Clinical Trials

Alpelisib (BYL719) is evaluated across numerous clinical trials for hormone receptor-positive, HER2-negative metastatic breast cancer, triple-negative breast cancer, PIK3CA-related overgrowth spectrum disorders, and vascular and lymphatic malformations. Sponsored by pharmaceutical entities such as Novartis Pharmaceuticals alongside academic institutions including Columbia University and the Murdoch Childrens Research Institute, these studies span Phase 1 through Phase 4 protocols across recruiting, completed, and terminated statuses. Representative research includes Phase 2 neoadjuvant combination therapy studies and Phase 2/3 trials targeting lymphatic malformations.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT07679269 NOT_YET_RECRUITING
PIK3CA-Related Overgrowth Spectrum (PROS); Breast Neoplasms (Including Advanced or Metastatic Breast Cancer With PIK3CA Mutation)
Novartis Pharmaceuticals
 2027-01-01 PHASE2
NCT05933395 RECRUITING
Advanced Breast Cancer
Dartmouth-Hitchcock Medical Center
 2024-09-23 PHASE2
NCT05564377 RECRUITING
Advanced Malignant Solid Neoplasm; Anatomic Stage III Breast Cancer AJCC v8; Anatomic Stage IV Breast Cancer AJCC v8; Locally Advanced Malignant Solid Neoplasm; Malignant Female Reproductive System Neoplasm; Metastatic HER2-Negative Breast Carcinoma; Metastatic Malignant Solid Neoplasm; Recurrent Endometrial Carcinoma; Recurrent Fallopian Tube Carcinoma; Recurrent Malignant Female Reproductive System Neoplasm; Recurrent Malignant Solid Neoplasm; Recurrent Ovarian Carcinoma; Recurrent Primary Peritoneal Carcinoma; Unresectable HER2-Negative Breast Carcinoma; Unresectable Malignant Solid Neoplasm
National Cancer Institute (NCI)
 2023-04-07 PHASE2
NCT07573696 NOT_YET_RECRUITING
Nonsmall Cell Lung Cancer Stage III
Wen-zhao ZHONG
 2026-06-15 PHASE2
NCT07543822 ACTIVE_NOT_RECRUITING
Vascular Anomalies; Vascular Anomaly; TIE2/PIK3CA Pathway Driven Vascular Anomalies
Children's Hospital of Philadelphia
 2025-08-27 PHASE2
NCT07778524 RECRUITING
Insulin Resistance; Type 2 Diabetes; Obesity & Overweight; Healthy Adult Participants
Columbia University
 2026-09-02 PHASE1
NCT06997588 RECRUITING
PIK3CA-related Overgrowth Spectrum (PROS)
Novartis Pharmaceuticals
 2025-10-09 PHASE2
NCT06145308 RECRUITING
Salivary Gland Carcinoma; Precision Therapy
Peking Union Medical College
 2023-08-15 PHASE2
NCT05625087 ACTIVE_NOT_RECRUITING
Breast Cancer Stage IV
UNICANCER
 2023-10-19 PHASE2
NCT05948943 RECRUITING
Lymphatic Malformations
Novartis Pharmaceuticals
 2023-11-24 PHASE2; PHASE3
NCT05154487 ACTIVE_NOT_RECRUITING
Endometroid Endometrial Cancer
GOG Foundation
 2024-09-11 PHASE2
NCT05472220 WITHDRAWN
Solid Tumors, Adult; PIK3CA Mutation-Related Tumors; PIK3CA Mutation; Human Papillomavirus-Related Carcinoma; HPV-Related Squamous Cell Carcinoma; Human Papillomavirus-Related Squamous Cell Carcinoma; Locally Advanced Malignant Solid Neoplasm; Metastatic Malignant Solid Neoplasm
Pamela Munster
 2022-11-01 PHASE1
NCT05508906 RECRUITING
Metastatic Breast Cancer; ER-positive Breast Cancer; HER2-negative Breast Cancer; Breast Cancer; Locally Advanced Breast Cancer
Olema Pharmaceuticals, Inc.
