Opdivo® is the first and only PD-1 inhibitor approved for subcutaneous (SC) use in the European Union SAN DIEGO, May 28, 2025 /PRNewswire/ -- Halozyme Therapeutics, Inc. (NASDAQ: HALO) (Halozyme) today announced that Bristol Myers Squibb received European Commission (EC) approval of a new Opdivo® (nivolumab) subcutaneous formulation developed with ENHANZE®, Halozyme's proprietary recombinant human hyaluronidase enzyme...Read more
Opdivo is the first and only PD-1 inhibitor approved for subcutaneous (SC) use in the European Union Approval is based on results from the Phase 3 CheckMate -67T clinical trial which demonstrated noninferiority in the co-primary endpoints of Cavgd28 and Cminss, and consistent efficacy in the secondary endpoint of overall response rate, for the subcutaneous formulation of Opdivo (Opdivo SC) vs. its intravenous formulation (IV...Read more
Positive CHMP opinion based on FELIX clinical trial of obecabtagene autoleucel (obe-cel) in adult patients with r/r B-ALL, demonstrating high and durable response rates and low toxicity Opinion follows FDA approval and MHRA conditional marketing authorisation European Commission (EC) decision on conditional marketing authorization application (MAA) expected within approximately two months LONDON, May 23, 2025 (GLOBE NEWSWIRE) --...Read more
If approved, mirdametinib is expected to be the first and only therapy in the European Union with marketing authorization for both adults and children with NF1-PN Decision from European Commission expected in the third quarter of 2025 STAMFORD, Conn., May 23, 2025 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, announced today...Read more
REDWOOD CITY, Calif., May 22, 2025 (GLOBE NEWSWIRE) -- Soleno Therapeutics, Inc. (Soleno) (NASDAQ: SLNO), a biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today announced that Soleno’s Marketing Authorization Application (MAA) seeking regulatory approval of Diazoxide Choline Prolonged-Release Tablets (previously referred to as DCCR) for the treatment of adults and children four years and older...Read more
Company continues to advance QRX003 in late-stage clinical trials in Netherton Syndrome patients Regulatory milestone provides 10 years of market exclusivity in Europe upon approval ASHBURN, Va., May 20, 2025 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the “Company” or “Quoin”), a late clinical stage, specialty pharmaceutical company focused on rare and orphan diseases, today announces that it has been granted...Read more
Approval based on results from the CheckMate-77T trial which showed perioperative Opdivo improved event-free survival compared to neoadjuvant chemotherapy and placebo followed by surgery and adjuvant placebo Opdivo is now the only PD-(L)1 inhibitor approved for both perioperative and neoadjuvant-only treatment of resectable non-small cell lung cancer in the European Union PRINCETON, N.J. / May 16, 2025 / Business Wire / Bristol Myers...Read more
Adds nine additional countries to the Company’s ongoing pivotal Phase 3 trial; Authorization granted in all EU countries requested Enrollment and dosing underway in Phase 3 clinical trial (the “MIRACLE” trial) evaluating Annamycin for the treatment of R/R AML; Interim data readout expected in the second half of 2025 Subjects targeted for the “MIRACLE” trial include venetoclax regimen failures where outcomes with currently available...Read more
New designation follows the U.S. Food and Drug Administration (FDA) Orphan Drug Designation for eRapa in FAP granted in 2019 Company nears start of Registrational Phase 3 study targeted at an addressable market of $7.3Bn May 12, 2025 - Biodexa Pharmaceuticals PLC (“Biodexa” or the “Company”) (Nasdaq: BDRX), a clinical stage biopharmaceutical company developing a pipeline of innovative products for the treatment of diseases with...Read more
INVINCIBLE-4 (SAKK/66/22) Study continues to recruit patients in eight sites in Switzerland SHELTON, Conn. and BERN, Switzerland, May 6, 2025 /PRNewswire/ -- Intensity Therapeutics, Inc. (Nasdaq: INTS), ("Intensity" or "the Company") a late-stage clinical biotechnology company focused on the discovery and development of proprietary, novel immune-based intratumoral cancer therapies designed to kill tumors and increase immune...Read more
European Commission converts conditional approval of FILSPARI (sparsentan) into standard marketing authorization for the treatment of IgA Nephropathy (IgAN) Decision follows positive recommendation from Committee for Medicinal Products for Human Use (CHMP) from February 2025 EU approval is based on the complete data set from the phase-III PROTECT study ST. GALLEN, Switzerland and SAN DIEGO, April 29, 2025 /CNW/ -- CSL Vifor...Read more
