BioMarin Pharmaceutical has patented a method using AvPAL variants to reduce blood phenylalanine levels in adolescents with PKU. The weekly dose of the formulation is administered for over 50 weeks, targeting subjects aged 12 to 17 years. GlobalData’s report on BioMarin Pharmaceutical gives a 360-degree view of the company including its patenting strategy. Buy the report here.

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According to GlobalData’s company profile on BioMarin Pharmaceutical, Human telomerase RT biomarker was a key innovation area identified from patents. BioMarin Pharmaceutical's grant share as of April 2024 was 40%. Grant share is based on the ratio of number of grants to total number of patents.

Treatment of adolescent pku patients using avpal variant

Source: United States Patent and Trademark Office (USPTO). Credit: BioMarin Pharmaceutical Inc

A recently granted patent (Publication Number: US11918633B2) discloses a method for reducing blood phenylalanine concentration in subjects aged 12 to 17 years old by administering a weekly dose of a formulation containing an AvPAL variant. The AvPAL variant, with specific amino acid sequences, is administered for over 50 weeks, with dosage variations ranging from 0.1 mg to 840 mg per week. The method involves induction, titration, and maintenance dosages to achieve the desired reduction in blood phenylalanine levels.

Furthermore, the patent outlines the administration frequency, duration, and dosage adjustments based on blood phenylalanine concentration. The method is specifically targeted at subjects with phenylketonuria (PKU) within the specified age range. The AvPAL variant can be pegylated and administered with stabilizers in a pharmaceutically acceptable carrier. The extension dosage, administered post-maintenance, is crucial for sustained reduction in blood phenylalanine levels. The pegylation process involves reacting the AvPAL variant with polyethylene glycol at specific ratios per lysine residue to enhance its therapeutic efficacy. Overall, the patent provides a detailed and structured approach to effectively reduce blood phenylalanine concentration in subjects with PKU, emphasizing the importance of dosage optimization and monitoring for optimal outcomes.

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GlobalData Patent Analytics tracks bibliographic data, legal events data, point in time patent ownerships, and backward and forward citations from global patenting offices. Textual analysis and official patent classifications are used to group patents into key thematic areas and link them to specific companies