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 |
EMA Drug Approvals
Approved Product | Authorization Holder | Status | Issued Date |
---|---|---|---|
Authorised | 7/27/2020 | ||
Authorised | 7/27/2020 |
HSA Drug Approvals
Approved Product | Manufacturer | Approval Number | Dosage Form | Strength | Approval Date |
---|---|---|---|---|---|
PIQRAY 200MG DAILY DOSE PACK | SIN15918P | TABLET, FILM COATED | 200mg | 3/25/2020 | |
PIQRAY 250MG DAILY DOSE PACK (200mg + 50mg) | SIN15916P | TABLET, FILM COATED | 50mg | 3/25/2020 | |
PIQRAY 300MG DAILY DOSE PACK (150mg + 150mg) | SIN15917P | TABLET, FILM COATED | 150mg | 3/25/2020 | |
PIQRAY 250MG DAILY DOSE PACK (200mg + 50mg) | SIN15916P | TABLET, FILM COATED | 200mg | 3/25/2020 |
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 |
TGA Drug Approvals
Approved Product | ARTG ID | Sponsor | Registration Type | Status | Registration Date |
---|---|---|---|---|---|
PIQRAY alpelisib 200 mg daily dose blister (200 mg tablet) | 315020 | Medicine | A | 3/20/2020 | |
PIQRAY alpelisib 250 mg daily dose blister (200+50 mg tablet) composite pack | 315055 | Medicine | A | 3/20/2020 | |
PIQRAY alpelisib 300 mg daily dose blister (150 mg tablet) | 315064 | Medicine | A | 3/20/2020 |