Recommended Approval Based on the Pivotal HELIOS-B Phase 3 Study in which Vutrisiran Demonstrated Significant Reductions in Mortality and Cardiovascular Events, While Preserving Functional Status and Quality of Life Offers a Clinically Differentiated Approach with Rapid and Sustained Transthyretin (TTR) Knockdown and Quarterly Subcutaneous Dosing Follows Recent Approvals in the U.S. and Brazil European Commission...Read more
European Commission (EC) decision on marketing authorization application expected within approximately two months SAN DIEGO, April 28, 2025 /PRNewswire/ -- Halozyme Therapeutics, Inc. (NASDAQ: HALO) (Halozyme) today announced that argenx received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency recommending European Commission (EC) approval of VYVGART® 1000mg...Read more
Approval of Lynozyfic is based on data showing deep and durable responses in relapsed/refractory multiple myeloma Lynozyfic will provide a new option with convenient dosing and administration to patients who face cycles of relapse and remission TARRYTOWN, N.Y., April 28, 2025 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that the European Commission (EC) has granted conditional marketing...Read more
VYJUVEK approved for the treatment of DEB from birth in Europe Approval allows for dosing at home or in a healthcare setting, as well as patient or caregiver administration if deemed appropriate by a healthcare professional PITTSBURGH, April 28, 2025 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS) announced today that on April 23, 2025, the European Commission (EC) granted marketing authorization to VYJUVEK®...Read more
VYVGART® is first-and-only targeted IgG Fc-antibody fragment for CIDP First novel mechanism of action for CIDP treatment in more than 30 years CHMP positive opinion based on ADHERE data, the largest ever CIDP clinical trial European Commission (EC) decision on marketing authorization application (MAA) expected within approximately two months April 28, 2025, 07:00 AM CET - Amsterdam, the Netherlands – argenx SE (Euronext...Read more
STAMFORD, Conn., April 27, 2025 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, announced today that the Company anticipates the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) will adopt an opinion on the marketing authorization application (MAA) for nirogacestat, an oral gamma secretase...Read more
DUBLIN, April 25, 2025 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending the conditional marketing authorization of zanidatamab, an investigational dual human epidermal growth factor receptor 2 (HER2)-targeted bispecific antibody, as monotherapy for the treatment of...Read more
Label includes full spectrum of PKU patients European launch preparations underway WARREN, N.J., April 25, 2025 /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 issued a positive opinion on the marketing authorization application for Sephience™ (sepiapterin) for the treatment of children and adults living...Read more
Recently presented data demonstrated sustained functional improvement with DYNE-251 treatment through 18 months Data from the fully enrolled DELIVER registrational expansion cohort is planned for late 2025 WALTHAM, Mass., April 24, 2025 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on advancing life-transforming therapeutics for people living with genetically driven...Read more
Company-sponsored clinical trial will be initiated at FAU Erlangen-Nuremberg in Germany and Università Cattolica del Sacro Cuore, Rome in Q2 2025 CLN-978 is also being studied in the U.S., Europe, and Australia for systemic lupus erythematosus CAMBRIDGE, Mass., April 16, 2025 (GLOBE NEWSWIRE) -- Cullinan Therapeutics, Inc. (Nasdaq: CGEM), a biopharmaceutical company focused on developing modality-agnostic targeted therapies,...Read more
In the European Union (EU), lecanemab is indicated for the treatment of adult patients with a clinical diagnosis of mild cognitive impairment and mild dementia due to Alzheimer’s disease (early AD) who are apolipoprotein E ε4 (ApoE ε4*) non-carriers or heterozygotes with confirmed amyloid pathology Lecanemab is the first therapy that targets an underlying cause of the disease to be authorized in the EU for eligible people with...Read more
Subcutaneous DARZALEX® is co-formulated with Halozyme's ENHANZE® drug delivery technology SAN DIEGO, April 9, 2025 /PRNewswire/ -- Halozyme Therapeutics, Inc. (NASDAQ: HALO) (Halozyme) today announced that Janssen-Cilag International NV, a Johnson & Johnson company, received European Commission (EC) approval for an indication extension of DARZALEX® (daratumumab) subcutaneous (SC) co-formulated with ENHANZE® in the frontline...Read more
