Atea Pharmaceuticals, Inc. a clinical-stage biopharmaceutical company engaged in the discovery and development of oral antiviral therapeutics for serious viral diseases, has announced that its Board of Directors unanimously rejected the unsolicited proposal from Tang Capital Partners, LP on behalf of Concentra Biosciences, LLC to acquire all outstanding common shares of Atea for $5.75 per share in cash, plus a contingent value right representing the right to receive 80% of the net proceeds payable from any license or disposition of Atea’s programs (the “Proposal”)
Braeburn announces that the FDA has approved Brixadi (buprenorphine) extended-release injection for subcutaneous use (CIII), a new weekly and monthly medication for moderate to severe opioid use disorder (OUD) in patients who have initiated treatment with a single dose of a transmucosal buprenorphine product or who are already being treated with a transmucosal buprenorphine-containing product
Amgen announced new research examining the use of Otezla (apremilast) in psoriatic arthritis, including the Phase IV MOSAIC study and an exploratory analysis of cardiometabolic risk factors, which are commonly elevated in patients with psoriatic disease. The findings will be presented at the 2023 European Congress of Rheumatology (EULAR), taking place May 31-June 3 in Milan, Italy
UCB, a global biopharmaceutical company, announced new long-term 52-week data from three Phase III studies BE COMPLETE with its long-term extension study, BE MOBILE 1 and BE MOBILE 2 – evaluating the efficacy and safety profile of bimekizumab, an inhibitor of IL-17F in addition to IL-17A, in adults with active psoriatic arthritis (PsA), active non-radiographic axial spondyloarthritis (nr-axSpA) and active ankylosing spondylitis (AS), also known as radiographic axSpA (r-axSpA), respectively
Akebia Therapeutics, Inc. announced that the company received a written response from the Office of New Drugs (OND) of the FDA to Akebia's Formal Dispute Resolution Request (FDRR) regarding the Complete Response Letter (CRL) received in March 2022 for vadadustat
Pfizer Inc. announced that the pivotal Phase III BASIS clinical trial (NCT03938792) evaluating marstacimab has met its primary endpoints, having demonstrated statically significant and clinically meaningful effects
Bristol Myers Squibb announced that the FDA has accepted the New Drug Application (NDA) for repotrectinib, a next-generation tyrosine kinase inhibitor (TKI), for the treatment of patients with ROS1-positive locally advanced or metastatic non-small cell lung cancer (NSCLC), based on results from the TRIDENT-1 trial
BeiGene, Ltd announced that Brukinsa (zanubrutinib), a Bruton's tyrosine kinase inhibitor (BTKi), has been approved by Health Canada for the treatment of adult patients with chronic lymphocytic leukemia (CLL)
Novavax, Inc. announced that Nuvaxovid (NVX-CoV2373) has been recommended for full Marketing Authorization (MA) for use as a primary series in individuals aged 12 and older and as a booster in individuals aged 18 and older for the prevention of COVID-19 in the European Union (EU) following a positive opinion issued by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency
Hansa Biopharma, a pioneer in enzyme technology for rare immunological conditions, announced the first patient has been dosed with imlifidase in the GOOD-IDES-02 trial, a global pivotal phase III trial in anti-glomerular basement membrane (anti-GBM) disease
BioXcel Therapeutics, Inc., announced promising results for BXCL 501, the Company’s proprietary, orally dissolving film of dexmedetomidine under investigation for the treatment of agitation, in Part 1 of its Phase III SERENITY III trial conducted in institutional settings for acute treatment of bipolar disorders- or schizophrenia-associated agitation
The FDA approved sulbactam and durlobactam (SUL+DUR; Xacduro, Innoviva Specialty Therapeutics) co-packaged for IV use to treat hospital-acquired bacterial pneumonia (HABP) and ventilator-associated bacterial pneumonia (VABP) caused by susceptible strains of Acinetobacter baumannii–calcoaceticus complex
Takeda and Hutchmed (China) Limited announced that the FDA has granted priority review of the New Drug Application (“NDA”) for fruquintinib, a highly selective and potent inhibitor of vascular endothelial growth factor receptors -1, -2 and -3 for the treatment of adult patients with previously treated metastatic colorectal cancer
Ribociclib (Kisqali) plus switch endocrine therapy induced a statistically significant progression-free survival (PFS) benefit compared with placebo plus switch endocrine therapy in patients with hormone receptor (HR)–positive, HER2-negative, metastatic breast cancer, according to findings from the phase II MAINTAIN trial (NCT02632045) previously presented at the 2022 ASCO Annual Meeting, now published in the Journal of Clinical Oncology
Verastem Oncology announced updated data from Part A of the ongoing registration-directed RAMP 201 (ENGOTov60/GOG3052) trial evaluating the safety and efficacy of avutometinib (VS 6766) alone and in combination with defactinib among patients with recurrent low-grade serous ovarian cancer (LGSOC)
Beta Bionics, Inc. — a medical technology company focused on diabetes — announces FDA 510(k) clearance and the commercial launch of the iLet Bionic Pancreas.