 2022-08-31 PHASE1
NCT05090358 ACTIVE_NOT_RECRUITING
Breast Cancer; Breast Cancer Stage IV; Metastatic Breast Cancer
Memorial Sloan Kettering Cancer Center
 2021-10-08 PHASE2
NCT02925234 RECRUITING
Cancer; Tumors; Neoplasm; Neoplasia
The Netherlands Cancer Institute
 2016-08 PHASE2
NCT04980833 ACTIVE_NOT_RECRUITING
PIK3CA-related Overgrowth Spectrum (PROS)
Novartis Pharmaceuticals
 2022-01-27 PHASE2
NCT06545682 RECRUITING
Melanoma (Skin Cancer); Breast Cancer; Brain Metastasases
M.D. Anderson Cancer Center
 2024-10-15 PHASE1
NCT05577754 RECRUITING
Megalencephaly-capillary Malformation Polymicrogyria Syndrome (MCAP)
Centre Hospitalier Universitaire Dijon
 2022-11-28 PHASE2
NCT01872260 ACTIVE_NOT_RECRUITING
Breast Cancer
Novartis Pharmaceuticals
 2013-10-22 PHASE1; PHASE2
NCT04208178 ACTIVE_NOT_RECRUITING
Advanced HER2+Breast Cancer
Novartis Pharmaceuticals
 2020-07-16 PHASE3
NCT04524000 ACTIVE_NOT_RECRUITING
Advanced Breast Cancer
Novartis Pharmaceuticals
 2021-01-08 PHASE2
NCT04544189 ACTIVE_NOT_RECRUITING
Breast Neoplasms
Novartis Pharmaceuticals
 2021-01-20 PHASE2
NCT05038735 ACTIVE_NOT_RECRUITING
Breast Cancer
Novartis Pharmaceuticals
 2021-12-17 PHASE3
NCT05983159 RECRUITING
Slow-Flow Vascular Malformation; Fast-Flow Vascular Malformation; Vascular Malformations; Venous Malformation; Lymphatic Malformation, Low Flow; Lymphatic Malformation; Lymphangioma; Arteriovenous Malformations; Venous Malformation, Low Flow; Cystic Hygroma; Vascular Anomaly; Vascular Anomalies; PI3K Gene Mutation; MAP2K1 Gene Mutation; PIK3CA-related Overgrowth Spectrum; Arteriovenous Malformation (AVM); KRAS G12C; KRAS G12D
Murdoch Childrens Research Institute
 2024-09-13 PHASE2
NCT06739395 RECRUITING
Solid Tumor; Precision Medicine
Tianjin Medical University Second Hospital
 2024-11-01 PHASE2
NCT05753657 RECRUITING
Metastatic Breast Cancer; Hyperinsulinism; Hyperglycemia Drug Induced
Rambam Health Care Campus
 2022-12-25 EARLY_PHASE1
NCT06354088 RECRUITING
Insulin Resistance; Prediabetic State; Overweight and Obesity; Non-Alcoholic Fatty Liver Disease
Columbia University
 2024-04-24 PHASE1
NCT05563220 ACTIVE_NOT_RECRUITING
Breast Cancer; Metastatic Breast Cancer
Stemline Therapeutics, Inc.
