Australia New Zealand Therapeutic Products Authority

Nanoscope Therapeutics Strengthens Clinical and Regulatory Expertise With Appointment of Samuel Barone, M.D., as Chief Medical Officer

Retrieved on: 
Thursday, September 21, 2023
University of California, San Diego Performance-Based Skills Assessment, New York Eye and Ear Infirmary, Adverum Biotechnologies, University, Therapy, Senior, Gene, Food and Drug Administration, Drug, Retina, AAV, Nanoscopic scale, Australia New Zealand Therapeutic Products Authority, Dermatology, Patient, Ophthalmology, Gemini, Air force, Entrepreneurship, Paratriathlon, Rare, FDA, Food, United States Department of the Air Force, Pharmaceutical industry, Medical device, DALLAS, Atomic de Broglie microscope

The regulatory expertise garnered during his tenure at the FDA will be instrumental as we continue to advance our pipeline through late-stage clinical development and navigate the regulatory process," said Sulagna Bhattacharya, Co-Founder and Chief Executive Officer of Nanoscope.

Key Points: 
  • The regulatory expertise garnered during his tenure at the FDA will be instrumental as we continue to advance our pipeline through late-stage clinical development and navigate the regulatory process," said Sulagna Bhattacharya, Co-Founder and Chief Executive Officer of Nanoscope.
  • "Dr. Barone will support our mission to bring sight-restoring therapies to patients with inherited retinal diseases in an expeditious manner."
  • Prior to that he was Chief Medical Officer for Veloce BioPharma, a topical therapeutics company focusing on unmet clinical needs in dermatology, ophthalmology and supportive oncology.
  • He was also Chief Medical Officer for Avalanche Biotechnologies and then Senior Vice President, Clinical Development, for Adverum Biotechnologies.

enCore Energy Provides Update on the South Texas Alta Mesa ISR Uranium Central Processing Plant and Wellfield

Retrieved on: 
Tuesday, September 5, 2023
Workforce, Maintenance, EU, GT, Australia New Zealand Therapeutic Products Authority, Encore, Rosita, ISR, Technische Universität Darmstadt, Environmental Defense v. Duke Energy Corp., NYSE, Uranium mining, Surveying, Alta Mesa AVA

DALLAS, Sept. 5, 2023 /PRNewswire/ - enCore Energy Corp. (NYSE American: EU) (TSXV: EU) (the "Company" or "enCore") today provides an update from the South Texas Alta Mesa In-Situ Recovery (ISR) Uranium Central Processing Plant and Wellfield.

Key Points: 
  • DALLAS, Sept. 5, 2023 /PRNewswire/ - enCore Energy Corp. (NYSE American: EU) (TSXV: EU) (the "Company" or "enCore") today provides an update from the South Texas Alta Mesa In-Situ Recovery (ISR) Uranium Central Processing Plant and Wellfield.
  • Site and production resumption work at the Rosita ISR Uranium Central Processing Plant and Wellfield (Rosita CPP) also remains on schedule for 2023 production.
  • The Alta Mesa ISR Uranium Central Processing Plant (Alta Mesa CPP) is advancing towards a restart of production and presently completing minor scheduled renovations with equipment upgrades and refurbishments underway.
  • To view the Alta Mesa project maps and enCore Energy's South Texas projects please visit: bit.ly/3fV9fTg .

Relief Therapeutics Announces Swissmedic Approval and Operation of New Good Manufacturing Practice-Compliant Laboratory

Retrieved on: 
Monday, May 15, 2023
GMP, Good manufacturing practice, Relief Therapeutics, International Council, OTCQB, Viscometer, ICH, Research, Australia New Zealand Therapeutic Products Authority, Relief, International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use, Documentation, CGMP, Patient, Agency, Rare, Harmonization, APR, Therapy, HPLC, Fine chemical, Pharmaceutical industry

Swissmedic is the national authorization and supervisory authority for drugs and medical products and devices.

Key Points: 
  • Swissmedic is the national authorization and supervisory authority for drugs and medical products and devices.
  • The next-generation laboratory in Balerna, Switzerland is run by Relief Therapeutics’ subsidiary APR Applied Pharma Research SA (APR) and offers a comprehensive suite of analytical and development services for internal projects and provides external clients with data and full documentation to meet current Good Manufacturing Practice (cGMP), International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use (ICH) and international regulatory authority requirements.
  • “GMP compliance is a critical achievement for our laboratory, enabling us to create, safeguard and supply high-quality products to patients and healthcare providers as well as enable the future submissions of product files to regulatory authorities,” said Paolo Galfetti, chief operating officer of Relief Therapeutics and chief executive officer of APR.
  • “We are excited that our new GMP-compliant lab in Balerna, where innovation is driven by our experienced and highly talented employees, is now Swissmedic-approved and operational,” said Jack Weinstein, chief executive officer of Relief Therapeutics.