RINVOQ is the first and only oral Janus kinase (JAK) inhibitor approved in the European Union (EU) to treat adult patients with giant cell arteritis (GCA) The approval is supported by data from the pivotal Phase 3 SELECT-GCA trial which demonstrated that RINVOQ achieved the primary endpoint of sustained remission* and key secondary endpoints, including reduction in disease flares, lower cumulative steroid exposure and complete...Read more
CAMBRIDGE, Mass., April 03, 2025 (GLOBE NEWSWIRE) -- Akebia Therapeutics®, Inc. (Nasdaq: AKBA), a biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending the European Commission (EC) to approve XOANACYL® (Ferric Citrate as Coordination...Read more
ZUG, Switzerland, April 01, 2025 (GLOBE NEWSWIRE) -- Pharvaris (Nasdaq: PHVS), a late-stage biopharmaceutical company developing novel, oral bradykinin B2 receptor antagonists to help address unmet needs of those living with bradykinin-mediated diseases such as hereditary angioedema (HAE) and acquired angioedema due to C1 inhibitor deficiency (AAE-C1INH), today announced that the European Commission (EC) has granted orphan designation to...Read more
ABRYSVO is the first and only RSV vaccine approved in the European Union (EU) for non-pregnant adults aged 18-49 NEW YORK / Apr 01, 2025 / Business Wire / Pfizer Inc. (NYSE: PFE) announced today that the European Commission (EC) has issued a decision amending the marketing authorization for ABRYSVO®, the company’s bivalent respiratory syncytial virus (RSV) prefusion F (RSVpreF) vaccine, to extend the indication to include prevention of...Read more
TIVDAK® is the first and only antibody-drug conjugate (ADC) approved to treat recurrent or metastatic cervical cancer with disease progression on or after systemic therapy In the global Phase 3 innovaTV 301 clinical trial TIVDAK demonstrated superior overall survival compared to chemotherapy TIVDAK is approved for the treatment of recurrent or metastatic cervical cancer in the European Union, United States and Japan COPENHAGEN,...Read more
A decision on the European marketing authorization extension for the subcutaneous formulation of Opdivo is expected by June 2, 2025 SAN DIEGO, March 31, 2025 /PRNewswire/ -- Halozyme Therapeutics, Inc. (NASDAQ: HALO) (Halozyme) today announced that Bristol Myers Squibb received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency, recommending approval of a new Opdivo®...Read more
Positive opinion for first-line treatment of extensive-stage small cell lung cancer based on results of RATIONALE-312 study demonstrating statistically significant overall survival benefit for TEVIMBRA in combination with chemotherapy SAN CARLOS, Calif. / Mar 31, 2025 / Business Wire / BeiGene, Ltd. (NASDAQ: ONC; HKEX: 06160; SSE: 688235), a global oncology company that intends to change its name to BeOne Medicines Ltd., today...Read more
WARREN, N.J., March 28, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the European Commission (EC) has adopted the opinion of the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) to not renew the authorization of Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy. While this action effectively removes the drug's...Read more
Recommendation based on Phase 3 CheckMate -77T trial showing significant event-free survival improvement when compared to neoadjuvant chemotherapy and placebo followed by surgery and adjuvant placebo If approved by the European Commission, the perioperative regimen would be the company’s second Opdivo-based regimen available for the treatment of resectable non-small cell lung cancer in the European Union PRINCETON, N.J. / Mar 28, 2025...Read more
Recommendation is based primarily on results from the Phase 3 CheckMate -67T trial which demonstrated noninferiority in the co-primary endpoints of Cavgd28 and Cminss and consistent efficacy in the secondary endpoint of overall response rate for the subcutaneous formulation of Opdivo vs. its intravenous formulation A decision on the EU Opdivo extension of marketing authorization is expected by June 2, 2025 PRINCETON, N.J. / Mar 28,...Read more
SHANGHAI, China & JERSEY CITY, N.J. / Mar 28, 2025 / Business Wire / Shanghai Henlius Biotech, Inc. (2696.HK) and Organon (NYSE: OGN) today announced that the European Medicines Agency (EMA) has validated the marketing authorization application (MAA) for HLX11, an investigational biosimilar of Perjeta® (pertuzumab). Pertuzumab has been approved in various countries and regions in combination with trastuzumab and chemotherapy for the...Read more
