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Alpelisib
open-in-new
<h2 id="medical-uses">Medical uses</h2> <p>Alpelisib is <a href="/facts/Indication_(medicine)/DLdGwxDY">indicated</a> 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.<a class="footnote-ref" id="fnref:10" href="#fn:10"><sup>10</sup></a> </p><p>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.<a class="footnote-ref" id="fnref:11" href="#fn:11"><sup>11</sup></a> </p><p>In April 2022, the indication for alpelisib was expanded in the US to include the treatment of severe manifestations of <a href="/facts/PIK3CA-related_overgrowth_spectrum/bMrrI7A0">PIK3CA-related overgrowth spectrum</a> (PROS) in those who require systemic therapy.<a class="footnote-ref" id="fnref:12" href="#fn:12"><sup>12</sup></a><a class="footnote-ref" id="fnref:13" href="#fn:13"><sup>13</sup></a><a class="footnote-ref" id="fnref:14" href="#fn:14"><sup>14</sup></a> </p> <h2 id="history">History</h2> <p>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 <a href="/facts/Hormone_receptor_positive_breast_tumor/v2mjTh4g">(HR)-positive</a>, human epidermal growth factor receptor 2 (<a href="/facts/HER2/xue3SgcA">HER2</a>)-negative, <a href="/facts/PIK3CA/8hv8qF2I">PIK3CA</a>-mutated, advanced or metastatic breast cancer following progression on or after an endocrine-based regimen.<a class="footnote-ref" id="fnref:15" href="#fn:15"><sup>15</sup></a><a class="footnote-ref" id="fnref:16" href="#fn:16"><sup>16</sup></a><a class="footnote-ref" id="fnref:17" href="#fn:17"><sup>17</sup></a> </p><p>The U.S. <a href="/facts/Food_and_Drug_Administration/rCEnh9WB">Food and Drug Administration</a> (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.<a class="footnote-ref" id="fnref:18" href="#fn:18"><sup>18</sup></a> </p><p>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.<a class="footnote-ref" id="fnref:19" href="#fn:19"><sup>19</sup></a><a class="footnote-ref" id="fnref:20" href="#fn:20"><sup>20</sup></a> </p><p>The FDA granted the application for alpelisib <a href="/facts/Priority_review/PBc14kY6">priority review</a> designation and granted approval of Piqray to Novartis.<a class="footnote-ref" id="fnref:21" href="#fn:21"><sup>21</sup></a> The FDA granted approval of the therascreen PIK3CA RGQ PCR Kit to Qiagen Manchester, Ltd.<a class="footnote-ref" id="fnref:22" href="#fn:22"><sup>22</sup></a> </p><p>On 28 May 2020, the <a href="/facts/Committee_for_Medicinal_Products_for_Human_Use/yCkJTISe">Committee for Medicinal Products for Human Use</a> (CHMP) of the <a href="/facts/European_Medicines_Agency/h9FTtCYc">European Medicines Agency</a> (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.<a class="footnote-ref" id="fnref:23" href="#fn:23"><sup>23</sup></a> The applicant for this medicinal product is Novartis Europharm Limited.<a class="footnote-ref" id="fnref:24" href="#fn:24"><sup>24</sup></a> Alpelisib was approved for medical use in the European Union in July 2020.<a class="footnote-ref" id="fnref:25" href="#fn:25"><sup>25</sup></a> </p> <h2 id="society-and-culture">Society and culture</h2> <h3>Legal status</h3> <p>Alpelisib was approved for medical use in the United States in May 2019,<a class="footnote-ref" id="fnref:26" href="#fn:26"><sup>26</sup></a><a class="footnote-ref" id="fnref:27" href="#fn:27"><sup>27</sup></a> in Australia in March 2020,<a class="footnote-ref" id="fnref:28" href="#fn:28"><sup>28</sup></a> and in the European Union in July 2020.