EC

CHMP Adopts Positive Opinion to Extend the Use of Veklury® (Remdesivir) to Treat COVID-19 in People With Hepatic Impairment

Retrieved on: 
Tuesday, September 19, 2023
Health, Infectious Diseases, Hospitals, COVID-19, Clinical Trials, Pharmaceutical, Biotechnology, Gilead Sciences, Liver disease, Safety, European Medicines Agency, Committee for Medicinal Products for Human Use, Pharmacokinetics, Virus, Emergent virus, Liver, European Commission, Committee, EMA, Hepatitis, Patient, Risk, European, Disease, Science, Mortality, EC, Infection, CHMP, Vaccine, Pharmaceutical industry, EU, MD, Remdesivir

Europe has the highest burden of liver disease in the world and cases are expected to grow across many countries.

Key Points: 
  • Europe has the highest burden of liver disease in the world and cases are expected to grow across many countries.
  • Safety data in people with severe hepatic impairment are limited and there are few treatment options that are safe and effective.
  • This positive opinion was based on results from a Phase 1 study of safety and pharmacokinetics in people with hepatic impairment (GS-US-540-9014).
  • “Expanding the use of Veklury to those with hepatic impairment will help more people gain access to treatment for COVID-19.”
    This positive opinion follows recent approvals in the U.S. and the EU which extended the approval of Veklury to treat COVID-19 in people with renal and hepatic impairment.

Pathways to Progress: How Orphan Drug Designation Sparks Biotech Transformation

Retrieved on: 
Wednesday, September 20, 2023
Atezolizumab, Diabetes, Neoplasm, Doctor of Philosophy, ODA, National library, Patient, GC, Disease, Prostate cancer, Carcinoma, CD47, Hope, Research, ACHL, Lung cancer, NLM, Orphan drug, Death, Drug development, Diagnosis, Growth, Rare, FDA, Legislation, Civil service commission, ODD, Roche, Orphan Drug Act of 1983, PharmaMar, Cancer, NSCLC, Treatment of lung cancer, ES, Therapy, Stomach, ORR, European Commission, File, United States National Library of Medicine, MedImmune, Inc. v. Genentech, Inc., Clinical trial, Woman, Lung, Fast track (FDA), SCLC, American Cancer Society, Achilles, Melanoma, OTCQX, Orphan, EC, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, FTD, RET, Food, Breast cancer, Pharmaceutical industry, Medical imaging, Genentech, Pembrolizumab

NEW YORK, Sept. 20, 2023 /PRNewswire/ -- More than 30 million people in the United States suffer with a rare disease; most of them are receiving no current treatment. Supporting the development and evaluation of new treatments, particularly for rare diseases, is a key priority for the U.S. Food & Drug Administration. As part of that process, the agency can grant Orphan Drug Designation (ODD) to a drug or biological product being developed to prevent, diagnose or treat a rare disease or condition. This designation is intended to spark innovation among biotech companies that are developing treatments for these patient populations, which by definition affect fewer than 200,000 people in the U.S., by providing incentives such as tax credits, user-fee exemptions and up to seven years of market exclusivity after FDA approval. Last month, Genprex Inc. (NASDAQ: GNPX) (Profile) was granted FDA orphan drug designation for its REQORSA(R) immunogene therapy in development for the treatment of small cell lung cancer (SCLC). The Phase 1/2 clinical trial, expected to dose the first patient in the fourth quarter of 2023, uses a combination of REQORSA and Genentech Inc.'s Tecentriq(R) as maintenance therapy in patients with extensive stage small cell lung cancer ("ES-SCLC") who did not develop tumor progression after receiving Tecentriq and chemotherapy as initial standard treatment. Genprex joins other companies — including Roche Holding AG ADR (OTCQX: RHHBY), Jazz Pharmaceuticals PLC (NASDAQ: JAZZ), ALX Oncology Holdings Inc. (NASDAQ: ALXO) and Achilles Therapeutics PLC (NASDAQ: ACHL) — that have been granted ODD status or may seek ODD status as they work to develop treatments for rare diseases.

Key Points: 
  • Four decades after the Orphan Drug Act was passed, hundreds of 'orphan' drugs have been approved for use.
  • Genprex's REQORSA Immunogene Therapy has gained both Orphan Drug Designation and Fast Track Designation.
  • Specifically, the report noted that 6,340 orphan drug designations were granted, representing drug development for 1,079 rare diseases.
  • For companies looking to help those in the underserved rare-disease space, an Orphan Drug Designation is the ideal pathway to progress.