SAN DIEGO, March 27, 2025 (GLOBE NEWSWIRE) -- Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) today announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for paltusotine, the first once-daily, oral, selectively-targeted somatostatin receptor type 2 nonpeptide agonist, for the proposed treatment and long-term maintenance therapy of acromegaly, a serious, rare and progressive endocrine...Read more
EC decision marks the fourth approval for CAPVAXIVE for pneumococcal vaccination in adults RAHWAY, N.J. / Mar 26, 2025 / Business Wire / Merck (NYSE: MRK), known as MSD outside of the United States and Canada, announced today that the European Commission (EC) has approved CAPVAXIVE® (Pneumococcal 21-valent Conjugate Vaccine) for active immunization for the prevention of invasive disease and pneumonia caused by Streptococcus pneumoniae...Read more
In the TRANSCEND FL trial, 97.1% of patients responded to Breyanzi, with 94.2% of patients achieving complete response* Breyanzi demonstrated sustained clinical benefit, with 75.7% of patients still in response at 18 months, and a consistent safety profile with no new safety signals observed PRINCETON, N.J. / Mar 14, 2025 / Business Wire / Bristol Myers Squibb (NYSE: BMY) today announced that the European Commission (EC) has granted...Read more
Approval across ESA ineligible and ESA relapsed/refractory non-del 5q patients with transfusion-dependent anemia due to LR-MDS, regardless of ring sideroblast (RS) status RYTELO is the first and only telomerase inhibitor approved in the U.S. and Europe FOSTER CITY, Calif. / Mar 11, 2025 / Business Wire / Geron Corporation (Nasdaq: GERN), a commercial-stage biopharmaceutical company aiming to change lives by changing the course of...Read more
Second major approval for WAINZUA, which is marketed in the U.S. as WAINUA™ EU approval based on NEURO-TTRansform Phase 3 results showing WAINZUA demonstrated consistent and sustained benefit improving neuropathy impairment and quality of life versus placebo CARLSBAD, Calif. / Mar 10, 2025 / Business Wire / Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that Ionis and AstraZeneca’s WAINZUA (eplontersen) has been...Read more
MALVERN, Pa., March 03, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the European Commission has provided a positive opinion from the European Medicines Agency’s (EMA) Committee for Advanced Therapies (CAT) for OCU410 and OCU410ST Advanced Therapy Medicinal Product (ATMP) classification. OCU410 is a novel,...Read more
TOKYO and CAMBRIDGE, Mass., Feb. 28, 2025 /PRNewswire/ -- Eisai Co., Ltd. (Headquarters: Tokyo, CEO: Haruo Naito, "Eisai") and Biogen Inc. (Nasdaq: BIIB, Corporate headquarters: Cambridge, Massachusetts, CEO: Christopher A. Viehbacher, "Biogen") announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has reaffirmed its positive opinion by consensus for the anti-Aβ monoclonal...Read more
The positive opinion is based on results from the Phase 3 BRUIN CLL-321 trial, recently presented at the 2024 American Society of Hematology Annual Meeting BRUIN CLL-321 is the first randomized Phase 3 study in CLL ever conducted exclusively in patients previously treated with a BTK inhibitor INDIANAPOLIS, Feb. 28, 2025 /PRNewswire/ -- Eli Lilly and Company (NYSE: LLY) announced today that the European Medicines Agency's (EMA)...Read more
PITTSBURGH, Feb. 28, 2025 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company, today welcomed the European Medicines Agency's (EMA’s) announcement that its Committee for Medicinal Products for Human Use (CHMP) has issued a positive recommendation for the European Commission (EC) to approve VYJUVEK® (beremagene geperpavec-svdt, or B-VEC) for the treatment of wounds in...Read more
TARRYTOWN, N.Y., Feb. 28, 2025 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending conditional marketing authorization of linvoseltamab to treat adults with relapsed and refractory (R/R) multiple myeloma (MM). The recommendation is specific to those who have received at...Read more
The positive opinion is based on results from the pivotal Phase 3 SELECT-GCA trial that evaluated the efficacy and safety of upadacitinib in adults with giant cell arteritis (GCA)1 The primary endpoint of sustained remission* and key secondary endpoints, including reduction in disease flares, lower cumulative steroid exposure, and complete remission,† were met1 GCA is an autoimmune disease that causes...Read more
