ALL represents 10% of all leukemia cases in the United States, progresses rapidly, and is typically fatal within weeks or months if left untreated1 There is an urgent need to develop new therapies for ALL for patients who are not candidates for hematopoietic stem cell transplantation (HSCT) or relapse after FDA ODD and RPDD designations for UCART22 marks an important step towards developing allogeneic CAR T products that would be...Read more
Sickle cell disease affects approximately 100,000 patients in the U.S. annually and is a debilitating and life-threatening condition with high unmet need Current conditioning with non-targeted chemotherapies provides limited access to potentially curative bone marrow transplant and recently approved gene therapies for sickle cell disease patients Initial trial focused on conditioning for bone marrow transplant intended to...Read more
FOSTER CITY, Calif. / Jul 25, 2024 / Business Wire / Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that the U.S. Food and Drug Administration (FDA) has approved a label expansion for LIVMARLI® (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC). The expanded label includes use in PFIC patients 12 months and older as well as the higher...Read more
Fast Track is designed to expedite FDA review of important new drugs to treat serious conditions and fill an unmet medical need Fast Track designation for Tonmya recognizes fibromyalgia as a serious condition impacting more than 10 million U.S. adults NDA submission on track for second half 2024 Tonmya has the potential to be the first new drug for treating fibromyalgia in more than 15 years CHATHAM, N.J., July 25, 2024 (GLOBE...Read more
BURLINGTON, Mass., July 25, 2024 (GLOBE NEWSWIRE) -- scPharmaceuticals Inc. (Nasdaq: SCPH) (the “Company”), a pharmaceutical company focused on developing and commercializing products that have the potential to optimize the delivery of infused therapies, advance patient care, and reduce healthcare costs, today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the Company’s Supplemental New Drug Application...Read more
WALTHAM, Mass., July 25, 2024 (GLOBE NEWSWIRE) -- Checkpoint Therapeutics, Inc. (“Checkpoint”) (Nasdaq: CKPT), a clinical-stage immunotherapy and targeted oncology company, today announced that the U.S. Food and Drug Administration (“FDA”) has accepted for review Checkpoint’s resubmission of its Biologics License Application (“BLA”) for cosibelimab, its anti-programmed death ligand-1 (“PD-L1”) antibody, as a potential new treatment for...Read more
Treatment of first randomized person with preclinical AD expected this quarter Phase 2b ReTain trial is fully funded and conducted by development partner Lausanne, Switzerland, July 25, 2024 – AC Immune SA (NASDAQ: ACIU), a clinical-stage biopharmaceutical company pioneering precision medicine for neurodegenerative diseases, today announced that its active-immunotherapy candidate, ACI-35.030 (now called JNJ-2056), targeting the...Read more
Now Approved for Children of All Ages with CLN2 Batten Disease, Regardless of Whether They Yet Show Symptoms SAN RAFAEL, Calif., July 24, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has approved the company's supplemental Biologics License Application (sBLA) for BRINEURA® (cerliponase alfa) to slow the loss of ambulation in children of all ages with...Read more
NEW YORK, July 24, 2024 (GLOBE NEWSWIRE) -- Tiziana Life Sciences, Ltd. (Nasdaq: TLSA) (“Tiziana” or the “Company”), a biotechnology company developing breakthrough immunomodulation therapies with its lead development candidate, intranasal foralumab, a fully human, anti-CD3 monoclonal antibody, today announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for its intranasal formulation of foralumab, a...Read more
WINNIPEG, Manitoba, July 23, 2024 (GLOBE NEWSWIRE) -- Kane Biotech Inc. (TSX- V:KNE; OTCQB:KNBIF) (“Kane Biotech”, “Kane” or the “Company”) announces that the US Food and Drug Administration (FDA) has eliminated its usage limitation on the Company’s 510(k) cleared revyve™ Antimicrobial Wound Gel (“revyve™”). Prior to the removal of this restriction, there was a 90 grams/month limit to the amount of revyve™ product that could be...Read more
IND follows encouraging findings of necrosis, inflammation and T cell infiltration in tumor biopsy of patient in lowest dose cohort SAN JOSE, Calif., July 23, 2024 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced that its collaborator, Moffitt Cancer Center (Moffitt), has received approval by the U.S. Food and...Read more
