Trial of the century

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:

Jeudi, avril 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:

Mercredi, avril 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.

Optimi Health Signs Psilocybin Supply Agreement With New Zealand-Based Mātai Medical Research Institute

Retrieved on:

Mardi, avril 9, 2024

Psilocybin, First Nations, Wharerātā, Certificate, Patient, Ethics, FRA, COA, Synthetic, Health Canada, Trial of the century, GMP, Clinical trial, Ground substance, Safety, MDMA, Medsafe, Medicine, Pharmaceutical industry

VANCOUVER, British Columbia, April 09, 2024 (GLOBE NEWSWIRE) -- Optimi Health Corp. (CSE: OPTI) (OTCQX: OPTHF) (FRA: 8BN), a leading Health Canada licensed psychedelics pharmaceutical manufacturer, specializing in controlled substances such as natural psilocybin and MDMA, is proud to announce the signing of an international natural psilocybin supply agreement with New Zealand-Based Mātai Medical Research Institute (Mātai), on behalf of the Tū Wairua Project.

Key Points:

VANCOUVER, British Columbia, April 09, 2024 (GLOBE NEWSWIRE) -- Optimi Health Corp. (CSE: OPTI) (OTCQX: OPTHF) (FRA: 8BN), a leading Health Canada licensed psychedelics pharmaceutical manufacturer, specializing in controlled substances such as natural psilocybin and MDMA, is proud to announce the signing of an international natural psilocybin supply agreement with New Zealand-Based Mātai Medical Research Institute (Mātai), on behalf of the Tū Wairua Project.
This agreement marks Optimi's first supply deal to New Zealand, a significant milestone for the company's global expansion efforts.
Under the terms of the agreement, Optimi will provide Mātai with a quantity of its validated GMP Full Spectrum Natural Psilocybin extract.
Further, the agreement includes tracked stability of the extract, ensuring the highest standards of GMP quality and compliance.

Nuwellis Announces Launch of Ultrafiltration Therapy Using Aquadex Smartflow® System at Henry Ford Health

Retrieved on:

Mardi, avril 9, 2024

Ultrafiltration, Diuretic, Trial of the century, Henry Ford, Patient, Heart failure, The Henry Ford, Pharmaceutical industry

MINNEAPOLIS, April 09, 2024 (GLOBE NEWSWIRE) -- Nuwellis, Inc. (Nasdaq: NUWE) today announced that Detroit, Michigan-based health system, Henry Ford Health, has begun offering ultrafiltration therapy to heart failure patients suffering from fluid overload using the company’s Aquadex SmartFlow system as part of Nuwellis’ pivotal REVERSE-HF clinical study.

Key Points:

MINNEAPOLIS, April 09, 2024 (GLOBE NEWSWIRE) -- Nuwellis, Inc. (Nasdaq: NUWE) today announced that Detroit, Michigan-based health system, Henry Ford Health, has begun offering ultrafiltration therapy to heart failure patients suffering from fluid overload using the company’s Aquadex SmartFlow system as part of Nuwellis’ pivotal REVERSE-HF clinical study.
“We are excited to study the impact of Aquadex SmartFlow therapy on our heart failure patients who are suffering from severe fluid overload,” said Dr. Jennifer Cowger, Section Head of the heart failure program at Henry Ford.
“We are eager to see if ultrafiltration can affect clinical outcomes of our heart failure patients resistant to diuretics.
“We are proud and honored to be working with Henry Ford Health and look forward to partnering with Dr. Cowger and the team of heart failure experts through the REVERSE-HF trial.

Vanqua Bio Announces First Patient Dosed in Phase 1 Clinical Trial Evaluating VQ-101, its Small Molecule GCase Activator for GBA-Parkinson’s Disease and Related Disorders

Retrieved on:

Mardi, avril 9, 2024

Cell, Recycling, PD, Patient, Glucocerebrosidase, Doctor of Philosophy, Alpha-synuclein, Trial of the century, Protein, Neuron, Safety, Pharmacokinetics, Lysosome

CHICAGO, April 09, 2024 (GLOBE NEWSWIRE) -- Vanqua Bio, a clinical-stage biopharmaceutical company dedicated to discovering and developing next-generation medicines for the treatment of neurodegenerative diseases, announced that the first patient has been dosed in a first-in-human Phase 1 clinical study evaluating VQ-101 in healthy individuals and patients with various forms of Parkinson’s disease (PD). VQ-101 is an orally administered brain-penetrant small molecule allosteric activator of the lysosomal enzyme glucocerebrosidase (GCase).

Key Points:

VQ-101 is an orally administered brain-penetrant small molecule allosteric activator of the lysosomal enzyme glucocerebrosidase (GCase).
“VQ-101 demonstrated promising efficacy, safety, pharmacokinetics, and target engagement in preclinical studies.
Mutations in GBA1 are the most common genetic risk factor for PD, representing approximately 10% of patients with PD worldwide.
In developing VQ-101, Vanqua is adopting a precision-medicine approach that focuses initially on patients with GBA-PD, the largest genetically defined segment of PD.

