Advanced Therapy Medicinal Product

European Commission Approves Merck’s KEYTRUDA® (pembrolizumab) Plus Chemotherapy for New First-Line Indications in Advanced HER2-Negative Gastric or GEJ Adenocarcinoma in Tumors Expressing PD-L1 (CPS ≥1) and Advanced Biliary Tract Cancer

Retrieved on: 
Monday, December 18, 2023
Biotechnology, Health, Pharmaceutical, Clinical Trials, Oncology, MSD, Cisplatin, PD-L1, NYSE, European Commission, Risk, Civil service commission, Gastrointestinal cancer, Neoplasm, Committee, Advanced Therapy Medicinal Product, HER2, Adenocarcinoma, Death, BTC, OS, CPS, GEJ, MRK, Pharmaceutical industry, Merck

In patients whose tumors expressed PD-L1 (CPS ≥1), KEYTRUDA plus chemotherapy reduced the risk of death by 26% (HR=0.74 [95% CI, 0.65-0.84]; p

Key Points: 
  • In patients whose tumors expressed PD-L1 (CPS ≥1), KEYTRUDA plus chemotherapy reduced the risk of death by 26% (HR=0.74 [95% CI, 0.65-0.84]; p
  • Median OS was 13.0 months (95% CI, 11.6-14.2) for patients treated with KEYTRUDA plus chemotherapy vs 11.4 months (95% CI, 10.5-12.0) for chemotherapy alone.
  • Median OS was 12.7 months (95% CI, 11.5-13.6) for KEYTRUDA plus chemotherapy versus 10.9 months (95% CI, 9.9-11.6) for chemotherapy alone.
  • In KEYNOTE-859, the incidence of Grade 3-5 adverse reactions in patients with gastric cancer was 75% for KEYTRUDA plus chemotherapy and 70% for chemotherapy.

Vertex Receives CHMP Positive Opinion for the First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

Retrieved on: 
Friday, December 15, 2023
Research, FDA, Genetics, Clinical Trials, Stem Cells, Biotechnology, Health, Pharmaceutical, Science, Quality, Pediatrics, European Commission, Sickle cell disease, European Medicines Agency, HSC, Bambino, Vertex, Civil service commission, Patient, European, Pediatric Hematology and Oncology, CHMP, Gene, Committee, Advanced Therapy Medicinal Product, Hematology, VRTX, CRISPR, TDT, Beta thalassemia, Volatile organic compound, Catholic higher education, SCD, Quality of life, Vertex Pharmaceuticals, Pharmaceutical industry

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the conditional approval of CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

Key Points: 
  • Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the conditional approval of CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).
  • An approval decision by the European Commission is expected in February 2024.
  • “This positive opinion is yet another important regulatory milestone underscoring the potentially transformative benefit of CASGEVY for eligible patients with sickle cell and transfusion-dependent beta thalassemia,” said Nia Tatsis, Ph.D., Executive Vice President and Chief Regulatory and Quality Officer at Vertex.
  • “As an investigator, I have witnessed first-hand the transformative impact exa-cel can have on patients’ lives and I eagerly await the approval in the European Union.”

Takeda Receives Positive CHMP Opinion for HYQVIA® as Maintenance Therapy in Patients with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

Retrieved on: 
Friday, December 15, 2023
Pharmaceutical, Science, Health, Research, Peripheral nervous system, European Commission, European Academy of Neurology, Research and development, Chronic inflammatory demyelinating polyneuropathy, European Medicines Agency, Disease, Clinical trial, EMA, Patient, European, CHMP, Marketing, Committee, Caregiver, Advanced Therapy Medicinal Product, CIDP, Neurology, Recombinant, IGS, IVIG, Pharmaceutical industry, Takeda

Takeda ( TSE:4502/NYSE:TAK ) today announced the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of HYQVIA® [Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase] in patients with chronic inflammatory demyelinating polyneuropathy (CIDP) as maintenance therapy after stabilization with intravenous immunoglobulin therapy (IVIG).