<a class="footnote-ref" id="fnref:29" href="#fn:29"><sup>29</sup></a> </p> <h2 id="external-links">External links</h2> <ul><li>Clinical trial number <i><a href="https://www.clinicaltrials.gov/show/NCT02437318">NCT02437318</a></i> for "Study Assessing the Efficacy and Safety of Alpelisib Plus Fulvestrant in Men and Postmenopausal Women With Advanced Breast Cancer Which Progressed on or After Aromatase Inhibitor Treatment. (SOLAR-1)" at <a href="/facts/ClinicalTrials.gov/X6WVgItm">ClinicalTrials.gov</a></li></ul> <h2 id="references">References</h2> <ol> <li id="fn:1"><p>"FDA approves first PI3K inhibitor for breast cancer". U.S. Food and Drug Administration (FDA) (Press release). 24 May 2019. Archived from the original on 25 November 2019. Retrieved 29 May 2019. This article incorporates text from this source, which is in the public domain. <a href="https://www.fda.gov/news-events/press-announcements/fda-approves-first-pi3k-inhibitor-breast-cancer" target="_blank">https://www.fda.gov/news-events/press-announcements/fda-approves-first-pi3k-inhibitor-breast-cancer</a> <a href="#fnref:1" class="footnote-back-ref">↩</a></p></li> <li id="fn:2"><p>"FDA approves first PI3K inhibitor for breast cancer". U.S. Food and Drug Administration (FDA) (Press release). 24 May 2019. Archived from the original on 25 November 2019. Retrieved 29 May 2019. This article incorporates text from this source, which is in the public domain. <a href="https://www.fda.gov/news-events/press-announcements/fda-approves-first-pi3k-inhibitor-breast-cancer" target="_blank">https://www.fda.gov/news-events/press-announcements/fda-approves-first-pi3k-inhibitor-breast-cancer</a> <a href="#fnref:2" class="footnote-back-ref">↩</a></p></li> <li id="fn:3"><p>"FDA approves first PI3K inhibitor for breast cancer". U.S. Food and Drug Administration (FDA) (Press release). 24 May 2019. Archived from the original on 25 November 2019. Retrieved 29 May 2019. This article incorporates text from this source, which is in the public domain. <a href="https://www.fda.gov/news-events/press-announcements/fda-approves-first-pi3k-inhibitor-breast-cancer" target="_blank">https://www.fda.gov/news-events/press-announcements/fda-approves-first-pi3k-inhibitor-breast-cancer</a> <a href="#fnref:3" class="footnote-back-ref">↩</a></p></li> <li id="fn:4"><p>"FDA approves first PI3K inhibitor for breast cancer". U.S. Food and Drug Administration (FDA) (Press release). 24 May 2019. Archived from the original on 25 November 2019. Retrieved 29 May 2019. This article incorporates text from this source, which is in the public domain. <a href="https://www.fda.gov/news-events/press-announcements/fda-approves-first-pi3k-inhibitor-breast-cancer" target="_blank">https://www.fda.gov/news-events/press-announcements/fda-approves-first-pi3k-inhibitor-breast-cancer</a> <a href="#fnref:4" class="footnote-back-ref">↩</a></p></li> <li id="fn:5"><p>"FDA approves first PI3K inhibitor for breast cancer". U.S. Food and Drug Administration (FDA) (Press release). 24 May 2019. Archived from the original on 25 November 2019. Retrieved 29 May 2019. This article incorporates text from this source, which is in the public domain. <a href="https://www.fda.gov/news-events/press-announcements/fda-approves-first-pi3k-inhibitor-breast-cancer" target="_blank">https://www.fda.gov/news-events/press-announcements/fda-approves-first-pi3k-inhibitor-breast-cancer</a> <a href="#fnref:5" class="footnote-back-ref">↩</a></p></li> <li id="fn:6"><p>"FDA approves first PI3K inhibitor for breast cancer". U.S. Food and Drug Administration (FDA) (Press release). 24 May 2019. Archived from the original on 25 November 2019. Retrieved 29 May 2019. This article incorporates text from this source, which is in the public domain. <a href="https://www.fda.gov/news-events/press-announcements/fda-approves-first-pi3k-inhibitor-breast-cancer" target="_blank">https://www.fda.gov/news-events/press-announcements/fda-approves-first-pi3k-inhibitor-breast-cancer</a> <a href="#fnref:6" class="footnote-back-ref">↩</a></p></li> <li id="fn:7"><p>André F, Ciruelos E, Rubovszky G, Campone M, Loibl S, Rugo HS, et al. (May 2019). "PIK3CA-Mutated, Hormone Receptor-Positive Advanced Breast Cancer". The New England Journal of Medicine. 