Pathways to Progress: How Orphan Drug Designation Sparks Biotech Transformation

Retrieved on: 
Wednesday, September 20, 2023
Atezolizumab, Diabetes, Neoplasm, Doctor of Philosophy, ODA, National library, Patient, GC, Disease, Prostate cancer, Carcinoma, CD47, Hope, Research, ACHL, Lung cancer, NLM, Orphan drug, Death, Drug development, Diagnosis, Growth, Rare, FDA, Legislation, Civil service commission, ODD, Roche, Orphan Drug Act of 1983, PharmaMar, Cancer, NSCLC, Treatment of lung cancer, ES, Therapy, Stomach, ORR, European Commission, File, United States National Library of Medicine, MedImmune, Inc. v. Genentech, Inc., Clinical trial, Woman, Lung, Fast track (FDA), SCLC, American Cancer Society, Achilles, Melanoma, OTCQX, Orphan, EC, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, FTD, RET, Food, Breast cancer, Pharmaceutical industry, Medical imaging, Genentech, Pembrolizumab

NEW YORK, Sept. 20, 2023 /PRNewswire/ -- More than 30 million people in the United States suffer with a rare disease; most of them are receiving no current treatment. Supporting the development and evaluation of new treatments, particularly for rare diseases, is a key priority for the U.S. Food & Drug Administration. As part of that process, the agency can grant Orphan Drug Designation (ODD) to a drug or biological product being developed to prevent, diagnose or treat a rare disease or condition. This designation is intended to spark innovation among biotech companies that are developing treatments for these patient populations, which by definition affect fewer than 200,000 people in the U.S., by providing incentives such as tax credits, user-fee exemptions and up to seven years of market exclusivity after FDA approval. Last month, Genprex Inc. (NASDAQ: GNPX) (Profile) was granted FDA orphan drug designation for its REQORSA(R) immunogene therapy in development for the treatment of small cell lung cancer (SCLC). The Phase 1/2 clinical trial, expected to dose the first patient in the fourth quarter of 2023, uses a combination of REQORSA and Genentech Inc.'s Tecentriq(R) as maintenance therapy in patients with extensive stage small cell lung cancer ("ES-SCLC") who did not develop tumor progression after receiving Tecentriq and chemotherapy as initial standard treatment. Genprex joins other companies — including Roche Holding AG ADR (OTCQX: RHHBY), Jazz Pharmaceuticals PLC (NASDAQ: JAZZ), ALX Oncology Holdings Inc. (NASDAQ: ALXO) and Achilles Therapeutics PLC (NASDAQ: ACHL) — that have been granted ODD status or may seek ODD status as they work to develop treatments for rare diseases.

Key Points: 
  • Four decades after the Orphan Drug Act was passed, hundreds of 'orphan' drugs have been approved for use.
  • Genprex's REQORSA Immunogene Therapy has gained both Orphan Drug Designation and Fast Track Designation.
  • Specifically, the report noted that 6,340 orphan drug designations were granted, representing drug development for 1,079 rare diseases.
  • For companies looking to help those in the underserved rare-disease space, an Orphan Drug Designation is the ideal pathway to progress.

EBGLYSS® (lebrikizumab) Receives Positive CHMP Opinion for Moderate-to-severe Atopic Dermatitis

Retrieved on: 
Friday, September 15, 2023
Biotechnology, Other Health, Health, Pharmaceutical, Clinical Trials, Trinity College, Maintenance, Eczema Area and Severity Index, Disease, Civil service commission, AE, Week, Dermatitis, European Medicines Agency, Adherence, Committee for Medicinal Products for Human Use, Quality of life, Atopic dermatitis, Almirall, TCS, Committee, EMA, Trinity College Dublin, BME, Advocate, ALM, Patient, European, Adolescence, United Kingdom, Eli Lilly and Company, Itch, Hospital, AD, EudraCT, Conjunctivitis, EC, Human, Skin, European Commission, Safety, CHMP, Vaccine, Pharmaceutical industry, Dermatology

The positive CHMP opinion is now being reviewed by the European Commission (EC).

Key Points: 
  • The positive CHMP opinion is now being reviewed by the European Commission (EC).
  • “Atopic dermatitis, commonly known as atopic eczema, can have a profound impact on the quality of life for those it affects.
  • "The positive CHMP recommendation for EBGLYSS in moderate to severe AD represents a significant milestone in bringing a next-generation biologic therapy to people living with atopic dermatitis, providing a much needed additional treatment option.
  • Almirall has licensed the rights to develop and commercialize lebrikizumab for the treatment of dermatology indications, including atopic dermatitis, in Europe.