Approximately 40% of the 9 million individuals in the United States with plaque psoriasis experience involvement of the scalp Once-daily ZORYVE foam significantly improved both scalp and body psoriasis in a Phase 2b and a pivotal Phase 3 trial Data also show rapid reduction in scalp itch as soon as 24 hours after first application ZORYVE foam demonstrated a favorable safety and tolerability profile WESTLAKE VILLAGE, Calif., July...Read more
PALM BEACH GARDENS, Fla., July 23, 2024 (GLOBE NEWSWIRE) -- ZimVie Inc. (Nasdaq: ZIMV), a global life sciences leader in the dental market, today announced the U.S. launch of its GenTek® Genuine Restorative Components product portfolio. The launch expands ZimVie’s portfolio of end-to-end prosthetic offerings and follows the recent receipt of FDA 510(k) clearance. ZimVie first launched the GenTek portfolio in Europe in 2019 and has seen...Read more
Ozuriftamab vedotin, the Company’s conditionally and reversibly active antibody drug conjugate directed against ROR2, has shown promising clinical activity with a manageable safety profile in treatment-refractory patients with squamous cell carcinoma of the head and neck (SCCHN) in its Phase 2 clinical trial The Company is on track to meet with the FDA for guidance on a potentially registrational trial in 2H 2024 SAN DIEGO, July 23,...Read more
NEW YORK, July 23, 2024 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, announced that the United States Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) resubmission for Ryoncil® (remestemcel-L) in the treatment of children with steroid-refractory acute graft versus host disease (SR-aGVHD). FDA considers the...Read more
RESEARCH TRIANGLE PARK, N.C., July 23, 2024 (GLOBE NEWSWIRE) -- Asensus Surgical, Inc. (NYSE American: ASXC), a global leader of innovative digital solutions for the operating room, today announced that it has received 510(k) clearance from the U.S. Food and Drug Administration (“FDA”) for an expanded indication to treat adult and pediatric Urology patients with the Senhance® Surgical System. “This FDA clearance marks another milestone...Read more
Phase 4 SPRAVATO® monotherapy data shows rapid improvement in depressive symptoms at ~24 hours, sustained through at least 4 weeks Monotherapy submission builds on more than a decade of research, 31 clinical trials and more than five years of real-world use that reinforce the safety and efficacy of SPRAVATO® TITUSVILLE, N.J., July 22, 2024 /PRNewswire/ -- Johnson & Johnson (NYSE: JNJ) announced today the submission of a...Read more
VANCOUVER, British Columbia, July 22, 2024 (GLOBE NEWSWIRE) -- Zymeworks Inc. (Nasdaq: ZYME), a clinical-stage biotechnology company developing a diverse pipeline of novel, multifunctional biotherapeutics to improve the standard of care for difficult-to-treat diseases, today announced that the United States Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for ZW191, the Company’s novel folate...Read more
VOQUEZNA is now approved and available to treat the largest category of Gastroesophageal Reflux Disease (GERD) VOQUEZNA met its primary endpoint in its Phase 3 pivotal trial by demonstrating a significant and rapid reduction of heartburn with daily treatment VOQUEZNA represents the first major innovation in GERD treatment in over 30 years and the only FDA-approved treatment of its kind available in the U.S. FLORHAM PARK, N.J., July...Read more
Prescription Drug User Fee Act (PDUFA) Target Action Date of January 15, 2025 If Approved, Tab-cel Would Be First Approved Therapy in U.S. for EBV+ PTLD BLA Acceptance Triggers $20 Million Milestone Payment from Pierre Fabre Laboratories, with Additional $60 Million Milestone if Approved by FDA THOUSAND OAKS, Calif. / Jul 17, 2024 / Business Wire / Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leader in T-cell immunotherapy,...Read more
The total number of Hyperfine AI-powered marketing authorizations places the company in a leading position on recently published FDA list. GUILFORD, Conn. / Jul 17, 2024 / Business Wire / Hyperfine (Nasdaq: HYPR), the groundbreaking medical device company that has redefined brain imaging with the world’s first FDA-cleared portable magnetic resonance brain imaging system—the Swoop® system—today announced the clearance of the ninth...Read more
AUDUBON, Pa., July 17, 2024 (GLOBE NEWSWIRE) -- Globus Medical, Inc. (NYSE: GMED), a leading musculoskeletal solutions company, today announced it recently received 510(k) clearance by the U.S. Food and Drug Administration (FDA) for ExcelsiusFlex™ with Total Knee Arthroplasty (TKA) application. This new robotic navigation platform joins the already best-in-class Excelsius™ ecosystem, designed to offer surgeons control, resection accuracy,...Read more