Lexicon Pharmaceuticals, Inc. announced that the FDA has approved Inpefa (sotagliflozin), a once-daily oral tablet to reduce the risk of cardiovascular death, hospitalization for heart failure, and urgent heart failure visit in adults with: heart failure or type 2 diabetes mellitus, chronic kidney disease, and other cardiovascular risk factors.
Marinus Pharmaceuticals 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 Ztalmy (ganaxolone) oral suspension for the adjunctive treatment of epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients two to 17 years of age. Ztalmy may be continued in patients 18 years of age and older.
The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the revocation of the conditional marketing authorization (MA) for Adakveo (crizanlizumab), from Novartis, a once-a-month, humanized anti-P-selectin monoclonal antibody infusion indicated for the prevention of recurrent vaso?occlusive crises (pain crises) in sickle cell disease patients aged 16 years and older.
Celltrion USA announced that the FDA has approved Yuflyma (adalimumab-aaty), a high-concentration (100mg/mL) and citrate-free formulation of Humira (adalimumab) biosimilar.
Bristol Myers Squibb announced first results from the Phase III COMMANDS study, an open-label, randomized trial evaluating Reblozyl (luspatercept-aamt) versus epoetin alfa, an erythropoiesis-stimulating agent (ESA), for the treatment of anemia in adult patients with very low-, low- or intermediate-risk myelodysplastic syndromes (MDS) who require red blood cell (RBC) transfusions and are ESA-naïve. Results from the study will be featured at the American Society of Clinical Oncology (ASCO) Annual Meeting and in an oral presentation of select abstracts during a plenary session at the European Hematology Association (EHA) Congress.
SpringWorks Therapeutics, Inc. announced that additional data from the Phase III DeFi trial assessing the impact of nirogacestat, an investigational gamma secretase inhibitor, on pain, tumor volume and T2 hypersensitivity in adults with desmoid tumors will be presented at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting, being held June 2-6, 2023. Data from the DeFi trial were previously presented at the European Society for Medical Oncology Congress in September 2022 and published in the March 9, 2023 edition of the New England Journal of Medicine.
Pfizer Inc. announced that the FDA approved Paxlovid(nirmatrelvir tablets and ritonavir tablets) for the treatment of mild-to-moderate COVID-19 in adults who are at high risk for progression to severe COVID-19, including hospitalization or death.
Bristol Myers Squibb announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended approval of Opdivo (nivolumab) in combination with platinum-based chemotherapy for the neoadjuvant treatment of resectable non-small cell lung cancer (NSCLC) at a high risk of recurrence in adult patients with tumor cell PD-L1 expression greater than 1%.
AbbVie announced the New England Journal of Medicine (NEJM) published results from the pivotal Phase III clinical trials – U-EXCEL, U-EXCEED and U-ENDURE – evaluating upadacitinib (Rinvoq) in adult patients with moderately to severely active Crohn's disease who have had an inadequate response, lost response or were intolerant to conventional therapy or a biologic agent.
Novo Nordisk announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion, recommending once-weekly Sogroya (somapacitan) for replacement of endogenous growth hormone (GH) in children aged three years and above, and adolescents with growth failure due to growth hormone deficiency.