CHMP Delivers Opinion on Translarna™ for Conversion to Full Authorization

Retrieved on: 
Friday, September 15, 2023
PTC Therapeutics, Duchenne muscular dystrophy, CHMP-FM, PTC, European Medicines Agency, Committee for Medicinal Products for Human Use, Committee, EMA, DMD, Patient, STRIDE, Therapy, EC, News, Disease, CHMP, Vaccine, Dietary supplement, Ataluren

SOUTH PLAINFIELD, N.J., Sept. 15, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has given a negative opinion on the conversion of the conditional marketing authorization to full marketing authorization of Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). The negative opinion also applies to the renewal of the existing conditional authorization. PTC plans to submit a request for re-examination per EMA guidelines. Translarna will remain on the market and available to patients with nmDMD until the re-examination process is completed. Based on CHMP procedural guidance, the opinion following the re-examination process would be expected to occur in January 2024, with EC ratification of the opinion within the following 67 days.

Key Points: 
  • The negative opinion also applies to the renewal of the existing conditional authorization.
  • Based on CHMP procedural guidance, the opinion following the re-examination process would be expected to occur in January 2024, with EC ratification of the opinion within the following 67 days.
  • We will be submitting a request for re-examination to the CHMP to reverse this opinion, as we have done previously in the regulatory history of Translarna in Europe."
  • Translarna received conditional marketing authorization in Europe in 2014 based on the results of Study 007, and the conditional authorization was renewed in 2017.

Ascendis Pharma Launches SKYTROFA® (lonapegsomatropin) in Germany for the Once-Weekly Treatment of Children and Adolescents with Growth Hormone Deficiency

Retrieved on: 
Friday, September 15, 2023
Acceleration, GHD, US Foods, Civil service commission, Growth hormone, Southern Transcon, European, FDA, EC, European Commission, Birth control

COPENHAGEN, Denmark, Sept. 15, 2023 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced the launch in Germany of SKYTROFA® (lonapegsomatropin), its growth hormone approved in the European Union for the once-weekly treatment of children and adolescents ages 3 to 18 years with growth failure due to insufficient endogenous growth hormone secretion (growth hormone deficiency, or GHD).

Key Points: 
  • COPENHAGEN, Denmark, Sept. 15, 2023 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced the launch in Germany of SKYTROFA® (lonapegsomatropin), its growth hormone approved in the European Union for the once-weekly treatment of children and adolescents ages 3 to 18 years with growth failure due to insufficient endogenous growth hormone secretion (growth hormone deficiency, or GHD).
  • “SKYTROFA is designed to deliver unmodified somatropin, the same growth hormone used in daily pediatric GHD treatments for more than 30 years, and we are pleased now to make it available in Germany,” said Camilla Harder Hartvig, Executive Vice President and Global Chief Commercial Officer at Ascendis Pharma.
  • “This first European Union launch of SKYTROFA begins acceleration of its commercialization beyond the U.S. as we work to expand our leadership to new geographies.”
    SKYTROFA (developed as TransCon hGH) was approved for pediatric GHD by the US Food & Drug Administration (FDA) in 2021 and by the European Commission (EC) in 2022.

argenx Announces Positive CHMP Opinion for Subcutaneous Efgartigimod for Generalized Myasthenia Gravis

Retrieved on: 
Friday, September 15, 2023
1991 World Sportscar Championship, IV, Marketing, SC, GMG, European Medicines Agency, Committee for Medicinal Products for Human Use, Quality of life, Committee, EMA, Euronext, Patient, Physician, ADAPT, Autoimmune disease, Ghent University, Immunoglobulin G, SE, Acetylcholine receptor, Ghent University Hospital, EC, CHMP, Pharmaceutical industry, Vaccine

SC efgartigimod is formulated with Halozyme’s ENHANZE® drug delivery technology to facilitate subcutaneous delivery of biologics.

Key Points: 
  • SC efgartigimod is formulated with Halozyme’s ENHANZE® drug delivery technology to facilitate subcutaneous delivery of biologics.
  • “Generalized myasthenia gravis is a complex and devastating disease that is debilitating for people who live with it, making routine movements exhausting and challenging to perform,” said Prof. dr. Jan De Bleecker, Ghent University Hospital and Ghent University.
  • The positive CHMP opinion is a scientific recommendation for marketing authorization, serving as a basis for the EC’s final decision on argenx’s application for SC efgartigimod.
  • The decision will apply to all 27 European Union Member States, and also to Iceland, Norway and Liechtenstein.