IRVINE, Calif., July 17, 2024 /PRNewswire/ -- InMode Ltd. (NASDAQ: INMD), a leading global provider of innovative medical technologies, is pleased to announce an additional FDA 510(k) clearance for the Morpheus8 technology. Morpheus8 is the first and only fractional radiofrequency (FRF) microneedling technology cleared for contraction of soft tissue. The U.S. Food and Drug Administration (FDA) has cleared the use of the Morpheus8...Read more
FDA designation enables expedited clinical development and regulatory review timelines for Lomecel-B™ Second designation received for Lomecel-B™ for the treatment of mild Alzheimer’s Disease after Regenerative Medicine Advanced Therapeutics (RMAT) Designation announced on July 9, 2024 Phase 2a Lomecel-B™ data in mild Alzheimer’s Disease selected for Featured Research Session oral presentation and poster...Read more
Acute Myeloid Leukemia (AML) is the Second Most Common Hematological Malignancy in Children Often Associated with Poorer Prognosis Compared to Other Pediatric Cancers This Recognition Marks SELLAS’ Second RPDD Following the Designation for ALL Received Last Month Opened Enrollment for Pediatric AML Patients in Ongoing Phase 2 Clinical Trial RPDD Provides Eligibility for SLS009 to Receive a Priority Review Voucher...Read more
Vancouver, Canada, July 16, 2024 (GLOBE NEWSWIRE) -- Clearmind Medicine Inc. (Nasdaq: CMND), (FSE: CWY0) (“Clearmind” or the "Company"), a clinical-stage biotech company focused on discovery and development of novel psychedelic-derived therapeutics to solve major under-treated health problems, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) application for its...Read more
Engineered for precision, confidence and efficiency in surgery - and compatible with the CORI◊ Surgical System Smith+Nephew (LSE:SN, NYSE:SNN), the global medical technology company, today announces it has received 510(k) clearance for its new CATALYSTEM Primary Hip System from the United States Food and Drug Administration. The system is designed to address the evolving demands of primary hip surgery including the increased adoption...Read more
Launch Preparations Actively Underway; Launch Planned for Early 2025 Lexicon Seeks Approval for Zynquista™ (sotagliflozin) as an Adjunct to Insulin Therapy for Glycemic Control in Adults with Type 1 Diabetes and Chronic Kidney Disease THE WOODLANDS, Texas, July 16, 2024 (GLOBE NEWSWIRE) -- Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX), today announced that the U.S. Food and Drug Administration (FDA) has acknowledged the resubmission...Read more
The application was evaluated based on the positive clinical outcomes of KYV-101 in patients treated in Germany under a named-patient treatment option The Regenerative Medicine Advanced Therapies designation will allow Kyverna to receive expert guidance on efficient drug development and use of surrogate endpoints from senior FDA officials EMERYVILLE, Calif., July 15, 2024 /PRNewswire/ -- Kyverna Therapeutics, Inc. (Kyverna), a...Read more
- Prescription Drug User Fee Act (PDUFA) target action date is February 28, 2025 - - Product has patent protection through 2043 - DEER PARK, Ill., July 15, 2024 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc (“Eton” or the “Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, today announced the Company’s New Drug Application (NDA) for ET-400, a...Read more
ART26.12 seeks to address a critical need in painful neuropathies, including chemotherapy-induced peripheral neuropathy for which there is no FDA-approved treatment Phase 1 trial results expected in the first half of 2025 SOLANA BEACH, Calif., July 15, 2024 (GLOBE NEWSWIRE) -- Artelo Biosciences, Inc. (Nasdaq: ARTL), a clinical-stage pharmaceutical company focused on modulating lipid-signaling pathways to develop treatments for...Read more
PRINCETON, N.J., July 15, 2024 (GLOBE NEWSWIRE) -- ANI Pharmaceuticals, Inc. (ANI or the Company) (Nasdaq: ANIP) today announced that following final approval from the U.S. Food and Drug Administration (FDA) for its Abbreviated New Drug Application (ANDA), the Company launched L-Glutamine Oral Powder. ANI’s L-Glutamine Oral Powder is the generic version of the reference listed drug (RLD) Endari®. "The approval and launch of L-Glutamine...Read more
Submissions are supported by the Phase 3 SELECT-GCA study demonstrating upadacitinib 15 mg with a 26-week steroid taper regimen achieved the primary endpoint of sustained remission from week 12 through week 521 The safety profile of upadacitinib in patients with GCA was generally consistent with that in approved indications1 NORTH CHICAGO, Ill., July 12, 2024 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced that it has...Read more
PETACH TIKVA, Israel, July 11, 2024 (GLOBE NEWSWIRE) -- Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE: CANF), a biotechnology company advancing a pipeline of proprietary small molecule drugs that address oncological and inflammatory diseases, today announced that it has submitted an application to the U.S. Food and Drug Administration (FDA) for Orphan Drug Designation for its drug candidate Namodenoson in the treatment...Read more
