Medicines and Healthcare products Regulatory Agency

CEL-SCI Concludes Positive Meeting With U.S. FDA Concerning Multikine’s Approval for Head & Neck Cancer

Retrieved on: 
Friday, July 14, 2023
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During the recent meeting, the FDA acknowledged the longstanding need for improved treatments for head and neck cancer.

Key Points: 
  • During the recent meeting, the FDA acknowledged the longstanding need for improved treatments for head and neck cancer.
  • Preliminary feedback from the FDA included that the selection criteria developed by CEL-SCI could be used to determine which patients should receive Multikine.
  • Based on the feedback received at this recent meeting, CEL-SCI is preparing additional information about its development plan for the next meeting with the agency.
  • These are priority markets for CEL-SCI, as Europe has more than twice the number of head and neck cancer cases diagnosed each year as compared to the U.S.

BPSA Releases Guide on X-Ray Sterilization of Single-Use Bioprocessing Equipment, Part 2: Representative Qualification Data

Retrieved on: 
Thursday, July 13, 2023
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To support the risk assessments needed for such implementation, BPSA has generated supporting data evaluating the performance levels of X-ray sterilization and gamma-ray sterilization on certain single-use materials.

Key Points: 
  • To support the risk assessments needed for such implementation, BPSA has generated supporting data evaluating the performance levels of X-ray sterilization and gamma-ray sterilization on certain single-use materials.
  • The resulting data in the new BPSA guide demonstrates the equivalent impact of X-ray irradiation of single-use materials as gamma.
  • Advancing X-ray as a mode of sterilization is critical, for BPSA members and the broader bioprocessing industry."
  • Please contact BPSA Executive Director, Chris Clark, at [email protected] , and/or visit the BPSA website to download a copy of the white paper.

Dr. Reddy’s Proposed Rituximab Biosimilar Application Accepted for Review by USFDA, EMA and MHRA

Retrieved on: 
Wednesday, July 12, 2023
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Reddy’s”), a global pharmaceutical company, announced that its Biologics License Application (BLA) for its proposed biosimilar rituximab candidate DRL_RI has been accepted for a substantive review by the U.S. Food and Drug Administration (USFDA).

Key Points: 
  • Reddy’s”), a global pharmaceutical company, announced that its Biologics License Application (BLA) for its proposed biosimilar rituximab candidate DRL_RI has been accepted for a substantive review by the U.S. Food and Drug Administration (USFDA).
  • This closely follows acceptance of its rituximab biosimilar dossier for review by two other regulatory agencies – the European Medicines Agency (EMA) and the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA).
  • DRL_RI is being developed as a biosimilar of Rituxan® / MabThera® (rituximab), a cluster of differentiation 20 (CD20) directed cytolytic antibody.
  • generic drugs for critical and chronic conditions, to commercialise its proposed biosimilar of rituximab in the United States.

Sensorion Submits Clinical Trial Application for Lead Gene Therapy Candidate OTOF-GT in the UK

Retrieved on: 
Monday, July 10, 2023
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, US Foods, Audiogenic, Dental implant, Congenital hearing loss, Health care, European Medicines Agency, Cochlea, Clinical trial, Quality of life, Mutation, CTA, AAV, IHC, EMA, Patient, Hearing, Approved Drug Products with Therapeutic Equivalence Evaluations, Medicines and Healthcare products Regulatory Agency, Orphan, Ageing, FDA, Safety, Food, MHRA, Pharmaceutical industry, Europe 1

The phase 1/2 clinical trial (Audiogene), aims to evaluate the safety, tolerability and efficacy of intra-cochlear injection of OTOF-GT, for the treatment of otoferlin gene-mediated hearing loss in pediatric patients aged up to 31 months.