Revolution Medicines Announces Publications on the Discovery and Preclinical Profile of Representative of a New Class of RAS(ON) Multi-Selective Inhibitors Designed to Block Full Spectrum of Oncogenic RAS(ON) Proteins

Retrieved on:

Lundi, avril 8, 2024

NSCLC, Colorectal cancer, CYPA, RAS, Patient, Research, PDAC, Lung cancer, Protein, Trial of the century, GTP, Cancer, CRC, Pharmacology, Ras GTPase, Drug resistance, Pharmaceutical industry

REDWOOD CITY, Calif., April 08, 2024 (GLOBE NEWSWIRE) -- Revolution Medicines, Inc. (Nasdaq: RVMD), a clinical-stage oncology company developing targeted therapies for patients with RAS-addicted cancers, today announced the publication of two peer-reviewed research papers in Nature. The first paper highlights the discovery and preclinical characterization of RMC-7977, a preclinical tool compound representative of a class of oral RAS(ON) multi-selective inhibitors, including the investigational drug candidate RMC-6236, that target multiple RAS variants. The second paper highlights the systematic evaluation of RMC-7977 in a wide range of preclinical models of PDAC. This original research was led by scientists at Revolution Medicines and conducted in collaboration with researchers from across the U.S. and Europe.

Key Points:

The first paper highlights the discovery and preclinical characterization of RMC-7977, a preclinical tool compound representative of a class of oral RAS(ON) multi-selective inhibitors, including the investigational drug candidate RMC-6236, that target multiple RAS variants.
The second paper highlights the systematic evaluation of RMC-7977 in a wide range of preclinical models of PDAC.
This original research was led by scientists at Revolution Medicines and conducted in collaboration with researchers from across the U.S. and Europe.
Oncogenic RAS proteins drive up to 30 percent of all human cancers, most notably non-small cell lung cancer (NSCLC), PDAC and colorectal cancer (CRC).

PETVIVO HOLDINGS, INC. TO EXHIBIT AT THE IVAPM PAIN MANAGEMENT FORUM IN INDIANAPOLIS, INDIANA

Retrieved on:

Lundi, avril 8, 2024

PetVivo intends to exhibit its lead product, SPRYNG™ with OsteoCushion™ Technology.

Key Points:

PetVivo intends to exhibit its lead product, SPRYNG™ with OsteoCushion™ Technology.
Spryng is an intra-articular injectable veterinary medical device composed of sterilized, extra-cellular matrix microparticles.
Such action promotes the restoration of proper joint mechanics, thereby aiding in the management of noninfectious sources of joint pain such as joint instability, degenerative joint disease and osteoarthritis.
For more information about PetVivo Holdings, Inc. please contact [email protected] or visit https://petvivo.com/
Pain Management Forum Exhibit Details:
Location: The JW Marriott in Indianapolis, Indiana
Presentation Title: How do we Elevate Our Strategy for Managing Osteoarthritis in Pets?

Junshi Biosciences Announces Approval of the sNDA for Toripalimab for the 1st-Line Treatment of Renal Cancer

Retrieved on:

Dimanche, avril 7, 2024

Renal carcinoma is the third most common malignancy of the urinary system globally, and RCC accounts for 80%~90% of all cases of renal carcinoma.

Key Points:

Renal carcinoma is the third most common malignancy of the urinary system globally, and RCC accounts for 80%~90% of all cases of renal carcinoma.
There were approximately 77,000 new cases of and 46,000 deaths due to renal carcinoma in China in 2022.
Distant metastasis occurred in about one-third of renal carcinoma patients at initial diagnosis, and in 20%-50% of localized patients after nephrectomy.
“The approval of toripalimab combined with axitinib addresses the gap in first-line immunotherapy for renal cancer in China.

Glancy Prongay & Murray LLP Reminds Investors of Looming Deadline in the Class Action Lawsuit Against Anavex Life Sciences Corp. (AVXL)

Retrieved on:

Vendredi, avril 5, 2024

GPM, News, Avatar, Suite, Trial of the century, Excellence, Court

You can also contact Charles H. Linehan, of GPM at 310-201-9150, Toll-Free at 888-773-9224, or via email at [email protected] to learn more about your rights.

Key Points:

You can also contact Charles H. Linehan, of GPM at 310-201-9150, Toll-Free at 888-773-9224, or via email at [email protected] to learn more about your rights.
On January 2, 2024, Anavex announced results from a Phase 2/3 EXCELLENCE Clinical Study in Pediatric Rett Syndrome, reporting that the “co-primary endpoint .
To be a member of the class action you need not take any action at this time; you may retain counsel of your choice or take no action and remain an absent member of the class action.
Glancy Prongay & Murray LLP, Los Angeles

Protara Therapeutics Announces Oversubscribed $45 Million Private Placement Financing

Retrieved on:

Vendredi, avril 5, 2024

NMIBC, Private, Exercise, Patient, Oppenheimer Holdings, Sale, Public expenditure, Cancer, Trial of the century, Guggenheim Partners, Share (finance), Therapy, Security (finance)

Gross proceeds from the private placement are expected to be approximately $45 million, before deducting expenses.

Key Points:

Gross proceeds from the private placement are expected to be approximately $45 million, before deducting expenses.
Proceeds from the private placement, along with existing cash and cash equivalents, are expected to be sufficient to fund the Company’s planned operations into 2026.
The Company intends to use the net proceeds from the Private Placement for general corporate and working capital purposes, including funding clinical trials.
Guggenheim Securities, LLC acted as lead placement agent and Oppenheimer & Co. acted as a placement agent in the transaction.