Metabolite

Draft revised Heads of Medicines Agency / European Medicines Agency guidance document on the identification of personal data and commercially confidential information within the structure of the marketing authorisation application dossier

Retrieved on: 
Thursday, April 18, 2024
Steps, Union, Patient, CTD, Syndrome, CCI, Local, Disclosure, Toxicity, Process validation, MAH, Clinical trial, IP, RMP, Pharmacovigilance, Cell, Legislation, Annex, Trial of the century, Escherichia coli, Safety, Pediatrics, INTRODUCTION, Documentation, Prevalence, Vital signs, Tablet, Design, Transparency, Conclusion, Pip, Analysis, European Parliament, INN, Record, Quality, Generic, Biology, CMO, Genotoxicity, Composition, CTIS, Uncontrolled, Health care, European Medicines Agency, Prejudice, Committee, Policy, HCP, Animal, Characterization, Cell bank, Fertility, IRB, CMOS, Risk management, Private law, European Pharmacopoeia, Telephone, Research, Good, Data Protection Directive, Ampere-hour, IEC, QP, Human, Personal data, Labelling, Bibliography, Figure, MAA, R4, Institutional review board, Elucidation, Marketing, M4, ChromeOS, Contract research organization, Mental, Impairment, Toxicokinetics, NCA, Independent, Metabolite, Drug, Risk, Metabolism, GMO, Organ, EMA, Common Technical Document, General Data Protection Regulation, Confidentiality, PPD, PI, Language, DRUG, Privacy, Result, Claimed, Medication, Comparison, Ethics, Drive, PD, Narrative, EEA, Developmental toxicity, Saccharomyces cerevisiae, Pharmacopoeia, PIP, MCB, HMA, Physical chemistry, Midol, Particle size, Council, GCP, European Economic Area, Draft, Fermentation, Overview, Justification, Control, Dicarboxylic acid, Pharmacology, WCB, Expert, Immunogenicity, Data, Study, Publication, European, ICH, Element, Analytical procedures (finance auditing), Name, Common, Guideline, Exceptional circumstances, ID, Liver, Chin Na, Toxicology, Protein primary structure, Immunosuppressive drug, Vaccine

See websites for contact details

Key Points: 
    • See websites for contact details
      Heads of Medicines Agencies www.hma.eu
      European Medicines Agency www.ema.europa.eu

      11

      Table of contents

      12

      Abbreviations .............................................................................................. 3

      13

      Definitions ................................................................................................... 4

      14

      1.

    • redaction, masking,

      68

      hiding) in such a manner that the recipient can no longer attribute the resulting information to a data

      69

      subject and make it identifiable.

    • 81

      Contract Manufacturing Organisation (CMO): shall mean an arrangement under which a

      82

      manufacturer provides upstream manufacturing services under contract on behalf of third-party

      83

      pharmaceutical companies.

    • 94

      Protected Personal Data (PPD): shall mean any personal data which should be protected from

      95

      disclosure.

    • ?Finalised? shall mean that the marketing

      102

      authorisation (MA) has been granted or refused or that the MAA has been withdrawn.

    • The application of the general principles laid down in this guidance is without prejudice to

      106

      national rules on transparency.

    • The guidance should be read in conjunction with the relevant applicable

      107

      legislation and case law on transparency and data protection.

    • 117

      This guidance document is intended to apply to information/documents on medicinal products for

      118

      human use, for which the procedure has been finalised under the national, mutual recognition,

      119

      decentralised and centralised procedures.

    • Third

      124

      parties shall be informed or consulted as needed depending on respective national and European legal

      125

      frameworks.

    • 140

      In the following sections, the agreed principles on PD and CCI are presented, including guidance on

      141

      whether such information can be disclosed.

    • EMA/131365/2024

      Page 5/50

      142

      Any information identified as PD or CCI must be subject to a preliminary review by the EMA/NCA prior

      143

      to the possible disclosure of the information/documents.

    • Principles on the protection of personal data (PD)

      145

      The protection of PD is enshrined in EU legislation; it is a fundamental right of EU citizens.

    • In

      146

      compliance with the applicable European/national legislation, PD should be anonymised in order to

      147

      avoid the disclosure of the document undermining the privacy and integrity of any individual.

    • EMA/NCA applies a risk-based approach to assess which PD elements are to be

      152

      removed from the information/documents in order to limit the risk of re-identification.

    • are included in the MAA dossier because they have a legally

      164

      defined role or responsibility and it is in the public interest to disclose this data.

