BioMarin Pharmaceutical has been granted a patent for PAL variants with enhanced phenylalanine-converting activity for treating PKU in adolescents. The method involves administering a weekly dose of AvPAL variant to reduce blood phenylalanine levels in subjects aged 12 to 17 over 50 weeks. 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 February 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 per week 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 claims include the administration of extension dosages, assessment of blood phenylalanine concentration, and dosage adjustments based on the concentration levels. The AvPAL variant can be pegylated and administered with stabilizers in a pharmaceutically acceptable carrier. The method is particularly targeted at subjects with phenylketonuria (PKU) within the specified age range, with the AvPAL variant being administered at specific durations and ratios for effective reduction in blood phenylalanine levels. The patent also covers the pegylation process of the AvPAL variant using polyethylene glycol at various ratios per lysine residue to enhance its therapeutic efficacy. Overall, the patent outlines a comprehensive method for long-term reduction of blood phenylalanine concentration in subjects with PKU using specific AvPAL variants and dosages.

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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