Sarepta Therapeutics Announces First Quarter 2023 Financial Results and Recent Corporate Developments

Retrieved on: 
Tuesday, May 2, 2023
Science, Other Science, Biotechnology, Research, Pharmaceutical, Health, Genetics, Other Health, United, University, Duchenne muscular dystrophy, Gene, BioMarin Pharmaceutical, SRPT, Sarepta, Clinical trial, Sino-Japanese Journalist Exchange Agreement, GAAP, Part, RNA, Interest, Australia New Zealand Therapeutic Products Authority, PIN, Acquisition, Patient, Investment, Patent, Depreciation, Ageing, Rare, FDA, Therapy, Safety, BLA, Food, May 29, Pharmaceutical industry, Cryptocurrency, Fine chemical, Construction, Security (finance), Real estate, Bank, Note, Duchenne

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the first quarter 2023.

Key Points: 
  • Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the first quarter 2023.
  • If successful, Sarepta anticipates Part B to serve as the pivotal study for SRP-5051 and plans to seek accelerated approval for the candidate.
  • These non-GAAP measures are not intended to be considered in isolation or to replace the presentation of the Company’s financial results in accordance with GAAP.
  • All relevant non-GAAP measures are reconciled from their respective GAAP measures in the attached table “Reconciliation of GAAP Financial Measures to Non-GAAP Financial Measures.”

Sarepta Therapeutics Announces that the Date of the Advisory Committee Meeting for SRP-9001 will be May 12, 2023

Retrieved on: 
Monday, April 10, 2023
Research, FDA, Genetics, Clinical Trials, Biotechnology, Health, Other Science, Science, Oncology, SRPT, Australia New Zealand Therapeutic Products Authority, Sarepta, Center for Biologics Evaluation and Research, Rare, Therapy, Duchenne muscular dystrophy, Gene, BLA, Food, Pharmaceutical industry

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the date of the U.S. Food and Drug Administration’s Cellular, Tissue and Gene Therapies Advisory Committee meeting for the SRP-9001 (delandistrogene moxeparvovec) biologics license application (BLA) is May 12, 2023.

Key Points: 
  • Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the date of the U.S. Food and Drug Administration’s Cellular, Tissue and Gene Therapies Advisory Committee meeting for the SRP-9001 (delandistrogene moxeparvovec) biologics license application (BLA) is May 12, 2023.
  • The advisory committee meeting will be hosted as a virtual meeting.
  • “We look forward to sharing the wealth of evidence supporting the transformative potential of SRP-9001 for the treatment of Duchenne muscular dystrophy with the advisory committee on May 12, 2023,” said Doug Ingram, president and chief executive officer, Sarepta.
  • “We would again like to thank Center for Biologics Evaluation and Research and the Office of Therapeutic Products for working swiftly to schedule the advisory committee in advance of our regulatory action date of May 29, 2023.”

FDA Accepts Mesoblast’s Resubmission of the Biologic License Application for Remestemcel-L In Children with Steroid-Refractory Acute Graft Versus Host Disease as a Complete Response and Sets Goal Date of August 2, 2023

Retrieved on: 
Wednesday, March 8, 2023
MSB, Bone marrow, PDUFA, Australia New Zealand Therapeutic Products Authority, Biomarker, OTP, FDA, MESO, Safety, Agency, Survival, Priority review, BLA, Mesoblast, Inflammation, Pharmaceutical industry

FDA considers the resubmission to be a complete response and has set a Prescription Drug User Fee Act (PDUFA) goal date of August 2, 2023.

Key Points: 
  • FDA considers the resubmission to be a complete response and has set a Prescription Drug User Fee Act (PDUFA) goal date of August 2, 2023.
  • “Over the last two years we have worked tirelessly to address the issues previously raised by FDA.
  • We look forward to working closely with the Agency over the review period with the aim to make remestemcel-L available as a therapy for children suffering from SR-aGVHD,” said Mesoblast Chief Executive Silviu Itescu.
  • If remestemcel-L receives FDA approval, it will be the first allogeneic “off-the-shelf” cellular medicine to be approved in the United States, and the first therapy for children under 12 years old with SR-aGVHD.

Alimera Announces Submission of Marketing Authorization Application in Switzerland for ILUVIEN®

Retrieved on: 
Wednesday, February 22, 2023
Alimera Sciences, Australia New Zealand Therapeutic Products Authority, DME, Agency, Partnership, Diabetic retinopathy, Pharma, Patient, Eye, Physician, Pharmaceutical industry, Trosia, Horus

ATLANTA, Feb. 22, 2023 (GLOBE NEWSWIRE) -- Alimera Sciences, Inc. (Nasdaq: ALIM) (“Alimera”), a global pharmaceutical company whose mission is to be invaluable to patients, physicians and partners concerned with retinal health and maintaining better vision longer, announces that its distribution partner, Horus Pharma S.A.S.