    • 168

      Applicants are advised that non-essential information (e.g., personal address, personal phone number)

      169

      should not be included in the MAA dossier.

    • The

      183

      confidentiality of records that could identify subjects should be protected, respecting the privacy and

      184

      confidentiality rules in accordance with the applicable regulatory requirement(s).

    • 185

      The applicant remains responsible for compliance with the relevant legislation in cases where such data

      186

      is inadvertently included in the MAA dossier.

    • 188

      EMA/NCA applies a risk-based approach to assess which personal data elements need to be removed

      189

      from the information/documents in order to limit the risk of re-identification.

    • 194

      EMA/NCA applies a risk-based approach to assess which personal data elements need to be removed

      195

      from the information/documents in order to limit the risk of re-identification.

    • 205

      Any proposal to consider information as commercially confidential should be properly justified by the

      206

      owner of the information.

    • In this respect, any reference(s) to the risk of that interest being

      209

      undermined should be foreseeable and not purely hypothetical.

    • 210

      Information that is already in the public domain is not considered to be commercially confidential.

    • Information on the Quality and Manufacturing of medicines

      226

      A general principle regarding quality and manufacturing information is that detailed information could

      227

      be considered commercially confidential but general information should be disclosed.

    • 234

      In general, and if not in the public domain, the names of manufacturers or suppliers of the active

      235

      substance or the excipients are considered commercially confidential.

    • 248

      A general description of the type of test methods used and the appropriateness of the specification is

      249

      not commercially confidential.

    • General information on the fermentation and purification process

      259

      is not commercially confidential, although details including operating parameters and specific material

      260

      requirements are commercially confidential.

    • 273

      A general description of the type of test methods used and the appropriateness of the specification is

      274

      not commercially confidential.

    • In general, the data included in clinical trial study reports is considered to be data that can be

      283

      disclosed once PD has been anonymised.

    • 338

      In each module, a non-exhaustive list of information that may be considered protected personal data (PPD) or commercially confidential information

      332
      333

      339

      (CCI) is included.

    • ?

      Direct contact details such as telephone

      Therefore, please refer to the appropriate sub-

      number, fax number, email, postal address,

      modules hereafter for guidance.

    • ?

      Information that may reveal strategic
      (contractual) agreements

      ?

      Any quality information on the clinical batches

      principal investigator

      that might be included here (such as e.g.

    • ?

      Information that may reveal strategic
      (contractual) agreements

      principal investigator

      Study Reports
      5.3.3.3

      as the evaluation of new formulation, innovative

      number, fax number, email, postal

      Paediatric Development Plan (PIP), etc.

    • This may include taking into

      More Than One Study
      5.3.5.4

      Other Clinical Study Reports

      5.3.6

      Reports of Post-Marketing
      Experience

      5.3.7

      Direct identifiers such as name,
      signature, contact details, etc.

Groundbreaking New Blood Test Offers Early Pancreatic Cancer Diagnosis Study Says

Retrieved on: 
Thursday, February 29, 2024
Neoplasm, Metabolism, Nagourney, Lipid, Dicarboxylic acid, Research, Pancreatic cancer, Blood, Metabolomics, Sugar, Proteomics, Survival, Breast, Metabolite, Mass spectrometry, Triglyceride, Death, Cancer, Patient, Protein, Disease, Genomics, Glucose, Plasma, Science, DNA, Fluid mechanics, Medical imaging

LONG BEACH, Calif., Feb. 29, 2024 /PRNewswire/ -- As reported in today's issue of the journal Metabolites, researchers at Metabolomycs, a California-based biotechnology company, for the first time applied targeted mass spectrometry to show that pancreatic cancer arises through changes in cellular metabolism and that these changes can be measured with a simple blood test.

Key Points: 
  • "Pancreatic cancer is the third leading cause of death from cancer largely because it is rarely caught early," said Dr. Robert Nagourney, senior author.
  • Using Mass Spectrometry to measure minute concentrations of bio-chemicals in the plasma, the technique identified pancreatic cancer in the blood with near perfect accuracy.
  • The results provide a "cancer signature" that distinguishes patients with pancreatic cancer from normal individuals and can distinguish pancreatic cancer from other forms of cancer, like breast and ovarian.
  • Following Genomics, the study of DNA and Proteomics, the study of cell proteins, metabolomics, is the new "omic" to watch.