One Application Seeks European Commission Authorization; Other Application Would Facilitate Availability in Low- and Lower-Middle-Income Countries Both Applications Will Be Assessed in Parallel Under Accelerated Assessment Review Timeline Based on Potential Public Health Interest in Lenacapavir for HIV Prevention EMA Validation Follows Acceptance of U.S. Applications by FDA Under Priority Review FOSTER CITY, Calif. / Feb...Read more
TAKHZYRO Solution for Injection in 2 mL Pre-Filled Pen is Now Approved to Facilitate Subcutaneous Administration in Adolescent (Aged 12 Years and Above) and Adult Patients with HAE.1 The Pre-Filled Pen Option is Designed to Allow for An Individualized Treatment Approach for Adolescent and Adult HAE Patients. In the EU, TAKHZYRO is Approved for Routine Prevention of Recurrent HAE Attacks in Patients Aged 2 Years and Older.1 ZURICH,...Read more
WALTHAM, Mass. and STOCKHOLM, Sweden, Feb. 20, 2025 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) and Sobi® (STO:SOBI) today announced the European Medicines Agency (EMA) has validated an indication extension application for Aspaveli® (pegcetacoplan) for the treatment of C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), which are rare, chronic kidney...Read more
KOSTAIVE represents a significant advancement in vaccine technology, demonstrating superior immunogenicity and antibody persistence for up to 12 months post-vaccination compared to conventional mRNA COVID-19 vaccines in clinical trials WALTHAM, Mass. & SAN DIEGO / Feb 14, 2025 / Business Wire / Global biotechnology leader CSL (ASX: CSL; USOTC: CSLLY) and sa-mRNA pioneer Arcturus Therapeutics (Nasdaq: ARCT) today announced that the...Read more
The approval is based on positive results from the Phase 3 ATTRibute-CM study, in which acoramidis demonstrated the most rapid benefit seen in any Phase 3 study of ATTR-CM to date In as few as 3 months, the time to first event (all-cause mortality (ACM) or cardiovascular-related hospitalization (CVH)) durably separated relative to placebo A 42% reduction in composite ACM and recurrent CVH events relative to placebo at Month 30 A...Read more
SAN DIEGO, Feb. 3, 2025 /PRNewswire/ -- Halozyme Therapeutics, Inc. (NASDAQ: HALO) (Halozyme) today announced that Janssen-Cilag International NV, a Johnson & Johnson company, received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency recommending an extension of marketing authorisation for a subcutaneous (SC) formulation of RYBREVANT® (amivantamab) in combination with...Read more
MALVERN, Pa., Feb. 03, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that the European Commission has provided a positive opinion from the European Medicines Agency’s (EMA) Committee for Advanced Therapies (CAT) for OCU400 Advanced Therapy Medicinal Product...Read more
RAHWAY, N.J. / Jan 31, 2025 / Business Wire / Merck (NYSE: MRK), known as MSD outside of the United States and Canada, announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommended the approval of CAPVAXIVE™ (Pneumococcal 21-valent Conjugate Vaccine) for active immunization for the prevention of invasive disease and pneumonia caused by Streptococcus pneumoniae in...Read more
Recommendation based on results of Phase 3 CheckMate -9DW clinical trial demonstrating statistically significant and clinically meaningful improvement in overall survival with Opdivo plus Yervoy compared to investigator’s choice of lenvatinib or sorafenib in this patient population PRINCETON, N.J. / Jan 31, 2025 / Business Wire / Bristol Myers Squibb (NYSE: BMY) today announced that the Committee for Medicinal Products for Human Use...Read more
Recommendation based on the Phase 2 TRANSCEND FL study in which 97.1% of patients responded to Breyanzi, with 94.2% of patients achieving complete response Across clinical trials, Breyanzi has delivered rapid and durable responses as well as a consistent and well-established safety profile PRINCETON, N.J. / Jan 31, 2025 / Business Wire / Bristol Myers Squibb (NYSE: BMY) today announced the Committee for Medicinal Products for Human...Read more
Application covers 30 countries in the European Union Submission is based on the ongoing QUILT 3.032 study in which 100 patients with Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer with carcinoma in situ (NMIBC CIS) have been treated with ANKTIVA® (nogapendekin alfa inbakicept-pmln) in combination with BCG, achieving a 71% (71/100) complete response (CR) rate In these responders, the range of duration is...Read more