Key Points: 
  • ATLANTA, Feb. 22, 2023 (GLOBE NEWSWIRE) -- Alimera Sciences, Inc. (Nasdaq: ALIM) (“Alimera”), a global pharmaceutical company whose mission is to be invaluable to patients, physicians and partners concerned with retinal health and maintaining better vision longer, announces that its distribution partner, Horus Pharma S.A.S.
  • (Horus) through its Swiss affiliate Horus Pharma Suisse SA, has filed a marketing authorization application dossier for ILUVIEN® for diabetic macular edema (DME) with the Swiss Agency for Therapeutic Products (Swissmedic).
  • “In our continued efforts for expanding our distribution, the partnership with Horus in Switzerland takes this critical first step toward an approval there,” said Rick Eiswirth, President and Chief Executive Officer of Alimera.
  • “Horus Pharma has been a close partner for us, with proven success selling ILUVIEN in other European markets, and has worked very quickly with our team to develop this dossier.”
    Claude Claret, CEO of Horus added, “Our teams are delighted to develop and expand their collaboration with Alimera.

Orchard Therapeutics Highlights Recent Progress Across HSC Gene Therapy Portfolio and Outlines Key 2023 Milestones

Retrieved on: 
Monday, January 9, 2023
IND, Disease, Agency, Therapy, Natural history, HSC, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Orchard, MLD, FDA, HAE, Type, Australia New Zealand Therapeutic Products Authority, Burn, Partnership, NOD2, U.S. FDA, Growth, Food, NBS, BLA, Patient, File, Investigational New Drug, Workforce, POC, Hereditary angioedema, Society, Investment, MAA, EU, Pharmaceutical industry, Medical device, Metachromatic leukodystrophy, Lancet

To date, over 170 patients have been treated with Orchard’s current and former HSC gene therapy programs across seven different diseases, reflecting the broad clinical applicability of the company’s approach.

Key Points: 
  • To date, over 170 patients have been treated with Orchard’s current and former HSC gene therapy programs across seven different diseases, reflecting the broad clinical applicability of the company’s approach.
  • “Orchard’s accomplishments in 2022 showcase the depth and strength of our neurometabolic portfolio,” said Bobby Gaspar, M.D., Ph.D., chief executive officer.
  • “We right sized our workforce and narrowed the focus of our HSC gene therapy portfolio to concentrate on severe neurometabolic diseases and research programs where alternative treatment options are limited or do not exist.
  • Refined the company’s HSC gene therapy portfolio and workforce to focus on severe neurometabolic diseases and pre-clinical research programs.

Orchard Therapeutics Announces Swissmedic Validation of the Marketing Authorization Application for Libmeldy (atidarsagene autotemcel)

Retrieved on: 
Thursday, December 1, 2022
United Kingdom, Literature, Australia New Zealand Therapeutic Products Authority, Therapy, Brain, Social media, Agency, Medication package insert, Risk, Patient, GSK, HSC, Late, Union, Infant, Dementia, ARSA, Royal commission, DOI, SAN, Systematic review, Nervous system, Seizure, Spasticity, MAA, Bone marrow, EMA, Partnership, Mortality, European Commission, Disease, Gallbladder, Liver, Safety, Multiple birth, Spleen, Mutation, Gene, MLD, File, Pharmaceutical industry, Child, Metachromatic leukodystrophy, EU, Orchard

The Swiss filing was based on the European Union (EU) MAA for Libmeldy, which was approved by the European Commission in December 2020.

Key Points: 
  • The Swiss filing was based on the European Union (EU) MAA for Libmeldy, which was approved by the European Commission in December 2020.
  • Over time, the nervous system is damaged, leading to neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures.
  • For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the EMA website.
  • At Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases.

GoodCell Launches in Concierge Practices to Meet the Demand for Proactive & Preventative Longevity Services

Retrieved on: 
Thursday, October 20, 2022
Primas, Quality of life, FACPM, Quest Diagnostics, Disease, Therapy, Science, Center for Biologics Evaluation and Research, OTP, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, FACP, Patient, Australia New Zealand Therapeutic Products Authority, Concierge medicine, PATH Biobank, Precision medicine, Food, Disorder, MD, Health, Blood, CEO, Genetic testing, Genetics, GLOBE, FDA, Medical device, Pharmaceutical industry, Tissue, Research

Atlanta, GA, Oct. 20, 2022 (GLOBE NEWSWIRE) -- At Concierge Medicine Todays annual Forum, GoodCell , a life sciences company, launched its Personal Biobanking service in concierge medicine practices nationwide.

Key Points: 
  • Atlanta, GA, Oct. 20, 2022 (GLOBE NEWSWIRE) -- At Concierge Medicine Todays annual Forum, GoodCell , a life sciences company, launched its Personal Biobanking service in concierge medicine practices nationwide.
  • As healthcare continues to move toward precision medicine, GoodCell will help to keep my patients at the cutting edge of science and ready for future cellular therapies.
  • GoodCell extracts and stores the most valuable components of the blood for future use in potential therapeutics and diagnostics.
  • We are thrilled to partner with concierge physicians across the country to deliver GoodCell directly to their patient populations, said Chris Garcia, CEO at GoodCell.