PMDA

Indegene acquires Trilogy Writing & Consulting GmbH, a medical writing consultancy

Retrieved on: 
Thursday, March 28, 2024

PRINCETON, N.J. and FRANKFURT, Germany, March 27, 2024 (GLOBE NEWSWIRE) -- Indegene, a digital-first, life sciences commercialization company, announced the acquisition of Trilogy Writing & Consulting GmbH (Trilogy), a global provider of specialty medical writing capabilities across clinical, regulatory, safety and medical content to life sciences companies.

Key Points: 
  • PRINCETON, N.J. and FRANKFURT, Germany, March 27, 2024 (GLOBE NEWSWIRE) -- Indegene, a digital-first, life sciences commercialization company, announced the acquisition of Trilogy Writing & Consulting GmbH (Trilogy), a global provider of specialty medical writing capabilities across clinical, regulatory, safety and medical content to life sciences companies.
  • The acquisition by Indegene Ireland, a subsidiary of Indegene Limited, augments Indegene’s depth of clinical and regulatory writing expertise for market authorization applications globally.
  • Trilogy Writing & Consulting is a Germany, UK, and US-based, medical writing consultancy with know-how in the development and delivery of clinical, regulatory, safety, and medical content.
  • It applies its expertise and unique approaches to deliver quality medical writing output.

Orchestra BioMed Reports Full Year 2023 Financial Results and Provides Fourth Quarter Business Update

Retrieved on: 
Wednesday, March 27, 2024

Globally, the treatment of coronary in-stent restenosis and other challenging artery disease indications is rapidly shifting toward drug-coated balloons.

Key Points: 
  • Globally, the treatment of coronary in-stent restenosis and other challenging artery disease indications is rapidly shifting toward drug-coated balloons.
  • Financial Results for the Year Ended December 31, 2023
    Cash and cash equivalents and marketable securities totaled $87.6 million as of December 31, 2023.
  • Net loss for 2023 was $49.1 million, or ($1.48) per share, compared with a net loss of $33.6 million, or ($2.24) per share, for 2022.
  • Net loss for the year-ended 2023 included non-cash stock-based compensation expense of $7.6 million, compared with $3.4 million for the same period in 2022.

Indegene acquires Trilogy Writing & Consulting GmbH, a medical writing consultancy

Retrieved on: 
Wednesday, March 27, 2024

PRINCETON, N.J. and FRANKFURT, Germany, March 27, 2024 (GLOBE NEWSWIRE) -- Indegene, a digital-first, life sciences commercialization company, announced the acquisition of Trilogy Writing & Consulting GmbH (Trilogy), a global provider of specialty medical writing capabilities across clinical, regulatory, safety and medical content to life sciences companies.

Key Points: 
  • PRINCETON, N.J. and FRANKFURT, Germany, March 27, 2024 (GLOBE NEWSWIRE) -- Indegene, a digital-first, life sciences commercialization company, announced the acquisition of Trilogy Writing & Consulting GmbH (Trilogy), a global provider of specialty medical writing capabilities across clinical, regulatory, safety and medical content to life sciences companies.
  • The acquisition by Indegene Ireland, a subsidiary of Indegene Limited, augments Indegene’s depth of clinical and regulatory writing expertise for market authorization applications globally.
  • Trilogy Writing & Consulting is a Germany, UK, and US-based, medical writing consultancy with know-how in the development and delivery of clinical, regulatory, safety, and medical content.
  • It applies its expertise and unique approaches to deliver quality medical writing output.

Belite Bio Announces PMDA Submission of Tinlarebant for Stargardt Disease Clinical Trial in Japan

Retrieved on: 
Friday, March 22, 2024

In the Phase 2 trial, 42% of Tinlarebant-treated subjects (5 out of 12) did not develop atrophic retinal lesions during the 24-month treatment period

Key Points: 
  • In the Phase 2 trial, 42% of Tinlarebant-treated subjects (5 out of 12) did not develop atrophic retinal lesions during the 24-month treatment period
    SAN DIEGO, March 22, 2024 (GLOBE NEWSWIRE) -- Belite Bio, Inc (NASDAQ: BLTE) (“Belite Bio” or the “Company”), a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, today announces its submission to the Pharmaceuticals and Medical Devices Agency (PMDA) to initiate a clinical trial of Tinlarebant in adolescent STGD1 in Japan (“DRAGON II”).
  • The DRAGON II trial is a combination of Phase 1b open-label study to evaluate the pharmacokinetics and pharmacodynamics of Tinlarebant in Japanese adolescent STGD1 subjects and a Phase 2/3, global, multicenter, double-masked, placebo-controlled, randomized study designed to evaluate the efficacy, safety and tolerability of Tinlarebant in adolescent STGD1 subjects.
  • Approximately 60 subjects, aged 12 to 20 years old, including approximately 10 Japanese subjects, are targeted for enrollment in the Phase 2/3 portion of the trial with a 1:1 randomization (tinlarebant:placebo).
  • The data from Japanese subjects is intended to facilitate future NDA applications in Japan.