Bagsværd, Denmark, 10 July 2024 – Novo Nordisk today announced that the US Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) covering the Biologics License Application for once-weekly basal insulin icodec for the treatment of diabetes mellitus. In the letter, the FDA has requests related to the manufacturing process and the type 1 diabetes indication before the review of the application can be completed....Read more
MIAMI, July 10, 2024 (GLOBE NEWSWIRE) -- Longeveron Inc. (NASDAQ: LGVN), a clinical stage regenerative medicine biotechnology company developing cellular therapies for certain life-threatening and chronic aging-related conditions, today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to Lomecel-B™ for the treatment of mild Alzheimer’s Disease....Read more
ZORYVE cream provides rapid relief, with efficacy, safety, and tolerability demonstrated up to 56 weeks of treatment ZORYVE cream rapidly and significantly reduces itch, the most bothersome symptom of atopic dermatitis (AD) Once-daily cream is for use anywhere on the body for any duration AD is the most common type of eczema, affecting approximately 9.6 million children and 16.5 million adults in the United States Third FDA approval...Read more
LEWISVILLE, Texas / Jul 09, 2024 / Business Wire / Orthofix Medical Inc. (NASDAQ:OFIX), a leading global spine and orthopedics company, today announced the 510(k) clearance and first U.S. implant of the Fitbone™ Transport and Lengthening System. Used to treat large bone defects in the femur and tibia due to trauma, infectious or malignant conditions, the device is the only intramedullary nail designed to transport or lengthen the bone...Read more
Following FDA review, Avalo’s IND application for AVTX-009 is active allowing Avalo to proceed with its Phase 2 trial (LOTUS) to evaluate the efficacy and safety of AVTX-009 in patients with hidradenitis suppurativa WAYNE, Pa. and ROCKVILLE, Md., July 09, 2024 (GLOBE NEWSWIRE) -- Avalo Therapeutics, Inc. (Nasdaq: AVTX) today announced that the Investigational New Drug (IND) for AVTX-009, an anti-IL-1β monoclonal antibody (mAb), for...Read more
On the basis of its positive clinical studies Mainz Biomed has now defined the final configuration including its novel mRNA biomarkers of the Next Generation Test to be used in pivotal registration study ReconAAsense A recent clinical analysis of this new configuration demonstrates sensitivity for colorectal cancer of 97% and 88% for advanced adenomas, with specificity of 93% BERKELEY, Calif. and MAINZ, Germany, July 09, 2024 (GLOBE...Read more
NEW YORK, July 08, 2024 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, announced today it has resubmitted its BLA for approval of Ryoncil® (remestemcel-L) in the treatment of children with SR-aGVHD. The filing comes after Mesoblast was informed by FDA at the end of March that, following additional consideration, the available clinical data from the...Read more
Provides expanded access to FDA and prioritized review of submission MIAMI / Jul 08, 2024 / Business Wire / Pulse Biosciences, Inc. (Nasdaq: PLSE) (the “Company” or “Pulse Biosciences”), a company leveraging its novel and proprietary Nanosecond Pulsed Field Ablation™ (nsPFA™) technology, today announced that it has received the Breakthrough Device Designation from the U.S. FDA for the Company’s Cardiac Surgery System for the ablation...Read more
REDWOOD CITY, Calif. & BOSTON / Jul 08, 2024 / Business Wire / Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer, today announced the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to ADI-270 for the potential treatment of patients with metastatic/advanced clear cell renal cell carcinoma...Read more
Vabysmo PFS is the first and only syringe prefilled with an FDA-approved bispecific antibody to treat retinal conditions that can cause blindness Designed to simplify administration, Vabysmo PFS provides retina specialists a ready-to-use option Vabysmo PFS will be available for people living with wet AMD, DME and RVO SOUTH SAN FRANCISCO, Calif. / Jul 05, 2024 / Business Wire / Genentech, a member of the Roche Group (SIX: RO, ROG;...Read more
DENVER, July 03, 2024 (GLOBE NEWSWIRE) -- SeaStar Medical Holding Corporation (Nasdaq: ICU), a commercial-stage medical device company developing proprietary solutions to reduce the consequences of hyperinflammation on vital organs, announces that the U.S. Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER) has agreed to the final labeling for SeaStar Medical’s QUELIMMUNE™, the Selective Cytopheretic...Read more