Positive high-level results from the DUO-E Phase III trial showed Imfinzi (durvalumab) in combination with platinum-based chemotherapy followed by either Imfinzi plus Lynparza (olaparib) or Imfinzi alone as maintenance therapy both demonstrated a statistically significant and clinically meaningful improvement in progression-free survival (PFS) compared to standard-of-care chemotherapy alone in patients with newly diagnosed advanced or recurrent endometrial cancer
Ultomiris (ravulizumab) has been approved in Japan as the first and only long-acting C5 complement inhibitor for the prevention of relapses in patients with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD), including neuromyelitis optica
PDS Biotechnology Corporation, announced promising interim data from the VERSATILE-002 (NCT04260126) Phase II clinical trial investigating PDS 0101 in combination with Merck’s anti-PD-1 therapy, Keytruda (pembrolizumab), in patients with unresectable, recurrent or metastatic human papillomavirus (HPV)16-positive head and neck cancer
Eisai and Merck & Co., Inc., announced data from the final pre-specified overall survival (OS) analysis of the pivotal Phase III CLEAR (Study 307)/KEYNOTE-581 trial investigating Lenvima, the orally available multiple receptor tyrosine kinase inhibitor discovered by Eisai, plus Keytruda, Merck’s anti-PD-1 therapy, for the first-line treatment of patients with advanced renal cell carcinoma (RCC)
Gilead Sciences, Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) granted a positive opinion for the use of Veklury (remdesivir) in COVID-19 patients with severe renal impairment, including those on dialysis. The European Commission (EC) will review the CHMP recommendation and, if adopted, Veklury will become the first and only authorized antiviral COVID-19 treatment that can be used across all stages of renal disease
Apellis Pharmaceuticals, Inc. announced that the Phase II MERIDIAN study investigating systemic pegcetacoplan for the treatment of amyotrophic lateral sclerosis (ALS) did not meet its primary endpoint of the Combined Assessment of Function and Survival (CAFS) rank score at Week 52
Vanda Pharmaceuticals Inc. announced the results from its Phase III study of tradipitant in motion sickness, confirming the previously reported results demonstrating that tradipitant is effective in the prevention of vomiting associated with motion sickness
Pyxis Oncology, Inc. a clinical-stage company focused on developing next-generation therapeutics to target difficult-to-treat cancers, and Apexigen, Inc., a clinical-stage biopharmaceutical company focused on discovering and developing innovative antibody therapeutics for oncology, announced a definitive agreement by which Pyxis Oncology will acquire Apexigen in an all-stock transaction for an implied value of $0.64 per Apexigen share
<p class="epg-standfirst-tinymce">Mirati Therapeutics, Inc. announced that the SAPPHIRE study did not meet its primary endpoint of overall survival at the final analysis</p>
Sandoz, a global leader in off-patent (generic and biosimilar) medicines, announced that the European Medicines Agency (EMA) has accepted the marketing authorization applications (MAA) for proposed biosimilar denosumab for regulatory review
Bayer announced that the Chinese National Medical Products Administration (NMPA) granted marketing authorization for a label extension for Kerendia (finerenone) to include results on cardiovascular (CV) outcomes from the Phase III FIGARO-DKD study
On May 22, 2023, Ayvakit became the first and only medicine approved by the FDA for the treatment of adults with ISM
Sarepta Therapeutics, Inc. provided the following update on the Biologics License Application (BLA) for SRP 9001 (delandistrogene moxeparvovec), which is currently under review for the treatment of ambulant individuals with Duchenne muscular dystrophy (DMD) who have a confirmed mutation of the DMD gene
New data presented from an investigator-sponsored European trial found managing indicated1 heart failure patients with Abbott's CardioMEMS HF System resulted in a significant improvement in patient-reported quality-of-life scores as early as three months after use with the remote monitoring sensor The MONITOR-HF trial also resulted in a significant 44% reduction in heart failure-related hospitalizations among chronic heart failure patients who used guideline-directed medical therapy (GDMT)
CohBar, Inc. and Morphogenesis, Inc. a privately-held Phase II/III clinical-stage biotechnology company developing novel personalized cancer vaccines and tumor microenvironment modulators to overcome resistance to current immunotherapies, announced that they have entered into a definitive agreement for an all-stock transaction forming a company combining expertise and resources to advance a late-stage oncology pipeline
Ironwood Pharmaceuticals, Inc. a GI-focused healthcare company, and VectivBio Holding AG, a clinical-stage biopharmaceutical company pioneering novel, transformational treatments for severe rare gastrointestinal conditions, announced that they have entered into a definitive agreement for Ironwood to acquire VectivBio for $17.00 per share in an all-cash transaction with an estimated aggregate consideration of approximately $1 billion, net of VectivBio cash and debt The acquisition price represents a premium of 80% relative to the volume-weighted average share price over the previous 90 trading days
PTC Therapeutics, Inc. reported topline results from the MOVE-FA trial of vatiquinone in patients with Friedreich ataxia
Cumberland Pharmaceuticals Inc. announced that the FDA has approved expanded labeling for Caldolor an intravenously delivered formulation of ibuprofen, to now include use in infants
AbbVie announced that the FDA has approved Rinvoq (upadacitinib) for the treatment of adults with moderately to severely active Crohn’s disease who have had an inadequate response or intolerance to one or more TNF blockers
Bausch + Lomb Corporation and Novaliq GmbH, a biopharmaceutical company focusing on first- and best-in class ocular therapeutics, announced that the FDA has approved Miebo (perfluorohexyloctane ophthalmic solution; formerly known as NOV03), for the treatment of the signs and symptoms of dry eye disease (DED)
Astellas Pharma Inc. announced that the FDA has approved Vezoah (fezolinetant) 45 mg once daily for the treatment of moderate to severe vasomotor symptoms (VMS) due to menopause.
