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Alpelisib

Generic Name
Alpelisib
Brand Names
Piqray 300 Mg Daily Dose, Vijoice 50 Mg 28 Day, Piqray
Drug Type
Small Molecule
Chemical Formula
C19H22F3N5O2S
CAS Number
1217486-61-7
Unique Ingredient Identifier
08W5N2C97Q

Overview

Alpelisib is a phosphatidylinositol 3-kinase (PI3K) inhibitor with potent antitumor activity. It works by selectively inhibiting class I PI3K p110α , which is the catalytic subunit of PI3K, a lipid kinase that plays a role in various biological processes, including proliferation, survival, differentiation, and metabolism. Alpelisib was designed to target this enzyme that appears to be mutated at a rate of nearly 30% in human cancers, leading to hyperactivation. There are several isoform-specific PI3K inhibitors that are under clinical development or currently approved, such as idelalisib used for chronic lymphocytic leukemia (CLL). Approved by the FDA in May 2019, alpelisib is the first approved PI3K inhibitor indicated for the treatment of hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, PIK3CA-mutated, advanced or metastatic breast cancer in combination with fulvestrant for postmenopausal women and male patients. To initiate alpelisib therapy, it is required that the presence of a PIK3CA mutation in the tissue and/or liquid biopsy sample collection should be confirmed via FDA-approved diagnostic tests. Alpelisib is marketed under the trade name Piqray and is available as oral tablets. Studies evaluating the therapeutic effectiveness of alpelisib in other cancers, such as ovarian cancer and colorectal cancer , are under ongoing investigations. Alpelisib was granted FDA approval on 24 May 2019. In April 2022, the FDA granted the use of alpelisib in the treatment of PIK3CA-Related Overgrowth Spectrum (PROS) in adults and children who require systemic therapy.

Background

Alpelisib is a phosphatidylinositol 3-kinase (PI3K) inhibitor with potent antitumor activity. It works by selectively inhibiting class I PI3K p110α , which is the catalytic subunit of PI3K, a lipid kinase that plays a role in various biological processes, including proliferation, survival, differentiation, and metabolism. Alpelisib was designed to target this enzyme that appears to be mutated at a rate of nearly 30% in human cancers, leading to hyperactivation. There are several isoform-specific PI3K inhibitors that are under clinical development or currently approved, such as idelalisib used for chronic lymphocytic leukemia (CLL). Approved by the FDA in May 2019, alpelisib is the first approved PI3K inhibitor indicated for the treatment of hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, PIK3CA-mutated, advanced or metastatic breast cancer in combination with fulvestrant for postmenopausal women and male patients. To initiate alpelisib therapy, it is required that the presence of a PIK3CA mutation in the tissue and/or liquid biopsy sample collection should be confirmed via FDA-approved diagnostic tests. Alpelisib is marketed under the trade name Piqray and is available as oral tablets. Studies evaluating the therapeutic effectiveness of alpelisib in other cancers, such as ovarian cancer and colorectal cancer , are under ongoing investigations. Alpelisib was granted FDA approval on 24 May 2019. In April 2022, the FDA granted the use of alpelisib in the treatment of PIK3CA-Related Overgrowth Spectrum (PROS) in adults and children who require systemic therapy.

Indication

Alpelisib is indicated in combination with fulvestrant to treat postmenopausal women, and men, with advanced or metastatic breast cancer. This cancer must be hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, and PIK3CA­ mutated. The cancer must be detected by an FDA-approved test following progression on or after an endocrine-based regimen. Alpelisib is also used to treat adult and pediatric patients two years of age and older with severe manifestations of PIK3CA-Related Overgrowth Spectrum (PROS) who require systemic therapy. This indication is approved under accelerated approval based on response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

Associated Conditions

  • HR+, HER2-, Advanced Breast Cancer
  • PIK3CA-Related Overgrowth Spectrum (PROS)
  • Metastatic HR + HER2 - breast cancer

Clinical Trials

Title
Posted
Study ID
Phase
Status
Sponsor
2025/05/30
Phase 2
Not yet recruiting
2024/12/18
Phase 2
Recruiting
Tianjin Medical University Second Hospital
2024/08/09
Phase 1
Recruiting
2024/04/09
Phase 1
Recruiting
2023/10/13
N/A
Active, not recruiting
2023/08/09
Phase 2
Recruiting
2023/08/01
Phase 2
Withdrawn
2023/07/17
Phase 2
Recruiting
2023/07/06
Phase 2
Recruiting
2023/02/17
Phase 1
Completed

FDA Drug Approvals

Approved Product
Manufacturer
NDC Code
Route
Strength
Effective Date
Novartis Pharmaceuticals Corporation
0078-1028
ORAL
125 mg in 1 1
6/18/2025
Novartis Pharmaceuticals Corporation
0078-1021
ORAL
50 mg in 1 1
6/18/2025
Novartis Pharmaceuticals Corporation
0078-0708
ORAL
150 mg in 1 1
5/24/2019
Novartis Pharmaceuticals Corporation
0078-1175
ORAL
50 mg in 1 1
6/18/2025
Novartis Pharmaceuticals Corporation
0078-0701
ORAL
200 mg in 1 1
5/24/2019

NMPA Drug Approvals

Approved Product
Company
Approval Number
Drug Type
Dosage Form
Approval Date
No NMPA approvals found for this drug.

PPB Drug Approvals

Approved Product
Registration No.
Company
Licence No.
Strength
Registration Date
PIQRAY TABLETS 200MG
N/A
N/A
N/A
12/9/2020
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