Pharming Group reports fourth quarter and full year 2023 financial results

Retrieved on: 
Thursday, March 14, 2024

The U.S. market contributed 97% of 2023 revenues, while the EU and Rest of World contributed 3%.

Key Points: 
  • The U.S. market contributed 97% of 2023 revenues, while the EU and Rest of World contributed 3%.
  • Revenues increased to US$7.9 million in the fourth quarter of 2023, driven by the continued increase in patients on paid therapy, and revenues were US$18.2 million for 2023.
  • Pharming made continued progress in the fourth quarter of 2023 on leniolisib regulatory filings for APDS patients 12 years of age and older in key global markets.
  • Pharming filed regulatory submissions in Canada and Australia in the third quarter of 2023, and Israel in the second quarter.

Aurion Biotech to Present Clinical Data at the 2024 American Society of Cataract and Refractive Surgery (ASCRS) Annual Meeting

Retrieved on: 
Tuesday, April 2, 2024

Aurion Biotech , whose mission is to restore vision to millions of patients with life-changing regenerative therapies, today announced several presentations during the 2024 American Society of Cataract and Refractive Surgery (ASCRS) Annual Meeting to be held April 5-8, 2024 in Boston, Massachusetts.

Key Points: 
  • Aurion Biotech , whose mission is to restore vision to millions of patients with life-changing regenerative therapies, today announced several presentations during the 2024 American Society of Cataract and Refractive Surgery (ASCRS) Annual Meeting to be held April 5-8, 2024 in Boston, Massachusetts.
  • “We are delighted to share additional insights into these clinical studies, in multiple sessions at the Eyecelerator and ASCRS 2024 meetings,” said Michael Goldstein, M.D., president and chief medical officer, Aurion Biotech.
  • The ABA-1 / CLARA trial is designed to assess the safety, tolerability, and efficacy of AURN001 for the treatment of corneal edema secondary to corneal endothelial dysfunction.
  • The primary endpoint is the percentage of subjects who gain 3 lines of vision at 6 months.

Partner Therapeutics' Leukine® (sargramostim) Receives Approval in Japan to Treat Autoimmune Pulmonary Alveolar Proteinosis (aPAP)

Retrieved on: 
Wednesday, April 3, 2024

LEXINGTON, Mass., April 3, 2024 /PRNewswire/ -- Partner Therapeutics, Inc. (PTx) announced that its partner Nobelpharma received approval from the Japanese Pharmaceuticals and Medical Device Agency (PMDA) for the inhaled use of Leukine (sargramostim), branded in Japan as Sargmalin, to treat aPAP. PTx licensed rights for certain indications in Japan to Nobelpharma in 2022. Leukine is a glycosylated recombinant human granulocyte-macrophage colony-stimulating factor (rhu GM-CSF) produced by recombinant DNA technology in yeast. Leukine is not approved outside Japan for aPAP or as an inhalation therapy. Partner Therapeutics will manufacture Sargmalin for Nobelpharma at its manufacturing facility in Lynnwood, WA.

Key Points: 
  • Leukine is not approved outside Japan for aPAP or as an inhalation therapy.
  • Partner Therapeutics will manufacture Sargmalin for Nobelpharma at its manufacturing facility in Lynnwood, WA.
  • Inhaled recombinant GM-CSF reduces the need for whole lung lavage and improves gas exchange in autoimmune pulmonary alveolar proteinosis patients.
  • Autoimmune pulmonary alveolar proteinosis.

Procedural advice for orphan medicinal product designation: Guidance for sponsors

Retrieved on: 
Wednesday, April 3, 2024

Frequently asked questions ................................................................... 10

Key Points: 
    • Frequently asked questions ................................................................... 10

      Procedural advice for orphan medicinal product designation
      EMA/420706/2018

      Page 2/13

      1.