Sai Life Sciences augments DMPK capabilities to ace large-scale collaborations

Retrieved on: 
Thursday, February 1, 2024
Met, Pharmacokinetics, Growth, Peptide, Absorption, Pharmacology, Immunohistochemistry, Safety, IDD, TK, Appetite, Drug discovery, Proteolysis targeting chimera, DMPK, Automation, ADME, Metabolite, Metabolism, Toxicology, Development, Biology

HYDERABAD, India, Feb. 01, 2024 (GLOBE NEWSWIRE) -- To offer global big pharma clients cutting-edge, end-to-end drug metabolism and pharmacokinetics (DMPK) services, Sai Life Sciences, a global Contract Research, Development & Manufacturing Organization (CRO-CDMO) , has expanded its DMPK capabilities .

Key Points: 
  • HYDERABAD, India, Feb. 01, 2024 (GLOBE NEWSWIRE) -- To offer global big pharma clients cutting-edge, end-to-end drug metabolism and pharmacokinetics (DMPK) services, Sai Life Sciences, a global Contract Research, Development & Manufacturing Organization (CRO-CDMO) , has expanded its DMPK capabilities .
  • Making the announcement, Krishna Kanumuri, CEO & Managing Director, Sai Life Sciences, said, “The focus of innovator companies on diversifying their supply chains bodes well for Indian CROs-CDMOs like ours.
  • Sauri Gudlavalleti, Chief Operating Officer said, “The latest expansion of DMPK capabilities augments our integrated drug discovery (IDD) offerings.
  • Sai Life Sciences also has plans to invest in niche areas such as peptides, oligos and large molecules to strengthen its discovery capabilities.

TransCode Therapeutics Reports Third Quarter 2023 Results; Provides Business Update

Retrieved on: 
Tuesday, November 14, 2023
Magnetic resonance imaging, Therapy, Transcoding, Cancer, Glioblastoma, IND, Bank statement, Exercise, Tissue, GBM, TTX, Breast cancer, Protein, Mouse, Patient, DMPK, Metastasis, Research, FDA, Radioactive decay, Neoplasm, Investigational New Drug, Blood, CRISPR, Trial of the century, FDG, MRI, PET, Recurrence, Real-time, Cell, RNA, Metabolite, Pharmaceutical industry, Medical imaging, Vaccine, Cryptocurrency, Pharmacokinetics

BOSTON, Nov. 14, 2023 (GLOBE NEWSWIRE) -- TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology company committed to more effectively treating cancer using RNA therapeutics, today reported financial results for the third quarter ended September 30, 2023, and recent business progress.

Key Points: 
  • BOSTON, Nov. 14, 2023 (GLOBE NEWSWIRE) -- TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology company committed to more effectively treating cancer using RNA therapeutics, today reported financial results for the third quarter ended September 30, 2023, and recent business progress.
  • TransCode co-founder and Chief Technology Officer, Dr. Zdravka Medarova, commented, “We also announced encouraging preclinical data in glioblastoma multiforme (GBM) with our lead candidate.
  • Research and development expense was approximately $3.3 million in the third quarter of 2023, compared to approximately $3.0 million in the third quarter of 2022.
  • General and administrative expense was approximately $2.0 million in the third quarter of 2023, compared to approximately $1.9 million in the third quarter of 2022.

TransCode Therapeutics Announces Preliminary Clinical Results in First Patient in Phase 0 Clinical Study with Lead Therapeutic Candidate, TTX-MC138

Retrieved on: 
Tuesday, October 24, 2023
Massachusetts General Hospital, Anatomy, Trial of the century, IND, Molecular biology, Therapy, Physiology, RNA, Human, Radioactive decay, Liver, Doctor of Philosophy, Metastasis, PET, DMPK, Cancer, Blood, Metabolite, Transcoding, FDG, Patient, Hospital, NHP, Targeted therapy, Medical imaging, Pharmaceutical industry, Pharmacokinetics, MD

In addition, radiolabeled TTX-MC138 had pharmacokinetic behavior consistent with that expected based on non-clinical IND-enabling studies.

Key Points: 
  • In addition, radiolabeled TTX-MC138 had pharmacokinetic behavior consistent with that expected based on non-clinical IND-enabling studies.
  • Complete analysis of data from this first patient is in process and will be included in the final report for all patients enrolled in the study.
  • The trial is intended to yield important data regarding TTX-MC138 delivery to clinical metastases that could inform dose selection and frequency, for further clinical development.
  • “Our Phase 0 trial involves a single microdose of radiolabeled TTX-MC138 followed by noninvasive PET-MRI imaging and metabolite analysis.