Key Points: 
  • Takeda ( TSE:4502/NYSE:TAK ) today announced the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of HYQVIA® [Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase] in patients with chronic inflammatory demyelinating polyneuropathy (CIDP) as maintenance therapy after stabilization with intravenous immunoglobulin therapy (IVIG).
  • The European Commission (EC) will consider the CHMP positive opinion when determining the potential marketing authorization for HYQVIA for CIDP throughout the European Union.
  • This proposed extension of indication for HYQVIA is based on data from the pivotal Phase 3 ADVANCE-CIDP 1 clinical trial, which investigated HYQVIA as maintenance therapy in adult patients with CIDP.
  • HYQVIA is also under regulatory review in the United States for use as a maintenance therapy in adult patients with CIDP.

Melinta Therapeutics & Cidara Therapeutics Announce Publication of Pooled Data from Phase 3 Pivotal ReSTORE Trial & Phase 2 STRIVE Trial of REZZAYO® (rezafungin for injection) for the Treatment of Candidemia & Invasive Candidiasis in The Lancet ID

Retrieved on: 
Tuesday, December 5, 2023
Other Health, Research, Managed Care, Pharmaceutical, Hospitals, Clinical Trials, Science, Surgery, Biotechnology, FDA, Other Science, Health, CDTX, Lancet, NDA, Priority review, Disease, Safety, Food, Melinta Therapeutics, European Medicines Agency, Therapy, NTAP, Injection, Randomized controlled trial, QIDP, The Lancet, MAA, Mortality, Restore, EMA, Patient, Caspofungin, CHMP, Marketing, Advanced Therapy Medicinal Product, New Drug Application, CMS, MPH, Medicare, Hospital, Pharmaceutical industry, MD, Fungemia

These findings are of clinical importance given the ongoing morbidity and mortality caused by candidemia and invasive candidiasis.

Key Points: 
  • These findings are of clinical importance given the ongoing morbidity and mortality caused by candidemia and invasive candidiasis.
  • On March 22, 2023, the FDA approved REZZAYO for the treatment of candidemia and invasive candidiasis in adults with limited or no alternative treatment options.
  • REZZAYO is the first new treatment option approved for patients with candidemia and invasive candidiasis in over fifteen years.
  • In October, the EMA Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for rezafungin (rezafungin acetate) for the treatment of invasive candidiasis in adults.

Applied StemCell, Inc. Strengthens Executive Leadership with Two Key Appointments

Retrieved on: 
Monday, December 4, 2023
Construction & Property, Genetics, REIT, Health Technology, Stem Cells, Biotechnology, Health, Pharmaceutical, General Health, B cell, Sigma-Aldrich, Acquisition, Research, GMP, Molecular biology, Duke University, Commercial, CDMO, MSAT, ASC, Doctor of Philosophy, Marketing, Boston University, Advanced Therapy Medicinal Product, MBA, University, Champaign–Urbana metropolitan area, CCO, Dendreon, ATMP, Fine chemical, Management, Science, Biology

The recent acquisition of the company by QHP Capital in October has set the stage for an ambitious expansion focused on supporting cell manufacturing from development through commercialization.

Key Points: 
  • The recent acquisition of the company by QHP Capital in October has set the stage for an ambitious expansion focused on supporting cell manufacturing from development through commercialization.
  • George Hong joins Applied StemCell, Inc. as Chief Commercial Officer (CCO), bringing more than 20 years of experience in academic and industry roles within life sciences, serving as a sales, marketing, and commercial executive with increasing responsibilities.
  • Prior to joining Applied StemCell, Tim held key positions, including VP of Commercial Manufacturing at Adrian Biotech, VP of Manufacturing Science and Technology (MSAT) at RoslinCT, and over 8 years in commercial manufacturing and quality positions at Dendreon.
  • "We are thrilled to have George and Tim as valuable additions to the ASC leadership team," expressed Dr. Ruby Chen-Tsai, CEO of Applied StemCell.

