Next-generation non-cardiotoxic anthracycline Annamycin with notable signs of clinical efficacy in AML in combination with Cytarabine achieves preliminary CRc rate of 60% in 2nd line AML subjects in a European clinical trial There are approximately 160,000 people with AML worldwide Annamycin continues to show no signs of cardiotoxicity (N=82 across multiple studies); Lower toxicity profile than traditional intensive...Read more
MALVERN, Pa., April 10, 2024 (GLOBE NEWSWIRE) -- Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, and vaccines, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) reviewed the study design, endpoints and planned statistical analysis of the pivotal OCU400...Read more
Randomized Study with 18 Weeks Continuous Treatment Expected to Replicate and Extend Results from the First, Statistically Significant Phase 3 Study PRINCETON, N.J., April 3, 2024 /PRNewswire/ -- Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced today that it has...Read more
Approval of Reblozyl is based on head-to-head, pivotal Phase 3 COMMANDS study, in which Reblozyl nearly doubled the percentage of patients achieving transfusion independence and hemoglobin increase, along with increased durability compared to epoetin alfa This is the fourth authorized indication in Europe for Reblozyl, a first-in-class treatment for patients with disease-related anemia and the first therapy to demonstrate superior...Read more
Submission includes results from pivotal Phase 2 FELIX study evaluating obe-cel in r/r B-ALL The US marketing application is under review with a Prescription Drug User Fee Act (PDUFA) target action date of November 16, 2024 LONDON, April 02, 2024 (GLOBE NEWSWIRE) -- Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, today announces that the...Read more
Positive, previously disclosed results from the global Phase 1/2 trial demonstrated genetic and phenotypic correction combined with hematologic stabilization extending out to 42 months after treatment with RP-L102 CRANBURY, N.J. / Apr 02, 2024 / Business Wire / Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with...Read more
First marketing authorization submission for sepiapterin with additional global submissions to follow in 2024 SOUTH PLAINFIELD, N.J., March 28, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the submission of the sepiapterin MAA to the European Medicines Agency. The MAA submission is for the treatment of pediatric and adult patients with PKU, including the full spectrum of disease...Read more
Decision marks the first approval in Europe for an anti-PD-1/L1 therapy in resectable NSCLC based on positive overall survival results and represents the sixth approval for KEYTRUDA in lung cancer in Europe RAHWAY, N.J. / Mar 28, 2024 / Business Wire / Merck (NYSE: MRK), known as MSD outside of the United States and Canada, today announced that the European Commission (EC) has approved KEYTRUDA, Merck’s anti-PD-1 therapy, in...Read more
PRIME status is supported by positive data from the Phase 2b ENLIVEN trial of pegozafermin Phase 3 ENLIGHTEN-Fibrosis trial in non-cirrhotic MASH (fibrosis stage F2-F3) patients is enrolling and ENLIGHTEN-Cirrhosis in MASH patients with compensated cirrhosis (F4) is planned to initiate in the second quarter of 2024 SAN FRANCISCO, March 27, 2024 (GLOBE NEWSWIRE) -- 89bio, Inc. (Nasdaq: ETNB), a clinical-stage biopharmaceutical...Read more
San Diego, California--(Newsfile Corp. - March 25, 2024) - Thiogenesis Therapeutics, Corp. (TSXV: TTI) ("Thiogenesis" or the "Company") a clinical-stage biotechnology company developing disulfides that are precursors to thiol-active compounds and that have the potential to treat unmet pediatric diseases, today announced that the European Medicines Agency ("EMA") has accepted its Clinical Trial Application ("CTA") Part I - Scientific and...Read more
Positive CHMP Opinions Are Based On The Analyses Of The Phase 3 Of The CLEAR (Cholesterol Lowering via Bempedoic Acid, an ATP Citrate Lyase (ACL)-Inhibiting Regimen) Outcomes Trial1 European Commission Decision On Label Update Authorization Applications Are Expected To Be Made In 1H 2024 ANN ARBOR, Mich. and MUNICH, Germany, March 22, 2024 (GLOBE NEWSWIRE) -- Daiichi Sankyo Europe GmbH (hereafter, ‘Daiichi Sankyo’) and Esperion...Read more
Positive opinion serves as a basis for final decision for potential authorization from the European Commission (EC), expected within 67 days ISELIN, N.J., March 22, 2024 (GLOBE NEWSWIRE) -- Outlook Therapeutics, Inc. (Nasdaq: OTLK), a biopharmaceutical company working to achieve the first approval for an ophthalmic formulation of bevacizumab for the treatment of retinal diseases in the US and the EU, today announced that the...Read more
