Alpelisib Explained
Tradename: | Piqray, Vijoice |
Dailymedid: | Alpelisib |
Pregnancy Au: | D |
Pregnancy Au Comment: | [1] [2] |
Routes Of Administration: | By mouth |
Atc Prefix: | L01 |
Atc Suffix: | EM03 |
Legal Au: | S4 |
Legal Ca: | Rx-only |
Legal Ca Comment: | [3] |
Legal Us: | Rx-only |
Legal Us Comment: | [4] [5] |
Legal Eu: | Rx-only |
Legal Eu Comment: | [6] [7] |
Cas Number: | 1217486-61-7 |
Pubchem: | 56649450 |
Pubchemsubstance: | 347828332 |
Drugbank: | DB12015 |
Chemspiderid: | 28424123 |
Unii: | 08W5N2C97Q |
Kegg: | D11011 |
Chebi: | 93752 |
Chembl: | 2396661 |
Synonyms: | BYL719 |
Iupac Name: | (2S)-1-N-[4-Methyl-5-[2-(1,1,1-trifluoro-2-methylpropan-2-yl)pyridin-4-yl]-1,3-thiazol-2-yl]pyrrolidine-1,2-dicarboxamide |
C: | 19 |
H: | 22 |
F: | 3 |
N: | 5 |
O: | 2 |
S: | 1 |
Smiles: | CC1=C(SC(=N1)NC(=O)N2CCCC2C(=O)N)C3=CC(=NC=C3)C(C)(C)C(F)(F)F |
Stdinchi: | 1S/C19H22F3N5O2S/c1-10-14(11-6-7-24-13(9-11)18(2,3)19(20,21)22)30-16(25-10)26-17(29)27-8-4-5-12(27)15(23)28/h6-7,9,12H,4-5,8H2,1-3H3,(H2,23,28)(H,25,26,29)/t12-/m0/s1 |
Stdinchikey: | STUWGJZDJHPWGZ-LBPRGKRZSA-N |
Alpelisib, sold under the brand name Piqray among others, is a medication used to treat certain types of breast cancer. It is used together with fulvestrant.[8] It is taken by mouth.[8] It is marketed by Novartis.
Common side effects include high blood sugar, kidney problems, diarrhea, rash, low blood cells, liver problems, pancreatitis, vomiting, and hair loss.[8] It is an alpha-specific PI3K inhibitor.[8] [9] It was approved for medical use in the United States in May 2019.[8] [10]
Medical uses
Alpelisib is indicated in combination with fulvestrant for the treatment of postmenopausal women, and men, with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, PIK3CA-mutated, advanced or metastatic breast cancer as detected by an FDA-approved test following progression on or after an endocrine-based regimen.[11]
In the European Union, alpelisib is indicated in combination with fulvestrant for the treatment of postmenopausal women, and men, with hormone receptor (HR)‑positive, human epidermal growth factor receptor 2 (HER2)‑negative, locally advanced or metastatic breast cancer with a PIK3CA mutation after disease progression following endocrine therapy as monotherapy.
In April 2022, the indication for alpelisib was expanded in the US to include the treatment of severe manifestations of PIK3CA-related overgrowth spectrum (PROS) in those who require systemic therapy.[12] [13]
History
In May 2019, alpelisib was approved in the United States for use in combination with the endocrine therapy fulvestrant, to treat postmenopausal women, and men, with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, PIK3CA-mutated, advanced or metastatic breast cancer following progression on or after an endocrine-based regimen.
The U.S. Food and Drug Administration (FDA) also approved the companion diagnostic test, therascreen PIK3CA RGQ PCR Kit, to detect the PIK3CA mutation in a tissue and/or a liquid biopsy.
The efficacy of alpelisib was studied in the SOLAR-1 trial (NCT02437318), a randomized trial of 572 postmenopausal women and men with HR-positive, HER2-negative, advanced or metastatic breast cancer whose cancer had progressed while on or after receiving an aromatase inhibitor.[14]
The FDA granted the application for alpelisib priority review designation and granted approval of Piqray to Novartis. The FDA granted approval of the therascreen PIK3CA RGQ PCR Kit to Qiagen Manchester, Ltd.
On 28 May 2020, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product alpelisib (Piqray), intended for the treatment of locally advanced or metastatic breast cancer with a PIK3CA mutation. The applicant for this medicinal product is Novartis Europharm Limited. Alpelisib was approved for medical use in the European Union in July 2020.
Society and culture
Legal status
Alpelisib was approved for medical use in the United States in May 2019,[8] in Australia in March 2020,[15] and in the European Union in July 2020.
