Medexus will target a commercial launch in 1H CY2025; potential for annual product-level revenue to exceed US$100 million within five years after commercial launch Toronto, Ontario and Chicago, Illinois--(Newsfile Corp. - January 22, 2025) - Medexus Pharmaceuticals (TSX: MDP) (OTCQX: MEDXF) is pleased to provide a business update regarding the successful completion of the regulatory review process for GRAFAPEX™ (treosulfan) for...Read more
LONDON and NEW YORK, Jan. 22, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings Plc (Nasdaq: MGTX), a vertically integrated, clinical-stage genetic medicines company, today announced the U.S. Food and Drug Administration (FDA) has granted the Company Rare Pediatric Disease Designation to its AAV8-RK-RetGC program for the treatment of patients with Leber congenital amaurosis due to GUCY2D mutations (LCA1). This is the fourth Rare...Read more
Only dual route gene transfer therapy in development to treat Friedreich’s ataxia with FDA IND clearance and Fast Track designation CHARLESTOWN, Mass., Jan. 21, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that it has received Fast Track designation from the U.S. Food and Drug Administration...Read more
Following U.S. FDA Priority Review, approval is based on data demonstrating SPRAVATO® alone met its primary endpoint at 4 weeks and led to rapid and superior improvement in depressive symptoms compared to placebo as early as 24 hours1 SPRAVATO® alone showed a rapid and superior improvement vs. placebo in the Montgomery-Asberg Depression Rating Scale (MADRS) total score, with numerical improvements across all 10 MADRS items...Read more
WOBURN, Mass., Jan. 21, 2025 (GLOBE NEWSWIRE) -- Replimune Group, Inc. (NASDAQ: REPL), a clinical stage biotechnology company pioneering the development of novel oncolytic immunotherapies, today announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for RP1 (vusolimogene oderparepvec) in combination with nivolumab for patients with advanced melanoma. The FDA granted the BLA...Read more
Company Anticipates Submitting for U.S. Accelerated Approval in H1 2026 WALTHAM, Mass., Jan. 21, 2025 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage neuromuscular disease company focused on advancing life-transforming therapeutics for people living with genetically driven diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for DYNE-101 for...Read more
Orphan drug designation paves the way for potential of sebetralstat to be first oral on-demand treatment for HAE in Japan Submission advances Company’s strategic plan to address unmet needs in HAE on global scale CAMBRIDGE, Mass. & SALISBURY, England / Jan 21, 2025 / Business Wire / KalVista Pharmaceuticals, Inc. (NASDAQ: KALV), today announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted sebetralstat...Read more
ELYXYB®, a rapid onset and ready-to-use formulation of Celecoxib, delivers a first line non-opioid therapeutic alternative to habit-forming opioids and acetaminophen, the leading cause of acute liver failure in the U.S.(1) DelveInsight estimates there were approximately 100 million cases of acute pain in the United States and that the total acute pain market in the U.S. was approximately $3 billion in 2021.(2) Approximately 40 million...Read more
First approval in the US for AstraZeneca and Daiichi Sankyo’s DATROWAY based on TROPION-Breast01 results showing 37% reduction in the risk of disease progression or death vs. chemotherapy DATROWAY is the eighth new medicine of the 20 AstraZeneca has set out to deliver by 2030 WILMINGTON, Del. / Jan 17, 2025 / Business Wire / DATROWAY® (datopotamab deruxtecan-dlnk) has been approved in the US for the treatment of adult patients with...Read more
The New Drug Application is based on positive results from the Phase 3 PALISADE study People living with familial chylomicronemia syndrome have extremely high triglyceride levels and a substantially higher risk of developing acute pancreatitis and associated long-term complications, including poor quality of life PASADENA, Calif. / Jan 17, 2025 / Business Wire / Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that the...Read more
Pivotal Study Demonstrated the Combination More Than Doubled Progression-Free Survival Compared to Investigated SOC THOUSAND OAKS, Calif., Jan. 17, 2025 /PRNewswire/ -- Amgen (NASDAQ:AMGN) today announced that the U.S. Food and Drug Administration (FDA) has approved LUMAKRAS® (sotorasib) in combination with Vectibix® (panitumumab) for the treatment of adult patients with KRAS G12C-mutated metastatic colorectal cancer (mCRC), as...Read more
Based on ECHO Phase III trial results which showed more than 16 months of progression-free survival improvement vs. chemoimmunotherapy alone First and only BTK inhibitor approved for the 1st-line treatment of MCL in the US WILMINGTON, Del. / Jan 17, 2025 / Business Wire / AstraZeneca’s CALQUENCE® (acalabrutinib) in combination with bendamustine and rituximab has been approved in the US for the treatment of adult patients with...Read more
U.S. launch expected in early February Niktimvo is the first and only approved treatment for chronic GVHD that targets CSF-1R to reduce the drivers of inflammation and fibrosis Pivotal data from the AGAVE-201 trial supporting FDA approval show treatment with Niktimvo resulted in durable responses across all organs studied and patient subgroups WILMINGTON, Del. and WALTHAM, Mass., Jan. 15, 2025 /PRNewswire/ -- Incyte (Nasdaq:INCY)...Read more