 2023-01-24 PHASE1; PHASE2
NCT05660083 RECRUITING
HER2-negative Breast Cancer; Metastatic Breast Cancer; Metaplastic Breast Carcinoma; TNBC - Triple-Negative Breast Cancer
The Methodist Hospital Research Institute
 2023-01-12 PHASE2
NCT05646862 RECRUITING
Breast Cancer
Hoffmann-La Roche
 2023-06-07 PHASE3
NCT05501886 ACTIVE_NOT_RECRUITING
Breast Cancer
Celcuity Inc
 2022-12-08 PHASE3
NCT05063786 ACTIVE_NOT_RECRUITING
Advanced Breast Cancer
Spanish Breast Cancer Research Group
 2021-09-14 PHASE3
NCT05238831 WITHDRAWN
Advanced Breast Carcinoma; Advanced Malignant Solid Neoplasm; Advanced Ovarian Carcinoma; Advanced Pancreatic Carcinoma; Advanced Prostate Carcinoma; Advanced Sarcoma; Anatomic Stage III Breast Cancer AJCC v8; Anatomic Stage IV Breast Cancer AJCC v8; Recurrent Adult Soft Tissue Sarcoma; Recurrent Breast Carcinoma; Recurrent Ovarian Carcinoma; Recurrent Prostate Carcinoma; Stage II Pancreatic Cancer AJCC v8; Stage III Ovarian Cancer AJCC v8; Stage III Pancreatic Cancer AJCC v8; Stage IV Ovarian Cancer AJCC v8; Stage IV Pancreatic Cancer AJCC v8
OHSU Knight Cancer Institute
 2023-01-30 EARLY_PHASE1
NCT05392608 ACTIVE_NOT_RECRUITING
Neoplasm, Breast; Congenital, Familial and Genetic Disorders
Borstkanker Onderzoek Groep
 2022-06-02 PHASE2
NCT05230810 ACTIVE_NOT_RECRUITING
HER2-positive Metastatic Breast Cancer
Criterium, Inc.
 2022-08-25 PHASE1; PHASE2
NCT03631953 UNKNOWN
Meningioma
Assistance Publique Hopitaux De Marseille
 2019-09-30 PHASE1
NCT04762979 ACTIVE_NOT_RECRUITING
Hormone Receptor Positive Breast Carcinoma; HER2-negative Breast Cancer; PIK3CA Mutant Metastatic Breast Cancer
Marina N Sharifi
 2021-02-12 PHASE2
NCT04216472 TERMINATED
Anatomic Stage I Breast Cancer AJCC v8; Anatomic Stage IA Breast Cancer AJCC v8; Anatomic Stage IB Breast Cancer AJCC v8; Anatomic Stage II Breast Cancer AJCC v8; Anatomic Stage IIA Breast Cancer AJCC v8; Anatomic Stage IIB Breast Cancer AJCC v8; Anatomic Stage III Breast Cancer AJCC v8; Anatomic Stage IIIA Breast Cancer AJCC v8; Anatomic Stage IIIB Breast Cancer AJCC v8; Anatomic Stage IIIC Breast Cancer AJCC v8; Prognostic Stage I Breast Cancer AJCC v8; Prognostic Stage IA Breast Cancer AJCC v8; Prognostic Stage IB Breast Cancer AJCC v8; Prognostic Stage II Breast Cancer AJCC v8; Prognostic Stage IIA Breast Cancer AJCC v8; Prognostic Stage IIB Breast Cancer AJCC v8; Prognostic Stage III Breast Cancer AJCC v8; Prognostic Stage IIIA Breast Cancer AJCC v8; Prognostic Stage IIIB Breast Cancer AJCC v8; Prognostic Stage IIIC Breast Cancer AJCC v8; Refractory Breast Carcinoma; Triple-Negative Breast Carcinoma
M.D. Anderson Cancer Center
 2020-07-10 PHASE2
NCT04997902 COMPLETED
HNSCC
Kura Oncology, Inc.
 2021-12-07 PHASE1; PHASE2
NCT05631795 COMPLETED
Advanced Breast Cancer
Novartis Pharmaceuticals
 2022-08-09 PHASE4
NCT05967286 WITHDRAWN
Anatomic Stage III Breast Cancer AJCC v8; Anatomic Stage IV Breast Cancer AJCC v8; Metastatic HER2-Negative Breast Carcinoma; Unresectable HER2-Negative Breast Carcinoma
National Cancer Institute (NCI)
 2023-10-23 PHASE2
NCT05143229 ACTIVE_NOT_RECRUITING
Breast Cancer
University of Kansas Medical Center
 2022-03-28 PHASE1
NCT04591431 UNKNOWN
Breast Cancer; Gastrointestinal Cancer; Non Small Cell Lung Cancer; Other Cancer
Fondazione per la Medicina Personalizzata
 2020-10-07 PHASE2
NCT03284957 TERMINATED
Breast Cancer
Sanofi
 2017-09-20 PHASE1; PHASE2
NCT05966584 TERMINATED
Breast Cancer
Memorial Sloan Kettering Cancer Center
 2023-07-06 PHASE2
NCT05101564 COMPLETED
Breast Cancer; HER2-negative Breast Cancer; ER Positive Breast Cancer
Jennifer Lee Caswell-Jin
 2023-03-20 PHASE2
NCT04589650 ACTIVE_NOT_RECRUITING
PIK3CA-related Overgrowth Spectrum (PROS)
Novartis Pharmaceuticals
 2021-04-19 PHASE2
NCT04753203 UNKNOWN
Metastatic Colorectal Cancer; PIK3CA Gene Mutation
Korean Cancer Study Group
 2021-02-28 PHASE1; PHASE2
NCT05107388 UNKNOWN
Breast Cancer; Diabetes
Centre Hospitalier Universitaire de Besancon
 2021-12
NCT03207529 COMPLETED
Advanced Breast Carcinoma; Anatomic Stage III Breast Cancer AJCC v8; Anatomic Stage IIIA Breast Cancer AJCC v8; Anatomic Stage IIIB Breast Cancer AJCC v8; Anatomic Stage IIIC Breast Cancer AJCC v8; Anatomic Stage IV Breast Cancer AJCC v8; Androgen Receptor Positive; HER2/Neu Negative; Metastatic Breast Carcinoma; Prognostic Stage III Breast Cancer AJCC v8; Prognostic Stage IIIA Breast Cancer AJCC v8; Prognostic Stage IIIB Breast Cancer AJCC v8; Prognostic Stage IIIC Breast Cancer AJCC v8; Prognostic Stage IV Breast Cancer AJCC v8; PTEN Positive; Recurrent Breast Carcinoma; Refractory Breast Carcinoma; Triple-Negative Breast Carcinoma
M.D. Anderson Cancer Center
 2019-06-07 PHASE1
NCT03439046 TERMINATED
Breast Cancer
Novartis Pharmaceuticals
 2018-02-02 PHASE3
NCT05733455 COMPLETED
Insulin Resistance; Hyperinsulinemia; Dyslipidemias
Columbia University
 2023-05-09 PHASE1
NCT05948943 Recruiting
Lymphatic Malformations
Novartis Pharmaceuticals|Novartis
 2023-11-24 Phase 2|Phase 3
NCT04526470 UNKNOWN
Solid Tumor; Stomach Cancer
Seoul National University Bundang Hospital
 2020-09-01 PHASE1; PHASE2
NCT04251533 TERMINATED
Triple Negative Breast Neoplasms
Novartis Pharmaceuticals
 2020-06-08 PHASE3
NCT04899349 TERMINATED
Breast Cancer
Novartis Pharmaceuticals
 2022-04-06 PHASE2
NCT04729387 TERMINATED
Ovarian Cancer
Novartis Pharmaceuticals
 2021-07-22 PHASE3
NCT05660083 Recruiting
HER2-negative Breast Cancer|Metastatic Breast Cancer|Metaplastic Breast Carcinoma|TNBC - Triple-Negative Breast Cancer
The Methodist Hospital Research Institute|Novartis Pharmaceuticals
 2023-01-12 Phase 2
NCT04801966 TERMINATED
Cancer
Peter MacCallum Cancer Centre, Australia
 2021-09-23
NCT05025735 UNKNOWN
Metastatic Breast Cancer; HER2-negative Breast Cancer
Saint Luke's Health System
 2021-08-25 PHASE2
NCT03386162 TERMINATED
Breast Cancer, PI3K, Alpelisib
UNICANCER
 2018-03-15 PHASE2
NCT05195892 COMPLETED
Healthy Volunteers
Novartis Pharmaceuticals
 2022-02-03 PHASE1
NCT04862143 TERMINATED
Advanced Breast Cancer
Novartis Pharmaceuticals
 2022-03-08 PHASE2
NCT05508906 Recruiting
Metastatic Breast Cancer|Advanced Breast Cancer|HR-positive Breast Cancer|HER2-negative Breast Cancer
Olema Pharmaceuticals Inc.|Novartis
 2022-08-31 Phase 1
NCT05230810 Recruiting
HER2-positive Metastatic Breast Cancer
Criterium Inc.|Novartis|Seagen Inc.
 2022-08-25 Phase 1|Phase 2
NCT04300790 COMPLETED
Breast Cancer
MedSIR
 2020-10-23 PHASE2
NCT03601507 TERMINATED
CDKN2A-p16 Positive; HPV Positive Oropharyngeal Squamous Cell Carcinoma; Stage I Oropharyngeal Squamous Cell Carcinoma AJCC V7; Stage II Oropharyngeal Squamous Cell Carcinoma AJCC V7; Stage III Oropharyngeal Squamous Cell Carcinoma AJCC v7; Stage IVA Oropharyngeal Squamous Cell Carcinoma AJCC v7
University of Arizona
 2019-03-11 PHASE2
NCT01870505 COMPLETED
Metastatic or Locally-advanced Unresectable Breast Cancer
Memorial Sloan Kettering Cancer Center
 2013-05 PHASE1
NCT02506556 COMPLETED
Metastatic Breast Cancer
Peter MacCallum Cancer Centre, Australia
 2015-09-07 PHASE2
NCT02282371 COMPLETED
Head and Neck Squamous Cell Cancer
Memorial Sloan Kettering Cancer Center
 2014-10-30 PHASE1
NCT03138070 COMPLETED
Head and Neck Squamous Cell Cancer
London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's
 2017-10-20
NCT02734615 TERMINATED
Advanced or Metastatic ER+ Breast Cancer
Novartis Pharmaceuticals
 2016-06-14 PHASE1
NCT02167854 COMPLETED
Breast Cancer
Memorial Sloan Kettering Cancer Center
 2014-06-16 PHASE1
NCT03056755 COMPLETED
Breast Cancer
Novartis Pharmaceuticals
 2017-08-29 PHASE2
NCT04188548 ACTIVE_NOT_RECRUITING
Breast Cancer; Advanced Breast Cancer; Metastatic Breast Cancer; Endometrial Cancer
Eli Lilly and Company
 2019-12-10 PHASE1
NCT03292250 COMPLETED
HNSCC; Head and Neck Neoplasms
Seoul National University Hospital
 2017-09-10 PHASE2
NCT02276027 COMPLETED
Adenocarcinoma Lung Cancer; Squamous Cell Lung Carcinoma
Novartis Pharmaceuticals
 2015-01-20 PHASE2
NCT02298595 WITHDRAWN
Carcinoma, Squamous; Squamous Cell Carcinoma; Oropharyngeal Neoplasms; Oropharyngeal Cancer
Julie E. Bauman, MD, MPH
 2016-08 PHASE1; PHASE2
NCT01623349 COMPLETED
Ovarian Cancer; Breast Cancer
Dana-Farber Cancer Institute
 2012-09 PHASE1
NCT02145312 UNKNOWN
Recurrent or Metastatic Squamous Cell Carcinoma of Head and Neck
Yonsei University
 2016-10-01 PHASE2
NCT02077933 COMPLETED
Neoplasms; Breast Neoplasms; Kidney Neoplasms; Pancreatic Neuroendocine Neoplasms (pNETs)
Novartis Pharmaceuticals
 2014-05-14 PHASE1
NCT02620839 TERMINATED
Solid Tumors
Pamela Munster
 2016-12-01 PHASE1
NCT02550743 TERMINATED
Rectal Cancer
howard safran
 2016-06-03 PHASE1
NCT01735968 COMPLETED
3rd Line GIST
Novartis Pharmaceuticals
 2013-02-27 PHASE1
NCT02058381 COMPLETED
Pre-menopausal Breast Cancer; PI3K Pathway Inhibition
Novartis Pharmaceuticals
 2014-05-06 PHASE1
NCT02537223 COMPLETED
Squamous Cell Carcinoma of Head and Neck; Locoregionally Advanced
University Health Network, Toronto
 2015-09 PHASE1
NCT02437318 COMPLETED
Breast Cancer
Novartis Pharmaceuticals
 2015-07-23 PHASE3
NCT02088684 COMPLETED
Breast Cancer
Novartis Pharmaceuticals
 2014-05-19 PHASE1
NCT02273219 COMPLETED
Uveal Melanoma
Columbia University
 2014-11-19 PHASE1
NCT02624557 COMPLETED
Hepatic Impairment
