Clinical trial

Draft guideline on the pharmaceutical quality of inhalation and nasal medicinal products

Retrieved on: 
星期四, 四月 18, 2024
Reproduction, Midol, Medical device, Particle size, QRD, Patient, Chronic obstructive pulmonary disease, Food packaging, DPI, CTD, Isocyanate, European Pharmacopoeia, Roflumilast, GSD, Scope, Asthma, OIP, Clinical trial, European, COPD, APSD, Marketing, CQA, Nasal mucosa, Medical device regulation, Medication, Solvent, No-no, RH, Ageing, Draft, Gel, EMA, Freezing, Outline, Risk assessment, Uniformity, Chemistry, Pressurization, Micronization, Clutch (eggs), Blister, CHMP, Mass, MDI, Solubility, Documentation, Life cycle, Pharmacopoeia, Capsule, Bias, LDPE, Inhalation, Conformity, Publication, The central science, ICH, Orientation, NB, Lubricant, AIM, Excipient, Temperature, Monograph, Humidity, Cold, Light, Life, Medication package insert, Rugosity, Quality, Water, Yesa, Administration, Weight loss, Life insurance

17

Key Points: 
    • 17

      Guideline on the pharmaceutical quality of inhalation and
      nasal medicinal products

      18

      Table of contents

      19

      Executive summary ..................................................................................... 3

      20

      1.

    • Lifecycle management ........................................................................................ 28

      49

      Definitions ................................................................................................. 29

      16

      50
      51

      Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 2/30

      52

      Executive summary

      53

      This guideline is the first revision of the guideline on pharmaceutical quality of inhalation and nasal

      54

      products (EMEA/CHMP/QWP/49313/2005 Corr).

    • Quality aspects specific to inhalation and nasal medicinal products are discussed, the need for

      66

      safety testing (e.g., for excipients and leachables) is also considered.

    • 69

      Detailed guidance on pharmaceutical development study designs (e.g., priming studies) and the

      70

      analytical procedures primarily used for inhalation and nasal medicinal products (e.g., cascade

      71

      impactor analysis) is not included in this guideline.

    • Scope

      74

      The guideline addresses requirements "on the quality of inhalation and nasal medicinal products" in

      75

      new marketing authorisation applications, including abridged applications.

    • Liquid inhalation anaesthetics and nasal ointments, creams and gels are

      88

      excluded, however the general principles described in this guideline should be considered.

    • 118

      Different polymorphic forms including any amorphous content could affect the quality or performance

      119

      of the finished medicinal product.

    • Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 4/30

      132

      The primary packaging, type of inhaler and, if necessary, the secondary packaging or other

      133

      components required for reasons of stability should be described.

    • Pharmaceutical
      development study

      (a) Physical
      characterisation
      (b) Minimum fill
      justification
      (c) Extractable
      volume

      Pressurised

      Dry powder

      Preparations for

      Non-

      metered-

      inhalers (DPI)

      nebulisation

      pressurised

      dose

      metered-

      Device-

      Pre-

      Single-

      Multi-

      (pMDI)

      metered

      metered

      dose

      dose

      inhalers

      Yesa

      Yes

      Yes

      Yesa

      Yesa

      Yesa

      Yes

      Yes

      Yes

      Yes

      Yes

      Yes

      No

      No

      No

      Yes

      No

      No

      inhalers

      Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      dose

      Page 5/30

      Table 4.2.1.

    • The last doses delivered by

      Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 7/30

      179

      the inhaler as defined by the label claim, should meet the finished medicinal product specification limits

      180

      for delivered dose and fine particle dose.

    • Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 9/30

      263
      264

      4.2.2.8.

    • Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 11/30

      345

      Instructions regarding cold temperature use should be provided in the product information.