Bagsværd, Denmark, 21 March 2024 – Novo Nordisk (NYSE: NVO) today announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion, recommending marketing authorisation for Awiqli® (the brand name for once-weekly basal insulin icodec) for treatment of diabetes in adults. The positive CHMP opinion is based on results from the ONWARDS phase 3a clinical trial...Read more
Abecma demonstrated superiority over standard regimens in the Phase 3 KarMMa-3 trial, with a 51% reduction in risk of disease progression or death and a well-established safety profile with mostly low-grade and transient occurrences of cytokine release syndrome and neurotoxicity Approval reinforces Bristol Myers Squibb’s commitment to bring the transformative potential of cell therapy into earlier lines of treatment PRINCETON N.J. /...Read more
The Company will release important advanced new features, such as Pulse Wave Doppler and Auto B-Line Counter to European Butterfly iQ+ devices in the coming months. The certification also serves as the foundation for Butterfly’s active pursuit to launch its new Butterfly iQ3 device in Europe later this year. BURLINGTON, Mass. & NEW YORK / Mar 20, 2024 / Business Wire / Butterfly Network, Inc. (“Butterfly”, “the Company”) (NYSE:...Read more
IRVINE, Calif. / Mar 14, 2024 / Business Wire / Axonics, Inc. (Nasdaq: AXNX), a global medical technology company that is developing and commercializing novel products for the treatment of bladder and bowel dysfunction, today announced that the Axonics R20™ rechargeable sacral neuromodulation (SNM) system has received CE Mark approval. The R20 neurostimulator is labeled for a functional life in the body of at least 20 years and reduces how...Read more
PREVENAR 20® (20-valent Pneumococcal Conjugate Vaccine) offers the broadest serotype coverage of any pediatric pneumococcal conjugate vaccine to help protect infants and children from the 20 serotypes responsible for the majority of currently circulating pneumococcal disease in the EU and globally1,2,3,4,5,6,7,8 NEW YORK / Mar 13, 2024 / Business Wire / Pfizer Inc. (NYSE: PFE) today announced that the European Commission (EC) has...Read more
MAA submission is supported by positive results from MAESTRO-NASH, the only Phase 3 trial in NASH to achieve fibrosis reduction and NASH resolution primary endpoints Resmetirom new drug application is currently under review with the FDA with a PDUFA date of March 14, 2024 CONSHOHOCKEN, Pa., March 05, 2024 (GLOBE NEWSWIRE) -- Madrigal Pharmaceuticals, Inc. (NASDAQ:MDGL), a clinical-stage biopharmaceutical company pursuing novel...Read more
NEWARK, Calif., March 04, 2024 (GLOBE NEWSWIRE) -- CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a biopharmaceutical company focused on innovative therapies for patients with liver and other chronic diseases, today announced that the Marketing Authorization Application (MAA) for seladelpar, for the treatment of primary biliary cholangitis (PBC) including pruritus in adults without cirrhosis or with compensated cirrhosis (Child Pugh A) who...Read more
BridgeBio grants Bayer exclusive license to commercialize acoramidis as a treatment for patients with transthyretin amyloid cardiomyopathy (ATTR-CM) in Europe BridgeBio to receive royalties according to a tiered structure beginning in the low-thirties percent, designed to provide BridgeBio the opportunity to maximally share in the blockbuster potential of acoramidis BridgeBio will also receive up to $310 Million USD in upfront and...Read more
STAMFORD, Conn., Feb. 29, 2024 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, announced today that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for nirogacestat, an oral gamma secretase inhibitor, for the treatment of adults with desmoid tumors. If approved, nirogacestat will be...Read more
Recommendation based on results of three Phase 3 clinical trials demonstrating benefit of tislelizumab as a first- and second-line treatment for patients with NSCLC BASEL, Switzerland & BEIJING & CAMBRIDGE, Mass. / Feb 26, 2024 / Business Wire / BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global oncology company, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European...Read more
CARVYKTI® is the first CAR-T therapy for patients with relapsed and refractory multiple myeloma to receive a positive CHMP opinion for second line treatment SOMERSET, N.J. / Feb 23, 2024 / Business Wire / Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global leader in cell therapy, announced today the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended expansion of...Read more