Key Points: 
  • The phase 1/2 clinical trial (Audiogene), aims to evaluate the safety, tolerability and efficacy of intra-cochlear injection of OTOF-GT, for the treatment of otoferlin gene-mediated hearing loss in pediatric patients aged up to 31 months.
  • The CTA submission follows extensive preclinical studies assessing the safety and efficacy of OTOF-GT and successful manufacturing of the gene therapy Drug Product for the clinical trial.
  • Sensorion’s OTOF-GT dual AAV vector gene therapy development program aims to restore hearing in patients with mutations in OTOF who suffer from severe to profound sensorineural prelingual non syndromic hearing loss.
  • Nawal Ouzren, Chief Executive Officer of Sensorion, commented: “This first gene therapy CTA filing is a major milestone for our OTOF-GT program and Sensorion’s broader gene therapy franchise.

MHRA Grants Marketing Authorisation for Opzelura® (ruxolitinib) Cream for the Treatment of Non-Segmental Vitiligo with Facial Involvement in Adults and Adolescents

Retrieved on: 
Wednesday, July 5, 2023
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Incyte Biosciences UK today announced that the Medicines and Healthcare Products Regulatory Agency (MHRA) has granted marketing authorisation for Opzelura® (ruxolitinib) cream 15mg/g for the treatment of non-segmental vitiligo with facial involvement in adults and adolescents from 12 years of age.

Key Points: 
  • Incyte Biosciences UK today announced that the Medicines and Healthcare Products Regulatory Agency (MHRA) has granted marketing authorisation for Opzelura® (ruxolitinib) cream 15mg/g for the treatment of non-segmental vitiligo with facial involvement in adults and adolescents from 12 years of age.
  • Opzelura is the first and only approved treatment in the United Kingdom (UK) to offer eligible patients with non-segmental vitiligo support for repigmentation.
  • “Today’s MHRA approval is welcome news for dermatologists and people with vitiligo seeking treatment who until now have had limited options.
  • “We are delighted to announce the MHRA approval of Opzelura for people living with non-segmental vitiligo with facial involvement,” said Peter Williams, General Manager, Incyte United Kingdom and Ireland.

Cosmo announces Cortiment® approval in Japan

Retrieved on: 
Wednesday, June 28, 2023
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Dublin, Ireland – 28 June 2023: Cosmo Pharmaceuticals N.V. (SIX: COPN, XETRA: C43) (“Cosmo”) today announced that its partner Ferring Pharmaceuticals (“Ferring”) has received the approval for Cortiment® (budesonide 9 mg MMX tablets) in Japan by the Japanese Pharmaceuticals and Medical Devices Agency (PMDA).

Key Points: 
  • Dublin, Ireland – 28 June 2023: Cosmo Pharmaceuticals N.V. (SIX: COPN, XETRA: C43) (“Cosmo”) today announced that its partner Ferring Pharmaceuticals (“Ferring”) has received the approval for Cortiment® (budesonide 9 mg MMX tablets) in Japan by the Japanese Pharmaceuticals and Medical Devices Agency (PMDA).
  • First sales of Cortiment® in Japan are expected to begin by end of Q3 2023.
  • Cortiment® has been developed by Cosmo and is globally distributed by Ferring, with the exception of the US, where it is distributed by Bausch.
  • Alessandro Della Chà, CEO of Cosmo Pharmaceuticals, comments: “We are very pleased with the new success our partner Ferring Pharmaceuticals has achieved with Cortiment®.

CXR-AID, Powered by Lunit, Nets Japan Nod for Reimbursement, Accelerating Market Expansion Opportunities

Retrieved on: 
Monday, June 26, 2023
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SEOUL, South Korea, June 26, 2023 /PRNewswire/ -- Lunit (KRX:328130.KQ), a global provider of cancer diagnostics solutions, today announced that CXR-AID, FujiFilm's AI solution for chest X-ray analysis, powered by Lunit INSIGHT CXR, has been officially included as an eligible solution for health insurance reimbursement in Japan.

Key Points: 
  • MHLW added the new Class 3 condition for hospitals utilizing AI image diagnostics software and fulfilling specific facility and safety criteria.
  • This significantly boosts Lunit and FujiFilm's expansion in the Japanese market and creates new growth prospects in the field of AI image diagnostics.
  • Its exceptional diagnostic performance led to the approval of the Japan Pharmaceuticals and Medical Devices Agency (PMDA) in 2021.
  • The market expansion of CXR-AID in Japan is further bolstered by FujiFilm's widespread presence, with its screening devices and solutions already being utilized by over 25,000 medical institutions nationwide.