    • Finished medicinal
      product

      Pressurised

      Dry powder inhalers

      Preparations for

      metered-

      (DPI)

      nebulisation

      dose

      Nonpressurised
      metered-dose

      Device-

      Pre-

      Single-

      Multi-

      (pMDI)

      metered

      metered

      dose

      dose

      inhalers

      (a) Description

      Yes

      Yes

      Yes

      Yes

      Yes

      Yes

      (b) Assay

      Yes

      Yes

      Yes

      Yes

      Yes

      Yes

      (c) Moisture content

      Yes

      Yes

      Yes

      No

      No

      No

      Yes

      Yes

      Yes

      No

      No

      Yes

      Yes

      Yes

      Yes

      No

      No

      Yes

      specification test

      (d) Mean delivered
      dose
      (e) Uniformity of
      delivered dose

      inhalers

      Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 15/30

      Table 4.2.2.

    • Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 16/30

      510

      4.2.5.4.

    • The proposed specification limits should take into account the shelf-life performance of the
      Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 17/30

      552

      medicinal product.

    • Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 18/30

      586

      All medical devices, including inhalers and nasal devices, have to fulfil the general requirements as

      587

      outlined in the Medical Device Regulation (EU) 2017/745.

    • Stability (CTD 3.2.P.8)

      598

      All inhalation medicinal products should be tested on stability against the stability indicating tests

      599

      included in the finished medicinal product specification.

    • Quality data requirements as

      619

      described in this guideline should be met, supplemented by appropriate comparative quality and

      620

      clinical data with respect to the chosen reference medicinal product.

    • 621

      For inhalation medicinal products comparative in vitro data between the abridged application medicinal

      622

      product and the reference medicinal product must be provided.

    • Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 20/30

      670

      Nature and contents of container: The type of the device and its components should be listed.

    • Nasal medicinal products

      695

      Inhalation and nasal medicinal products have many similarities and therefore, most of the

      696

      requirements specified for inhalation medicinal products in section 4 also apply for nasal medicinal

      697

      products.

    • One difference between inhalation and nasal medicinal products is the desired

      698

      particle/droplet size of the finished medicinal product.

    • Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 21/30

      704

      5.2.

    • Nasal liquids
      Pharmaceutical
      development
      study

      Pressurised

      Nasal

      metered-

      powders,

      dose nasal

      device-

      spray

      metered

      NonSingledose
      drops

      Multidose
      drops

      Single-

      pressurised

      dose

      multidose

      spray

      metereddose spray

      (a) Physical
      characterisation
      (b) Minimum fill
      justification
      (d) Extractables /
      leachables

      Yesa

      Yes

      Yesa

      Yesa

      Yesa

      Yesa

      Yes

      Yes

      Yes

      Yes

      Yes

      Yes

      Yes

      No

      Yes

      Yes

      Yes

      Yes

      Yes

      Yes

      No

      No

      Yes

      Yes

      Yes

      Yes

      No

      No

      No

      Yes

      Yes

      Yes

      No

      No

      Yes

      Yes

      (f) Particle /
      droplet size
      distribution
      (g) Uniformity of
      delivered dose
      through container
      life
      (j) Actuator /
      mouthpiece
      deposition

      Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 22/30

      Table 5.2.1.

    • Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 23/30

      728

      5.2.2.2.

    • Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 24/30

      769

      5.2.5.

    • Quality data requirements as described in

      799

      this guideline should be met, supplemented by appropriate comparative quality and clinical data with

      800

      respect to the chosen reference medicinal product.

    • Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 27/30

      849

      5.5.

    • 866

      Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 28/30

      867

      Definitions
      Activation:

      The act of setting in motion the delivery device.

    • Delivery device:

      The sum of component(s) of the container closure system responsible for
      delivering the active substance to the respiratory tract (inhalation medicinal
      product) or the nasal and/or pharyngeal region (nasal medicinal product).

    • Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 29/30

      Label claim:

      The amount of active substance (usually on a per actuation basis) declared
      on the label of the medicinal product.

    • Nasal medicinal

      A finished medicinal product (including the delivery device, where

      product:

      applicable) whose intended site of deposition is the nasal and/or pharyngeal
      region.