Kisunla slowed cognitive and functional decline by up to 35% compared to placebo at 18 months in its pivotal Phase 3 study and reduced participants' risk of progressing to the next clinical stage of disease by up to 39% Kisunla is the first and only amyloid plaque-targeting therapy that used a limited-duration treatment regimen based on amyloid plaque removal; nearly half of study participants completed their course of treatment...Read more
Vanza triple granted priority review with Prescription Drug User Fee Act (PDUFA) target action date of January 2, 2025 EU Marketing Authorization Application (MAA) submission also validated by European Medicines Agency (EMA) BOSTON / Jul 02, 2024 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for...Read more
BOSTON, July 02, 2024 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) (“the Company” or “Inozyme”), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of pathologic mineralization and intimal proliferation, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to INZ-701 for the treatment of ABCC6 Deficiency. “Through Fast Track...Read more
WALTHAM, Mass., July 02, 2024 (GLOBE NEWSWIRE) -- Checkpoint Therapeutics, Inc. (“Checkpoint”) (Nasdaq: CKPT), a clinical-stage immunotherapy and targeted oncology company, today announced it has completed the resubmission of its Biologics License Application (“BLA”) to the U.S. Food and Drug Administration (“FDA”) for cosibelimab, its anti-programmed death ligand-1 (“PD-L1”) antibody, as a potential new treatment for patients with...Read more
PRINCETON, N.J., July 02, 2024 (GLOBE NEWSWIRE) -- ANI Pharmaceuticals, Inc. (ANI or the Company) (Nasdaq: ANIP) today announced that it received U.S. Food and Drug Administration (FDA) approval for the Abbreviated New Drug Application (ANDA) and launched Naproxen Delayed-Release Tablets, USP. ANI’s Naproxen Delayed-Release Tablets is the generic version of the reference listed drug (RLD) EC-Naprosyn®. "We are pleased to announce...Read more
The U.S. Food and Drug Administration ("FDA") assigned a Prescription Drug User Fee Act ("PDUFA") goal date of September 23, 2024 SAN DIEGO, July 2, 2024 /PRNewswire/ -- Heron Therapeutics, Inc. (Nasdaq: HRTX) ("Heron" or the "Company"), a commercial-stage biotechnology company, today announced that the FDA acknowledged the receipt of the Company's Prior Approval Supplement ("PAS") application for ZYNRELEF® (bupivacaine and...Read more
U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for bexicaserin for the treatment of seizures associated with Developmental and Epileptic Encephalopathies (DEEs) LA JOLLA, Calif. / Jul 01, 2024 / Business Wire / Longboard Pharmaceuticals, Inc. (Nasdaq: LBPH), a clinical-stage biopharmaceutical company focused on developing novel, transformative medicines for neurological diseases, today announced...Read more
CAESAREA, Israel, July 1, 2024 /PRNewswire/ -- IceCure Medical Ltd. (NASDAQ: ICCM) ("IceCure", "IceCure Medical" or the "Company"), developer of minimally-invasive cryoablation technology that destroys tumors by freezing as an alternative to surgical tumor removal, today announced it has received marketing authorization from the United States Food and Drug Administration (the "FDA") for its next-generation single probe cryoablation...Read more
Third RMAT designation by FDA for ATEV RMAT will expedite development of ATEV in PAD DURHAM, N.C., July 01, 2024 (GLOBE NEWSWIRE) -- Humacyte, Inc. (Nasdaq: HUMA), a clinical-stage biotechnology platform company developing universally implantable, bioengineered human tissues at commercial scale, has been granted the U.S. Food and Drug Administration’s (FDA’s) Regenerative Medicine Advanced Therapy (RMAT) designation for patients...Read more
PDUFA Target Action Dates in Late December 2024 Highly Selective CRF1 Antagonist is the Potential First New Treatment for CAH in 70 Years SAN DIEGO, July 1, 2024 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced the U.S. Food and Drug Administration (FDA) has accepted its two New Drug Applications (NDA) with Priority Review designations for crinecerfont in the treatment of children, adolescents and...Read more
STAMFORD, Conn., July 01, 2024 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, announced today that the Company has completed the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for mirdametinib, an investigational MEK inhibitor, for the treatment of pediatric and adult patients with...Read more
Nyxoah Submits Fourth and Final Module in PMA Application for Genio to the US FDA Mont-Saint-Guibert (Belgium), July 1, 2024, 8:00am CET / 2:00am ET – Nyxoah (“Nyxoah” or the “Company”), a medical technology company focused on the development and commercialization of innovative solutions to treat Obstructive Sleep Apnea (OSA), today announced that the Company submitted the fourth and final module of its Premarket Approval (PMA)...Read more
