Metabolism

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.

Biophytis launches OBA phase 2 clinical study in obesity with BIO101 (20-hydroxyecdysone)

Retrieved on: 
Wednesday, April 10, 2024
Notifiable disease, Metabolism, Obesity, Euronext Growth, Skeletal muscle, Patient, Nature Biotechnology, Weight loss, Trial of the century, OBA, Facial muscles, Sarcopenia, Therapy, MAS, Pharmaceutical industry

These benefical effects of BIO101 (20-hydroxyecdysone) may translate into improved mobility and muscle strength in obese sarcopenic patients, as suggested in the SARA-INT phase 2 study.

Key Points: 
  • These benefical effects of BIO101 (20-hydroxyecdysone) may translate into improved mobility and muscle strength in obese sarcopenic patients, as suggested in the SARA-INT phase 2 study.
  • We believe that our leadership in developing drugs for muscular diseases and promising results obtained in obesity will be a strong accelerator of the OBA clinical plan."
  • The OBA Phase 2 clinical study is expected to start mid 2024, upon regulatory approvals, with first patients expected to be treated in the second half of 2024.
  • Further information on the OBA program and the clinical study is expected to be provided through the coming weeks.

EQS-News: G.ST Antivirals reports start of Phase II trial and announces appointment of Ronald Bruce Turner as new Chief Medical Officer

Retrieved on: 
Wednesday, April 10, 2024
JAMA, Recreational vehicle, Metabolism, CHDR, Entrepreneurship, Dean (education), Viral, Reproduction, University, Head, Volunteering, MD, Urinary tract infection, Infection, Medical school, Clinical trial, Pharmacokinetics, Southern Illinois University, Division, Virus, CMO, University School, Glycolysis, Pharmaceutical industry

An expert in the clinical research of respiratory viruses, Dr. Turner will oversee the Company’s current clinical trial and drive further clinical development.

Key Points: 
  • An expert in the clinical research of respiratory viruses, Dr. Turner will oversee the Company’s current clinical trial and drive further clinical development.
  • This study aims to assess the effectiveness of 2-DG in preventing illness from rhinoviruses, reducing infection rates, and easing symptom severity.
  • A total of 128 volunteers will participate, receiving up to four daily intranasal doses of either 2-DG or a placebo.
  • 2-DG is a unique and intriguing therapeutic approach to treating viral infections by blocking the virus’ nutrient access,” commented Ronald Bruce Turner, MD, CMO of G.ST Antivirals.

Galectin Therapeutics Reports the Positive Outcome of the Fifth Data and Safety Monitoring Board Meeting for NAVIGATE Phase 2b/3 Study of Belapectin in Patients with Cirrhotic Portal Hypertension Caused by Metabolic Dysfunction-Associated SteatoHepatitis

Retrieved on: 
Tuesday, April 9, 2024
Data monitoring committee, Cirrhosis, Metabolism, Disease, Risk, Patient, Hope, MASH, Final Analysis, Therapy, GALT, Safety, DSMB, Interim, Plasma protein binding, Esophageal varices, Liver, Pharmaceutical industry

The objective of this fifth DSMB meeting was to further review the emerging tolerance and safety profiles of belapectin.

Key Points: 
  • The objective of this fifth DSMB meeting was to further review the emerging tolerance and safety profiles of belapectin.
  • Based on its deliberation, which included an unblinded review of the data collected thus far, the DSMB concluded that NAVIGATE can continue as designed, without modifications.
  • The Phase 2b/3 NAVIGATE study is a global, seamless, adaptive, randomized, placebo-controlled, double-blind trial.
  • Enrolled patients have been randomized to receive every two weeks a blinded infusion of belapectin at 2 mg/kg/LBM or 4 mg/kg/LBM or placebo.

Protara Therapeutics Announces Alignment with FDA on Registrational Path Forward for IV Choline Chloride in Patients Dependent on Parenteral Nutrition

Retrieved on: 
Friday, April 5, 2024
Choline, Plasma, Liver, Nutritional science, Route of administration, Liver disease, Steatosis, Short bowel syndrome, Metabolism, Patient, Choline chloride, Cancer, ESPEN, Parenteral nutrition, Adolescence, Liver injury, Hepatology, Tubing (recreation), Fibrosis, Guideline, Society, Therapy, Copenhagen University Hospital, Safety, Pharmacokinetics, PN, FDA, ASPEN, Rigshospitalet, Food, Feeding tube, Extractive metallurgy

IV Choline Chloride has the potential to become the first FDA-approved IV formulation of choline for the 40,000 PN patients in the U.S.