Committee for Medicinal Products for Human Use (CHMP) recommends approval of the conditional marketing authorization (CMA) for sparsentan for the treatment of IgA nephropathy (IgAN) in Europe Positive CHMP opinion is based on pivotal Phase 3 PROTECT Study results European Commission decision is expected in Q2 2024 SAN DIEGO, Feb. 23, 2024 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc., (NASDAQ: TVTX) and CSL Vifor today announced...Read more
SOD1-ALS is a devastating, uniformly fatal, and ultra-rare genetic form of ALS affecting less than 1,000 people in Europe1 With QALSODY, Biogen has advanced the role of neurofilament in the development of new medicines for ALS, with the potential to accelerate further discovery in the field CAMBRIDGE, Mass., Feb. 23, 2024 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) announced the Committee for Medicinal Products for Human Use...Read more
Approval by European Commission would expand Reblozyl’s indication to include first-line treatment of anemia in adults with MDS in Europe In the pivotal Phase 3 COMMANDS study, Reblozyl nearly doubled the percentage of patients achieving primary endpoint of both transfusion independence and hemoglobin increase vs. epoetin alfa PRINCETON, N.J. / Feb 23, 2024 / Business Wire / Bristol Myers Squibb (NYSE: BMY) today announced the...Read more
Opinion granted based on positive overall survival and event-free survival results from the Phase 3 KEYNOTE-671 trial First opinion to be granted for an anti-PD-1/L1 therapy in resectable non-small cell lung cancer (NSCLC) based on positive overall survival results RAHWAY, N.J. / Feb 23, 2024 / Business Wire / Merck (NYSE: MRK), known as MSD outside of the United States and Canada, today announced that the European Medicines Agency’s...Read more
Committee for Medicinal Products for Human Use (CHMP) recommends approval of the conditional marketing authorization (CMA) for sparsentan for the treatment of IgA nephropathy (IgAN) in Europe Positive CHMP opinion is based on pivotal phase-III PROTECT study results European Commission decision is expected in Q2 2024 ST. GALLEN, Switzerland, Feb. 23, 2024 /CNW/ -- CSL Vifor and Travere Therapeutics, Inc., (NASDAQ: TVTX) today...Read more
Access to protocol assistance, centralized authorization process, fee reductions and 10 years of market exclusivity SAN DIEGO / Feb 22, 2024 / Business Wire / Arcturus Therapeutics Holdings Inc. (the “Company”, “Arcturus”, Nasdaq: ARCT), a global late-stage clinical messenger RNA medicines company focused on the development of infectious disease vaccines and medicines to treat unmet medical needs within liver and respiratory rare...Read more
TORONTO, Feb. 22, 2024 (GLOBE NEWSWIRE) -- Venus Concept Inc. (“Venus Concept” or the “Company”) (NASDAQ: VERO), a global medical aesthetic technology leader, today announced that it has received the CE Mark from DEKRA Certification B.V. to market the Venus Versa Pro system in the European Union. This follows the Company’s announcement on November 1, 2023 regarding the commercial launch of the device in the United...Read more
STAFFORD, Texas, Feb. 22, 2024 (GLOBE NEWSWIRE) -- Greenwich LifeSciences, Inc. (Nasdaq: GLSI) (the "Company"), a clinical-stage biopharmaceutical company focused on its Phase III clinical trial, Flamingo-01, which is evaluating GLSI-100, an immunotherapy to prevent breast cancer recurrences in patients who have previously undergone surgery, today provided the following update on the expansion of the clinical trial into Europe. The...Read more
VELSIPITY is the first and only oral advanced ulcerative colitis therapy approved for use in patients 16 years of age or older in the EU NEW YORK / Feb 19, 2024 / Business Wire / Pfizer Inc. (NYSE: PFE) announced today that the European Commission (EC) has granted marketing authorization for VELSIPITY® (etrasimod) in the European Union to treat patients 16 years of age and older with moderately to severely active ulcerative colitis...Read more
Over 8,000 patients 12 years of age and older with severe sickle cell disease or transfusion-dependent beta thalassemia may be eligible for treatment LONDON / Feb 13, 2024 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the European Commission has granted conditional marketing authorization to CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy. CASGEVY is approved...Read more
Friedreich’s ataxia is a rare, genetic, life-shortening, debilitating, and neurodegenerative disorder Treatment with SKYCLARYS improved patient function compared to placebo Biogen is leveraging its expertise and capabilities in rare disease to bring this groundbreaking treatment to patients CAMBRIDGE, Mass., Feb. 12, 2024 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) announced the European Commission (EC) has authorized...Read more