    • 868
      Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 30/30

Orphan designation: Recombinant adeno-associated viral vector serotype S3 containing codon-optimised expression cassette encoding human coagulation factor IX variant Treatment of haemophilia B, 26/10/2018 Withdrawn

Retrieved on: 
星期四, 四月 18, 2024
Eurostat, Diagnosis, Civil service commission, Brain, Disease, Coagulation, Patient, Committee, Knowledge, Internal, Regulation, Haemophilia, EMA, IRIS, Joint, Human, Wound healing, Clinical trial, European, Therapy, COMP, Bleeding, Spinal cord, Sponsor, European Commission, Immune system, Antibody, Safety, Factor IX, Blood, Marketing, Limited, Haemophilia B, Medicine, Pharmaceutical industry

Orphan designation: Recombinant adeno-associated viral vector serotype S3 containing codon-optimised expression cassette encoding human coagulation factor IX variant Treatment of haemophilia B, 26/10/2018 Withdrawn

Key Points: 


Orphan designation: Recombinant adeno-associated viral vector serotype S3 containing codon-optimised expression cassette encoding human coagulation factor IX variant Treatment of haemophilia B, 26/10/2018 Withdrawn

Orphan designation: Synthetic hypericin Treatment of cutaneous T-cell lymphoma, 28/07/2015 Positive

Retrieved on: 
星期四, 四月 18, 2024
Eurostat, Diagnosis, Civil service commission, X-ray, Growth, Medicine, Disease, Patient, Ageing, Committee, Knowledge, Tissue, Neoplasm, Light, Cutaneous T-cell lymphoma, Regulation, Cancer, EMA, IRIS, Quality of life, Reactive oxygen species, Lymph, CTCL, Clinical trial, European, Skin, COMP, Oxygen, European Commission, Immune system, Safety, Carmustine, Alfa, Marketing, Lymphatic system, Pharmaceutical industry

Orphan designation: Synthetic hypericin Treatment of cutaneous T-cell lymphoma, 28/07/2015 Positive

Key Points: 


Orphan designation: Synthetic hypericin Treatment of cutaneous T-cell lymphoma, 28/07/2015 Positive

Clinical Trials Information System (CTIS): Walk-in clinic - May 2024, Online, European Medicines Agency, Amsterdam, the Netherlands, Broadcast, from 15 May 2024, 16:00 (CEST) to 15 May 2024, 17:00 (CEST)

Retrieved on: 
星期四, 四月 18, 2024
Location, CTIS, FAQ, Clinical trial, SME, EMA, Flour

Questions about the interpretation of the Clinical Trial Regulation and /or national processes are out of the scope of this event.

Key Points: 
  • Questions about the interpretation of the Clinical Trial Regulation and /or national processes are out of the scope of this event.
  • The event is open to all sponsor organisations, including pharmaceutical companies, contract research organisations, small and medium-sized enterprises (SMEs) and academic organisations.
  • It will be broadcasted live; no registration is required for those wishing to follow the live broadcast on EMA’s website.
  • Please subscribe to the Clinical Trials Highlights newsletter for updates on the availability of CTIS event video recordings.

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: 
星期四, 四月 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.

Draft guideline on the pharmaceutical quality of inhalation and nasal medicinal products

Retrieved on: 
星期四, 四月 18, 2024
Reproduction, Midol, Medical device, Particle size, QRD, Patient, Chronic obstructive pulmonary disease, Food packaging, DPI, CTD, Isocyanate, European Pharmacopoeia, Roflumilast, GSD, Scope, Asthma, OIP, Clinical trial, European, COPD, APSD, Marketing, CQA, Nasal mucosa, Medical device regulation, Medication, Solvent, No-no, RH, Ageing, Draft, Gel, EMA, Freezing, Outline, Risk assessment, Uniformity, Chemistry, Pressurization, Micronization, Clutch (eggs), Blister, CHMP, Mass, MDI, Solubility, Documentation, Life cycle, Pharmacopoeia, Capsule, Bias, LDPE, Inhalation, Conformity, Publication, The central science, ICH, Orientation, NB, Lubricant, AIM, Excipient, Temperature, Monograph, Humidity, Cold, Light, Life, Medication package insert, Rugosity, Quality, Water, Yesa, Administration, Weight loss, Life insurance

17

Key Points: 
    • 17

      Guideline on the pharmaceutical quality of inhalation and
      nasal medicinal products

      18

      Table of contents

      19

      Executive summary ..................................................................................... 3

      20

      1.