Blueprint Medicines Corporation announced that the FDA has approved Ayvakit (avapritinib) for the treatment of adults with indolent systemic mastocytosis (ISM)
Indivior PLC announced that the FDA approved Opvee (nalmefene) nasal spray from Opiant Pharma /Invidor, for the emergency treatment of known or suspected opioid overdose induced by natural or synthetic opioids in adults and pediatric patients aged 12 years and older, as manifested by respiratory and/or central nervous system depression
Boehringer Ingelheim announced that the FDA has approved the Cyltezo Pen, a new autoinjector option for Cyltezo (adalimumab-adbm), an FDA-approved Interchangeable biosimilar to Humira (adalimumab)
Novo Nordisk announced headline results from OASIS 1, a phase IIIa trial in the global OASIS programme
Notable developments for the Watchman FLX LAAC Device: Data presented from two new sub-analyses of the SURPASS study out of the National Cardiovascular Data Registry (NCDR) LAAO Registry provided insights into real-world treatment strategies with the Watchman FLX LAAC Device
Effects of the EMBLEM MRI Subcutaneous Implantable Defibrillator (S-ICD) on tricuspid regurgitation Data from a secondary analysis of the investigator-sponsored, randomized ATLAS trial compared among nearly 450 patients the severity of tricuspid regurgitation at six months following the implantation of a transvenous implantable cardioverter-defibrillator (TV-ICD) versus the EMBLEM MRI S-ICD
Regeneron Pharmaceuticals, Inc. and Sanofi presented positive Phase III results evaluating the investigational use of Dupixent (dupilumab) compared to placebo in adults currently on maximal standard-of-care inhaled therapy (triple therapy) with uncontrolled chronic obstructive pulmonary disease (COPD) and evidence of type 2 inflammation
Primary results of the FROZEN-AF IDE trial with the POLARx Cryoablation System: Results from the global, prospective, non-randomized, single-arm FROZEN-AF IDE study of the POLARx Cryoablation System met the safety and effectiveness endpoints of the trial
Real-world outcomes from the multi-center EU-PORIA registry were highlighted in a late-breaking data presentation, further demonstrating the safety, efficacy and learning curve characteristics of the Farapulse PFA System
Medtronic plc announced longer-term follow-up results of its investigational EV ICD System, designed to treat dangerously fast heart rhythms that can lead to sudden cardiac arrest.
Alnylam Pharmaceuticals announced new results from an interim analysis of exploratory data from the open-label extension (OLE) period of the APOLLO-B Phase III study of Onpattro (patisiran), an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy.
Genmab/AbbVie announced that the FDA has approved Epkinly (epcoritamab-bysp) as the first and only T-cell engaging bispecific antibody for the treatment of adult patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL), not otherwise specified (NOS), including DLBCL arising from indolent lymphoma, and high-grade B?cell lymphoma, after two or more lines of systemic therapy.
HanAll Biopharma announced results from the Phase III VELOS-3 trial evaluating the safety and efficacy of tanfanercept, a novel, topical anti-inflammatory treatment being studied for the treatment of dry eye disease (DED) in subjects diagnosed with moderate to severe DED.
Krystal Biotech, Inc. announced that the FDA has approved Vyjuvek (beremagene geperpavec-svdt) for the treatment of patients six months of age or older with dystrophic epidermolysis bullosa (DEB). Vyjuvek is designed to address the genetic root cause of DEB by delivering functional copies of the human COL7A1 gene to provide wound healing and sustained functional COL7 protein expression with redosing.
Intercept Pharmaceuticals, Inc. announced the outcome of the FDA Gastrointestinal Drugs Advisory Committee (GIDAC) Meeting to review the Company’s New Drug Application (NDA) for obeticholic acid (OCA) for the treatment of pre-cirrhotic fibrosis due to nonalcoholic steatohepatitis (NASH).
Neurocrine Biosciences, Inc. announced that the complete study results from its Phase III KINECT-HD study investigating valbenazine for the treatment of chorea associated with Huntington's disease (HD) has been published in The Lancet Neurology online edition and will appear in the June 2023 print issue.
PTC Therapeutics, Inc. announced that the primary endpoint was achieved in the APHENITY, Phase III registration-directed clinical trial of sepiapterin in adult and pediatric patients with phenylketonuria (PKU).
Abbott announced that the FDA has approved the company's TactiFlex Ablation Catheter, Sensor Enabled, the world's first ablation catheter with a flexible tip and contact force technology.