Notes and References
- Web site: Piqray Australian prescription medicine decision summary . Therapeutic Goods Administration (TGA) . 27 March 2020 . 16 August 2020 . 27 August 2021 . https://web.archive.org/web/20210827211951/https://www.tga.gov.au/apm-summary/piqray . live .
- Web site: Updates to the Prescribing Medicines in Pregnancy database . Therapeutic Goods Administration (TGA) . 12 May 2022 . 13 May 2022 . 3 April 2022 . https://web.archive.org/web/20220403064059/https://www.tga.gov.au/updates-prescribing-medicines-pregnancy-database . live .
- Web site: Summary Basis of Decision (SBD) for Piqray . Health Canada . 23 October 2014 . 29 May 2022 . 30 May 2022 . https://web.archive.org/web/20220530055303/https://hpr-rps.hres.ca/reg-content/summary-basis-decision-detailTwo.php?linkID=SBD00493&lang=en . live .
- Web site: Piqray- alpelisib tablet Piqray- alpelisib kit . DailyMed . 12 June 2020 . 16 August 2020 . 28 October 2020 . https://web.archive.org/web/20201028032120/https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=b20b4e18-7a4b-4500-a08f-06c6dab0ee5b . live .
- Web site: Vijoice- alpelisib tablet Vijoice- alpelisib kit . DailyMed . 7 April 2022 . 27 April 2022 . 28 April 2022 . https://web.archive.org/web/20220428034846/https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=638069a4-aee0-46c9-9e21-54ad449c163c . live .
- Web site: Piqray EPAR . European Medicines Agency (EMA) . 26 May 2020 . 16 August 2020 . 14 August 2020 . https://web.archive.org/web/20200814032206/https://www.ema.europa.eu/en/medicines/human/EPAR/piqray . live . Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- Web site: Piqray Product information . Union Register of medicinal products . 3 March 2023.
- FDA approves first PI3K inhibitor for breast cancer . U.S. Food and Drug Administration (FDA) . 29 May 2019 . 24 May 2019 . https://web.archive.org/web/20191125012114/https://www.fda.gov/news-events/press-announcements/fda-approves-first-pi3k-inhibitor-breast-cancer . 25 November 2019 . live .
- André F, Ciruelos E, Rubovszky G, Campone M, Loibl S, Rugo HS, Iwata H, Conte P, Mayer IA, Kaufman B, Yamashita T, Lu YS, Inoue K, Takahashi M, Pápai Z, Longin AS, Mills D, Wilke C, Hirawat S, Juric D . 6 . PIK3CA-Mutated, Hormone Receptor-Positive Advanced Breast Cancer . The New England Journal of Medicine . 380 . 20 . 1929–1940 . May 2019 . 31091374 . 10.1056/NEJMoa1813904 . the SOLAR-1 Study Group . SOLAR-1 Study Group . free . 11577/3308753 . free .
- Web site: Drug Approval Package: Piqray . U.S. Food and Drug Administration (FDA) . 18 June 2019 . 25 November 2019 . 25 November 2019 . https://web.archive.org/web/20191125050717/https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/212526Orig1s000TOC.cfm . live .
- Web site: Piqray (alpelisib) tablets HR+/HER2- Advanced Breast Cancer Treatment. 24 August 2021. Novartis. 24 August 2021. https://web.archive.org/web/20210824091213/https://www.hcp.novartis.com/products/piqray/metastatic-breast-cancer/. live.
- Web site: FDA approves alpelisib for PIK3CA-related overgrowth spectrum . U.S. Food and Drug Administration (FDA) . 6 April 2022 . 8 April 2022 . 7 April 2022 . https://web.archive.org/web/20220407164055/https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-alpelisib-pik3ca-related-overgrowth-spectrum . live .
- FDA approves Novartis Vijoice (alpelisib) as first and only treatment for select patients with PIK3CA-Related Overgrowth Spectrum (PROS) . Novartis . 6 April 2022 . 9 April 2022 . 6 April 2022 . https://web.archive.org/web/20220406230750/https://www.novartis.com/news/media-releases/fda-approves-novartis-vijoice-alpelisib-first-and-only-treatment-select-patients-pik3ca-related-overgrowth-spectrum-pros . live .
- Web site: Drug Trials Snapshots: Piqray . U.S. Food and Drug Administration (FDA) . 14 June 2019 . https://web.archive.org/web/20191125013110/https://www.fda.gov/drugs/drug-trials-snapshots-piqray . 25 November 2019 . live . 24 November 2019.
- Web site: AusPAR: Alpelisib . Therapeutic Goods Administration (TGA) . 3 September 2020 . 23 September 2020 . 27 October 2020 . https://web.archive.org/web/20201027152621/https://www.tga.gov.au/auspar/auspar-alpelisib . live .