    • Lifecycle management ........................................................................................ 28

      49

      Definitions ................................................................................................. 29

      16

      50
      51

      Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 2/30

      52

      Executive summary

      53

      This guideline is the first revision of the guideline on pharmaceutical quality of inhalation and nasal

      54

      products (EMEA/CHMP/QWP/49313/2005 Corr).

    • Quality aspects specific to inhalation and nasal medicinal products are discussed, the need for

      66

      safety testing (e.g., for excipients and leachables) is also considered.

    • 69

      Detailed guidance on pharmaceutical development study designs (e.g., priming studies) and the

      70

      analytical procedures primarily used for inhalation and nasal medicinal products (e.g., cascade

      71

      impactor analysis) is not included in this guideline.

    • Scope

      74

      The guideline addresses requirements "on the quality of inhalation and nasal medicinal products" in

      75

      new marketing authorisation applications, including abridged applications.

    • Liquid inhalation anaesthetics and nasal ointments, creams and gels are

      88

      excluded, however the general principles described in this guideline should be considered.

    • 118

      Different polymorphic forms including any amorphous content could affect the quality or performance

      119

      of the finished medicinal product.

    • Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 4/30

      132

      The primary packaging, type of inhaler and, if necessary, the secondary packaging or other

      133

      components required for reasons of stability should be described.

    • Pharmaceutical
      development study

      (a) Physical
      characterisation
      (b) Minimum fill
      justification
      (c) Extractable
      volume

      Pressurised

      Dry powder

      Preparations for

      Non-

      metered-

      inhalers (DPI)

      nebulisation

      pressurised

      dose

      metered-

      Device-

      Pre-

      Single-

      Multi-

      (pMDI)

      metered

      metered

      dose

      dose

      inhalers

      Yesa

      Yes

      Yes

      Yesa

      Yesa

      Yesa

      Yes

      Yes

      Yes

      Yes

      Yes

      Yes

      No

      No

      No

      Yes

      No

      No

      inhalers

      Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      dose

      Page 5/30

      Table 4.2.1.

    • The last doses delivered by

      Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 7/30

      179

      the inhaler as defined by the label claim, should meet the finished medicinal product specification limits

      180

      for delivered dose and fine particle dose.

    • Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 9/30

      263
      264

      4.2.2.8.

    • Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 11/30

      345

      Instructions regarding cold temperature use should be provided in the product information.

    • Finished medicinal
      product

      Pressurised

      Dry powder inhalers

      Preparations for

      metered-

      (DPI)

      nebulisation

      dose

      Nonpressurised
      metered-dose

      Device-

      Pre-

      Single-

      Multi-

      (pMDI)

      metered

      metered

      dose

      dose

      inhalers

      (a) Description

      Yes

      Yes

      Yes

      Yes

      Yes

      Yes

      (b) Assay

      Yes

      Yes

      Yes

      Yes

      Yes

      Yes

      (c) Moisture content

      Yes

      Yes

      Yes

      No

      No

      No

      Yes

      Yes

      Yes

      No

      No

      Yes

      Yes

      Yes

      Yes

      No

      No

      Yes

      specification test

      (d) Mean delivered
      dose
      (e) Uniformity of
      delivered dose

      inhalers

      Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 15/30

      Table 4.2.2.

    • Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 16/30

      510

      4.2.5.4.

    • The proposed specification limits should take into account the shelf-life performance of the
      Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 17/30

      552

      medicinal product.

    • Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 18/30

      586

      All medical devices, including inhalers and nasal devices, have to fulfil the general requirements as

      587

      outlined in the Medical Device Regulation (EU) 2017/745.

