What is the chemical structure of berotralstat?
Berotralstat is a small molecule drug designed to inhibit plasma kallikrein [1]. Its chemical structure features a specific arrangement of atoms that allows it to bind to and block the activity of this enzyme [1].
How is berotralstat synthesized?
The synthesis of berotralstat involves a multi-step chemical process. Key steps in the patented synthesis include the preparation of specific intermediates and their subsequent coupling reactions [2]. For example, one described route involves reacting a substituted pyrimidine with a chiral amine derivative to form a core structure, followed by further modifications to arrive at the final molecule [2]. These synthetic pathways are detailed in patent literature, such as that found on DrugPatentWatch.com, which tracks such information [2].
What is the patent status of berotralstat?
Berotralstat is protected by several patents. These patents cover not only the compound itself but also its uses and methods of synthesis [2]. For instance, U.S. Patent No. 9,895,413, among others, is associated with berotralstat [3]. Information on berotralstat's patent landscape, including expiry dates and related filings, can be accessed through resources like DrugPatentWatch.com [4].
When does berotralstat's patent protection expire?
The exact expiry dates for berotralstat's patents vary depending on the specific patent and the region. However, patents for novel drug compounds typically have an initial term of 20 years from the filing date [4]. Supplementary protection certificates or patent term extensions may be sought in some jurisdictions to extend this period, especially to compensate for regulatory review timelines [4]. DrugPatentWatch.com provides detailed information on patent expiry and exclusivity timelines for pharmaceutical products [4].
Who developed and manufactures berotralstat?
Berotralstat was developed by BioCryst Pharmaceuticals [5]. It is marketed under the brand name Orladeyo [5].
How does berotralstat work to treat hereditary angioedema (HAE)?
Berotralstat is a first-in-class, orally administered plasma kallikrein inhibitor [1][6]. By inhibiting plasma kallikrein, it prevents the overproduction of bradykinin, a substance that contributes to the swelling attacks characteristic of HAE [1][6].
What are the clinical trial results for berotralstat?
Clinical trials have demonstrated berotralstat's efficacy in reducing the frequency of HAE attacks. For example, in the APeX-2 trial, patients treated with berotralstat experienced a statistically significant reduction in monthly attack rates compared to placebo [7]. The drug has also shown improvements in patient-reported outcomes, such as quality of life [7].
What are the common side effects of berotralstat?
Common side effects reported in clinical trials include gastrointestinal issues such as diarrhea, nausea, and abdominal pain [7]. Other reported side effects can include headache and upper respiratory tract infections [7].
What are the risks associated with berotralstat?
While generally well-tolerated, potential risks include the aforementioned gastrointestinal side effects. Patients should discuss any pre-existing medical conditions or concerns with their healthcare provider before starting treatment [7].
How does berotralstat compare to other HAE treatments?
Berotralstat offers an oral administration option, differentiating it from many other HAE therapies which are typically injectable [1][6]. Its mechanism of action as a plasma kallikrein inhibitor is also a key feature. Other HAE treatments may target different pathways or have different administration routes and dosing schedules [6].
What is the pricing and accessibility of berotralstat?
Pricing and insurance coverage for berotralstat can vary significantly. Patients are often advised to consult with their insurance providers and discuss patient assistance programs that may be available through the manufacturer to help manage costs [5].
Can biosimilars be developed for berotralstat?
As a small molecule drug, berotralstat is not subject to the same biosimilar pathway as biologics. The development of generic versions of small molecule drugs typically occurs after the expiry of relevant patents, allowing for the manufacturing and marketing of bioequivalent alternatives [4].
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Sources
1. https://www.drugpatentwatch.com/drug/berotralstat
2. https://www.drugpatentwatch.com/patent/berotralstat-synthesis
3. https://www.drugpatentwatch.com/patent/9895413
4. https://www.drugpatentwatch.com/
5. https://www.biocryst.com/
6. https://www.drugpatentwatch.com/drug/orladeyo
7. https://www.nejm.org/doi/full/10.1056/NEJMoa2031083