European Medicines Agency

Asahi Kasei Offers Pharmaceutical Excipient Ceolus™ with Nitrite Concentration of 0.1 ppm or Less

Retrieved on: 
Tuesday, February 27, 2024

To contribute to reducing the risk of potentially carcinogenic nitrosamine impurities in pharmaceuticals and nutritional supplements, Asahi Kasei now offers Ceolus™ microcrystalline cellulose (MCC) with nitrite levels of 0.1 μg/g (ppm) or less.

Key Points: 
  • To contribute to reducing the risk of potentially carcinogenic nitrosamine impurities in pharmaceuticals and nutritional supplements, Asahi Kasei now offers Ceolus™ microcrystalline cellulose (MCC) with nitrite levels of 0.1 μg/g (ppm) or less.
  • In order to reduce potential health hazards due to nitrosamine impurities, Asahi Kasei has succeeded in maintaining the nitrite concentration of Ceolus™ to 0.1 ppm or less (see image).
  • “Controlling the nitrite concentration in our products is an important step to ensure the provision of safer pharmaceuticals and nutritional supplements.
  • For further details on Ceolus™ and its contribution to pharmaceutical safety, please visit our website at https://www.ceolus.com/en/mcc/ .

Introducing Unlearn’s TrialPioneer: A Free Tool for Exploring Innovative Clinical Trial Designs Using AI

Retrieved on: 
Thursday, February 29, 2024

It also reveals the major advantages that novel AI-powered trial designs offer over traditional approaches.

Key Points: 
  • It also reveals the major advantages that novel AI-powered trial designs offer over traditional approaches.
  • TrialPioneer is designed to make clinical trial planning more efficient for professionals, including medical directors, clinicians, and statisticians.
  • It also illustrates how innovative trial designs, such as Unlearn’s TwinRCTs TM, can improve these metrics.
  • With TrialPioneer, we’re offering a free, powerful, and easy-to-use tool that enables anyone from any background to explore trial planning in a simple way, even with innovative trial designs that leverage tools like AI.

Rocket Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Financial Results and Highlights Recent Progress

Retrieved on: 
Monday, February 26, 2024

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and operational results for the fourth quarter and year ended December 31, 2023.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and operational results for the fourth quarter and year ended December 31, 2023.
  • “I am pleased with the strong results Rocket delivered in 2023, as we closed another successful year of progress across all six disclosed gene therapy programs spanning our AAV cardiovascular and LV hematology portfolios,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma.
  • Results from the study demonstrated the potential of RP-A601 as a gene therapy for patients with PKP2-ACM.
  • As of December 31, 2023, Rocket had cash, cash equivalents and investments of $407.5 million.

BeiGene Receives Positive CHMP Opinion for Tislelizumab as Treatment for Non-Small Cell Lung Cancer

Retrieved on: 
Monday, February 26, 2024

As monotherapy for the treatment of adult patients with locally advanced or metastatic NSCLC after prior platinum-based therapy.

Key Points: 
  • As monotherapy for the treatment of adult patients with locally advanced or metastatic NSCLC after prior platinum-based therapy.
  • Patients with EGFR mutant or ALK positive NSCLC should also have received targeted therapies before receiving tislelizumab.
  • Tislelizumab is also under review by the FDA as a first-line treatment for patients with unresectable, recurrent, locally advanced, or metastatic ESCC.
  • BeiGene has launched more than 17 potentially registration-enabling trials with tislelizumab with over 13,000 patients enrolled to-date, of which 15 have already reported positive readouts.

MHRA Issues Notice of Acceptance for POLARIS-AD, a Phase 3 Early Alzheimer’s Disease Trial Sponsored by AriBio Co., Ltd.

Retrieved on: 
Sunday, February 25, 2024

AriBio Co., Ltd. (AriBio) receives Notice of Acceptance for POLARIS-AD, a Phase 3 early Alzheimer’s disease clinical trial from the Medicines and Healthcare Products Regulatory Agency (MHRA) in the United Kingdom (UK).