Biodexa Pharmaceuticals PLC , a clinical stage biopharmaceutical company developing a pipeline of products aimed at primary and metastatic cancers of the brain is pleased to announce that it has signed non-binding letter of intent to potentially acquire Varian Biopharmaceuticals, Inc., a private US precision oncology company developing novel therapeutics for the treatment of cancer.
EyePoint Pharmaceuticals, Inc. announced that it has entered into a definitive agreement for the sale of Yutiq (fluocinolone acetonide intravitreal implant) 0.18mg to Alimera Sciences, Inc. (“Alimera”).
Sobi announced that the European Medicines Agency (EMA) has accepted and validated a marketing authorisation application for efanesoctocog alfa, a new class of high-sustained FVIII developed for the treatment of people with haemophilia A of all age groups.
Abbott announced its Assert-IQ insertable cardiac monitor (ICM) has received FDA clearance, giving physicians a new option for diagnostic evaluation and long-term monitoring of people experiencing irregular heartbeats.
The New England Journal of Medicine published detailed results from Eli Lilly and Company's phase IIa study of peresolimab in rheumatoid arthritis (RA), in which peresolimab met the primary endpoint for efficacy and had similar rates of adverse events between peresolimab and placebo arms.
Eisai and Biogen announced that Canadian health authorities have accepted for review an application for their anti-amyloid beta antibody lecanemab for the treatment of early Alzheimer’s disease (AD) with confirmed amyloid pathology in the brain.
Takeda announced that the FDA has accepted Takeda’s Biologics License Application (BLA) for TAK 755, an enzyme replacement therapy for the treatment of congenital thrombotic thrombocytopenic purpura (cTTP), an ADAMTS13 deficiency disorder. The TAK 755 application was accepted by the FDA on May 16th, and has been granted Priority Review.
Ardelyx, Inc. announced that the FDA has accepted its resubmission of a New Drug Application (NDA) for Xphozah (tenapanor) for the control of serum phosphate in adult patients with chronic kidney disease on dialysis who have had an inadequate response or intolerance to a phosphate binder therapy.
Bayer announced that the Chinese National Medical Products Administration (NMPA) granted marketing authorization for a label extension for Kerendia (finerenone) to include results on cardiovascular (CV) outcomes from the Phase III FIGARO-DKD study.
Allergan Aesthetics, an AbbVie company announced the FDA approval of Skinvive by Juvederm to improve skin smoothness of the cheeks in adults over the age of 21. Skinvive by Juvederm is the first and only hyaluronic acid (HA) intradermal microdroplet injection for skin smoothness available in the U.S. with results lasting through six months with optimal treatment.
Bristol Myers Squibb's Sotyktu (deucravacitinib) has been authorised for use in the UK to treat adults with moderate-to-severe plaque psoriasis who are candidates for systemic therapy.
Roche announced positive results from the Phase II FENopta study evaluating investigational oral fenebrutinib in adults with relapsing forms of multiple sclerosis (RMS).
Positive high-level results from the FLAURA2 Phase III trial showed AstraZeneca’s Tagrisso (osimertinib) in combination with chemotherapy demonstrated a statistically significant and clinically meaningful improvement in progression-free survival (PFS) compared to Tagrisso alone for patients with locally advanced (Stage IIIB-IIIC) or metastatic (Stage IV) epidermal growth factor receptor-mutated (EGFRm) non-small cell lung cancer (NSCLC).
Dermavant Sciences announced positive results from ADORING 1, the second of two double-blind, randomized, vehicle-controlled Phase III studies to evaluate the efficacy and safety of topical Vtama (tapinarof) cream, 1% in adults and pediatric subjects down to 2 years old with moderate to severe atopic dermatitis (AD). In ADORING 1 (N=407), Vtama met the primary endpoint of the trial and demonstrated highly statistically significant improvement in the Validated Investigator Global Assessment for Atopic Dermatitis (vIGA-ADTM) score of clear (0) or almost clear (1) with at least a 2-grade improvement from baseline at Week 8 (P<0.0001).
Arrowhead Pharmaceuticals Inc. announced that it has completed enrollment of its global Phase III clinical trial (PALISADE) evaluating ARO-APOC3 for the treatment of familial chylomicronemia syndrome.
Abbott announced that the FDA has approved its spinal cord stimulation (SCS) devices for the treatment of chronic back pain in people who have not had or are not eligible to receive back surgery, known as non-surgical back pain.
Gilead Sciences, Inc. and Arcus Biosciences, Inc. announced that the companies have expanded the previously announced research collaboration focused on oncology to include therapies for the treatment of inflammatory diseases.
