Priority review

Human Acellular Vessel™ (HAV™) Biologics License Application Granted Priority Review by U.S. FDA for the Treatment of Vascular Trauma

Retrieved on: 
Friday, February 9, 2024
MD, FDA, Food, HAV, Injury, Defense, Diagnosis, Sale, Amputation, PDUFA, Peripheral artery disease, Surgeon, Infection, Patient, Priority review, BLA, Regenerative medicine, Doctor of Philosophy, HUMA, Pharmaceutical industry

DURHAM, N.C., Feb. 09, 2024 (GLOBE NEWSWIRE) -- Humacyte, Inc. (Nasdaq: HUMA), a clinical-stage biotechnology platform company developing universally implantable, bioengineered human tissue at commercial scale, today announced that the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review to Humacyte’s Biologics License Application (BLA) seeking approval of the Human Acellular Vessel (HAV) in urgent arterial repair following extremity vascular trauma when synthetic graft is not indicated, and when autologous vein use is not feasible.

Key Points: 
  • This targeted PDUFA date is based on the Priority Review grant, which is a mechanism reserved by FDA for products that, if approved, would significantly improve the treatment, diagnosis, or prevention of serious conditions.
  • Priority Review applications have a six-month review time instead of ten months for a standard review.
  • The Priority Review aligns with the Regenerative Medicine Advanced Therapy (RMAT) designation granted by the FDA in May 2023 for urgent arterial repair following extremity vascular trauma.
  • The HAV is an investigational product and has not been approved for sale by the FDA or any other regulatory agency.

Madrigal Pharmaceuticals Announces Publication of the Phase 3 MAESTRO-NASH Trial of Resmetirom in the New England Journal of Medicine

Retrieved on: 
Thursday, February 8, 2024
Principal, ALT, Liver failure, Fatty liver disease, Cirrhosis, Hepatotoxicity, Nausea, GGT, Death, Therapy, Biomarker, Patient, Breakthrough therapy, Safety, Incidence, FDA, NAFLD, Lipoprotein, Priority review, Diarrhea, NAS, Woman, University of Oxford, Disease, Drug development, Fibrosis, Biopsy, Risk, Week, Heart rate, PDUFA, CAP, NASH, Medicine, Liver cancer, Liver, Pharmaceutical industry

NASH is a leading cause of liver-related mortality and an increasing burden on healthcare systems globally.

Key Points: 
  • NASH is a leading cause of liver-related mortality and an increasing burden on healthcare systems globally.
  • Resmetirom received Breakthrough Therapy designation from the FDA and is under review to become the first medicine approved to treat patients with NASH with liver fibrosis.
  • Approximately 50% of patients treated with resmetirom 100 mg with biopsies at Week 52 showed either NASH resolution or fibrosis improvement.
  • More than 80% of patients with biopsies at Week 52 had either fibrosis reversal or no progression of fibrosis.

Telix Signs Agreement to Acquire QSAM Biosciences and Its Bone Cancer Targeting Platform

Retrieved on: 
Wednesday, February 7, 2024
PRV, Steroid, Prostate cancer, COGS, Contingent value rights, TLX, Palliative care, Osteosarcoma, Patient, Safety, Nephrotoxicity, QSAM, FDA, Appendix, Sarcoma, Food, Term sheet, Brain, Disease, Pain management, Acquisition, Metastasis, Samarium, ODD, Radiopharmaceutical, Aplastic anemia, Glioma, ASX, Priority review, Medical imaging

MELBOURNE, Australia, Feb. 08, 2024 (GLOBE NEWSWIRE) -- Telix Pharmaceuticals Limited (ASX: TLX, Telix, the Company) today announces it has entered into an agreement to acquire QSAM Biosciences, Inc. (U.S. OTC: QSAM) and its lead investigational drug Samarium-153-DOTMP (153Sm-DOTMP).

