase-pqpz) Injection - BioSpace
3. Pharmacoeconomic Review - Pegvaliase (Palynziq) - NCBI Bookshelf
4. BioMarin, Pioneer in Rare Disease Treatments for Phenylketonuria (PKU), Receives FDA Approval of Label Expansion to Allow Maximum Dose of 60 mg for Palynziq® (pegvaliase-pqpz) Injection - BioMarin
5. Pegvaliase-pqpz (Palynziq) - Medical Coverage Guideline - MCG Health