380 (20): 1929–1940. doi:10.1056/NEJMoa1813904. hdl:11577/3308753. PMID 31091374. <a href="https://doi.org/10.1056%2FNEJMoa1813904" target="_blank">https://doi.org/10.1056%2FNEJMoa1813904</a> <a href="#fnref:7" class="footnote-back-ref">↩</a></p></li> <li id="fn:8"><p>"FDA approves first PI3K inhibitor for breast cancer". U.S. Food and Drug Administration (FDA) (Press release). 24 May 2019. Archived from the original on 25 November 2019. Retrieved 29 May 2019. This article incorporates text from this source, which is in the public domain. <a href="https://www.fda.gov/news-events/press-announcements/fda-approves-first-pi3k-inhibitor-breast-cancer" target="_blank">https://www.fda.gov/news-events/press-announcements/fda-approves-first-pi3k-inhibitor-breast-cancer</a> <a href="#fnref:8" class="footnote-back-ref">↩</a></p></li> <li id="fn:9"><p>"Drug Approval Package: Piqray". U.S. Food and Drug Administration (FDA). 18 June 2019. Archived from the original on 25 November 2019. Retrieved 25 November 2019. <a href="https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/212526Orig1s000TOC.cfm" target="_blank">https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/212526Orig1s000TOC.cfm</a> <a href="#fnref:9" class="footnote-back-ref">↩</a></p></li> <li id="fn:10"><p>"Piqray (alpelisib) tablets HR+/HER2- Advanced Breast Cancer Treatment". Novartis. Archived from the original on 24 August 2021. Retrieved 24 August 2021. <a href="https://www.hcp.novartis.com/products/piqray/metastatic-breast-cancer/" target="_blank">https://www.hcp.novartis.com/products/piqray/metastatic-breast-cancer/</a> <a href="#fnref:10" class="footnote-back-ref">↩</a></p></li> <li id="fn:11"><p>"Piqray EPAR". European Medicines Agency (EMA). 26 May 2020. Archived from the original on 14 August 2020. Retrieved 16 August 2020. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged. <a href="https://www.ema.europa.eu/en/medicines/human/EPAR/piqray" target="_blank">https://www.ema.europa.eu/en/medicines/human/EPAR/piqray</a> <a href="#fnref:11" class="footnote-back-ref">↩</a></p></li> <li id="fn:12"><p>"Vijoice- alpelisib tablet Vijoice- alpelisib kit". DailyMed. 7 April 2022. Archived from the original on 28 April 2022. Retrieved 27 April 2022. <a href="https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=638069a4-aee0-46c9-9e21-54ad449c163c" target="_blank">https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=638069a4-aee0-46c9-9e21-54ad449c163c</a> <a href="#fnref:12" class="footnote-back-ref">↩</a></p></li> <li id="fn:13"><p>"FDA approves alpelisib for PIK3CA-related overgrowth spectrum". U.S. Food and Drug Administration (FDA). 6 April 2022. Archived from the original on 7 April 2022. Retrieved 8 April 2022. This article incorporates text from this source, which is in the public domain. <a href="https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-alpelisib-pik3ca-related-overgrowth-spectrum" target="_blank">https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-alpelisib-pik3ca-related-overgrowth-spectrum</a> <a href="#fnref:13" class="footnote-back-ref">↩</a></p></li> <li id="fn:14"><p>"FDA approves Novartis Vijoice (alpelisib) as first and only treatment for select patients with PIK3CA-Related Overgrowth Spectrum (PROS)". Novartis (Press release). 