Novartis Pharmaceuticals
 2015-12-21 PHASE1
NCT02379247 COMPLETED
Breast Cancer
Priyanka Sharma
 2015-02 PHASE1; PHASE2
NCT01923168 COMPLETED
Breast Cancer
Novartis Pharmaceuticals
 2014-03-11 PHASE2
NCT01300962 COMPLETED
Metastatic Breast Cancer
UNC Lineberger Comprehensive Cancer Center
 2011-08 PHASE1
NCT02155088 COMPLETED
Pancreatic Cancer
H. Lee Moffitt Cancer Center and Research Institute
 2014-10-30 PHASE1
NCT01602315 TERMINATED
Recurrent Head and Neck Squamous Cell Carcinoma; Metastatic Head and Neck Squamous Cell Carcinoma
Novartis Pharmaceuticals
 2012-11-12 PHASE1; PHASE2
NCT01449058 COMPLETED
Advanced and Selected Solid Tumors; AML; High Risk and Very High Risk MDS
Array BioPharma
 2012-03 PHASE1
NCT02051751 COMPLETED
Neoplasms, Breast Neoplasms, Head and Neck Neoplasms
Novartis Pharmaceuticals
 2014-03-05 PHASE1
NCT01928459 COMPLETED
Advanced Solid Tumors; Metastatic Solid Tumors
Novartis Pharmaceuticals
 2013-10 PHASE1
NCT01822613 COMPLETED
Esophageal Squamous Cell Carcinoma
Novartis Pharmaceuticals
 2013-07-26 PHASE1
NCT01387321 COMPLETED
Advanced Solid Tumor
Novartis Pharmaceuticals
 2011-09-22 PHASE1
NCT01719380 COMPLETED
Colorectal Cancer
Pfizer
 2012-11-23 PHASE2
NCT02144038 COMPLETED
Relapsed and Refractory Multiple Myeloma
Novartis Pharmaceuticals
 2014-07-23 PHASE1
NCT02038010 COMPLETED
HER2-positive Breast Cancer; Recurrent Breast Cancer; Stage IIIA Breast Cancer; Stage IIIB Breast Cancer; Stage IIIC Breast Cancer; Stage IV Breast Cancer
Northwestern University
 2014-05-21 PHASE1
NCT01219699 COMPLETED
Advanced Solid Tumors With an Alteration of the PIK3CA Gene; Estrogen Receptor Positive Breast Cancer
Novartis Pharmaceuticals
 2010-10-05 PHASE1
NCT01708161 TERMINATED
PIK3CA Mutated Advanced Solid Tumors; PIK3CA Amplified Advanced Solid Tumors
Novartis Pharmaceuticals
 2012-11-27 PHASE1; PHASE2
NCT01791478 COMPLETED
Estrogen Receptor-positive Breast Cancer; HER2-negative Breast Cancer; Invasive Ductal Breast Carcinoma; Progesterone Receptor-positive Breast Cancer; Recurrent Breast Cancer; Stage IV Breast Cancer
Vanderbilt-Ingram Cancer Center
 2013-04 PHASE1
NCT01613950 COMPLETED
Stomach Neoplasms Esophageal Neoplasms Metastatic Gastric Cancer Mutated PI3KCA Protein Overexpressed HER2 Protein
Novartis Pharmaceuticals
 2012-12 PHASE1

(data from https://clinicaltrials.gov, updated on 2026-07-01)

Check the Alpelisib (BYL719) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Alpelisib selectively binds to and inhibits the catalytic subunit of class I phosphoinositide 3-kinase alpha (PI3Kα), preventing PIP3 generation and suppressing downstream AKT signaling. This signal inhibition impairs cellular proliferation and survival, underlying its therapeutic efficacy in inhibiting tumor growth and structural anomalies in clinical conditions such as PIK3CA-mutated breast cancers and vascular malformations.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.