Submission based on the positive Phase 3 RESTORE study Mirum holds orphan designation for chenodiol in CTX Potential to have first and only therapy indicated for CTX in the US FOSTER CITY, Calif. / Jun 28, 2024 / Business Wire / Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced the submission of a new drug application (NDA) for chenodiol for the treatment of patients in the U.S. with cerebrotendinous xanthomatosis...Read more
REDWOOD CITY, Calif., June 28, 2024 (GLOBE NEWSWIRE) -- Soleno Therapeutics, Inc. (“Soleno”) (NASDAQ: SLNO), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for approval of DCCR (diazoxide choline) extended-release tablets for the treatment of Prader-Willi syndrome (PWS)...Read more
CRANBURY, N.J. / Jun 28, 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 high unmet needs, today announced a regulatory update for KRESLADI™ (marnetegragene autotemcel; marne-cel), a lentiviral (LV) vector-based gene therapy to treat severe leukocyte adhesion deficiency-I (LAD-I). The U.S....Read more
GTB-3650 Phase 1 trial initiation expected in H2 2024; initial clinical data expected in H1 2025 GTB-5550 TriKE® IND submission for treatment of B7H3 positive solid tumors expected in Q1 2025 GTB-5550 Phase 1 dose escalation basket trial initiation expected in 2025 evaluating GTB-5550 in six solid tumor cancers, including prostate, breast, head and neck, ovarian, lung, and GI Cash runway anticipated to be sufficient to fund...Read more
WALTHAM, Mass. and BOULDER, Colo., June 27, 2024 (GLOBE NEWSWIRE) -- Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, today announced it has reached alignment with the U.S. Food and Drug Administration (FDA) on the Company’s novel patient reported outcome measure, Mastocytosis Symptom Severity Daily Diary (MS2D2), for use in Part 2...Read more
Clear path forward on key elements of the planned potential registration-enabling trial in HR+/HER2- metastatic breast cancer On track to report overall survival results from the randomized HR+/HER2- metastatic breast cancer BRACELET-1 trial in H2 2024 SAN DIEGO and CALGARY, AB, June 27, 2024 /CNW/ -- Oncolytics Biotech® Inc. (NASDAQ: ONCY) (TSX: ONC), a leading clinical-stage company specializing in immunotherapeutics for...Read more
The letter did not identify any issues with the efficacy or safety data submitted in the application BASKING RIDGE, N.J. & RAHWAY, N.J. / Jun 26, 2024 / Business Wire / The U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for the Biologics License Application (BLA) seeking accelerated approval of Daiichi Sankyo (TSE: 4568) and Merck’s (known as MSD outside of the United States and Canada) (NYSE:...Read more
Approval based on results from Phase 1/2 EPCORE® NHL-1 study, which demonstrated durable, clinically meaningful treatment responses in patients with challenging-to-treat R/R FL EPKINLY offers an off-the-shelf, T-cell engaging treatment option that enables treatment across practice settings to address high clinical need EPKINLY is the first and only bispecific antibody approved in the U.S. to treat both relapsed or refractory (R/R)...Read more
EPKINLY is now the first and only bispecific antibody approved in the U.S. to treat both relapsed or refractory (R/R) follicular lymphoma (FL) and R/R diffuse large B-cell lymphoma (DLBCL) Bispecific antibodies are designed to induce targeted cell killing by engaging the immune system FL is considered incurable with current standard of care therapies. Many people living with FL who have relapsed or are refractory to existing...Read more
Ohtuvayre is indicated for the maintenance treatment of COPD allowing for broad use in COPD patients First inhaled COPD treatment providing bronchodilation and non-steroidal anti-inflammatory effects Conference call tomorrow at 8:30 a.m. EDT / 1:30 p.m. BST LONDON and RALEIGH, N.C., June 26, 2024 (GLOBE NEWSWIRE) -- Verona Pharma plc (Nasdaq: VRNA) (“Verona Pharma” or the “Company”), announces the US Food and Drug Administration...Read more
CHICAGO / Jun 26, 2024 / Business Wire / Tempus AI, Inc. (NASDAQ: TEM), a leader in artificial intelligence and precision medicine, today announced it has received 510(k) clearance from the U.S. Food and Drug Administration (FDA) for its Tempus ECG-AF device that uses AI to help identify patients who may be at increased risk of atrial fibrillation/flutter (AF). This is the first FDA clearance for an AF indication in the category known as...Read more