In the pivotal Phase 3 VIVID-1 trial, patients treated with Omvoh experienced significant improvement in clinical remission and endoscopic response at one year Among those who achieved clinical remission and endoscopic response at one year, nearly 90% of patients maintained clinical remission with two years of continuous Omvoh treatment in open-label extension INDIANAPOLIS, Jan. 15, 2025 /PRNewswire/ -- Eli Lilly and Company (NYSE:...Read more
Application accepted for U.S. FDA Real-Time Oncology Review (RTOR) based on Phase 2b SunRISe-1 study showing highest single-agent complete response rate of 83.5 percent1 RARITAN, N.J., Jan. 15, 2025 /PRNewswire/ -- Johnson & Johnson (NYSE: JNJ) announced it has initiated the submission of an original New Drug Application with the U.S. Food and Drug Administration (FDA) for TAR-200 for the treatment of patients with Bacillus...Read more
Independent Data Safety Monitoring Board (DSMB) recommends study to proceed without modifications based on initial safety assessment of 400 participant 30-day data The company plans to progress the trial to enrollment of 10,000 participants, upon favorable review from the U.S. Food and Drug Administration (FDA) and upon Biomedical Advanced Research and Development Authority (BARDA) approval SOUTH SAN FRANCISCO, Calif., Jan. 13, 2025...Read more
LEQEMBI is the only FDA-approved anti-amyloid therapy that potentially could offer the convenience of a subcutaneous injection with at-home administration option TOKYO and CAMBRIDGE, Mass., Jan. 13, 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 U.S....Read more
Study to be conducted under $3.6 million NIH grant DENVER, Jan. 13, 2025 (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 (FDA) has approved an investigational device exemption (IDE) application to evaluate the...Read more
The Company will present at the Annual J.P. Morgan Healthcare Conference, providing business updates on its sarcoma franchise and other cell therapy pipeline assets Adaptimmune Allo-T program to be featured at the Biotech ShowCase(TM) and the Wuxi Global Forum 2025 Philadelphia, Pennsylvania and Oxford, United Kingdom--(Newsfile Corp. - January 13, 2025) - Adaptimmune Therapeutics plc (NASDAQ: ADAP), a company working to redefine...Read more
Application based on TROPION-Lung05 trial and supported by data from TROPION-Lung01 and TROPION-PanTumor01 trials Approval would mark the first for AstraZeneca and Daiichi Sankyo’s datopotamab deruxtecan in lung cancer WILMINGTON, Del. / Jan 13, 2025 / Business Wire / AstraZeneca and Daiichi Sankyo’s Biologics License Application (BLA) for datopotamab deruxtecan (Dato-DXd) has been accepted and granted Priority Review in the US for...Read more
The VENTANA Kappa and Lambda Dual ISH mRNA Probe Cocktail assay is the first clinically approved in-situ hybridisation (ISH) test with the sensitivity to assess the full spectrum of B-cell lymphoma subtypes.1,2 The test helps differentiate a B-cell cancer from a normal, reactive immune response, offering diagnostic certainty for healthcare providers and their patients. B-cell lymphoma accounts for approximately 85 percent of...Read more
TYRA appoints urologic oncologist, Erik Goluboff, M.D., as SVP, Clinical Development to lead NMIBC First patient expected to be dosed in SURF302 in Q2 2025 CARLSBAD, Calif., Jan. 10, 2025 /PRNewswire/ -- Tyra Biosciences, Inc. (Nasdaq: TYRA), a clinical-stage biotechnology company focused on developing next-generation precision medicines that target large opportunities in Fibroblast Growth Factor Receptor (FGFR) biology, announced...Read more
WALTHAM, Mass. / Jan 10, 2025 / Business Wire / Revvity, Inc. (NYSE: RVTY), today announced that it received 510(k) clearance from the U.S. Food and Drug Administration (FDA) for EUROIMMUN’s automated chemiluminescence-based immunoassay (ChLIA) test for free testosterone. This innovative test is the first of its kind to receive FDA clearance for direct quantitative measurement of free testosterone levels, marking a significant advancement...Read more
CAMBRIDGE, Mass., Jan. 10, 2025 (GLOBE NEWSWIRE) -- Mersana Therapeutics, Inc. (NASDAQ: MRSN), a clinical-stage biopharmaceutical company focused on discovering and developing a pipeline of antibody-drug conjugates (ADCs) targeting cancers in areas of high unmet medical need, today announced the U.S. Food and Drug Administration (FDA) recently granted an additional Fast Track designation to XMT-1660. The company also announced that the...Read more
SOUTH SAN FRANCISCO, Calif., Jan. 9, 2025 /PRNewswire/ -- Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL), a commercial stage biotechnology company focused on hematologic disorders and cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to R289 for the treatment of myelodysplastic syndromes (MDS). R2891, Rigel's potent and selective dual inhibitor of IRAK1 and IRAK4, is being...Read more
Biologics License Application acceptance supported by results from the Phase 3 Vivacity-MG3 study Results demonstrate sustained disease control over 24 weeks in a broad population of antibody positive adult patients: anti-AChR, anti-MuSK, anti-LRP4 SPRING HOUSE, Pa., