Berotralstat

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Berotralstat
Berotralstat Structure.svg
Names
Trade namesOrladeyo
Other namesBCX7353, BCX-7353
  • 2-[3-(aminomethyl)phenyl]-N-[5-[(R)-(3-cyanophenyl)-(cyclopropylmethylamino)methyl]-2-fluorophenyl]-5-(trifluoromethyl)pyrazole-3-carboxamide
Clinical data
Drug classPlasma kallikrein inhibitor
Main usesHereditary angioedema (HAE)[1]
Side effectsAbdominal pain, vomiting, diarrhea, back pain, heartburn[1]
Routes of
use		By mouth
Typical dose150 mg OD[1]
External links
AHFS/Drugs.comMonograph
Legal
License data
Legal status
Chemical and physical data
FormulaC30H26F4N6O
Molar mass562.573 g·mol−1
3D model (JSmol)
  • NCC1=CC(=CC=C1)N1N=C(C=C1C(=O)NC1=CC(=CC=C1F)[C@H](NCC1CC1)C1=CC=CC(=C1)C#N)C(F)(F)F
  • InChI=1S/C30H26F4N6O/c31-24-10-9-22(28(37-17-18-7-8-18)21-5-1-3-19(11-21)15-35)13-25(24)38-29(41)26-14-27(30(32,33)34)39-40(26)23-6-2-4-20(12-23)16-36/h1-6,9-14,18,28,37H,7-8,16-17,36H2,(H,38,41)/t28-/m1/s1
  • Key:UXNXMBYCBRBRFD-MUUNZHRXSA-N

Berotralstat, sold under the brand name Orladeyo, is a medication used to prevent attacks of hereditary angioedema (HAE).[1] It is used in people aged twelve years and older.[1] It is taken by mouth.[1]

Common side effects include abdominal pain, vomiting, diarrhea, back pain, and heartburn.[1] Other side effects may include QT prolongation.[1] While there is no evidence of harm in pregnancy, such use has not been well studied.[1] It is a plasma kallikrein inhibitor.[1]

Berotralstat was approved for medical use in the United States in 2020 and Europe in 2021.[1][2] In the United Kingdom 4 weeks costs the NHS about £10,200 as of 2022.[5] This amount in the United States costs about 40,500 USD.[6]

Medical uses

It is used to prevent attacks of hereditary angioedema (HAE).[1] It decreases the rate of attacks from about 2.4 per month on placebo to 1.3 with treatment.[2]

Dosage

It is generally taken at a dose of 150 mg once per day.[1]

History

Berotralstat was approved based on evidence from one clinical trial (Trial 1 /NCT03485911) of 120 participants with hereditary angioedema.[4] The trial was conducted at 40 sites in the United States, the European Union, and Canada.[4] Trial investigators evaluated participants 12 years and older[7] with hereditary angioedema for eight weeks to determine the number of attacks for each participant.[4] The trial enrolled only participants who had at least two attacks during the eight-week period.[4] Participants were assigned to receive one of two doses of berotralstat or placebo once every day for 24 weeks.[4] Neither the participants nor the investigators knew which treatment was being given until after the trial was completed.[4] All participants could use other medications for treatment of attacks.[4]

References

  1. ↑ 1.00 1.01 1.02 1.03 1.04 1.05 1.06 1.07 1.08 1.09 1.10 1.11 1.12 1.13 "Orladeyo- berotralstat hydrochloride capsule". DailyMed. Archived from the original on 1 November 2022. Retrieved 25 December 2020.
  2. ↑ 2.0 2.1 2.2 "Orladeyo EPAR". European Medicines Agency (EMA). 24 February 2021. Archived from the original on 12 July 2021. Retrieved 12 July 2021.
  3. ↑ https://pdf.hres.ca/dpd_pm/00066149.PDF Archived 1 October 2022 at the Wayback Machine[bare URL PDF]
  4. ↑ 4.0 4.1 4.2 4.3 4.4 4.5 4.6 4.7 "Drug Trials Snapshot: Orladeyo". U.S. Food and Drug Administration. 3 December 2020. Archived from the original on 24 October 2021. Retrieved 25 December 2020. Public Domain This article incorporates text from this source, which is in the public domain.
  5. ↑ "Berotralstat". SPS - Specialist Pharmacy Service. 21 July 2018. Archived from the original on 12 December 2021. Retrieved 3 November 2022.
  6. ↑ "Orladeyo Prices, Coupons, Copay & Patient Assistance". Drugs.com. Archived from the original on 21 May 2022. Retrieved 3 November 2022.
  7. ↑ "Berotralstat (Oral Route) Side Effects - Mayo Clinic". www.mayoclinic.org. Archived from the original on 17 April 2021. Retrieved 3 March 2021.

External links

External sites:
Identifiers:
  • Clinical trial number NCT03485911 for "Efficacy and Safety Study of BCX7353 as an Oral Treatment for the Prevention of Attacks in HAE (APeX-2)" at ClinicalTrials.gov
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