Key Points: 
  • MELBOURNE, Australia, Feb. 08, 2024 (GLOBE NEWSWIRE) -- Telix Pharmaceuticals Limited (ASX: TLX, Telix, the Company) today announces it has entered into an agreement to acquire QSAM Biosciences, Inc. (U.S. OTC: QSAM) and its lead investigational drug Samarium-153-DOTMP (153Sm-DOTMP).
  • QSAM is a United States (U.S.) based company developing therapeutic radiopharmaceuticals for primary and metastatic bone cancer.
  • Telix believes that 153Sm-DOTMP may benefit patients with metastatic lung and breast cancer, where many patients develop brain and bone metastases, and disease management often focusses on quality-of-life palliative care.
  • Telix will issue ordinary shares to the stockholders of QSAM at closing within its Listing Rule 7.1 placement capacity as consideration for the acquisition.

GSK’s RSV Vaccine, AREXVY, Accepted Under Priority Review in US for the Prevention of RSV Disease in Adults Aged 50-59 at Increased Risk

Retrieved on: 
Tuesday, February 6, 2024
Research, Infectious Diseases, Seniors, FDA, Clinical Trials, Biotechnology, Pharmaceutical, Consumer, Health, Science, GSK plc, Chronic obstructive pulmonary disease, Pneumonia, Respiratory syncytial virus vaccine, Safety, Hospital, US FDA, Ageing, Priority review, Food, COPD, Risk, RSV, Respiratory syncytial virus, Asthma, GSK, Pharmaceutical industry

GSK plc (LSE/NYSE: GSK) today announced that the US Food and Drug Administration (FDA) has accepted under Priority Review an application to extend the indication of GSK’s adjuvanted respiratory syncytial virus (RSV) vaccine to adults aged 50-59 who are at increased risk for RSV disease.

Key Points: 
  • GSK plc (LSE/NYSE: GSK) today announced that the US Food and Drug Administration (FDA) has accepted under Priority Review an application to extend the indication of GSK’s adjuvanted respiratory syncytial virus (RSV) vaccine to adults aged 50-59 who are at increased risk for RSV disease.
  • If approved, GSK’s RSV vaccine would be the first vaccine available to help protect this population.
  • AREXVY (Respiratory Syncytial Virus Vaccine, Adjuvanted) is currently approved in the US in adults aged 60 and over for the prevention of lower respiratory tract disease (LRTD) caused by RSV.
  • GSK used a Priority Review Voucher to reduce the US FDA review period of a supplemental Biologics License Application (sBLA) by four months.

Valneva Announces Sale of Priority Review Voucher for $103 Million

Retrieved on: 
Monday, February 5, 2024
VLA, PRV, Chikungunya, FDA, Euronext Paris, Saint-Herblain, Food, Disease, Vaccine, Risk, Lyme disease, Sale, Priority review, U.S. FDA, Pharmaceutical industry

Saint-Herblain (France), February 5, 2024 – Valneva SE (Nasdaq: VALN; Euronext Paris: VLA), a specialty vaccine company, today announced it sold the Priority Review Voucher (PRV) it received from the U.S. Food and Drug Administration (FDA) for $103 million (€95 million).

Key Points: 
  • Saint-Herblain (France), February 5, 2024 – Valneva SE (Nasdaq: VALN; Euronext Paris: VLA), a specialty vaccine company, today announced it sold the Priority Review Voucher (PRV) it received from the U.S. Food and Drug Administration (FDA) for $103 million (€95 million).
  • With this approval, IXCHIQ® became the world’s first licensed chikungunya vaccine available to address this unmet medical need.
  • As shown with the recent approval of our chikungunya vaccine, we remain committed to growing our portfolio of vaccines addressing unmet medical needs which have the potential to transform people’s lives.”
    Under the tropical Disease Priority Review Voucher Program, FDA awards priority review vouchers to sponsors of tropical disease product applications that meet certain criteria.
  • PRVs can be redeemed to receive priority review of a subsequent marketing application for a different product, sold or transferred.