Collaborative study will evaluate drug's potential to improve synaptic health and cognitive function in MS patients NEW YORK, June 26, 2024 /PRNewswire/ -- Synaptogenix, Inc. (Nasdaq: SNPX) ("Synaptogenix" or the "Company"), an emerging biopharmaceutical company developing therapeutics for neurodegenerative disorders, today announced that the Food & Drug Administration (FDA) has authorized an Investigational New Drug (IND)...Read more
Fast Track designation follows positive preliminary Phase 2 trial data presented at the European Association for the Study of the Liver Congress 2024 and underscores the unmet need for people living with chronic hepatitis delta SAN FRANCISCO / Jun 26, 2024 / Business Wire / Vir Biotechnology, Inc. (Nasdaq: VIR) today announced that the U.S. Food and Drug Administration (FDA) has cleared its investigational new drug (IND) application...Read more
NEW YORK, June 26, 2024 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today announced it has reached alignment with the U.S. Food and Drug Administration (FDA) on the Chemistry, Manufacturing, and Controls (CMC) aspects of Brainstorm's Phase 3b clinical trial for NurOwn®, its investigational therapy for amyotrophic lateral...Read more
Twelve Required Registration Batches Completed New Drug Application Being Prepped for Submission KANSAS CITY, Kan., June 25, 2024 (GLOBE NEWSWIRE) -- In alignment with U.S. Food and Drug Administration (FDA) requirements, Cingulate Inc. (NASDAQ: CING), a biopharmaceutical company utilizing its proprietary Precision Timed Release™ (PTR™) drug delivery platform technology to build and advance a pipeline of next-generation...Read more
SAN DIEGO, June 25, 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 U.S. Food and Drug Administration (FDA) has scheduled a Pre-BLA (Biologics License Application) meeting with the Company in the third quarter of 2024 for deramiocel (CAP-1002), for the treatment of Duchenne...Read more
U.S. Food and Drug Administration (FDA) issues Complete Response Letter (CRL) for ABBV-951 based on observations from an inspection that did not involve ABBV-951 at one of AbbVie's third-party manufacturing facilities The CRL does not identify any issues related to the safety, efficacy or labeling of ABBV-951, including the device, and does not request that AbbVie conduct additional efficacy or safety trials related to the drug or...Read more
Fast Track designation highlights potential for CT-0525 to address unmet need for patients with solid tumors Initial Phase 1 data expected by year-end 2024 PHILADELPHIA, June 25, 2024 /PRNewswire/ -- Carisma Therapeutics Inc. (Nasdaq: CARM) ("Carisma" or the "Company"), a clinical stage biopharmaceutical company focused on discovering and developing innovative immunotherapies, announced today that the U.S. Food and Drug...Read more
Olezarsen PDUFA date set for December 19, 2024 for treatment of familial chylomicronemia syndrome Phase 3 enrollment completed in CORE, CORE2 and ESSENCE evaluating olezarsen for the treatment of severe hypertriglyceridemia, with results expected in 2H 2025 CARLSBAD, Calif., June 25, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has accepted...Read more
NEWPORT BEACH, Calif. / Jun 24, 2024 / Business Wire / Evolus, Inc. (NASDAQ: EOLS), a performance beauty company with a focus on building an aesthetic portfolio of consumer brands, today announced that it has submitted the final module of its premarket approval (PMA) application to the U.S. Food and Drug Administration (FDA) for the Evolysse™ Lift and Evolysse™ Smooth dermal filler products for the nasolabial fold (NLF). “We are pleased to...Read more
4D-175 comprises the proprietary low-dose intravitreal R100 AAV vector and a codon-optimized transgene encoding a highly functional shortened form of human complement factor H (sCFH) Complement factor H (CFH) variants with reduced function are a well-validated genetic risk factor for geographic atrophy (GA), with approximately 75% of age-related macular degeneration (AMD) patients carrying a high-risk variant of CFH Over 150 patients...Read more
PLYMOUTH MEETING, Pa., June 24, 2024 /PRNewswire/ -- Harmony Biosciences (Nasdaq: HRMY) today announced that the U.S. Food and Drug Administration (FDA) has approved its supplemental New Drug Application (sNDA) for WAKIX® (pitolisant) tablets for the treatment of excessive daytime sleepiness (EDS) in pediatric patients 6 years of age and older with narcolepsy. The FDA separated the submission into two sNDAs for administrative purposes to...Read more
ADI-270 is the first gamma delta 1 CAR T candidate to enter clinical development for solid tumors Phase 1 clinical study to evaluate safety and anti-tumor activity of ADI-270 in relapsed/refractory RCC patients Phase 1 clinical study to be initiated in 2H 2024; preliminary clinical data expected in 1H 2025 REDWOOD CITY, Calif. & BOSTON / Jun 24, 2024 / Business Wire / Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage...Read more