6 April 2022. Archived from the original on 6 April 2022. Retrieved 9 April 2022. <a href="https://www.novartis.com/news/media-releases/fda-approves-novartis-vijoice-alpelisib-first-and-only-treatment-select-patients-pik3ca-related-overgrowth-spectrum-pros" target="_blank">https://www.novartis.com/news/media-releases/fda-approves-novartis-vijoice-alpelisib-first-and-only-treatment-select-patients-pik3ca-related-overgrowth-spectrum-pros</a> <a href="#fnref:14" class="footnote-back-ref">↩</a></p></li> <li id="fn:15"><p>"FDA approves first PI3K inhibitor for breast cancer". U.S. Food and Drug Administration (FDA) (Press release). 24 May 2019. Archived from the original on 25 November 2019. Retrieved 29 May 2019. This article incorporates text from this source, which is in the public domain. <a href="https://www.fda.gov/news-events/press-announcements/fda-approves-first-pi3k-inhibitor-breast-cancer" target="_blank">https://www.fda.gov/news-events/press-announcements/fda-approves-first-pi3k-inhibitor-breast-cancer</a> <a href="#fnref:15" class="footnote-back-ref">↩</a></p></li> <li id="fn:16"><p>"Piqray- alpelisib tablet Piqray- alpelisib kit". DailyMed. 12 June 2020. Archived from the original on 28 October 2020. Retrieved 16 August 2020. <a href="https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=b20b4e18-7a4b-4500-a08f-06c6dab0ee5b" target="_blank">https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=b20b4e18-7a4b-4500-a08f-06c6dab0ee5b</a> <a href="#fnref:16" class="footnote-back-ref">↩</a></p></li> <li id="fn:17"><p>"Drug Approval Package: Piqray". U.S. Food and Drug Administration (FDA). 18 June 2019. Archived from the original on 25 November 2019. Retrieved 25 November 2019. <a href="https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/212526Orig1s000TOC.cfm" target="_blank">https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/212526Orig1s000TOC.cfm</a> <a href="#fnref:17" class="footnote-back-ref">↩</a></p></li> <li id="fn:18"><p>"FDA approves first PI3K inhibitor for breast cancer". U.S. Food and Drug Administration (FDA) (Press release). 24 May 2019. Archived from the original on 25 November 2019. Retrieved 29 May 2019. This article incorporates text from this source, which is in the public domain. <a href="https://www.fda.gov/news-events/press-announcements/fda-approves-first-pi3k-inhibitor-breast-cancer" target="_blank">https://www.fda.gov/news-events/press-announcements/fda-approves-first-pi3k-inhibitor-breast-cancer</a> <a href="#fnref:18" class="footnote-back-ref">↩</a></p></li> <li id="fn:19"><p>"FDA approves first PI3K inhibitor for breast cancer". U.S. Food and Drug Administration (FDA) (Press release). 24 May 2019. Archived from the original on 25 November 2019. Retrieved 29 May 2019. This article incorporates text from this source, which is in the public domain. <a href="https://www.fda.gov/news-events/press-announcements/fda-approves-first-pi3k-inhibitor-breast-cancer" target="_blank">https://www.fda.gov/news-events/press-announcements/fda-approves-first-pi3k-inhibitor-breast-cancer</a> <a href="#fnref:19" class="footnote-back-ref">↩</a></p></li> <li id="fn:20"><p>"Drug Trials Snapshots: Piqray". U.S. Food and Drug Administration (FDA). 14 June 2019. Archived from the original on 25 November 2019. Retrieved 24 November 2019. This article incorporates text from this source, which is in the public domain. <a href="https://www.fda.gov/drugs/drug-trials-snapshots-piqray" target="_blank">https://www.fda.gov/drugs/drug-trials-snapshots-piqray</a> <a href="#fnref:20" class="footnote-back-ref">↩</a></p></li> <li id="fn:21"><p>"FDA approves first PI3K inhibitor for breast cancer". U.S. Food and Drug Administration (FDA) (Press release). 24 May 2019. Archived from the original on 25 November 2019. Retrieved 29 May 2019. This article incorporates text from this source, which is in the public domain. <a href="https://www.fda.gov/news-events/press-announcements/fda-approves-first-pi3k-inhibitor-breast-cancer" target="_blank">https://www.fda.gov/news-events/press-announcements/fda-approves-first-pi3k-inhibitor-breast-cancer</a> <a href="#fnref:21" class="footnote-back-ref">↩</a></p></li> <li id="fn:22"><p>"FDA approves first PI3K inhibitor for breast cancer". U.S. Food and Drug Administration (FDA) (Press release). 