Orchard Therapeutics Announces Multiple Presentations at the 20th Annual WORLDSymposium

Retrieved on: 
Friday, February 2, 2024
Abstract, Sanfilippo, Poster, Knowledge, MLD, Patient, Diagnosis, Priority review, Biologics license application, Food, Therapy, Kyowa Kirin, BLA, Flour

BOSTON and LONDON, Feb. 02, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, a global gene therapy leader recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, today announced 10 presentations from across its neurometabolic portfolio will be featured at the 20th Annual WORLDSymposium™ taking place February 4-9, 2024, in San Diego, California.

Key Points: 
  • BOSTON and LONDON, Feb. 02, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, a global gene therapy leader recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, today announced 10 presentations from across its neurometabolic portfolio will be featured at the 20th Annual WORLDSymposium™ taking place February 4-9, 2024, in San Diego, California.
  • Featured presentations include updated neurocognitive and biochemical results from an ongoing proof-of-concept study of investigational OTL-201 in mucopolysaccharidosis type IIIA (MPS-IIIA), also known as Sanfilippo syndrome type A, with median follow-up now extending to 2.75 years (ranging from 24 to 39 months).
  • Additional data highlights include several accepted abstracts detailing newborn screening efforts to support the timely and accurate diagnosis of metachromatic leukodystrophy (MLD), as well as an encore presentation showcasing long-term results from an updated integrated analysis of 39 patients with MLD treated with investigational OTL-200, which comprises key components of the clinical data package of the Biologics License Application (BLA) currently under Priority Review by the U.S. Food and Drug Administration.
  • In addition to the data presentations outlined below, the company will also host a sponsored symposium on Monday, February 5, at 11:45 a.m., titled “Momentum, Leadership & Diagnosis: Changing the paradigm for MLD,” featuring expert speakers from different regions sharing their knowledge and experience to establish new standards and advance newborn screening through interdisciplinary collaboration.

Appendix 4C Quarterly Activity Report for Quarter Ended December 31, 2023

Retrieved on: 
Wednesday, January 31, 2024
Congenital heart defect, FDA, Appendix, MSB, Journal, Bangladesh Technical Education Board, PRV, Survival, Randomized controlled trial, Biopharmaceutical, GVHD, Phases of clinical research, BLA, Hypoplastic left heart syndrome, RPD, HLHS, Growth, Patient, Blood, Inflammation, Priority review, Heart, NIH, Pain, Mesoblast, Ageing, BMT, Executive, MESO, Pharmaceutical industry

NEW YORK, Jan. 30, 2024 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today provided an activity report for the second quarter ended December 31, 2023.

Key Points: 
  • NEW YORK, Jan. 30, 2024 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today provided an activity report for the second quarter ended December 31, 2023.
  • Mesoblast will provide the Phase 3 trial protocol to FDA ahead of the upcoming meeting this quarter.
  • Mesoblast will meet with FDA this quarter to address potential pathways to approval for REVASCOR under our Regenerative Medicine Advanced Therapies (RMAT) designation.
  • Revenue from royalties on sales of TEMCELL® HS Inj.5 sold in Japan by our licensee for the quarter were US$1.5 million.

Regulatory Applications Accepted in the U.S. and Japan for Bristol Myers Squibb’s Breyanzi (lisocabtagene maraleucel) in Relapsed or Refractory Follicular Lymphoma (FL) and Relapsed or Refractory Mantle Cell Lymphoma (MCL)

Retrieved on: 
Tuesday, January 30, 2024
Biotechnology, Health, Pharmaceutical, Clinical Trials, Oncology, Follicular lymphoma, Mantle cell lymphoma, Bristol Myers Squibb, ICML, MCL, Clinical trial, Lymphocytosis, Patient, New Drug Application, Shanda, Lymphoma, FDA, BMY, Priority review, Food, Society, MHLW, PDUFA, ASH, NHL, Chronic lymphocytic leukemia, Conference, Pharmaceutical industry

In the U.S., the FDA has accepted the company’s two supplemental Biologics License Applications (sBLA) for Breyanzi to expand into new indications to include the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) and relapsed or refractory mantle cell lymphoma (MCL) after a Bruton tyrosine kinase inhibitor (BTKi).