The Janssen Pharmaceutical Companies of Johnson & Johnson announced new efficacy and safety data from the Phase III QUASAR Induction Study evaluating the investigational use of Tremfya (guselkumab) in adults with moderately to severely active ulcerative colitis (UC)(defined as a baseline modified Mayo score of 5 to 9, inclusive of a rectal bleeding subscore greater than 1 and an endoscopy subscore greater than 2 evaluated during central review of video endoscopy) who had an inadequate response or intolerance to conventional and/or advanced therapies (thiopurines or corticosteroids).
Kyowa Kirin Co., Ltd. announces the discontinuation of the clinical development program for bardoxolone methyl (RTA 402), a small-molecule compound licensed from Reata Pharmaceuticals, Inc.
Sarepta Therapeutics, Inc. announced that the FDA)Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) voted 8 to 6 in support of accelerated approval of SRP 9001 (delandistrogene moxeparvovec) for the treatment of ambulatory patients with Duchenne muscular dystrophy with a confirmed mutation in the DMD gene.
Oramed Pharmaceuticals Inc. announced that Hefei Tianhui Biotechnology Co. Ltd. (HTIT), a strategic partner of Oramed, has successfully completed its Phase III trials of oral insulin in type 2 diabetes in China under a differentiated study protocol. HTIT is now moving toward regulatory approval and has submitted the data to the National Medical Products Administration (NMPA, formerly the CFDA).
Orchard Therapeutics held a productive pre-BLA meeting with the FDA which led to the initiation of a rolling BLA for OTL 200 for the treatment of early-onset metachromatic leukodystrophy (MLD).
EQRx resets to focus on clinically differentiated medicines: Prioritizing development of lerociclib (CDK 4/6 inhibitor); initiated Phase III trial in first-line advanced endometrial cancer; enrollment in Phase II trial in first- and second-line advanced breast cancer near completion, providing a foundation for future combination development opportunities.
Bayer and Bicycle Therapeutics plc, a biotechnology company pioneering a new and differentiated class of therapeutics by utilizing proprietary bicyclic peptides technology (Bicycle), announced that they have entered into a strategic collaboration agreement to discover, develop, manufacture, and commercialize Bicycle radioconjugates for multiple agreed upon oncology targets.
Janssen Biotech, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson, announced that it has entered into a worldwide collaboration and license agreement with Cellular Biomedicine Group Inc. (CBMG) to develop, manufacture and commercialize next-generation chimeric antigen receptor (CAR) T-cell therapies for the treatment of B-cell malignancies.
GSK plc presented preliminary positive results from the phase III trial (NCT04502693) evaluating the immunological vaccine effectiveness and safety of its MenABCWY combination vaccine candidate, administered as two doses given six months apart in healthy individuals aged 10-25 years. The preliminary data were disclosed at the 41st Annual Meeting of the European Society for Paediatric Infectious Diseases (ESPID) in Lisbon, Portugal.
ARS Pharmaceuticals, Inc., a biopharmaceutical company dedicated to empowering at-risk patients and caregivers to better protect themselves from severe allergic reactions that could lead to anaphylaxis, announced that the FDA Pulmonary-Allergy Drug Advisory Committee (PADAC) voted 16:6 in favor for adults, and 17:5 in favor for children (<18 years of age and ?30 kg), that available data support a favorable benefit-risk assessment for neffy in the treatment of severe allergic reaction (type 1), including anaphylaxis,for adults and children who weigh more than 30kg.
New data from the HARMONIE Phase IIIb clinical trial show an 83.21% (95% CI 67.77 to 92.04; P<0.001) reduction in hospitalizations due to rsv-related lrtd (lower respiratory tract disease) in infants under 12 months of age who received a single dose of nirsevimab, compared to infants who received no rsv intervention.
H. Lundbeck A/S (Lundbeck) and Otsuka Pharmaceutical, Co. Ltd. (Otsuka) announce the FDA has approved the supplemental New Drug Application (sNDA) of Rexulti (brexpiprazole) for use in the treatment of agitation associated with dementia due to Alzheimer’s disease.
Health Canada has approved the combination of cemiplimab-rwlc (Libtayo) and platinum-based chemotherapy for the first-line treatment of patients with locally advanced or metastatic non–small cell lung cancer (NSCLC) without EGFR, ALK, or ROS1 aberrations who are not candidates for definitive chemoradiation.
Idorsia Ltd announced that Health Canada has granted marketing authorization for Quviviq (daridorexant) for the management of adult patients with insomnia, characterized by difficulties with sleep onset and/or sleep maintenance, on April 28, 2023.
Vertex Pharmaceuticals Incorporated announced the FDA approved Kalydeco (ivacaftor) for use in children with cystic fibrosis (CF) ages 1 month to less than four months old who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to Kalydeco based on clinical and/or in vitro assay data.