24 May 2019. Archived from the original on 25 November 2019. Retrieved 29 May 2019. This article incorporates text from this source, which is in the public domain. <a href="https://www.fda.gov/news-events/press-announcements/fda-approves-first-pi3k-inhibitor-breast-cancer" target="_blank">https://www.fda.gov/news-events/press-announcements/fda-approves-first-pi3k-inhibitor-breast-cancer</a> <a href="#fnref:22" class="footnote-back-ref">↩</a></p></li> <li id="fn:23"><p>"Piqray EPAR". European Medicines Agency (EMA). 26 May 2020. Archived from the original on 14 August 2020. Retrieved 16 August 2020. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged. <a href="https://www.ema.europa.eu/en/medicines/human/EPAR/piqray" target="_blank">https://www.ema.europa.eu/en/medicines/human/EPAR/piqray</a> <a href="#fnref:23" class="footnote-back-ref">↩</a></p></li> <li id="fn:24"><p>"Piqray EPAR". European Medicines Agency (EMA). 26 May 2020. Archived from the original on 14 August 2020. Retrieved 16 August 2020. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged. <a href="https://www.ema.europa.eu/en/medicines/human/EPAR/piqray" target="_blank">https://www.ema.europa.eu/en/medicines/human/EPAR/piqray</a> <a href="#fnref:24" class="footnote-back-ref">↩</a></p></li> <li id="fn:25"><p>"Piqray EPAR". European Medicines Agency (EMA). 26 May 2020. Archived from the original on 14 August 2020. Retrieved 16 August 2020. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged. <a href="https://www.ema.europa.eu/en/medicines/human/EPAR/piqray" target="_blank">https://www.ema.europa.eu/en/medicines/human/EPAR/piqray</a> <a href="#fnref:25" class="footnote-back-ref">↩</a></p></li> <li id="fn:26"><p>"FDA approves first PI3K inhibitor for breast cancer". U.S. Food and Drug Administration (FDA) (Press release). 24 May 2019. Archived from the original on 25 November 2019. Retrieved 29 May 2019. This article incorporates text from this source, which is in the public domain. <a href="https://www.fda.gov/news-events/press-announcements/fda-approves-first-pi3k-inhibitor-breast-cancer" target="_blank">https://www.fda.gov/news-events/press-announcements/fda-approves-first-pi3k-inhibitor-breast-cancer</a> <a href="#fnref:26" class="footnote-back-ref">↩</a></p></li> <li id="fn:27"><p>"Drug Approval Package: Piqray". U.S. Food and Drug Administration (FDA). 18 June 2019. Archived from the original on 25 November 2019. Retrieved 25 November 2019. <a href="https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/212526Orig1s000TOC.cfm" target="_blank">https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/212526Orig1s000TOC.cfm</a> <a href="#fnref:27" class="footnote-back-ref">↩</a></p></li> <li id="fn:28"><p>"AusPAR: Alpelisib". Therapeutic Goods Administration (TGA). 3 September 2020. Archived from the original on 27 October 2020. Retrieved 23 September 2020. <a href="https://www.tga.gov.au/auspar/auspar-alpelisib" target="_blank">https://www.tga.gov.au/auspar/auspar-alpelisib</a> <a href="#fnref:28" class="footnote-back-ref">↩</a></p></li> <li id="fn:29"><p>"Piqray EPAR". European Medicines Agency (EMA). 26 May 2020. Archived from the original on 14 August 2020. Retrieved 16 August 2020. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged. <a href="https://www.ema.europa.eu/en/medicines/human/EPAR/piqray" target="_blank">https://www.ema.europa.eu/en/medicines/human/EPAR/piqray</a> <a href="#fnref:29" class="footnote-back-ref">↩</a></p></li> </ol>