U.S. Food and Drug Administration (FDA) advises that a single study with up to 25 patients, in combination with confirmatory evidence, may be acceptable pathway to Biologics License Application (BLA) submission for isaralgagene civaparvovec, which would significantly reduce anticipated complexity, cost and time to potential approval. European Medicines Agency (EMA) granted priority medicines (PRIME) eligibility to isaralgagene...Read more
European Orphan Drug Designation (“ODD”) qualifies NXC-201 for: 10 years of market exclusivity once authorized in the EU Access to the EU centralized authorization procedure Reduced fees for EU protocol assistance, marketing authorization applications, inspections before authorization, applications for changes to marketing authorizations made after approval, and reduced annual fees U.S. observed prevalence of relapsed/refractory...Read more
Applications based on results from CheckMate -77T, the company’s second positive Phase 3 randomized trial with an immunotherapy-based combination for the treatment of non-metastatic non-small cell lung cancer The perioperative Opdivo-based regimen demonstrated significant improvement in event-free survival compared to neoadjuvant chemotherapy and placebo followed by surgery and adjuvant placebo The U.S. Food and Drug Administration...Read more
Omnipod 5 is the world’s first tubeless automated insulin delivery system to achieve CE mark approval with multiple continuous glucose monitoring (CGM) sensor brands. Latest Omnipod 5 integration is expected to be available in the United Kingdom and Netherlands in the first half of 2024, with additional markets to follow. ACTON, Mass. / Feb 07, 2024 / Business Wire / Insulet Corporation (NASDAQ: PODD) (Insulet or the Company), the...Read more
GTX-102 is the first Angelman syndrome therapeutic candidate to receive PRIME designation Phase 1/2 study fully enrolled; expansion data expected in first half of 2024 NOVATO, Calif., Feb. 05, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the European Medicines Agency (EMA) has granted Priority Medicine (PRIME) designation to GTX-102 for the treatment of Angelman syndrome (AS). GTX-102...Read more
TARRYTOWN, N.Y., Feb. 02, 2024 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that the European Medicines Agency (EMA) has accepted for review the Marketing Authorization Application (MAA) for linvoseltamab to treat adult patients with relapsed/refractory (R/R) multiple myeloma (MM) who have progressed after at least three prior therapies. Linvoseltamab is an investigational bispecific...Read more
Validation is supported by data from Phase 3 innovaTV 301 trial COPENHAGEN, Denmark / Feb 02, 2024 / Business Wire / Genmab A/S (Nasdaq: GMAB) and Pfizer, Inc. (NYSE PFE) today announced that the European Medicines Agency (EMA) has validated for review the marketing authorization application (MAA) of tisotumab vedotin, an antibody-drug conjugate (ADC), developed for the treatment of adult patients with recurrent or metastatic cervical...Read more
CEL-SCI has cleared a significant hurdle that mandates cancer drugs in Europe comply with strict requirements for pediatric usage and clinical evidence VIENNA, Va. / Jan 31, 2024 / Business Wire / CEL-SCI Corporation (NYSE American: CVM) today announced that the European Medicines Agency (EMA) Paediatric Committee granted CEL-SCI a product-specific waiver of strict requirements for commercialization of cancer drugs in the European...Read more
TEL AVIV, Israel and MIAMI, Jan. 31, 2024 (GLOBE NEWSWIRE) -- InspireMD, Inc. (Nasdaq: NSPR), developer of the CGuard™ Embolic Prevention Carotid Stent System (EPS) for the prevention of stroke, today announced that it has received CE Mark recertification under the European Union’s new Medical Device Regulation (MDR) regulatory framework. MDR replaced the previous MDD framework, which had governed the approval and marketing of medical...Read more
HYQVIA [Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase] becomes the Only Facilitated Subcutaneous Immunoglobulin, Offering Patients an up to Once-Monthly Treatment Option At-Home or In-Office Administration Provides CIDP Patients with a Personalized Treatment Experience Approval Expands Takeda’s Portfolio of Differentiated Immunoglobulin Therapies for Patients with Neuroimmunological Disorders OSAKA, Japan...Read more