Key Points: 
  • AriBio Co., Ltd. (AriBio) receives Notice of Acceptance for POLARIS-AD, a Phase 3 early Alzheimer’s disease clinical trial from the Medicines and Healthcare Products Regulatory Agency (MHRA) in the United Kingdom (UK).
  • The MHRA completed their review on the 21 of February and confirmed a favorable ethical opinion, the Company expects to initiate the clinical trial before the end of Q1’2024 in the UK.
  • POLARIS-AD is a double-blind, randomized, placebo-controlled, multi-center registration trial to evaluate the efficacy and safety of AR1001 (Mirodenafil) over 52 weeks of treatment in patients with early Alzheimer’s disease.
  • “Acceptance of the POLARIS-AD clinical study in the UK is another achievement in the development of AR1001.

Arcturus Therapeutics Receives Orphan Medicinal Product Designation from the European Commission (EC), for ARCT-032, for the Treatment of Cystic Fibrosis

Retrieved on: 
Thursday, February 22, 2024

In November 2023, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to ARCT-032 for CF.

Key Points: 
  • In November 2023, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to ARCT-032 for CF.
  • Orphan medicinal product designation by the EC is available to novel therapeutics that prevent or treat life-threatening or chronically debilitating conditions that affect fewer than five in 10,000 persons in the EU.
  • “We are pleased to receive orphan medicinal product designation from the EC for ARCT-032, as it represents a significant milestone for our CF program,” said Joseph Payne, President & CEO of Arcturus Therapeutics.
  • Arcturus remains on track to share interim Phase 1b data in H1 2024.

Bristol Myers Squibb Receives Positive CHMP Opinion for Reblozyl® (luspatercept) for Treatment of Adults with Transfusion-Dependent Anemia due to Low- to Intermediate-Risk Myelodysplastic Syndromes (MDS)

Retrieved on: 
Friday, February 23, 2024

The recommendation will now be reviewed by the European Commission (EC), which has the authority to approve medicines for the European Union (EU).

Key Points: 
  • The recommendation will now be reviewed by the European Commission (EC), which has the authority to approve medicines for the European Union (EU).
  • Upon approval, this would represent the fourth authorized indication for Reblozyl in the EU.
  • The CHMP adopted a positive opinion based on results from the pivotal Phase 3 COMMANDS trial.
  • Safety results were consistent with previous MDS studies and were in line with expected symptoms in this patient population.

Questions and answers on the European Union framework for (traditional) herbal medicinal products, including those from a ‘non-European’ tradition

Retrieved on: 
Sunday, March 10, 2024

1

Key Points: 
    • 1
      Committee on Herbal Medicinal Products (HMPC)

      Questions & Answers on the European Union framework
      for (traditional) herbal medicinal products, including those
      from a ?non-European? tradition

      Table of Content
      1.

    • European Pharmacopoeia

      SAWP

      Scientific Advice Working Party

      SmPC

      Summary of Product Characteristics

      THMP

      Traditional Herbal Medicinal Product

      TUR

      Traditional Use Registration

      WEU

      Well-Established Use

      Questions & Answers on the European Union framework for (traditional) herbal
      medicinal products, including those from a ?non-European? tradition
      EMA/HMPC/402684/2013 Rev.

    • Terminology of herbal medicinal products (Q&A 1-4)

      Question 1
      What are herbal substances, herbal preparations, and herbal medicinal products?

    • Questions & Answers on the European Union framework for (traditional) herbal
      medicinal products, including those from a ?non-European? tradition
      EMA/HMPC/402684/2013 Rev.
    • Question 4
      Are food supplements regulated under the European Union (EU) pharmaceutical legislation
      for (traditional) herbal medicinal products ((T)HMPs)?
    • Questions & Answers on the European Union framework for (traditional) herbal
      medicinal products, including those from a ?non-European? tradition
      EMA/HMPC/402684/2013 Rev.
    • Regulation of herbal medicinal products in the European Union (Q&A 511)

      Question 5
      Where to find the pharmaceutical legislation and dossier requirements for herbal medicinal
      products (HMPs), including traditional herbal medicinal products (THMPs), in the European
      Union (EU)?

