Immunosuppressive drug

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.

Eledon Pharmaceuticals Announces 12th Participant Enrolled in Phase 2 BESTOW Trial Evaluating Tegoprubart for the Prevention of Organ Rejection

Retrieved on:

Monday, March 25, 2024

Toxicity, Nephrotoxicity, Transplant, Organ transplantation, Central nervous system, Risk, Patient, Kidney transplantation, Tacrolimus, Thumbay Hospital, Life, Kidney, Immune system, Tremor, Hypertension, Immunosuppressive drug

IRVINE, Calif., March 25, 2024 (GLOBE NEWSWIRE) -- Eledon Pharmaceuticals, Inc. (“Eledon”) (NASDAQ: ELDN) today announced the enrollment of the 12th participant on March 23, 2024, in the Company’s ongoing Phase 2 BESTOW trial assessing tegoprubart head-to-head with tacrolimus for the prevention of rejection in kidney transplantation.

Key Points:

IRVINE, Calif., March 25, 2024 (GLOBE NEWSWIRE) -- Eledon Pharmaceuticals, Inc. (“Eledon”) (NASDAQ: ELDN) today announced the enrollment of the 12th participant on March 23, 2024, in the Company’s ongoing Phase 2 BESTOW trial assessing tegoprubart head-to-head with tacrolimus for the prevention of rejection in kidney transplantation.
In transplantation procedures, organ rejection is a major cause of graft failure, which can be a life-threatening condition.
Rejection occurs due to allorecognition, wherein the recipient's immune system identifies the transplanted organ as foreign tissue, triggering an immune response against the transplanted organ.
Calcineurin inhibitors (“CNIs”) are a critical component of most immunosuppressive regimens to prevent acute and long-term organ transplant rejection.

North America Kidney Transplant Market Report 2023 - 2027: Featuring Sanofi, Pfizer, Novartis and B.Braun Melsungen Among Others - ResearchAndMarkets.com

Retrieved on:

Wednesday, March 1, 2023

The success of a kidney transplant is dependent on various factors, including donor-recipient compatibility, good post-operative care, and the recipient's overall health.

Key Points:

The success of a kidney transplant is dependent on various factors, including donor-recipient compatibility, good post-operative care, and the recipient's overall health.
According to our study, the number of kidney transplants in 2022 will be 30.67 thousand, rising to 39.44 thousand by 2027.
Growing demand for kidney transplantation is also one of the key drivers driving expansion potential in the North American sector in the next years.
Nevertheless, the report shows that the North American kidney transplant market was at US$ 11.36 Billion in 2022.

Global Multiple Sclerosis Therapeutics Market Analysis to 2029 - Featuring Teva Pharmaceuticals, Pfizer and Novartis Among Others - ResearchAndMarkets.com

Retrieved on:

Thursday, July 28, 2022

Health, Pharmaceutical, Administration, Therapy, CAGR, Multiple sclerosis, Region, Growth, Injection (medicine), Sclerosis, Disease, Mode, USD, Immunosuppressive drug, Pharmaceutical industry

The "Multiple Sclerosis Therapeutics Market Analysis by Drug Class (Immunosuppressant, Immunomodulators) By Mode of Administration (Injectable, Oral, Intravenous) and by Region - Forecast to 2029" report has been added to ResearchAndMarkets.com's offering.

Key Points:

The "Multiple Sclerosis Therapeutics Market Analysis by Drug Class (Immunosuppressant, Immunomodulators) By Mode of Administration (Injectable, Oral, Intravenous) and by Region - Forecast to 2029" report has been added to ResearchAndMarkets.com's offering.
The global multiple sclerosis therapeutics market size is estimated to be USD 27.84 billion in 2020 and is expected to witness a CAGR of 6.71% during the forecast period.
Increasing global prevalence of target disease is a key driver for the growth of the global multiple sclerosis therapeutics market.
Nevertheless, side-effects and risks associated with multiple sclerosis therapeutics and indeterminate etiology of the disease are expected to restrain the global market growth.

Complement 3 Glomerulopathy (C3G) in 7 Major Markets (US, Germany, Spain, Italy, France, UK, Japan) | Insights, Epidemiology, and Market Forecasts 2018-2030 - ResearchAndMarkets.com

Retrieved on:

Friday, November 19, 2021

Treatments for C3G include Immunosuppressant, Steroids, Renin-angiotensin-aldosterone system Inhibitors (RAAS), and other Supportive therapies (also including Antibody regimens: Eculizumab and Rituximab).

Key Points:

Treatments for C3G include Immunosuppressant, Steroids, Renin-angiotensin-aldosterone system Inhibitors (RAAS), and other Supportive therapies (also including Antibody regimens: Eculizumab and Rituximab).
The market size of Complement 3 Glomerulopathy (C3G) in the seven major markets was estimated to be worth USD 40.6 Million in 2017.
The companies and academics are working to assess challenges and seek opportunities that could influence Complement 3 Glomerulopathy (C3G) R&D.
As per the analysis, Complement 3 Glomerulopathy (C3G) can be divided into two types, namely, Dense Deposit Disease (DDD) and C3 glomerulonephritis (C3GN).

U.S. Food and Drug Administration Expands Indication for PROGRAF® for Prevention of Organ Rejection in Adult and Pediatric Lung Transplant Recipients

Retrieved on:

Tuesday, July 20, 2021

Medical specialties, Clinical medicine, Medicine, Organ transplantation, Transplantation medicine, Immunosuppressants, Macrolides, Cardiac surgery, Tacrolimus, Transplant rejection, Immunosuppressive drug, Heart transplantation

NORTHBROOK, Ill., July 20, 2021 /PRNewswire/ -- Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., "Astellas") announced that the U.S. Food and Drug Administration (FDA) has approved its supplemental New Drug Application (sNDA) for PROGRAF® (tacrolimus) for the prevention of organ rejection in adult and pediatric lung transplant recipients.