Submission supported by positive results from global, Phase 3 4WHIM clinical trial; U.S. regulatory approval in WHIM syndrome granted in 2024 Decision on MAA expected in 1H 2026 BOSTON, Jan. 24, 2025 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today announced that its Marketing Authorization Application (MAA) for mavorixafor for the...Read more
CAMBRIDGE, Mass., Jan. 23, 2025 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s supplemental New Drug Application (sNDA) and the European Medicines Agency (EMA) has validated the application for a higher dose regimen of nusinersen for spinal muscular atrophy (SMA). The higher dose regimen of nusinersen comprises a more rapid loading regimen, two 50 mg...Read more
MELBOURNE, Australia and LIÈGE, Belgium, Jan. 17, 2025 (GLOBE NEWSWIRE) -- Telix Pharmaceuticals Limited (ASX: TLX, Nasdaq: TLX, Telix, the Company) today announces that it has received a positive decision on the Marketing Authorization Application (MAA) for its prostate cancer PET1 imaging agent Illuccix® (kit for the preparation of gallium-68 gozetotide injection), which was submitted in Europe via a decentralized procedure (DCP). This...Read more
Marketing authorization application for BCG Unresponsive Non-Muscle Invasive Bladder Cancer (NMIBC) with Carcinoma in situ (CIS) submitted to European Medicines Agency (EMA) in December 2024 and anticipated acceptance of application in 2025 Marketing authorization application for BCG Unresponsive NMIBC CIS submitted to United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) in November 2024 and anticipated acceptance...Read more
Filing marks company’s first Marketing Authorization Application in Europe SAN DIEGO / Jan 14, 2025 / Business Wire / Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the company has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for trofinetide for the treatment of Rett syndrome in adults and pediatric patients two years of age and older. If granted marketing authorization,...Read more
CHICAGO and FORT WORTH, Texas, Jan. 07, 2025 (GLOBE NEWSWIRE) -- Actuate Therapeutics, Inc. (NASDAQ: ACTU) (“Actuate” or the “Company”), a clinical-stage biopharmaceutical company focused on developing therapies for the treatment of high-impact, difficult-to-treat cancers through the inhibition of glycogen synthase kinase-3 beta (GSK-3β), today announced that the European Medicines Agency (EMA) has granted Orphan Medicinal Product...Read more
Acurx has now received positive written responses from the EMA (European Medicines Agency) under its Scientific Advice Procedure that the clinical, non-clinical and CMC (Chemistry Manufacturing and Controls) information package submitted supports advancement of the ibezapolstat Phase 3 program The responses also included guidance on ibezapolstat's regulatory pathway for a Marketing Authorization Application for ibezapolstat in...Read more
SOUTH SAN FRANCISCO, Calif., Dec. 23, 2024 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) today announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for aficamten, a next-in-class cardiac myosin inhibitor, for the treatment of obstructive hypertrophic cardiomyopathy (HCM). The MAA will now be reviewed by the Committee for Medicinal Products for Human Use...Read more
Blarcamesine: Potential novel oral treatment to target upstream Alzheimer’s disease pathology through autophagy enhancement Submission based on favorable ANAVEX®2-73-AD-004 trial results in patients with early Alzheimer’s disease Once daily oral administration of blarcamesine meaningfully slowed clinical decline in early Alzheimer's disease patients, demonstrating a favorable safety profile with no associated neuroimaging adverse...Read more
CAMBRIDGE, Mass., Dec. 18, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, today announced that the European Commission has adopted a positive decision for the designation of mitapivat, an oral, small molecule PK activator, as an orphan medicinal product (OMP) for the treatment of sickle cell disease. Earlier,...Read more
The Phase 3 VIVID-1 trial evaluated the safety and efficacy of Omvoh in patients with or without prior biologic failure and was the basis for the positive opinion VIVID-1 was the first pivotal Crohn's disease trial to show benefits in hard-to-treat symptom of bowel urgency using a patient-centric scale Omvoh will be the first treatment for Crohn's disease with results demonstrating improvements of histologic measures of...Read more
The Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of acoramidis in the EU based on positive results from the Phase 3 ATTRibute-CM study; final approval decision, typically consistent with the CHMP recommendation, is expected from the European Commission within the coming months Acoramidis, a near-complete (≥90%) stabilizer of transthyretin (TTR), was approved on November 22, 2024, by the U.S. Food...Read more