Pivotal trial found the enfortumab vedotin plus pembrolizumab combination significantly extended overall and progression-free survival If approved, PADCEV with KEYTRUDA would be the first combination in the EU to offer an alternative to platinum-containing chemotherapy, the current standard of care in first-line locally advanced or metastatic urothelial cancer TOKYO and NEW YORK, Jan. 26, 2024 /PRNewswire/ -- Astellas Pharma Inc....Read more
Recommendation for approval based on Phase 3 KarMMa-3 study in which Abecma demonstrated superiority over standard regimens, significantly improved progression-free survival and a well-established safety profile with mostly low-grade occurrences of cytokine release syndrome and neurotoxicity This is the first positive CHMP opinion in earlier lines of therapy for a chimeric antigen receptor (CAR) T cell therapy in the treatment of...Read more
WALTHAM, Mass., Jan. 26, 2024 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a negative opinion on the marketing authorization application (MAA) of intravitreal pegcetacoplan for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). As previously announced,...Read more
SOUTH PLAINFIELD, N.J., Jan. 25, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a negative opinion following the re-examination procedure for the conditional marketing authorization of Translarna™ (ataluren). "We are disappointed that the CHMP has maintained its negative opinion on the Translarna...Read more
SOLANA BEACH, Calif., Jan. 22, 2024 (GLOBE NEWSWIRE) -- ClearPoint Neuro, Inc. (Nasdaq: CLPT) (the “Company”), a global device, cell, and gene therapy-enabling company offering precise navigation to the brain and spine, today announced receipt of European Medical Device Regulation (EU MDR) clearance for the manual SmartTwist® MR Hand Drill and SmartTip® MR Drill Kit. Additionally, the Company received updated certification from its...Read more
Designation is an important step toward bringing this potentially life-changing therapy to European patients EU Orphan Drug Designation is the first regulatory designation for a gorilla adenovector based immunotherapy outside of the United States for the Company GERMANTOWN, Md., Jan. 16, 2024 /PRNewswire/ -- Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative gene and...Read more
Reduces treatment time by approximately 80% compared to standard intravenous (IV) infusion Subcutaneous (SC) injection offers the potential for a faster, more convenient alternative to IV infusion and is preferred by patients and healthcare practitioners1 - 6 SAN DIEGO, Jan. 16, 2024 /PRNewswire/ -- Halozyme Therapeutics, Inc. (NASDAQ: HALO) ("Halozyme") today announced that Roche received European Commission (EC) marketing...Read more
SAN DIEGO and ZUG, Switzerland, Jan. 10, 2024 /PRNewswire/ -- Mirati Therapeutics, Inc.® (NASDAQ: MRTX), a commercial stage biotechnology company, today announced that the European Commission (EC) granted conditional marketing authorization for KRAZATI® (adagrasib) as a targeted treatment option for adult patients with KRASG12C -mutated advanced non-small cell lung cancer (NSCLC) and disease progression after at least one prior...Read more
Marketing authorization issued in European Economic Area for Uzpruvo®, the first biosimilar to Stelara®, a biologic therapy within gastroenterology, dermatology, and rheumatology Authorization paves way for biosimilar competition in the approximately €2.5 billion (US$2.7 billion) EU ustekinumab market as soon as possible after expiry of intellectual-property rights in July 2024 Approval for the Uzpruvo biosimilar is based on...Read more
TALZENNA is the first and only PARP inhibitor approved in combination with standard of care XTANDI for mCRPC patients in the Europe Union NEW YORK / Jan 08, 2024 / Business Wire / Pfizer Inc. (NYSE: PFE) today announced that the European Commission (EC) has approved TALZENNA® (talazoparib), an oral poly ADP-ribose polymerase (PARP) inhibitor, in combination with XTANDI® (enzalutamide), for the treatment of adult patients with...Read more
Application based on data from the registrational TRIDENT-1 and CARE trials showing robust responses and durable activity in these patient populations If approved, repotrectinib will offer a potential best-in-class treatment for patients with locally advanced or metastatic ROS1-positive non-small cell lung cancer and a new option for patients with NTRK-positive solid tumors, in the European Union PRINCETON, N.J. / Jan 02, 2024 /...Read more
Approval based on positive results from the pivotal ReSTORE Phase III clinical trial and supported by the STRIVE Phase II clinical trials and extensive nonclinical development program. REZZAYO represents the first new treatment option in over 15 years for patients with invasive candidiasis. Cidara is entitled to receive a milestone payment of approximately $11.14 million from Mundipharma for the European Medicines Agency (EMA)...Read more