Key Points:

"The current approval expands the indications for PROGRAF from liver, kidney and heart transplants to adult and pediatric patients who receive a lung transplant in combination with other medicines to help prevent organ rejection."
The FDA granted Orphan Drug Designation (ODD) to PROGRAF for the prevention of rejection after lung transplant in September 2019.
PROGRAFis a calcineurin-inhibitor immunosuppressant indicated for the prophylaxis of organ rejection in adult and pediatric patients receiving allogeneic liver, kidney, heart or lung transplants, in combination with other immunosuppressants.
PROGRAF is a prescription medicine used with other medicines to help prevent organ rejection in people who have had a kidney, liver, heart or lung transplant.

Medeor Therapeutics to Provide Interim Results of Phase 3 Mercury Study of MDR-101 Cell Therapy in Living Donor Kidney Transplants During Rapid Fire Oral Presentation at ATC Virtual Congress

Retrieved on:

Thursday, May 20, 2021

Biotechnology, Surgery, Health, Pharmaceutical, Clinical trials, Medical specialties, Clinical medicine, Medicine, Organ transplantation, Transplantation medicine, Immunology, Immunosuppressive drug, Immunosuppression

MDR-101 is a single-dose cellular therapy derived from a living kidney donor\xe2\x80\x99s blood.

Key Points:

MDR-101 is a single-dose cellular therapy derived from a living kidney donor\xe2\x80\x99s blood.
MDR-101 is intended to induce donor-specific immune tolerance in order to avert transplant kidney rejection and thereby preserve transplant kidney function and survival without the cumulative and serious side effects associated with immunosuppressive drugs.
Medeor\xe2\x80\x99s Phase 3 trial is supported in part by a grant from the California Institute for Regenerative Medicine.
Medeor\xe2\x80\x99s Phase 3 clinical study is demonstrating the significant opportunities of this one-time therapy.

FDA Approves Merck’s KEYTRUDA® (pembrolizumab) Combined With Trastuzumab and Chemotherapy as First-line Treatment in Locally Advanced Unresectable or Metastatic HER2-Positive Gastric or Gastroesophageal Junction Adenocarcinoma

Retrieved on:

Wednesday, May 5, 2021

There are currently more than 1,400 trials studying KEYTRUDA across a wide variety of cancers and treatment settings.

Key Points:

There are currently more than 1,400 trials studying KEYTRUDA across a wide variety of cancers and treatment settings.
Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Institute medical management promptly, including specialty consultation as appropriate.\nWithhold or permanently discontinue KEYTRUDA depending on severity of the immune-mediated adverse reaction.
Consider administration of other systemic immunosuppressants in patients whose adverse reactions are not controlled with corticosteroid therapy.\nKEYTRUDA can cause immune-mediated pneumonitis.

Worldwide Industry for Uveitis Treatment to 2025 - by Treatment Type, Disease Type, Cause, Distribution Channel and Region

Retrieved on:

Thursday, December 31, 2020

Medical specialties, Clinical medicine, Medicine, Uveitis, Medication, Immunosuppressive drug, Antibiotic, Antiviral drug, Corticosteroid

The global uveitis treatment market grew at a CAGR of around 6% during 2014-2019.

Key Points:

The global uveitis treatment market grew at a CAGR of around 6% during 2014-2019.
Some of the commonly used uveitis treatment methods include topical, systemic and local corticosteroids, immunosuppressants, monoclonal antibodies, cycloplegic agents, antibiotics, analgesics and antivirals and antifungal medicines.
Looking forward, the publisher expects the global uveitis treatment market to continue its moderate growth during the next five years.
What is the structure of the global uveitis treatment market and who are the key players?

AST Applauds Congressional Leaders for Passage of the Comprehensive Immunosuppressive Drug Coverage for Kidney Transplant Patients Act

Retrieved on:

Monday, December 28, 2020

Medical specialties, Clinical medicine, Surgery, Kidney transplantation, Organ transplantation, Immunosuppressive drug

MOUNT LAUREL, N.J., Dec. 28, 2020 /PRNewswire/ --After nearly 20 years of tireless advocacy from the transplant stakeholder community and some very committed Congressional leaders, the Comprehensive Immunosuppressive Drug Coverage for Kidney Transplant Patients Act (Immuno Bill) was passed by Congress and signed into law by President Trump.

Key Points:

MOUNT LAUREL, N.J., Dec. 28, 2020 /PRNewswire/ --After nearly 20 years of tireless advocacy from the transplant stakeholder community and some very committed Congressional leaders, the Comprehensive Immunosuppressive Drug Coverage for Kidney Transplant Patients Act (Immuno Bill) was passed by Congress and signed into law by President Trump.
"The passage of the Immuno Bill is a huge victory for kidney transplant recipients, donors, and their families," stated AST President Dr. Richard Formica.
The AST, representing a majority of medical professionals engaged in solid organ transplantation, has long advocated for removing the 36-month immunosuppressive drug coverage limitation for kidney transplant patients.
Additionally, the AST partnered on many occasions with members of Congress to host Congressional educational briefings to garner support for the Immuno Bill and hosted a Transplant Patient Summit with Congressional leaders that focused on the need to eliminate kidney patients' 36-month immunosuppressive drug coverage limitation.