If approved, WELIREG would be the first and only oral hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor available for these patients in the European Union Positive opinion granted based on data from the Phase 2 LITESPARK-004 trial and the Phase 3 LITESPARK-005 trial RAHWAY, N.J. / Dec 13, 2024 / Business Wire / Merck (NYSE: MRK), known as MSD outside of the United States and Canada, today announced that the European Medicines...Read more
FOSTER CITY, Calif. / Dec 13, 2024 / Business Wire / Geron Corporation (Nasdaq: GERN), a commercial-stage biopharmaceutical company aiming to change lives by changing the course of blood cancer, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending the approval of RYTELO (imetelstat) for the treatment of adult patients with...Read more
Recommendation Based on the RESPONSE Study Which Demonstrated ALP Normalization in 25% of Participants at 12 Months and Statistically Significant Reduction of Pruritus Versus Placebo If Approved by the European Commission, Seladelpar Would Provide an Important Treatment Option for People Living with the Rare Liver Disease in the EU FOSTER CITY, Calif. / Dec 13, 2024 / Business Wire / Gilead Sciences, Inc. (Nasdaq: GILD) today...Read more
Bagsværd, Denmark, 12 December 2024 – Novo Nordisk today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for an update of the Ozempic® (once-weekly subcutaneous semaglutide) label to reflect data from the FLOW kidney outcomes trial. The FLOW trial assessed the risk reduction from Ozempic® therapy in chronic kidney disease-related events,...Read more
Application to label a less frequent dosing regimen at a dose of 2 mg/kg body weight administered every four weeks in adult patients with Fabry disease in European Union PARMA, Italy and CARMIEL Israel, Dec. 9, 2024 /PRNewswire/ -- Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, and Protalix BioTherapeutics, Inc....Read more
New indications based on two Phase 3 studies demonstrating statistically significant overall survival benefit for patients treated with TEVIMBRA in combination with chemotherapy SAN MATEO, Calif. / Nov 27, 2024 / Business Wire / BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global oncology company that intends to change its name to BeOne Medicines, today announced that the European Commission has approved TEVIMBRA®...Read more
First marketing authorization submission for blarcamesine NEW YORK, Nov. 26, 2024 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of Alzheimer's disease, Parkinson's disease, schizophrenia, neurodevelopmental, neurodegenerative, and rare diseases, including Rett syndrome, and other central...Read more
The PRIME initiative provides enhanced support to developers of promising medicines to optimize development plans and accelerate evaluation Pivotal trials of NX-5948 are planned to initiate in 2025 SAN FRANCISCO, Nov. 20, 2024 (GLOBE NEWSWIRE) -- Nurix Therapeutics, Inc. (Nasdaq: NRIX), a clinical stage biopharmaceutical company developing targeted protein modulation drugs designed to treat patients with cancer and inflammatory...Read more
SAN DIEGO, Nov. 20, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced that the European Medicines Agency (EMA) has granted both Orphan Drug and Advanced Therapy Medicinal Product (ATMP) designations to its lead asset, deramiocel, for the treatment of Duchenne muscular dystrophy (DMD). The...Read more
MALVERN, Pa., Nov. 20, 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, biologics, and vaccines, today announced that the European Medicines Agency (EMA) has granted orphan medicinal product designation for OCU410ST for the treatment of ABCA4-associated retinopathies including Stargardt disease,...Read more
In the EU, HYMPAVZI is the first once-weekly subcutaneous treatment approved for eligible people living with severe hemophilia B and the first to be administered via a pre-filled pen or syringe for people living with severe hemophilia A or B HYMPAVZI’s approval is based on Phase 3 study results demonstrating non-inferiority and superiority compared to routine prophylaxis in eligible patients with hemophilia A or B without...Read more
Orphan designation in E.U. supports development of treatments for life-threatening or chronically debilitating conditions with significant unmet medical need Phase 2 SOLSTICE 24-week primary endpoint data for tobevibart and elebsiran in chronic hepatitis delta to be presented today at AASLD The Liver Meeting Positive opinion on E.U. orphan drug designation follows U.S. FDA fast track designation, highlighting growing...Read more