    • Stability (CTD 3.2.P.8)

      598

      All inhalation medicinal products should be tested on stability against the stability indicating tests

      599

      included in the finished medicinal product specification.

    • Quality data requirements as

      619

      described in this guideline should be met, supplemented by appropriate comparative quality and

      620

      clinical data with respect to the chosen reference medicinal product.

    • 621

      For inhalation medicinal products comparative in vitro data between the abridged application medicinal

      622

      product and the reference medicinal product must be provided.

    • Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 20/30

      670

      Nature and contents of container: The type of the device and its components should be listed.

    • Nasal medicinal products

      695

      Inhalation and nasal medicinal products have many similarities and therefore, most of the

      696

      requirements specified for inhalation medicinal products in section 4 also apply for nasal medicinal

      697

      products.

    • One difference between inhalation and nasal medicinal products is the desired

      698

      particle/droplet size of the finished medicinal product.

    • Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 21/30

      704

      5.2.

    • Nasal liquids
      Pharmaceutical
      development
      study

      Pressurised

      Nasal

      metered-

      powders,

      dose nasal

      device-

      spray

      metered

      NonSingledose
      drops

      Multidose
      drops

      Single-

      pressurised

      dose

      multidose

      spray

      metereddose spray

      (a) Physical
      characterisation
      (b) Minimum fill
      justification
      (d) Extractables /
      leachables

      Yesa

      Yes

      Yesa

      Yesa

      Yesa

      Yesa

      Yes

      Yes

      Yes

      Yes

      Yes

      Yes

      Yes

      No

      Yes

      Yes

      Yes

      Yes

      Yes

      Yes

      No

      No

      Yes

      Yes

      Yes

      Yes

      No

      No

      No

      Yes

      Yes

      Yes

      No

      No

      Yes

      Yes

      (f) Particle /
      droplet size
      distribution
      (g) Uniformity of
      delivered dose
      through container
      life
      (j) Actuator /
      mouthpiece
      deposition

      Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 22/30

      Table 5.2.1.

    • Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 23/30

      728

      5.2.2.2.

    • Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 24/30

      769

      5.2.5.

    • Quality data requirements as described in

      799

      this guideline should be met, supplemented by appropriate comparative quality and clinical data with

      800

      respect to the chosen reference medicinal product.

    • Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 27/30

      849

      5.5.

    • 866

      Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 28/30

      867

      Definitions
      Activation:

      The act of setting in motion the delivery device.

    • Delivery device:

      The sum of component(s) of the container closure system responsible for
      delivering the active substance to the respiratory tract (inhalation medicinal
      product) or the nasal and/or pharyngeal region (nasal medicinal product).

    • Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 29/30

      Label claim:

      The amount of active substance (usually on a per actuation basis) declared
      on the label of the medicinal product.

    • Nasal medicinal

      A finished medicinal product (including the delivery device, where

      product:

      applicable) whose intended site of deposition is the nasal and/or pharyngeal
      region.

    • 868
      Guideline on the pharmaceutical quality of inhalation and nasal medicinal products
      EMA/CHMP/20607/2024

      Page 30/30

Biophytis announces its 2023 financial results and provides an update on its business activities

Retrieved on: 
星期三, 四月 10, 2024
Annual, Notifiable disease, Annual report, Conference, Obesity, Euronext Growth, Goldman Sachs, Disease, MDA, Partnership, Risk, COVID-19, Patient, Duchenne muscular dystrophy, Sarcopenia, University, Respiratory failure, Clinical trial, Therapy, FDA, Pharmaceutical industry

2024: The actual start of the study will depend on the conclusion of partnership agreements and Biophytis' financial resources.

Key Points: 
  • 2024: The actual start of the study will depend on the conclusion of partnership agreements and Biophytis' financial resources.
  • Promising preclinical results for BIO101 (20-hydroxyecdysone) in obesity, suggesting beneficial metabolic effects on muscle and fat mass.
  • On this basis, Biophytis plans to start a phase 1/2 clinical trial in 2024, depending on its financial resources.
  • There is therefore significant doubt about the Company's ability to continue its business activities.