NEW YORK, Dec. 19, 2023 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders, announced today that the Committee for Medicinal Products for Human Use (CHMP) within the European Medicines Agency (EMA) agreed that oral blarcamesine for Alzheimer’s...Read more
NOVATO, Calif., Dec. 18, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the European Commission (EC) has extended the approval of Evkeeza® (evinacumab) as an adjunct to diet and other lipid-lowering therapies to treat children aged 5 to 11 years with homozygous familial hypercholesterolemia (HoFH). Evkeeza is the first angiopoietin-like 3 (ANGPTL3) inhibitor treatment indicated for children as...Read more
KEYTRUDA now approved for 26 indications in the EU, including seven in gastrointestinal cancers RAHWAY, N.J. / Dec 18, 2023 / Business Wire / Merck (NYSE: MRK), known as MSD outside of the United States and Canada, today announced the European Commission (EC) has approved two new indications for KEYTRUDA, Merck’s anti-PD-1 therapy, in gastrointestinal cancers: KEYTRUDA in combination with fluoropyrimidine- and platinum-containing...Read more
Friedreich’s ataxia is a genetic, debilitating and life-shortening neuromuscular disease1 Milestone highlights Biogen’s growing portfolio in rare diseases and focus on addressing unmet needs of patients living with neuromuscular diseases CAMBRIDGE, Mass., Dec. 15, 2023 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommended...Read more
If approved by the European Commission, patients 12 years of age and older with severe sickle cell disease or transfusion-dependent beta thalassemia, for whom hematopoietic stem cell transplantation is appropriate and a human leukocyte antigen matched related donor is not available, would be eligible for treatment Approval decision from the European Commission is expected in Q1 2024 BOSTON / Dec 15, 2023 / Business...Read more
If Approved, HYQVIA [Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase] Would Offer an up to Once-Monthly Facilitated Subcutaneous At-Home or In-Office Treatment Option Positive Opinion Based on Phase 3 ADVANCE-CIDP 1 Study, Which Met its Primary Endpoint Demonstrating a Statistically Significant Reduction in Relapse Rate 1 OSAKA, Japan & CAMBRIDGE, Mass. / Dec 15, 2023 / Business Wire / Takeda...Read more
WALTHAM, Mass., Dec. 14, 2023 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) announced today an update on the ongoing review of its marketing authorization application (MAA) for intravitreal pegcetacoplan for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD) by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). Following the oral...Read more
In the European Union, patients with indolent systemic mastocytosis now have an approved medicine that treats the primary driver of disease Approval based on data from PIONEER trial, in which AYVAKYT achieved significant improvements across a broad range of symptoms with a safety profile comparable to placebo1 CAMBRIDGE, Mass., Dec. 12, 2023 /PRNewswire/ -- Blueprint Medicines Corporation (Nasdaq: BPMC) today announced...Read more
Submissions based on positive data from the Phase 3 BASIS trial, which were presented this past weekend at the American Society of Hematology (ASH) Annual Meeting If approved in the U.S. and EU, marstacimab could become the first once-weekly subcutaneous treatment for people living with hemophilia B and the first treatment administered as a flat dose for people living with hemophilia A or B NEW YORK / Dec 11, 2023 / Business Wire /...Read more
Conditional marketing authorization is based on clinically meaningful response rates, duration of response, and safety from the Phase 2 MagnetisMM-3 trial ELREXFIO is an off-the-shelf (ready-to-use), fixed-dose, subcutaneous BCMA-directed bispecific antibody immunotherapy with reduced dosing after 24 weeks for responding patients NEW YORK / Dec 08, 2023 / Business Wire / Pfizer Inc. (NYSE:PFE) today announced the European...Read more
Alignment reached on key elements of the NASH development strategy, including accelerated approval pathway for both F4 and F2-F3 NASH patients using histology Outcomes trial in F4 cirrhotic NASH patients expected to support full approval across F2-F4 NASH; potential to accelerate timeline to outcomes readout based on agreement with FDA on modified definition of some events Safety database will be inclusive of data from the...Read more