    • Questions & Answers on the European Union framework for (traditional) herbal
      medicinal products, including those from a ?non-European? tradition
      EMA/HMPC/402684/2013 Rev.
    • These countries have,
      through the EEA agreement, adopted the complete Union acquis on medicinal products and are
      consequently parties to the Union procedures.
    • Questions & Answers on the European Union framework for (traditional) herbal
      medicinal products, including those from a ?non-European? tradition
      EMA/HMPC/402684/2013 Rev.
    • Questions & Answers on the European Union framework for (traditional) herbal
      medicinal products, including those from a ?non-European? tradition
      EMA/HMPC/402684/2013 Rev.
    • Questions & Answers on the European Union framework for (traditional) herbal
      medicinal products, including those from a ?non-European? tradition
      EMA/HMPC/402684/2013 Rev.
    • Questions & Answers on the European Union framework for (traditional) herbal
      medicinal products, including those from a ?non-European? tradition
      EMA/HMPC/402684/2013 Rev.
    • Specific provisions for traditional herbal medicinal products (Q&A 1221)

      Question 12
      Which indications can be granted for traditional herbal medicinal products (THMPs)?

    • Questions & Answers on the European Union framework for (traditional) herbal
      medicinal products, including those from a ?non-European? tradition
      EMA/HMPC/402684/2013 Rev.
    • Nov. 2023
      Answer 17

      Questions & Answers on the European Union framework for (traditional) herbal
      medicinal products, including those from a ?non-European? tradition
      EMA/HMPC/402684/2013 Rev.

    • Questions & Answers on the European Union framework for (traditional) herbal
      medicinal products, including those from a ?non-European? tradition
      EMA/HMPC/402684/2013 Rev.
    • Question 29
      Does the Committee on Herbal Medicinal Products (HMPC) hold a specific database on
      (registered) authorised (traditional) herbal medicinal products ((T)HMPs)?
    • Discussion with Member States intended to be

      Questions & Answers on the European Union framework for (traditional) herbal
      medicinal products, including those from a ?non-European? tradition
      EMA/HMPC/402684/2013 Rev.

    • Questions & Answers on the European Union framework for (traditional) herbal
      medicinal products, including those from a ?non-European? tradition
      EMA/HMPC/402684/2013 Rev.

Procedural advice on the accelerated assessment of marketing authorisation applications pursuant to Article 44 (3) of Regulation (EU) No 2019/6

Retrieved on: 
Sunday, March 10, 2024

2

Key Points: 
    • 2
      Committee for Veterinary Medicinal Products (CVMP)

      Procedural advice on the accelerated assessment of
      marketing authorisation applications pursuant to Article
      44 (3) of Regulation (EU) No 2019/6

      Table of contents
      1.

    • Accelerated assessment of the marketing authorisation application ....... 5
      6.1.
    • It only applies to marketing authorisation applications and does not cover any postauthorisation procedure.
    • The document replaces the ?Guideline on the procedure for accelerated
      assessment pursuant to Article 39(8) of Regulation (EC) No 726/2004' (EMEA/CVMP/32995/2006).
    • General considerations
      The accelerated assessment procedure is applicable to marketing authorisation applications for
      veterinary medicinal products falling within the scope of Article 44(3) of Regulation (EU) 2019/6.
    • An accelerated assessment request has to be agreed by the CVMP before submission of
      a marketing authorisation application as it introduces changes in the operation of the CVMP and
      procedure timelines.
    • A
      decision on accelerated assessment will be taken without prejudice to the (future) CVMP opinion
      (positive or negative) on the granting of a marketing authorisation.
    • It forms the basis for requesting an accelerated assessment and should be
      followed unless otherwise justified.
    • Procedural advice on the accelerated assessment of marketing authorisation
      applications pursuant to Article 44 (3) of Regulation (EU) No 2019/6
      EMA/CVMP/32995/2006 ? Rev.2

      Page 3/8

      5.3.

    • Request for an accelerated assessment procedure
      The formal request for an accelerated assessment should be submitted approximately 3-2 months prior
      to the actual submission of the marketing authorisation application.
    • Applicants are also reminded that evaluation under accelerated assessment is subject to
      the same evidence requirements for marketing authorisation as an evaluation under standard
      timetable.
    • Procedural advice on the accelerated assessment of marketing authorisation
      applications pursuant to Article 44 (3) of Regulation (EU) No 2019/6
      EMA/CVMP/32995/2006 ? Rev.2

      Page 5/8

      6.2.

    • ** The timing of the documents should be at least 11 working days in advance of the CVMP plenary meeting

      Procedural advice on the accelerated assessment of marketing authorisation
      applications pursuant to Article 44 (3) of Regulation (EU) No 2019/6
      EMA/CVMP/32995/2006 ? Rev.2

      Page 6/8

      7.2.