Biophytis announces its 2023 financial results and provides an update on its business activities

Retrieved on: 
星期三, 四月 10, 2024
Annual, Notifiable disease, Annual report, Conference, Obesity, Euronext Growth, Goldman Sachs, Disease, MDA, Partnership, Risk, COVID-19, Patient, Duchenne muscular dystrophy, Sarcopenia, University, Respiratory failure, Clinical trial, Therapy, FDA, Pharmaceutical industry

2024: The actual start of the study will depend on the conclusion of partnership agreements and Biophytis' financial resources.

Key Points: 
  • 2024: The actual start of the study will depend on the conclusion of partnership agreements and Biophytis' financial resources.
  • Promising preclinical results for BIO101 (20-hydroxyecdysone) in obesity, suggesting beneficial metabolic effects on muscle and fat mass.
  • On this basis, Biophytis plans to start a phase 1/2 clinical trial in 2024, depending on its financial resources.
  • There is therefore significant doubt about the Company's ability to continue its business activities.

EQS-News: Heidelberg Pharma announces financial figures and reports on successful business performance in 2023

Retrieved on: 
星期三, 四月 10, 2024
Malignancy, Spanish mythology, State university system, Patient, Sale, Renal cell carcinoma, Clinical trial, Neoplasm, Therapy, Depreciation, HPHA, USD, Marketing, GLP, Health, Research, ATACS, EPO, Immune system, ADC, BLA, ATAC, Food, Biologics license application, Disease, Magenta, Multiple myeloma, AACR, GCC, Annual report, FDA, Diagnosis, EUR, Supervisory board, Accounting, PET, EDT, Spacecraft, Amanitin, European Patent Convention, Patent, Eli Lilly and Company, Binghamton University, Pharmaceutical industry

Minority interest in Emergence sold: In summer 2023, Heidelberg Pharma sold its minority interest in Emergence Therapeutics AG, Duisburg, Germany, (Emergence).

Key Points: 
  • Minority interest in Emergence sold: In summer 2023, Heidelberg Pharma sold its minority interest in Emergence Therapeutics AG, Duisburg, Germany, (Emergence).
  • As a result, Heidelberg Pharma lost sales revenue in the low single-digit millions for the 2023 financial year.
  • In April 2023, Heidelberg Pharma signed a termination agreement with Magenta under which all licensed ATAC rights and some Magenta patents were assumed by Heidelberg Pharma.
  • The Heidelberg Pharma Group includes two entities, Heidelberg Pharma AG and Heidelberg Pharma Research GmbH.

EQS-News: ActiTrexx treats first patient with novel regulatory T cell therapy against Graft-versus-Host Disease

Retrieved on: 
星期三, 四月 10, 2024
Cell, Bone marrow, Disease, Transplant, Risk, Clinic, Patient, Gastrointestinal tract, Mortality, Haematopoietic system, Leukemia, Quality of life, Graft-versus-host disease, Clinical trial, Skin, Safety, CSO, Liver, Acute leukemia, Vaccine

GvHD is a severe and potentially life-threatening complication that can arise after an allogeneic haematopoietic stem cell transplantation and affects about 50 % of recipients.

Key Points: 
  • GvHD is a severe and potentially life-threatening complication that can arise after an allogeneic haematopoietic stem cell transplantation and affects about 50 % of recipients.
  • GvHD occurs when the stem cell donor’s T cells attack the patient’s tissues, leading to a range of symptoms that can affect the skin, gastrointestinal tract, and liver.
  • Prof. Dr. Andrea Tüttenberg, CEO of ActiTrexx, said: “GvHD can lead to life-long symptoms for the patient and is a major cause for the high mortality rate of blood stem cell transplantation.
  • Ten patients who have recently received a blood stem cell transplantation as treatment for leukemia will receive a single treatment with Actileucel.