Boca Raton, Florida, Nov. 27, 2023 (GLOBE NEWSWIRE) -- INmune Bio, Inc. (NASDAQ: INMB) (the “Company”), a clinical-stage immunology company dedicated to advancing treatments that leverage the patient’s innate immune system to combat disease, announced today it has received European Medicines Agency’s (EMA) Authorized Decision from the Agence Nationale de Securite du Medicament et des Produits de Sante (ANSM) in France and the Agencia...Read more
U.S. Food and Drug Administration (FDA) grants Breakthrough Therapy Designation (BTD) for epcoritamab-bysp for the treatment of relapsed or refractory (R/R) follicular lymphoma (FL) after two or more lines of systemic therapy European Medicines Agency (EMA) validates regulatory application for epcoritamab for the same indication The regulatory actions are supported by data from the phase 1/2 EPCORE™ NHL-1 trial COPENHAGEN, Denmark /...Read more
Updates are supported by data from the Phase 1/2 EPCORE™ NHL-1 clinical trial NORTH CHICAGO, Ill., Nov. 27, 2023 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced 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, for relapsed or refractory (R/R) follicular lymphoma (FL). The FDA has...Read more
If approved, ~3,000 people with DEB in the European Union could receive VYJUVEK to treat the underlying cause of the disease for the first time VYJUVEK received Orphan Drug Designation and PRIME designation from the EMA PITTSBURGH, Nov. 27, 2023 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company focused on the discovery, development and commercialization of...Read more
More than 1,200 children are newly eligible for a medicine that could treat the underlying cause of their disease BOSTON / Nov 23, 2023 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Commission has granted approval for the label expansion of KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor for the treatment of children with cystic fibrosis (CF)...Read more
YORVIPATH (developed as TransCon PTH) is a parathyroid hormone (PTH) replacement therapy indicated for the treatment of adults with chronic hypoparathyroidism Ascendis plans its first EU launch of YORVIPATH in Germany in January 2024 COPENHAGEN, Denmark, Nov. 20, 2023 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that the European Commission (EC) has granted marketing authorization for YORVIPATH®...Read more
NEW YORK, Nov. 20, 2023 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders announced today that representatives of Anavex met with team members of the European Medicines Agency (EMA). These meetings discussed the debilitating pathology of...Read more
BRUKINSA is the first and only BTK inhibitor approved for follicular lymphoma in the European Union Approval was based on results from the ROSEWOOD trial in which BRUKINSA plus the anti-CD20 monoclonal antibody obinutuzumab achieved higher overall response rate compared to obinutuzumab alone BASEL, Switzerland & BEIJING & CAMBRIDGE, Mass. / Nov 17, 2023 / Business Wire / BeiGene, Ltd. (Nasdaq: BGNE; HKEX: 06160; SSE: 688235),...Read more
VYVGART® SC Now Approved for Subcutaneous Administration, Adding to the Already Approved IV Administration SAN DIEGO, Nov. 16, 2023 /PRNewswire/ -- Halozyme Therapeutics, Inc. (NASDAQ: HALO) ("Halozyme") today announced that argenx received European Commission (EC) approval of VYVGART® SC (efgartigimod alfa and hyaluronidase-qvfc) co-formulated with ENHANZE® for the treatment of generalized myasthenia gravis (gMG) in adult...Read more
BOSTON, Nov. 16, 2023 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today announced that the European Medical Agency (EMA) has awarded its Priority Medicines (PRIME) designation for elsunersen (PRAX-222) for the...Read more
VYVGART® is now approved for both intravenous (IV) and self-administered subcutaneous (SC) use in Europe argenx is committed to continued collaboration with local authorities across the region to enable broad access to VYVGART SC for eligible patients Amsterdam, The Netherlands— November 16, 2023 — argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from...Read more
CAMBRIDGE, Mass., Nov. 14, 2023 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative single-dose therapeutics leveraging CRISPR-based technologies, today announced that the European Commission (EC) has granted orphan drug designation to NTLA-2002 for the treatment of hereditary angioedema (HAE). NTLA-2002 is an in vivo CRISPR-based...Read more
YAVNE, Israel, Nov. 13, 2023 (GLOBE NEWSWIRE) -- MediWound Ltd. (Nasdaq: MDWD), a fully integrated biopharmaceutical company focused on next-generation enzymatic therapeutics for tissue repair, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a positive opinion recommending a change to the terms of the marketing authorization for NexoBrid in Europe to include all...Read more