    • Procedural advice on the accelerated assessment of marketing authorisation
      applications pursuant to Article 44 (3) of Regulation (EU) No 2019/6
      EMA/CVMP/32995/2006 ? Rev.2

      Page 8/8

Compilation of quality review of documents (QRD) on stylistic matters in product information

Retrieved on: 
Sunday, March 10, 2024

29 February 2024

Key Points: 
    • 29 February 2024
      EMA/25090/2002 rev.23*
      Human Medicines Division

      Compilation of QRD decisions on stylistic matters in product information
      Issues
      Abbreviations

      Connected problems
      Subscript and superscript

      QRD Suggestions
      Acronyms must be written in their standard form; e.g.

    • Cmax, Cmax
      Abbreviations and

      Not always understood,

      Non-standard abbreviations and acronyms should be avoided, and the term should be written out in full.

    • The same applies when stating the pharmaceutical form in section 3 of the SmPC and section 4 of the labelling;
      i.e.
    • critical steps prior to administration of the product should also be included (section 5 of Annex IIIA).
    • Compilation of QRD decisions on stylistic matters in product information
      EMA/25090/2002

      Page 2/23

      Issues

      Connected problems

      QRD Suggestions

      emphasise in the labelling
      the special handling prior
      to administration of the
      product.

    • Consistency

      Inconsistencies in style are

      Once a particular style or house style has been selected, it must be used consistently throughout the text.

    • Tradename 150 mg solution for injection in pre-filled syringe
      Tradename 150 mg solution for injection in pre-filled pen

      information annexes?

    • Compilation of QRD decisions on stylistic matters in product information
      EMA/25090/2002

      Page 3/23

      Issues
      Desiccant

      Connected problems

      QRD Suggestions

      For medicinal products

      The foil of blister packs containing a desiccant must be clearly labelled to show which blister pocket contains

      packaged with a desiccant

      the desiccant.

    • For bottles containing a desiccant, a similar statement should also be considered provided there

      mistake the desiccant for a

      is available space.

    • It can only be included in brackets in section 3 of the

      where can this be

      SmPC and section 4 of the labelling.

    • Direct speech should only
      be used in section 6 of the SmPC for instructions about shelf-life, storage, handling and disposal.
    • The term ?drug? though can be used in the product information annexes when it is part of a standard set of
      terms (e.g.
    • Compilation of QRD decisions on stylistic matters in product information
      EMA/25090/2002

      Page 5/23

      Issues
      Foreign terms

      Connected problems

      QRD Suggestions

      Foreign terms, particularly

      Foreign terms must be written in italics; e.g.

    • Patients can be referred to as ?he? or

      physician is often referred

      as ?she? when the medicinal product is exclusively for use by males or females.

    • if the product in question might be

      measurements are

      used by elderly patients), in brackets after the metric measures in the English text.

    • Compilation of QRD decisions on stylistic matters in product information
      EMA/25090/2002

      Page 6/23

      Issues

      Connected problems

      QRD Suggestions

      product name is composed
      of MAH+INN?

    • Compilation of QRD decisions on stylistic matters in product information
      EMA/25090/2002

      Page 7/23

      Issues

      Connected problems

      QRD Suggestions
      MT: English
      BG: Bulgarian
      NL: Dutch
      CZ: Czech or English at applicant?s discretion.

    • Compilation of QRD decisions on stylistic matters in product information
      EMA/25090/2002

      Page 14/23

      Issues

      Connected problems

      QRD Suggestions
      considered, e.g.

    • Compilation of QRD decisions on stylistic matters in product information
      EMA/25090/2002

      Page 16/23

      Issues
      Symbols: Non-Unicode

      Connected problems

      QRD Suggestions

      The use of non-Unicode

      Only Unicode symbols must be used in submitted product information annexes.

    • Compilation of QRD decisions on stylistic matters in product information
      EMA/25090/2002

      Page 20/23

      Issues

      Connected problems

      Units: SI base units -

      International Standard

      litre

      base units have been
      introduced in the European
      Union with Council
      Directive 80/181/EEC of
      20.12.79 (O.J.

    • Compilation of QRD decisions on stylistic matters in product information
      EMA/25090/2002

      Page 23/23