Savara Completes Enrollment of Pivotal Phase 3 IMPALA-2 Trial of Molgramostim Nebulizer Solution (Molgramostim) in Autoimmune Pulmonary Alveolar Proteinosis (aPAP)

Retrieved on: 
Monday, June 26, 2023
Health, FDA, Other Health, General Health, Clinical Trials, Pharmaceutical, Biotechnology, APAP, The New England Journal of Medicine, Inc., Food, COVID-19, IMPALA, MHRA, Patient, PIM, Medicines and Healthcare products Regulatory Agency, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Safety, Chair, Pharmaceutical industry

Savara Inc. (Nasdaq: SVRA), a clinical stage biopharmaceutical company focused on rare respiratory diseases, today announced that enrollment has been completed for the pivotal Phase 3 IMPALA-2 trial.

Key Points: 
  • Savara Inc. (Nasdaq: SVRA), a clinical stage biopharmaceutical company focused on rare respiratory diseases, today announced that enrollment has been completed for the pivotal Phase 3 IMPALA-2 trial.
  • Target enrollment for the trial was 160 patients.
  • IMPALA-2 is a global, 48-week, placebo-controlled clinical trial evaluating molgramostim, a novel inhaled biologic, for the treatment of aPAP, a rare lung disease with no approved pharmacological treatments.
  • “Enrollment completion marks an important milestone in the molgramostim development program,” said Matt Pauls, Chair and CEO, Savara.

CSL Behring Announces the First Patient Has Received FDA-Approved HEMGENIX® (etranacogene dezaparvovec-drlb) for Hemophilia B

Retrieved on: 
Tuesday, June 20, 2023
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"As part of our longtime promise to patients, CSL Behring is thrilled to mark this latest milestone of the first commercial patient having received HEMGENIX and look forward to continuing to deliver this paradigm-shifting treatment to the hemophilia B community," said Bob Lojewski, Senior Vice President and General Manager, North America, CSL Behring.

Key Points: 
  • "As part of our longtime promise to patients, CSL Behring is thrilled to mark this latest milestone of the first commercial patient having received HEMGENIX and look forward to continuing to deliver this paradigm-shifting treatment to the hemophilia B community," said Bob Lojewski, Senior Vice President and General Manager, North America, CSL Behring.
  • While prophylactic hemophilia B treatments have significantly advanced over the past several decades and are effective, treatments are typically administered on a strict infusion schedule and breakthrough bleeds into joints are still possible.
  • HEMGENIX has the potential to fundamentally transform the treatment paradigm through a single, one-time infusion in people living with hemophilia B by addressing the genetic cause of the condition.
  • "As of May 2023, payers covering roughly 60 percent of the U.S. population have established clear medical policies covering HEMGENIX.

HUTCHMED and Takeda Announce Publication of Phase III FRESCO-2 Results in The Lancet

Retrieved on: 
Friday, June 16, 2023
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HONG KONG and SHANGHAI, China and FLORHAM PARK, N.J. and OSAKA, Japan and CAMBRIDGE, Mass., June 15, 2023 (GLOBE NEWSWIRE) -- HUTCHMED (China) Limited (“HUTCHMED”) (Nasdaq/AIM:​HCM, HKEX:​13) and Takeda (TSE:4502, NYSE:TAK) today announced that results of the Phase III FRESCO-2 study evaluating fruquintinib in patients with previously treated metastatic colorectal cancer (“CRC”) were published in The Lancet.  

Key Points: 
  • The publication provides details of the FRESCO-2 study results as of June 24, 2022.
  • Summary results from this cut-off date were presented on September 12, 2022, at the European Society for Medical Oncology Congress 2022 (“ESMO22”).
  • Fruquintinib is a highly selective and potent inhibitor of vascular endothelial growth factor receptors (“VEGFR”) -1, -2 and -3.
  • In March 2023, HUTCHMED and Takeda closed an exclusive license agreement to further the global development, commercialization and manufacture of fruquintinib outside of China.