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Piqray (alpelisib)
Alpelisib inhibits PI3Kα, blocking Akt-signaling and preventing tumor growth and organ abnormalities in PROS disorders.
Piqray (alpelisib) is a small molecule developed by Novartis that targets the PI3Kα enzyme. It is approved to treat germline BRCA-mutated, HER2-negative metastatic breast cancer and PIK3CA-related overgrowth spectrum (PROS). Piqray was first approved by the FDA in 2019 and remains a patented product with no generic manufacturers. Key safety considerations include monitoring for hyperglycemia and other metabolic effects. As a targeted therapy, Piqray works by inhibiting the PI3Kα enzyme, which is involved in cell growth and survival.
At a glance
| Generic name | alpelisib |
|---|---|
| Sponsor | Novartis |
| Target | PI3Kα |
| Therapeutic area | Oncology |
| Phase | FDA-approved |
| First approval | 2019 |
Mechanism of action
Alpelisib works by inhibiting the PI3Kα enzyme, which is often activated due to mutations in the PIK3CA gene. This activation leads to increased Akt-signaling, promoting cell transformation and tumor formation. By blocking this pathway, alpelisib can prevent or improve symptoms in conditions like CLOVES syndrome, a form of PROS.
Approved indications
- Germline BRCA-mutated, HER2-negative metastatic breast cancer
- PIK3CA-related overgrowth spectrum (PROS)
Common side effects
- Diarrhea
- Rash
- Fatigue
- Glucose increased
- Creatinine increased
- Nausea
- Lymphocyte count decreased
- Gamma glutamyl transferase (GGT) increased
- Alanine aminotransferase (ALT) increased
- Hemoglobin decreased
- Lipase increased
- Decreased appetite
Key clinical trials
- Targeted Therapy Directed by Genetic Testing in Treating Patients With Locally Advanced or Advanced Solid Tumors, The ComboMATCH Screening Trial (PHASE2)
- Study Assessing the Efficacy and Safety of Alpelisib + Nab-paclitaxel in Subjects With Advanced TNBC Who Carry Either a PIK3CA Mutation or Have PTEN Loss (PHASE3)
- Phase 1/2 Study of ETX-636 in Participants With Advanced Solid Tumors (PHASE1,PHASE2)
- Clinical Trial of Alpelisb and Tucatinib in Patients With PIK3CA-Mutant HER2+ Metastatic Breast Cancer. (PHASE1,PHASE2)
- Alpelisib Plus Olaparib in Platinum-resistant/Refractory, High-grade Serous Ovarian Cancer, With no Germline BRCA Mutation Detected (PHASE3)
- A Study of Alpelisib and Fulvestrant to Treat Endometrial Cancer (PHASE2)
- First-in-Human Study of STX-478 as Monotherapy and in Combination With Other Antineoplastic Agents in Participants With Advanced Solid Tumors (PHASE1,PHASE2)
- EPIK-P4: A Phase II Single-arm Study to Assess the Efficacy, Safety and Pharmacokinetics of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum (PROS) (PHASE2)
Patents
| Patent | Expiry | Type |
|---|---|---|
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Piqray CI brief — competitive landscape report
- Piqray updates RSS · CI watch RSS
- Novartis portfolio CI