PTC Therapeutics has been granted a patent for crystalline forms of sepiapterin free base, which can be used in the treatment of diseases associated with low cellular levels of BH4, such as phenylketonuria. The patent also covers pharmaceutical compositions containing these crystalline forms and methods for their preparation. One specific crystalline form, Form D, is described in the patent claim, which has peaks at specific diffraction angles as measured by X-ray diffractometry. GlobalData’s report on PTC Therapeutics gives a 360-degree view of the company including its patenting strategy. Buy the report here.

Smarter leaders trust GlobalData


Data Insights PTC Therapeutics Inc - Company Profile

Buy the Report

Data Insights

The gold standard of business intelligence.

Find out more

According to GlobalData’s company profile on PTC Therapeutics, Human telomerase RT biomarker was a key innovation area identified from patents. PTC Therapeutics's grant share as of September 2023 was 36%. Grant share is based on the ratio of number of grants to total number of patents.

Patent granted for pharmaceutical composition containing crystalline form d of sepiapterin

Source: United States Patent and Trademark Office (USPTO). Credit: PTC Therapeutics Inc

A recently granted patent (Publication Number: US11773097B2) discloses a pharmaceutical composition comprising crystalline Form D of sepiapterin free base. This composition includes peaks at specific diffraction angles as measured by X-ray diffractometry. The crystalline Form D is present in the composition in an amount of at least 90 percent by weight and is formulated as particles less than 100 µm in size. The patent also claims methods of treating tetrahydrobiopterin (BH4)-related disorders, such as primary BH4 deficiency and phenylketonuria, by administering the pharmaceutical composition to a subject in need. The composition can be administered as a liquid suspension.

The patent further describes a method for decreasing phenylalanine levels in a subject by administering the pharmaceutical composition of crystalline Form D. This method can also be carried out using a liquid suspension. Additionally, the patent discloses a method of preparing a liquid formulation by dispersing the crystalline Form D in a liquid. The resulting formulation can be a suspension.

Overall, this patent provides a pharmaceutical composition and methods for treating BH4-related disorders and decreasing phenylalanine levels using crystalline Form D of sepiapterin free base. The composition is formulated as particles and can be administered as a liquid suspension. The patent also includes a method for preparing a liquid formulation containing the crystalline Form D. These innovations have the potential to improve the treatment options for individuals with BH4-related disorders and phenylketonuria.

To know more about GlobalData’s detailed insights on PTC Therapeutics, buy the report here.

Data Insights


The gold standard of business intelligence.

Blending expert knowledge with cutting-edge technology, GlobalData’s unrivalled proprietary data will enable you to decode what’s happening in your market. You can make better informed decisions and gain a future-proof advantage over your competitors.


GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

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