Genmab Announces Positive Regulatory Updates for Epcoritamab (EPKINLY®/TEPKINLY®) for the Treatment of Relapsed/Refractory Follicular Lymphoma

Retrieved on: 
Monday, November 27, 2023
Oncology, FDA, Health, Clinical Trials, Pharmaceutical, Biotechnology, Food, European Medicines Agency, Type, ASH, Disease, EMA, Patient, European, BTD, Breakthrough therapy, AbbVie, CHMP, Advanced Therapy Medicinal Product, Exposition, Society, NHL, Pharmaceutical industry, Vaccine

Genmab A/S (Nasdaq: GMAB) today announced regulatory updates from the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) for epcoritamab, an investigational T-cell engaging bispecific antibody administered subcutaneously.

Key Points: 
  • Genmab A/S (Nasdaq: GMAB) today announced regulatory updates from the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) for epcoritamab, an investigational T-cell engaging bispecific antibody administered subcutaneously.
  • The U.S. FDA has granted Breakthrough Therapy Designation (BTD) to epcoritamab-bysp for the treatment of adult patients with relapsed or refractory (R/R) follicular lymphoma (FL) after two or more lines of systemic therapy.
  • Additionally, the EMA has validated a Type II variation application for epcoritamab for the same indication.
  • “Despite recent treatment advances in relapsed or refractory follicular lymphoma, a need still exists for more treatment options,” said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

Human medicines European public assessment report (EPAR): Casgevy, Exagamglogene autotemcel, Status: Opinion

Retrieved on: 
Monday, December 18, 2023
Medicine, Vomiting, B cell, Anatomy, ATC, Gene, Sickle cell disease, European Commission, Health, International, Transplant, HSC, Headache, INN, Thalassemia, Tissue, Vertex Pharmaceuticals, Liver disease, Disease, Febrile neutropenia, EMA, Civil service commission, Patient, Medication package insert, HBF, Hemoglobinopathy, Limited, CHMP, Mouth ulcer, Marketing, Committee, Advanced Therapy Medicinal Product, HLA, Medical Subject Headings, ATMP, Cas9, Nausea, TDT, BCL11A, Hematology, Volatile organic compound, Public, SCD, Physician, Cell, CD34, Haematopoiesis, Instrumentation, Vaccine, Veterinary medicine, Dentistry, Dietary supplement, Pharmaceutical industry, Medicinal plants, Anemia

Human medicines European public assessment report (EPAR): Casgevy, Exagamglogene autotemcel, Status: Opinion

Key Points: 


Human medicines European public assessment report (EPAR): Casgevy, Exagamglogene autotemcel, Status: Opinion

Merck Receives Positive EU CHMP Opinion for KEYTRUDA® (pembrolizumab) Plus Gemcitabine and Cisplatin as First-Line Treatment for Locally Advanced Unresectable or Metastatic Biliary Tract Cancer

Retrieved on: 
Friday, November 10, 2023
Oncology, Health, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, MSD, Cisplatin, NYSE, Food, European Commission, Head, European Medicines Agency, Merck & Co., Disease, Civil service commission, Patient, Gastrointestinal cancer, European, MRK, FDA, CHMP, Advanced Therapy Medicinal Product, Survival, Immunotherapy, Pharmaceutical industry, Merck

Merck (NYSE: MRK), known as MSD outside of the United States and Canada, today announced the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending approval of KEYTRUDA, Merck’s anti-PD-1 therapy, in combination with gemcitabine and cisplatin, for the first-line treatment of adult patients with locally advanced unresectable or metastatic biliary tract carcinoma.