Recommendation by Committee for Medicinal Products for Human Use (CHMP) based on positive results from PULSAR clinical trial in neovascular (wet) age-related macular degeneration (nAMD) and PHOTON trial in diabetic macular edema (DME) If approved by European Commission, aflibercept 8 mg will be the only drug providing extended treatment intervals of up to 5 months for patients with nAMD and DME Extended treatment intervals with...Read more
Opinion granted based on positive overall survival results from the Phase 3 KEYNOTE‑966 trial RAHWAY, N.J. / Nov 10, 2023 / Business Wire / 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...Read more
SAN DIEGO, Calif. and ZUG, Switzerland, Nov. 10, 2023 /PRNewswire/ -- Mirati Therapeutics, Inc.® (NASDAQ: MRTX), a commercial stage biotechnology company, today announced that following a re-examination procedure, the Company has received a positive opinion from the European Medicine Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) on KRAZATI® (adagrasib) as a targeted treatment option for adult patients with KRASG12C...Read more
For the first time, the estimated 40,000 patients with indolent SM in the EU1,2* would have a medicine that treats the primary disease driver3,4 Opinion based on the positive PIONEER trial, in which once-daily AYVAKYT achieved significant improvements across a broad range of symptoms with a comparable safety profile to placebo3,4 CAMBRIDGE, Mass., Nov. 10, 2023 /PRNewswire/ -- Blueprint Medicines Corporation (Nasdaq:...Read more
CHMP positive opinion in the European Economic Area for first biosimilar to Stelara, a biologic therapy within gastroenterology, dermatology and rheumatology Positive CHMP opinion for Uzpruvo biosimilar is based on comprehensive package comprising analytical, non-clinical and clinical similarity data, including AVT04-GL-301 study for pharmacokinetic similarity in healthy subjects and confirmatory comparative efficacy and safety...Read more
Novavax's updated COVID-19 vaccine is the only updated protein-based non-mRNA COVID-19 vaccine available in the European Union for individuals aged 12 and older Novavax is working closely with EU member states to bring its updated COVID-19 vaccine to those that have requested doses through the advance purchase agreement GAITHERSBURG, Md., Oct. 31, 2023 /PRNewswire/ -- Novavax, Inc. (Nasdaq: NVAX), a global company advancing...Read more
If approved, the Opdivo-based regimen would be the first immunotherapy-chemotherapy combination approved for the first-line treatment of untreated, unresectable or metastatic urothelial carcinoma in the European Union Application based on CheckMate -901 trial, the first Phase 3 trial with an immunotherapy-based combination to show statistically significant and clinically meaningful survival benefit over standard-of-care cisplatin-based...Read more
WALTHAM, Mass. / Oct 27, 2023 / Business Wire / ImmunoGen, Inc. (Nasdaq: IMGN), a leader in the expanding field of antibody-drug conjugates (ADCs) for the treatment of cancer, today announced that the European Medicines Agency (EMA) has accepted the Marketing Authorization Application (MAA) for mirvetuximab soravtansine (ELAHERE®) for the treatment of patients with folate receptor alpha (FRα)-positive, platinum-resistant epithelial ovarian,...Read more
PRIME Designation Granted Based on Phase 2 ARCHER Trial Results, which Showed Meaningful Preservation of Visual Function in Patients with Geographic Atrophy ANX007 is the First Therapeutic Candidate to Receive PRIME Designation by the EMA for the Indication of Geographic Atrophy Company Engaging with U.S. and EU Regulatory Authorities to Determine the Optimal Global Pivotal Phase 3 Program for ANX007 BRISBANE, Calif., Oct. 24, 2023...Read more
The European Medicines Agency’s priority medicines status is granted to drug candidates that may offer a major therapeutic advantage over existing treatments or benefit patients without treatment options 4D-150 combines a novel, targeted intravitreal next generation AAV vector with a dual transgene that inhibits four VEGF pathway targets (VEGF A, B, C, and PlGF) in wet age-related macular degeneration (wet AMD) and diabetic macular...Read more
Surmodics is the global leader in surface modification technologies for intravascular medical devices and a leading provider of chemical components for in vitro diagnostic immunoassay tests and microarrays. Surmodics is pursuing highly differentiated whole-product solutions that are designed to address...
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