Avadel Pharmaceuticals plc that the FDA has granted final approval to Lumryz, an extended-release formulation of sodium oxybate indicated to be taken once at bedtime for the treatment of cataplexy or excessive daytime sleepiness (EDS) in adults with narcolepsy.
Cassava Sciences, Inc. announced the completion of patient dosing in a 6-month, randomized controlled trial of simufilam in over 125 patients with Alzheimer’s disease. This trial is known as the Cognition Maintenance Study (CMS).
Swedish Orphan Biovitrum AB (publ) (Sobi) announced that it has entered into an agreement and plan of merger with CTI BioPharma Corp. (CTI) under which Sobi has agreed to acquire CTI, a biopharmaceutical company focused on blood related cancers and rare diseases, by means of a tender offer.
Bayer announced the initiation of the phase III QUASAR study, designed to evaluate the efficacy and safety of aflibercept 8 mg dosed at extended treatment intervals compared to the standard of care, Eylea (aflibercept 2 mg) in macular edema secondary to retinal vein occlusion (RVO).
Minerva Neurosciences, Inc. announced that on May 8, 2023, it received confirmation from the FDA that the Company’s New Drug Application (NDA) for roluperidone for the treatment of negative symptoms in patients with schizophrenia has been filed in accordance with the recent Appeal Granted letter dated April 27, 2023 and assigned a standard review classification.
Ultomiris (ravulizumab) from AstraZeneca has been approved in the European Union (EU) as the first and only long-acting C5 complement inhibitor for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD).
Gilead Sciences, Inc. announced the acquisition of all outstanding shares of XinThera, a privately held biotech company in San Diego. The acquisition complements Gilead’s existing clinical development priorities by adding additional pipeline assets for well-validated targets in oncology and inflammation.
Eli Lilly and Company announced new investigational data for mirikizumab in patients with moderately to severely active ulcerative colitis (UC) that further support the efficacy and safety seen in previous pivotal studies.
AstraZeneca’s Farxiga (dapagliflozin) has been approved in the US to reduce the risk of cardiovascular (CV) death, hospitalisation for heart failure (hHF) and urgent heart failure (HF) visits in adults with HF.
Genentech, a member of the Roche Group announced that the FDA has accepted the company’s supplemental Biologics License Application (sBLA) for Vabysmo (faricimab-svoa) for the treatment of macular edema following retinal vein occlusion (RVO).
Eyenovia Inc. announced the FDA approval of Mydcombi (tropicamide and phenylephrine hydrochloride ophthalmic spray) 1%/2.5% for inducing mydriasis for diagnostic procedures and in conditions where short-term pupil dilation is desired.
Trevena, Inc. a biopharmaceutical company focused on the development and commercialization of novel medicines for patients with central nervous system (CNS) disorders, announced that its partner in China, Jiangsu Nhwa, received formal approval from the National Medical Products Administration for Olinvyk.
Takeda announced that the FDA has accepted for review its Biologics License Application (BLA) resubmission for the investigational subcutaneous (SC) administration of Entyvio (vedolizumab) for maintenance therapy in adults with moderately to severely active ulcerative colitis (UC) after induction therapy with Entyvio intravenous. The resubmission is intended to address FDA feedback in a December 2019 Complete Response Letter (CRL).
Koselugo (selumetinib) from AstraZeneca + Merck Inc., has been approved in China for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in paediatric patients with neurofibromatosis type 1 (NF1) aged three years and above.
BeiGene announced the China National Medical Products Administration (NMPA) approved four applications for Brukinsa (zanubrutinib), the company’s Bruton’s tyrosine kinase inhibitor (BTKi), including two Supplemental New Drug Applications for treatment-naïve adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) and Waldenström's macroglobulinemia (WM), and two Supplemental Applications for conversions from conditional approval to regular approval.
FibroGen announced that , a Phase III clinical study of Evrenzo (roxadustat) for treatment of anaemia in patients with transfusion-dependent lower risk myelodysplastic syndromes (MDS) did not meet its primary efficacy endpoint. The proportion of patients who achieved red blood cell transfusion independence in the first 28 weeks was 47.5% for the roxadustat arm compared to 33.3% for placebo (p=0.217).
Chiesi Group and Protalix BioTherapeutics announced that the European Commission has granted marketing authorization to PRX 102 (pegunigalsidase alfa) in the European Union for the treatment of adult patients with Fabry disease.
Sobi North America, the North American affiliate of Swedish Orphan Biovitrum AB (Sobi) announced that Health Canada has approved Empaveli (pegcetacoplan), a complement inhibitor, for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who have an inadequate response to, or are intolerant of, a C5 inhibitor. Empaveli is a targeted C3 therapy designed to regulate the activity of the complement cascade, which can lead to the onset and progression of many serious diseases.