Key Points: 
  • IV Choline Chloride has the potential to become the first FDA-approved IV formulation of choline for the 40,000 PN patients in the U.S.
  • The Company plans to advance the development of IV Choline Chloride as a source of choline for adult and adolescent patients on long-term PN.
  • The FDA has granted IV Choline Chloride Orphan Drug Designation for the prevention of choline deficiency in PN patients.
  • “We look forward to advancing the clinical development of IV Choline Chloride, which we believe has the potential to become the first FDA approved IV choline therapy for patients dependent on PN.

BetterLife Announces Closing of $1.168 Million Convertible Debentures to Further Advance IND-enabling Studies of its Non-hallucinogenic LSD-based Drug Candidate BETR-001

Retrieved on: 
Wednesday, April 3, 2024
GAD, Metabolism, MDD, LSD, Patient, Research, Lysergic acid diethylamide, DSM-IV codes, IND, Hallucination, Safety, GLP, Multilateral, Medical device

The IND-enabling cardio-pulmonary GLP studies have been completed, and the metabolism and genotoxicity GLP studies are to be initiated shortly.

Key Points: 
  • The IND-enabling cardio-pulmonary GLP studies have been completed, and the metabolism and genotoxicity GLP studies are to be initiated shortly.
  • Dr. Ahmad Doroudian, CEO of BetterLife, commented, “We are very encouraged with FDA’s recent decision to grant breakthrough therapy designation to LSD for generalized anxiety disorder (GAD) patients.
  • In simple words, we believe BETR-001 will deliver all the therapeutic benefits of LSD without its side effects.”
    BetterLife issued a total of $1.168 million 10% convertible debentures (the “Convertible Debentures”).
  • $800,000 of the Convertible Debentures mature on March 27, 2026 and $368,000 mature on April 1, 2026.

Surrozen Announces Safety, Pharmacodynamic and Liver Function Data for SZN-043

Retrieved on: 
Monday, April 1, 2024
MELD, Metabolism, Regeneration, Dicarboxylic acid, Survival, Liver disease, PD, Safety, Patient, History, Alkaline phosphatase, CYP1A2, Crosstalk (biology), ASGR1, Pharmacokinetics, Therapy, ALP, Pharmaceutical industry

SOUTH SAN FRANCISCO, Calif., April 01, 2024 (GLOBE NEWSWIRE) -- Surrozen, Inc. (“Surrozen” or the “Company”) (Nasdaq: SRZN), a company pioneering targeted therapeutics that selectively activate the Wnt Pathway for tissue repair and regeneration, today provided an update on the Phase 1a clinical trial of SZN-043 in healthy volunteers and patients with cirrhosis.   The Phase 1a study was completed in February 2024. SZN-043 demonstrated acceptable safety and tolerability in all subjects, with evidence of target engagement, Wnt signal activation and effects on liver function.   The observed safety and pharmacodynamic activity were the basis for the Company’s previous announcement that it planned to initiate enrollment in the Phase 1b study in alcohol-associated hepatitis.

Key Points: 
  • SZN-043 demonstrated acceptable safety and tolerability in all subjects, with evidence of target engagement, Wnt signal activation and effects on liver function.
  • The observed safety and pharmacodynamic activity were the basis for the Company’s previous announcement that it planned to initiate enrollment in the Phase 1b study in alcohol-associated hepatitis.
  • The randomized, placebo-controlled Phase 1a trial enrolled a total of 48 subjects, including 40 healthy volunteers and 8 patients with cirrhosis and a history of liver disease.
  • Cirrhotic patients also showed evidence of liver function effects after treatment with SZN-043 as measured by HepQuant which is a test that measures cholate clearance, a liver specific function that quantifies liver function.