OPUVIZ™ is one of the first wave aflibercept biosimilars in Europe OPUVIZ is the second European Commission (EC)-approved ophthalmology biosimilar under Samsung Bioepis and Biogen’s partnership EC approval based on robust totality of evidence confirming biosimilarity to reference aflibercept in terms of quality, efficacy, and safety INCHEON, South Korea and CAMBRIDGE, Mass., Nov. 18, 2024 (GLOBE NEWSWIRE) -- Samsung Bioepis Co.,...Read more
ELAHERE is the first and only novel therapy approved in the European Union specifically for patients with folate receptor-alpha (FRα) positive, platinum-resistant ovarian cancer ELAHERE represents the first treatment to demonstrate an overall survival benefit in a Phase 3 trial in platinum-resistant ovarian cancer compared with chemotherapy VENTANA FOLR1 (FOLR1-2.1) RxDx Assay, the companion diagnostic to identify ovarian cancer...Read more
Opinion based on results from the Phase 3 CheckMate -8HW trial, in which the dual immunotherapy combination of Opdivo and Yervoy demonstrated statistically significant and clinically meaningful improvement in progression-free survival compared to investigator’s choice of chemotherapy PRINCETON, N.J. / Nov 15, 2024 / Business Wire / Bristol Myers Squibb (NYSE: BMY) today announced that the Committee for Medicinal Products for Human Use...Read more
JENA, Germany, Nov. 15, 2024 (GLOBE NEWSWIRE) -- InflaRx N.V. (Nasdaq: IFRX), a biopharmaceutical company pioneering anti-inflammatory therapeutics targeting the complement system, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending marketing authorization of GOHIBIC (vilobelimab), under exceptional circumstances...Read more
Opinion granted based on positive overall survival results from the IND.227/KEYNOTE-483 trial RAHWAY, N.J. / Nov 15, 2024 / Business Wire / Merck (NYSE: MRK), known as MSD outside of the United States and Canada, today announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending approval of KEYTRUDA® (pembrolizumab), Merck’s anti-PD-1 therapy, in...Read more
Recommendation based on data from registrational TRIDENT-1 and CARE trials, which demonstrated robust responses and durable activity with repotrectinib in these patient populations If approved, repotrectinib has the potential to be a best-in-class treatment for patients with advanced ROS1-positive non-small cell lung cancer in the European Union PRINCETON, N.J. / Nov 15, 2024 / Business Wire / Bristol Myers Squibb (NYSE: BMY) today...Read more
TOKYO and CAMBRIDGE, Mass., Nov. 14, 2024 (GLOBE NEWSWIRE) -- Eisai Co., Ltd. (Headquarters: Tokyo, CEO: Haruo Naito, “Eisai”) and Biogen Inc. (Nasdaq: BIIB, Corporate headquarters: Cambridge, Massachusetts, CEO: Christopher A. Viehbacher, “Biogen”) announced today a positive opinion has been received from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommending approval of the...Read more
TORONTO and HAIFA, Israel, Nov. 13, 2024 (GLOBE NEWSWIRE) -- NurExone Biologic Inc. (TSXV: NRX) (OTCQB: NRXBF) (Germany: J90) ("NurExone" or the "Company"), a biopharmaceutical company developing exosome-based regenerative therapies, is pleased to announce that the European Medicines Agency (the “EMA”) has granted Orphan Medicinal Product Designation for the Company’s ExoPTEN therapy, marking a significant step towards making this...Read more
If approved, DARZALEX FASPRO® will become the first treatment option for patients with smoldering multiple myeloma at high-risk of developing multiple myeloma, offering a novel approach to treat before the onset of active disease and the occurrence of end organ damage RARITAN, N.J., Nov. 8, 2024 /PRNewswire/ -- Johnson & Johnson (NYSE:JNJ) today announced the submission of regulatory applications to the U.S. Food and...Read more
Approval based on Phase 3 data showing significantly more children aged 1 to 11 years on Dupixent achieved histological disease remission at 16 weeks compared to placebo, which was sustained up to one year Dupixent is the first-ever medicine in the EU indicated to treat these young patients, who persistently struggle to eat at a critical stage in life where growth is crucial TARRYTOWN, N.Y. and PARIS, Nov. 06, 2024 (GLOBE NEWSWIRE)...Read more
Astria Therapeutics is a biopharmaceutical company, and our mission is to bring life-changing therapies to patients and families affected by rare and niche allergic and immunological diseases. Our lead program, STAR-0215, is a monoclonal antibody inhibitor of plasma kallikrein in clinical development...
CLICK TO LEARN MORE