FDA Provided Constructive Feedback to Help Guide Next Steps in TFF TAC Clinical Development Company to Provide More Detailed Regulatory Update in 3Q 2024 FORT WORTH, Texas, June 24, 2024 (GLOBE NEWSWIRE) -- TFF Pharmaceuticals, Inc. (NASDAQ: TFFP) (“the Company”), a clinical-stage biopharmaceutical company focused on developing and commercializing innovative drug products based on its patented Thin Film Freezing (TFF) technology...Read more
Designation is based on pre-clinical data and data from an ongoing Phase 1/2 trial for BNT324/DB-1311, with antitumor activity and a manageable safety profile demonstrated by preliminary Phase 1/2 clinical data from patients with advanced or metastatic solid tumors1,2 With the Fast Track designation, the development of BNT324/DB-1311 can benefit from more frequent engagement with the U.S. Food and Drug Administration (“FDA”) to support...Read more
Acute Lymphoblastic Leukemia (ALL) is the Most Common Type of Cancer in Children Rare Pediatric Disease Designation (RPDD) Provides Eligibility for SLS009 to Receive a Priority Review Voucher (PRV) Upon Marketing Approval that can be Transferred/Sold to Other Parties Past Sales of PRVs Have Averaged More Than $100 Million NEW YORK, June 24, 2024 (GLOBE NEWSWIRE) -- SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) (“SELLAS” or the...Read more
Combined vitreoretinal-cataract system (VCS) and standalone cataract system (CS) are cleared for use in the U.S. New, proprietary technologies designed to deliver transformative surgical innovation Alcon to immediately begin collecting real-world user experience in the U.S. prior to broad commercialization in 2025 First innovations to be introduced from Alcon’s cutting-edge Unity portfolio of surgical equipment Ad Hoc Announcement...Read more
Approval based on results from the Phase 1/2 KRYSTAL-1 study where KRAZATI in combination with cetuximab showed an objective response rate of 34% in pretreated patients with locally advanced or metastatic CRC harboring a KRASG12C mutation1 Second FDA approval for KRAZATI - reinforcing its potential across tumor types PRINCETON, N.J. / Jun 21, 2024 / Business Wire / Bristol Myers Squibb (NYSE: BMY) today announced that the U.S....Read more
VYVGART® Hytrulo is first and only neonatal Fc receptor (FcRn) blocker approved to treat chronic inflammatory demyelinating polyneuropathy (CIDP) First novel, precision mechanism of action in more than 30 years for patients with CIDP Third approved indication for VYVGART® and VYVGART Hytrulo franchise Management to host conference call on June 21, 2024 at 11:00pm CET (5:00pm ET) Amsterdam, the Netherlands – argenx SE (Euronext...Read more
DUBLIN, Ireland and CHICAGO, June 21, 2024 (GLOBE NEWSWIRE) -- Iterum Therapeutics plc (Nasdaq: ITRM) (the Company), a clinical-stage pharmaceutical company focused on developing next generation oral and IV antibiotics to treat infections caused by multi-drug resistant pathogens in both community and hospital settings, today announced that the U.S. Food and Drug Administration (FDA) has determined that the New Drug Application (NDA) for...Read more
Lexicon Seeks Approval for Sotagliflozin as an Adjunct to Insulin Therapy for Glycemic Control in People with Type 1 Diabetes and Chronic Kidney Disease Company Anticipates Six Month Review and Potential Launch in Early 2025 THE WOODLANDS, Texas, June 21, 2024 (GLOBE NEWSWIRE) -- Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX), today announced that, following multiple interactions with and recent feedback from the U.S. Food and Drug...Read more
FDA grants traditional approval to ELEVIDYS for ambulatory Duchenne patients FDA grants accelerated approval to ELEVIDYS for non-ambulatory Duchenne patients Sarepta will host an investor conference call on June 21, 2024, at 8:30 a.m. ET CAMBRIDGE, Mass. / Jun 20, 2024 / Business Wire / Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced U.S. Food and Drug...Read more
Submission is supported by 48-week results from the Phase 3 GALAXI and GRAVITI programs TREMFYA® is the only IL-23 inhibitor to demonstrate strong endoscopic outcomes with subcutaneous (SC) induction, consistent with intravenous (IV) induction, and has the potential to be the first in its class to offer the option of both SC and IV induction therapy in Crohn's disease GALAXI includes data demonstrating superior outcomes for...Read more
Compass Therapeutics is a clinical-stage, oncology-focused biopharmaceutical company developing proprietary antibody-based therapeutics to treat multiple human diseases. The company's scientific focus is on the relationship between angiogenesis, the immune system, and tumor growth...
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