Key Points: 
  • In the U.S., the FDA has accepted the company’s two supplemental Biologics License Applications (sBLA) for Breyanzi to expand into new indications to include the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) and relapsed or refractory mantle cell lymphoma (MCL) after a Bruton tyrosine kinase inhibitor (BTKi).
  • The FDA has granted both applications Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) goal date of May 23, 2024 for Breyanzi in relapsed or refractory FL and May 31, 2024 for Breyanzi in relapsed or refractory MCL.
  • Japan's MHLW has also accepted Bristol Myers Squibb’s supplemental New Drug Application (sNDA) for Breyanzi for the treatment of relapsed or refractory FL.
  • In both studies, Breyanzi demonstrated a consistent safety profile with no new safety signals reported.

Dupixent® (dupilumab) FDA Approved as First and Only Treatment Indicated for Children Aged 1 Year and Older with Eosinophilic Esophagitis (EoE)

Retrieved on: 
Thursday, January 25, 2024
Ageing, Regeneron Pharmaceuticals, Patient, Histology, Failure, Diagnosis, Medicine, Heartburn, Risk, Lower respiratory tract infection, Food, Arthralgia, Chronic pain, Safety, Disease, Esophagus, Inflammation, Helminthiasis, Priority review, FDA, Eosinophilic esophagitis, Prevalence, Vomiting, International development, Growth, Pharmaceutical industry

TARRYTOWN, N.Y. and PARIS, Jan. 25, 2024 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced that the U.S. Food and Drug Administration (FDA) has approved Dupixent® (dupilumab) for the treatment of pediatric patients aged 1 to 11 years, weighing at least 15 kg, with eosinophilic esophagitis (EoE). Dupixent is now the first and only medicine approved in the U.S. specifically indicated to treat these patients. This approval expands the initial FDA approval for EoE in May 2022 for patients aged 12 years and older, weighing at least 40 kg. The FDA evaluated Dupixent for this expanded indication under Priority Review, which is reserved for medicines that represent potentially significant improvements in efficacy or safety in treating serious conditions.

Key Points: 
  • Dupixent is now the first and only medicine approved in the U.S. specifically indicated to treat these patients.
  • This approval expands the initial FDA approval for EoE in May 2022 for patients aged 12 years and older, weighing at least 40 kg.
  • Approximately 21,000 children under the age of 12 in the U.S. are currently being treated for EoE with unapproved therapies.
  • “With this approval, Dupixent becomes the first and only treatment option for EoE patients aged 1 and older, weighing at least 15 kg.

Orchard Therapeutics Announces Agreement with the Beneluxa Consortium Enabling Reimbursed Access to Libmeldy

Retrieved on: 
Thursday, January 25, 2024
Death, Pharmaceutical policy, Patient, MHRA, Therapy, Busulfan, Health care, Civil service commission, European Commission, Health, Mutation, Food, MLD, Central nervous system, Traffic barrier, ARSA, FDA, Priority review, Genome, HSC, Nerve, PDUFA, Orchard, Pharmaceutical industry

BOSTON and LONDON, Jan. 25, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, a global gene therapy leader, today announced the company has reached an agreement with the Beneluxa Initiative on Pharmaceutical Policy (Beneluxa) enabling reimbursed access to Libmeldy® (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, approved for the treatment of early-onset metachromatic leukodystrophy (MLD).

Key Points: 
  • BOSTON and LONDON, Jan. 25, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, a global gene therapy leader, today announced the company has reached an agreement with the Beneluxa Initiative on Pharmaceutical Policy (Beneluxa) enabling reimbursed access to Libmeldy® (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, approved for the treatment of early-onset metachromatic leukodystrophy (MLD).
  • With more than a cumulative 250 patient-years of follow-up, Libmeldy was generally well-tolerated, with no treatment-related serious adverse events or deaths.
  • “We are pleased to have reached an agreement with the Beneluxa consortium that enables sustainable access to treatment.
  • Multiple eligible MLD patients are currently in the treatment process and expected to receive Libmeldy in the coming months.