Santhera Announces Half-Year 2023 Financial Results and Provides Corporate Update

Retrieved on: 
Thursday, September 7, 2023
Vamorolone, Marketing, European Medicines Agency, Lead, Prescription Drug User Fee Act, Committee, U.S. FDA, Patient, Committee for Medicinal Products for Human Use, News, Duchenne muscular dystrophy, Glucocorticoid, Becker muscular dystrophy, European Commission, EMA, CEST, PDUFA, Rare, FDA, Safety, Health care, LR, Duchenne, Publication, MAA, MHRA, CHMP, Catalyst Pharmaceuticals, Partnership, European Directive on Traditional Herbal Medicinal Products, NDA, BMD, EC, BST, Medical device, Pharmaceutical industry, Security (finance), Idebenone, EU, Chiesi Farmaceutici, LHON, Atlantic Ocean, Catalysis, DMD

Furthermore, we have secured a solid financial footing which allows us to press ahead toward our goal of European commercialization,” said Dario Eklund, CEO of Santhera.

Key Points: 
  • Furthermore, we have secured a solid financial footing which allows us to press ahead toward our goal of European commercialization,” said Dario Eklund, CEO of Santhera.
  • Total consideration to Santhera is up to USD 231 million (including equity investment) plus royalty payments from product sales.
  • After closing of the transaction in July 2023, Santhera received an upfront payment of USD 90 million (USD 75 million in cash and USD 15 million equity investment).
  • During the six months ending June 30, 2023, Santhera committed to the sale of Raxone/idebenone upon settlement of the French case and the transaction closed on July 28, 2023.

Smartapp ONETM for SAP® Solutions Now Available on SAP® Store as Part of SAP’s Industry Cloud Portfolio

Retrieved on: 
Tuesday, September 12, 2023
Software, Internet, Hardware, Data Management, Apps, Applications, Other Construction & Property, Technology, Residential Building & Real Estate, Commercial Building & Real Estate, Construction & Property, Engineering, Manufacturing, Cloud, Partnership, WBS, Head, Construction, ONE, ERP, SAP, CBS, Forecasting, Automation, Ecosystem, Financial management, EC, Real estate, Sales, Video game, Risk management

Smartapp.com TM announced today that its Smartapp ONETM for SAP® Solutions is now available on SAP Store and is part of SAP’s industry cloud portfolio for Construction and Real Estate companies.

Key Points: 
  • Smartapp.com TM announced today that its Smartapp ONETM for SAP® Solutions is now available on SAP Store and is part of SAP’s industry cloud portfolio for Construction and Real Estate companies.
  • Smartapp.com leverages the integration capabilities of SAP Business Technology Platform (SAP BTP) to integrate with SAP S/4HANA® Cloud.
  • Smartapp ONE™ for SAP® Solutions delivers a high-value vertical solution that connects jobsite activities to the back-office and bridges the gap between FINANCE and FIELD in ONE integrated automation platform.
  • Partnering with SAP, we’re able to bring together the very best of both worlds - jobsite automation and ERP platforms!

European Commission Expands Merck’s ERVEBO® [Ebola Zaire Vaccine, (rVSVΔG-ZEBOV-GP) live] Indication to Include Children 1 Year of Age and Older

Retrieved on: 
Thursday, September 7, 2023
Biotechnology, General Health, Infectious Diseases, Health, Pharmaceutical, MSD, Vaccination, Chills, Public, Anaphylaxis, Arthralgia, Rash, Committee, Milk, Plasma, Risk, Committee for Medicinal Products for Human Use, European, EVD, NYSE, Saliva, Fatigue, Disease, Antigen, Pregnancy, Diarrhea, Urine, Somnolence, MRK, Appetite, UNICEF, Merck & Co., Myalgia, Vomiting, Child, Immunodeficiency, Headache, Ebola, Irritability, Mouth, Safety, Screaming, Fever, Civil service commission, Arthritis, History, Abdominal pain, Hyperhidrosis, IG, Allergy, Dizziness, Blood, European Medicines Agency, European Commission, Breast milk, CHMP, Infection, European Directive on Traditional Herbal Medicinal Products, RNA, Nausea, EC, Vaccine, Birth control, Merck, EU, Ebolavirus, Marburgvirus

The vaccine was previously approved for use in the European Union (EU) for individuals 18 years of age or older.

Key Points: 
  • The vaccine was previously approved for use in the European Union (EU) for individuals 18 years of age or older.
  • “Ebola virus disease is severe and potentially life-threatening for both children and adults.
  • INDICATIONS AND USAGE IN THE U.S.
    ERVEBO® is indicated for the prevention of disease caused by Zaire ebolavirus in individuals 12 months of age and older.
  • Effectiveness of the vaccine when administered concurrently with antiviral medication, immune globulin (IG), and/or blood or plasma transfusions is unknown.