    • This Regulation lays down the procedure for
      the designation of medicinal products as orphan medicinal products and provides incentives for the
      development and placing on the market of designated orphan medicinal products.
    • The Regulation also
      establishes the Committee for Orphan Medicinal Products (COMP) within the European Medicines
      Agency (EMA), which is responsible for examining applications for orphan medicinal product
      designation.
    • As of 28 April 2000, the date this Regulation entered into force, sponsors have been able to
      submit applications for orphan medicinal product designation to the EMA.
    • Procedural advice for orphan medicinal product designation
      EMA/420706/2018

      Page 3/13

      2.

    • Objectives
      In examining an application for orphan medicinal product designation, the COMP will focus on
      determining whether the sponsor has established that the designation criteria are met, i.e.
    • To assist in the development of a policy on orphan medicinal products, an expert network will
      be built up by the Committee, with expert(s) identified as appropriate to be involved in the evaluation
      of applications for orphan medicinal product designation.
    • ?

      The complete application should include:

      Procedural advice for orphan medicinal product designation
      EMA/420706/2018

      Page 5/13

      Document

      Format

      General administrative and scientific information completed online via the portal.

    • Important:
      In preparing an application for orphan medicinal product designation, sponsors are requested to follow
      the Commission guideline (2022/C 440/02) on the format and content of applications for designation
      as orphan medicinal products.
    • Please refer to the Procedural advice on
      appeal procedure for Orphan Medicinal Product Designation (EMEA/2677/01 Rev.3).
    • The grounds for the

      Procedural advice for orphan medicinal product designation
      EMA/420706/2018

      Page 9/13

      appeal should be based only on the original information provided in the application for orphan
      designation.

    • ?

      The European Commission will enter the designated medicinal product in the Community Register
      of Orphan Medicinal Products.

    • General advice
      ?

      Full information on the procedure for orphan medicinal products designation is available on the
      EMA orphan designation website.

    • In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the sponsor may submit an
      application for orphan medicinal product designation to the Agency at any stage of development of the

      Procedural advice for orphan medicinal product designation
      EMA/420706/2018

      Page 10/13

      medicinal product as long as the criteria for designation may be justified.

    • A request for orphan medicinal product designation may be made for
      a new orphan indication for an already authorised medicinal product.
    • Procedural advice for orphan medicinal product designation
      EMA/420706/2018

      Page 11/13

      Which are the sponsor?s options in case of negative outcome for orphan designation?

EQS-News: Eckert & Ziegler Extends Ga-68 Activities in Japan

Retrieved on: 
Wednesday, March 13, 2024

Berlin, Germany – 28 February 2024 – Eckert & Ziegler (ISIN DE0005659700, SDAX) expands its activities on Gallium-68 (Ga-68) labeled diagnostics in Japan together with Novartis Pharma K.K.

Key Points: 
  • Berlin, Germany – 28 February 2024 – Eckert & Ziegler (ISIN DE0005659700, SDAX) expands its activities on Gallium-68 (Ga-68) labeled diagnostics in Japan together with Novartis Pharma K.K.
  • (Japan).
  • For this purpose, a clinical trial was notified with the Japanese Pharmaceuticals and Medical Devices Agency (PMDA), stating Eckert & Ziegler’s proprietary Ge-68/Ga-68 generator GalliaPharm® as the single source for Ga-68.
  • “We are happy to extend our radioisotope supply business in Japan by contributing our GalliaPharm® to this important clinical research project,” explained Dr. Harald Hasselmann, CEO of Eckert & Ziegler AG.

OrbusNeich Achieves Record-High Revenue and Net Profit of US$154 Million and US$45.1 Million Respectively, Recommends Final Dividend Payment of HK10 cents per Share

Retrieved on: 
Thursday, March 7, 2024

-- Revenue hit record high at US$153.9 million, up 12.5% year-on-year

Key Points: 
  • -- Revenue hit record high at US$153.9 million, up 12.5% year-on-year
    -- Revenue from PCI balloons and PTA balloons grew by 12.1% and 29.0% year-on-year, respectively.
  • In particular, revenue from PCI balloons and PTA balloons grew by 12.1% and 29.0% year-on-year to US$115.4 million and US$14.7 million, respectively.
  • As at December 31, 2023, the Group was in a strong financial position with cash and bank balances amounting to US$255.8 million (2022: US$229.1 million).
  • Having considered future capital requirements, the Board has proposed a final dividend of HK10 cents per share in cash, as a token of appreciation for the continuous support of shareholders.