Olaris, Inc. to present data on metabolomic changes observed during DCD heart perfusion prior to successful transplant at the American Transplant Congress 2023 Meeting

Retrieved on: 
Monday, June 5, 2023
Heart, Surgeon, MGH, Surgery, Perfusion, DRS, Research, Biomarker, Ischemia, Patient, DCD, Machine learning, Metabolomics, Medicine, Annual general meeting, Reperfusion, Hospital, ATC, Transplant, Massachusetts General Hospital, Metabolite, Heart transplantation, Dentistry, Medical device

FRAMINGHAM, Mass., June 5, 2023 /PRNewswire-PRWeb/ -- Olaris, Inc., a precision medicine company leveraging metabolomics and machine learning to discover and develop its myOLARIS™ diagnostics products, is pleased to announce that the company's study on the metabolic changes occurring in donor hearts after Donation after Circulatory Death (DCD) heart transplant has been selected for a poster presentation on June 5th at the upcoming American Transplant Congress (ATC) 2023 Annual Meeting, to be held June 3rd-7th in San Diego, CA.

Key Points: 
  • Metabolite reprogramming observed in donor hearts after circulatory death and prior to transplant suggests exciting possibility for improved biomarkers to assess DCD heart quality.
  • The ATC 2023 presentation will describe data from a multi-year collaboration between Olaris and an elite team of heart transplant surgeons and researchers at Massachusetts General Hospital, including Drs.
  • The collaboration was formed with the goal of identifying biomarkers of heart damage due to ischemia and reperfusion during DCD heart transplant.
  • They found that several metabolites were consistently altered over time, reflecting metabolic reprogramming in the DCD hearts during machine perfusion.

Here's how a new AI tool may predict early signs of Parkinson's disease

Retrieved on: 
Tuesday, May 9, 2023
Delayed onset muscle soreness, Food, Optimism, Depression, Cell death, Prevalence, Methamphetamine, Face, Friends, UNSW, ACS Central Science, Fox, Method, Metabolite, The Michael J. Fox Foundation, EPIC, Breast, Research, Brain, DSM-IV codes, Boston University, Future, Dementia, European Prospective Investigation into Cancer and Nutrition, Parkinson's disease, Oxidative stress, ACS, Risk, Blood, Artificial intelligence, Diagnosis, AI, Family, Disease, Film industry, Medical device, Pharmaceutical industry, Health insurance, Dietary supplement, Frankincense, Cancer

He was just 29 years old and at the height of Hollywood fame, a year after the release of the blockbuster Back to the Future III.

Key Points: 
  • He was just 29 years old and at the height of Hollywood fame, a year after the release of the blockbuster Back to the Future III.
  • Parkinson’s is a debilitating neurological disease characterised by motor symptoms including slow movement, body tremors, muscle stiffness, and reduced balance.
  • While this AI tool showed promise for accurate early diagnosis, it also revealed chemicals that were strongly linked to a correct prediction.

More common than ever

    • For people over 50, the chance of developing Parkinson’s is higher than many cancers including breast, colorectal, ovarian and pancreatic cancer.
    • Read more:
      Drooling is a common symptom of Parkinson's.

Our research

    • For this study, we looked at the Spanish European Prospective Investigation into Cancer and Nutrition (EPIC) study which involved over 41,000 participants.
    • To train the AI model we used a subset of data consisting of a random selection of 39 participants who later developed Parkinson’s.
    • The AI tool was given blood data from participants, all of whom were healthy at the time of blood donation.

Key metabolites

    • Metabolites are chemicals produced or used as the body digests and breaks down things like food, drugs, and other substances from environmental exposure.
    • Our bodies can contain thousands of metabolites and their concentrations can differ significantly between healthy people and those affected by disease.
    • Our research identified a chemical, likely a triterpenoid, as a key metabolite that could prevent Parkinson’s disease.

A high financial and personal burden

    • The burden of living with the disease can be intolerable.
    • Fox acknowledges the disease can be a “nightmare” and a “living hell”, but he has also found that “with gratitude, optimism is sustainable”.
    • She is supported by a Scientia PhD and RTP scholarship from the University of New South Wales.

