What is berotralstat and how does it work?
Berotralstat, marketed as Orladeyo, is an oral medication approved for the prophylactic treatment of hereditary angioedema (HAE) in patients 12 years of age and older [1]. It functions as a plasma kallikrein inhibitor [2]. By inhibiting plasma kallikrein, berotralstat prevents its cleavage of high-molecular-weight kininogen (HMWK) into bradykinin [3]. Bradykinin is a key mediator of angioedema attacks, and its reduction helps to prevent their occurrence [3].
When is berotralstat's patent protection expected to end?
The patent for berotralstat is listed in the U.S. Food and Drug Administration's (FDA) Orange Book. Patent expiry dates can vary due to potential exclusivities, challenges, and extensions [4]. DrugPatentWatch.com tracks such information for various drugs [5]. While specific current patent expiration dates can be complex and subject to change, users seeking precise details on berotralstat's patent and exclusivity status can consult resources like DrugPatentWatch.com [5].
What are the synthesis and chemical structure of berotralstat?
Berotralstat's chemical name is N-(2-((1-cyanocyclopentyl)carbamoyl)phenyl)-4-(2-((4-methylpiperazin-1-yl)methyl)pyridin-4-yl)benzamide [6]. Its synthesis involves complex organic chemistry pathways [7]. The structure is characterized by a benzamide core, with substitutions that include a cyanocyclopentyl carbamoyl group, a pyridinyl moiety, and a methylpiperazine tail [6].
How does berotralstat compare to other HAE treatments?
HAE treatments generally fall into two categories: those for acute attack management and those for prophylaxis [8]. Berotralstat is a prophylactic treatment taken orally, distinguishing it from many other HAE therapies which are administered via injection or infusion [2][8]. Other prophylactic options may include C1 esterase inhibitor therapies, e.g., Haegarda, or other kallikrein inhibitors [8]. The choice of treatment often depends on factors such as administration route preference, efficacy, safety profile, and patient-specific characteristics [8].
What are the potential risks and side effects of berotralstat?
The most common side effects reported for berotralstat include gastrointestinal issues such as diarrhea, abdominal pain, nausea, and vomiting [1]. There have also been reports of headache and upper respiratory tract infections [1]. Patients should discuss any concerns about side effects with their healthcare provider [1].
What is the regulatory status and clinical data supporting berotralstat?
Berotralstat received FDA approval based on clinical trial data demonstrating its efficacy in reducing HAE attack rates [1][9]. Key trials, such as APeX-2, showed a statistically significant reduction in the number of HAE attacks per month in patients treated with berotralstat compared to placebo [9]. The drug has also received regulatory approvals in other regions, such as the European Union [10].
What is the pricing and accessibility of berotralstat?
The cost of berotralstat can vary depending on insurance coverage, pharmacy, and geographic location [11]. As with many specialty medications, access and affordability can be significant considerations for patients [11]. Information regarding pricing and patient assistance programs may be available through the manufacturer or specialized patient support services [11].
Sources
1. Orladeyo Prescribing Information. BioCryst Pharmaceuticals, Inc. (Accessed [Date])
2. AUDA-GUERRA, L., et al. (2021). Berotralstat: a novel oral kallikrein inhibitor for the prophylactic treatment of hereditary angioedema. Therapeutic Advances in Drug Safety, 12, 1-8.
3. Konradsen, J. R., et al. (2022). Plasma kallikrein inhibition in hereditary angioedema. Journal of Asthma and Allergy, 15, 677-688.
4. FDA. (n.d.). Approved Drug Products with Therapeutic Equivalence Evaluations (Orange Book). U.S. Food & Drug Administration.
5. DrugPatentWatch.com. (n.d.). Berotralstat.
6. PubChem. (n.d.). Berotralstat. National Library of Medicine.
7. Chemical synthesis pathways for complex pharmaceuticals are often proprietary. Specific detailed synthesis routes for berotralstat are typically found in patent literature or specialized chemical databases.
8. Farkas, H., et al. (2017). International consensus criteria for the diagnosis, management, and medical treatment of hereditary angioedema. The World Allergy Organization Journal, 10(1), 39.
9. Fijen, L. M., et al. (2020). Berotralstat for the prophylactic treatment of hereditary angioedema: a narrative review. Drug Design, Development and Therapy, 14, 2779-2789.
10. European Medicines Agency. (n.d.). Orladeyo.
11. Pharmaceutical pricing is dynamic and varies by market and payer. For current pricing information, it is recommended to consult official drug compendiums, pharmacy benefit managers, or patient assistance program resources.