Key Points: 
  • Merck (NYSE: MRK), known as MSD outside of the United States and Canada, today announced the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending approval of KEYTRUDA, Merck’s anti-PD-1 therapy, in combination with gemcitabine and cisplatin, for the first-line treatment of adult patients with locally advanced unresectable or metastatic biliary tract carcinoma.
  • “Patients diagnosed with locally advanced unresectable or metastatic biliary tract cancer face a challenging disease with poor survival outcomes, underscoring the need for new treatment options that may help extend their lives,” said Dr. Marjorie Green, senior vice president and head of late-stage oncology, global clinical development, Merck Research Laboratories.
  • In October 2023, the U.S. Food and Drug Administration (FDA) approved the use of KEYTRUDA in combination with gemcitabine and cisplatin for the treatment of patients with locally advanced unresectable or metastatic biliary tract cancer.
  • Merck has an extensive clinical development program evaluating KEYTRUDA in gastrointestinal cancers and is continuing to study KEYTRUDA for multiple uses in gastric, hepatobiliary, esophageal and colorectal cancers.

EQS-News: Rentschler Biopharma SE appoints Gunnar Voss von Dahlen as Chief Financial Officer and completes Executive Board

Retrieved on: 
Wednesday, December 13, 2023
Region, Heidelberger Druckmaschinen, Supervisory board, BDO Global, Patient, Knowledge, Advanced Therapy Medicinal Product, CDMO, Strategic planning, Growth, CFO, Service, ATMP, Financial management, Management

Laupheim, Milford, MA, USA, and Stevenage, UK, December 07, 2023 – Rentschler Biopharma SE, a leading contract development and manufacturing organization (CDMO) for biopharmaceuticals, including Advanced Therapy Medicinal Products (ATMPs), announced today that Gunnar Voss von Dahlen (54) has been appointed as Chief Financial Officer, effective December 1, 2023.

Key Points: 
  • Laupheim, Milford, MA, USA, and Stevenage, UK, December 07, 2023 – Rentschler Biopharma SE, a leading contract development and manufacturing organization (CDMO) for biopharmaceuticals, including Advanced Therapy Medicinal Products (ATMPs), announced today that Gunnar Voss von Dahlen (54) has been appointed as Chief Financial Officer, effective December 1, 2023.
  • Gunnar Voss von Dahlen succeeds Alexander Dettmer, who left the company in best mutual agreement in October 2023, as agreed with the Supervisory Board.
  • Prof. Dr. Nikolaus F. Rentschler, Chairman of the Supervisory Board of Rentschler Biopharma SE, said: “On behalf of the entire Supervisory Board, I am pleased to welcome Gunnar Voss von Dahlen as our new CFO.
  • Mr. Voss von Dahlen is an experienced financial expert with a successful track record in various companies and industries.

Plasmid DNA Manufacturing Markets, 2035 - ResearchAndMarkets.com

Retrieved on: 
Wednesday, October 18, 2023
Health, Pharmaceutical, Good manufacturing practice, Genetics, Biotechnology, Advanced Therapy Medicinal Product, Engineering, Genetic engineering, Pfizer, Geography, GenScript Biotech, COVID-19, Messenger, ATMP, Thermo Fisher Scientific, GMP, Plasmid, Education minister, DNA, Vaccine, Fine chemical

The plasmid DNA manufacturing market is a dynamic and swiftly expanding sector with a crucial role in biotechnology and genetic research.

Key Points: 
  • The plasmid DNA manufacturing market is a dynamic and swiftly expanding sector with a crucial role in biotechnology and genetic research.
  • The current market landscape features the presence of over 70 companies offering a range of plasmid DNA services, including plasmid design, plasmid engineering, plasmid construction, process development and optimization, and plasmid manufacturing.
  • They are vigorously advancing their capabilities to serve as a fully integrated one-stop-shop by offering a range of plasmid services, including process development and optimization, plasmid design, plasmid engineering and plasmid construct, and GMP plasmid manufacturing.
  • Stakeholders in the plasmid manufacturing industry have forged several partnerships to enhance their service portfolios and augment the reach of their proprietary plasmid DNA technology.