Galectin Therapeutics Reports 2023 Financial Results and Provides Business Update

Retrieved on: 
Friday, March 29, 2024
Research, Data monitoring committee, Cirrhosis, Disease, Metabolism, AASLD, Safety, Patient, Food and Drug Administration, Liver, Mortality, Liver transplantation, Hope, Hepatology, Fibrosis, Clinical trial, Annual report, Therapy, GALT, Pharmacokinetics, Data, DSMB, NASH, Food, Esophageal varices

NORCROSS, Ga., March 29, 2024 (GLOBE NEWSWIRE) -- Galectin Therapeutics, Inc. (NASDAQ: GALT), the leading developer of therapeutics that target galectin proteins, today reported financial results and provided a business update for the year ended December 31, 2023.

Key Points: 
  • NORCROSS, Ga., March 29, 2024 (GLOBE NEWSWIRE) -- Galectin Therapeutics, Inc. (NASDAQ: GALT), the leading developer of therapeutics that target galectin proteins, today reported financial results and provided a business update for the year ended December 31, 2023.
  • Joel Lewis, Chief Executive Officer and President of Galectin Therapeutics, said “We have been focused on advancing our Metabolic Dysfunction-Associated Steatohepatitis (MASH, formerly known as NASH) cirrhosis program.
  • Dr. Jamil’s extensive experience in advanced liver disease, specifically for late-stage cirrhotic patients, is a tremendous asset to our program.
  • These results are included in the Company's Annual Report on Form 10-K, which has been filed with the U.S. Securities and Exchange Commission and is available at www.sec.gov .

Amylyx Pharmaceuticals to Host Virtual Webcast to Discuss Interim Data from Phase 2 HELIOS Study of AMX0035 in Wolfram Syndrome on April 10, 2024

Retrieved on: 
Monday, April 8, 2024
Research, Neurology, Diabetes, Clinical Trials, Biotechnology, Health, Pharmaceutical, Optical, Science, Wolfram syndrome, Medication, Metabolism, Patient, AMX0035, Samuel, Immunology, Doctor of Philosophy, MD, Trial of the century, Amylyx Pharmaceuticals, Division, Disorder, Pathology, Pharmaceutical industry

A live webcast of the presentation can be accessed under “Events and Presentations” in the Investor section of the Company’s website, https://investors.amylyx.com/news-events/events , and will be available for replay for 90 days following the event.

Key Points: 
  • A live webcast of the presentation can be accessed under “Events and Presentations” in the Investor section of the Company’s website, https://investors.amylyx.com/news-events/events , and will be available for replay for 90 days following the event.
  • Dr. Fumihiko Urano is a physician and medical researcher specializing in Wolfram syndrome.
  • Dr. Urano is a driving force in the study of Wolfram syndrome and related disorders, including WFS1-related disorders/Wolfram-like disorders.
  • As director of the Wolfram Syndrome Clinic and the Wolfram Syndrome International Registry & Clinical Study at Washington University, Dr. Urano treats patients with Wolfram syndrome and related disorders, and leads basic science, clinical, translational, and interventional studies of Wolfram syndrome and related disorders.

L-Nutra Reveals World's First Patent-Pending Protein Formula for Healthy Aging to Pioneer Nutrition-Focused Health Solutions

Retrieved on: 
Tuesday, April 9, 2024
Dicarboxylic acid, Health, Meat, Metabolism, Vitamin, Longevity, FMD, Facial muscles, Poultry, Environment, Rejuvenation, Lactose, Insulin, Research, GMO, Doctor of Philosophy, MD, Mineral, Life, Fruit, Ageing, Dietary supplement

LOS ANGELES, April 9, 2024 /PRNewswire/ -- In a remarkable leap forward for nutrition and healthy aging, L-Nutra , the leading nutrition technology company, proudly introduces the world's first patent-pending protein designed to support healthy aging – L-Protein .

Key Points: 
  • LOS ANGELES, April 9, 2024 /PRNewswire/ -- In a remarkable leap forward for nutrition and healthy aging, L-Nutra , the leading nutrition technology company, proudly introduces the world's first patent-pending protein designed to support healthy aging – L-Protein .
  • This first-of-its-kind protein formula was developed by L-Nutra to support muscle function and healthspan.
  • Similarly, inadequate protein intake, as demonstrated in some strict vegan diets, does not produce enough of the necessary growth factors for optimal muscle support and healthy aging.
  • L-Protein is another nutritional milestone to complement the healthy aging benefits of the Prolon Fasting Mimicking Diet (FMD) , L-Nutra's flagship program.