Magenta Therapeutics, Inc. and Dianthus Therapeutics, Inc. , a privately-held, clinical-stage biotechnology company dedicated to advancing the next generation of antibody complement therapeutics, announced that they have entered into a definitive merger agreement to combine the companies in an all-stock transaction.
GSK plc announced that the FDA has approved Arexvy (respiratory syncytial virus vaccine, adjuvanted) for the prevention of lower respiratory tract disease (LRTD) caused by respiratory syncytial virus (RSV) in individuals 60 years of age and older. This is the first RSV vaccine for older adults to be approved anywhere in the world.
Soligenix, Inc. announced positive clinical results from a compatibility study evaluating HyBryte (synthetic hypericin sodium) in the treatment of cutaneous T-cell lymphoma (CTCL) using the commercially ready Daavlin Series 7 visible light device, which recently received 510(k) clearance from FDA.
ImmunoGen Inc. announced positive top-line data from the Phase III confirmatory MIRASOL trial (GOG 3045/ENGOT OV-55) evaluating the safety and efficacy of Elahere (mirvetuximab soravtansine-gynx) compared to chemotherapy in patients with folate receptor alpha (FRalpha)-positive platinum-resistant ovarian cancer who have received one to three prior lines of therapy.
Bristol Myers Squibb announced that the European Commission (EC) has granted approval for Breyanzi (lisocabtagene maraleucel; liso-cel)
Eli Lilly and Company announced positive results of the TRAILBLAZER-ALZ 2 Phase III study showing that donanemab significantly slowed cognitive and functional decline in people with early symptomatic Alzheimer's disease.
Abivax SA announces the Journal of Crohn’s and Colitis (JCC) publication of a piece titled “Obefazimod: a first-in-class drug for the treatment of ulcerative colitis”, written by global Inflammatory Bowel Disease (IBD) experts
Arcutis Biotherapeutics, Inc. announced the enrollment of the last subject in the INTEGUMENT-PED Pivotal Phase III trial evaluating roflumilast cream 0.05% for the treatment of mild to moderate AD in children ages 2 to 5
Travere Therapeutics, Inc. announced topline primary efficacy results from the pivotal Phase III DUPLEX Study of sparsentan, a Dual Endothelin Angiotensin Receptor Antagonist (DEARA), in focal segmental glomerulosclerosis (FSGS)
Astellas Pharma Inc. and Iveric bio, Inc. announced that, the Companies have entered into a definitive agreement under which Astellas through Berry Merger Sub, Inc. a wholly-owned subsidiary of Astellas US Holding, Inc., has agreed to acquire 100% of the outstanding shares of Iveric Bio for US$40.00 per share in cash for a total equity value of approximately US$5.9 billion.
Meiji Holdings announced on April 28 that subsidiary Meiji Seika Pharma has filed an application seeking Japanese regulatory approval for ARCT -154, an mRNA vaccine in-licensed from CSL Seqirus, for a primary two-dose inoculation for the prevention of COVID-19 in adults.
Horizon Therapeutics plc announced that the first patient has been enrolled in a Phase III clinical trial in Japan evaluating Tepezza for the treatment of adults with chronic Thyroid Eye Disease (TED) and low clinical activity score (CAS), which is a measure of disease activity
Bausch Health Companies Inc. and its dermatology business, Ortho Dermatologics, announced that the FDA has accepted the NDA for investigational IDP 126 (clindamycin 1.2%/adapalene 0.15%/benzoyl peroxide 3.1%) Gel with a Prescription Drug User Fee Act (PDUFA) action date of October 20, 2023
Medtronic plc announced it has received FDA approval of its Micra AV2 and Micra VR2, the next generation of its industry-leading miniaturized, leadless pacemakers
Bristol Myers Squibb announced that the FDA has accepted the supplemental Biologics License Application (sBLA) and the European Medicines Agency (EMA) has validated the Type II Variation Application for Reblozyl (luspatercept-aamt), a first-in-class treatment option, to expand its current indication to include treatment of anemia without previous use of erythropoiesis-stimulating agents (ESA-naïve) in adult patients with very low- to intermediate-risk myelodysplastic syndromes (MDS) who may require red blood cell (RBC) transfusions
Minerva Neurosciences, Inc. a clinical-stage biopharmaceutical company focused on the development of therapies to treat central nervous system (CNS) disorders, announced that the FDA filed the Company’s New Drug Application (NDA) for roluperidone for the treatment of negative symptoms in patients with schizophrenia on April 27, 2023