Windtree Therapeutics Reports Fourth Quarter and Year-End 2022 Financial Results and Provides Key Business Updates

Retrieved on: 
Monday, April 3, 2023
Exercise, SBP, AHF, Cardiogenic shock, Heart failure, Efficacy, WINT, Research, Experiment, American Society for Pharmacology and Experimental Therapeutics, Biomarker, Arrhythmia, Doctor of Philosophy, Istaroxime, Science, CCU, THT, Heart, Journal, Publication, Patent, MD, Patient, Annual report, Risk, Average cost, Toxicology, Security (finance), ICU, Therapy, Metabolite, Safety, Seismology, Conference, FESC, Pharmaceutical industry, Medical device, Cryptocurrency, Life insurance, Windtree Therapeutics, Pharmacology

WARRINGTON, Pa., April 03, 2023 (GLOBE NEWSWIRE) -- Windtree Therapeutics, Inc. (NasdaqCM: WINT), a biotechnology company focused on advancing multiple late-stage interventions for cardiovascular disorders, today reported financial results for the fourth quarter and fiscal year ended December 31, 2022 and provided key business updates.

Key Points: 
  • WARRINGTON, Pa., April 03, 2023 (GLOBE NEWSWIRE) -- Windtree Therapeutics, Inc. (NasdaqCM: WINT), a biotechnology company focused on advancing multiple late-stage interventions for cardiovascular disorders, today reported financial results for the fourth quarter and fiscal year ended December 31, 2022 and provided key business updates.
  • Research and development expenses were $1.2 million for the fourth quarter of 2022, compared to $4.5 million for the fourth quarter of 2021.
  • General and administrative expenses for the fourth quarter of 2022 were $2.2 million, compared to $3.0 million for the fourth quarter of 2021.
  • For the fourth quarter ended December 31, 2022, the Company reported an operating loss of $10.8 million, compared to an operating loss of $14.7 million in the fourth quarter of 2021.

Windtree Announces Publication on its SERCA2a Activator Drug Candidate for Chronic and Acute Heart Failure

Retrieved on: 
Tuesday, February 28, 2023
Windtree Therapeutics, Metabolite, Therapy, Arrhythmia, Journal of Pharmacology and Experimental Therapeutics, CMO, Istaroxime, Acute decompensated heart failure, Treatment, Journal, Heart failure, Probability, Activity, Relaxation, Human, Calcium, Public health genomics, Cardiogenic shock, Experiment, Pharmaceutical industry, Medical device, Pharmacology

The publication emphasizes the interest in SERCA2a as a target in treating heart failure because it is instrumental in calcium management, critically important in heart muscle contraction and relaxation.

Key Points: 
  • The publication emphasizes the interest in SERCA2a as a target in treating heart failure because it is instrumental in calcium management, critically important in heart muscle contraction and relaxation.
  • Our SERCA2a activators uniquely interfere with phospholamban, a protein that inhibits SERCA2a function and does so excessively in heart failure.
  • The preclinical data discussed in the paper provides rationale for selective targeting of SERCA2A in the treatment of heart failure.
  • “Windtree’s SERCA2a activator drug candidates could represent a promising IV or oral approach to treat heart failure,” said Steve Simonson, CMO of Windtree Therapeutics.

Journal of Psychoactive Drugs Publishes Peer Reviewed Ketamine Research Foundation Study on the Duration of the Presence of Ketamine in Breast Milk

Retrieved on: 
Monday, August 8, 2022
Research, Philanthropy, Pharmaceutical, Consumer, Mental Health, Foundation, Parenting, Clinical Trials, Science, Baby, Maternity, Women, FDA, Health, Woman, Psychoactive drug, Depression, Psychotherapy, Breast milk, CEO, Research, Substance abuse, Metabolite, Journal of Psychoactive Drugs, Excoriation disorder, Infant, Journal, Mother, Pharmacokinetics, Medicine, Postpartum period, Crime, Birth control, Pharmaceutical industry, Ketamine

The research provides the first quantification of Ketamine and its major metabolites in human breast milk.

Key Points: 
  • The research provides the first quantification of Ketamine and its major metabolites in human breast milk.
  • The Ketamine Research Foundation believes this study will help open the door for further explorations of ketamine in the treatment of emotional disorders of the postpartum period, said Dr. Philip Wolfson, CEO of The Ketamine Research Foundation and a widely recognized leader in the development of Ketamine Assisted Psychotherapy.
  • The Ketamine Research Foundation is in the process of developing its protocol for postpartum depression and expects to launch it in the fall of 2022.
  • The Ketamine Research Foundation stands at the apex of Ketamine research and ketamine psychotherapy.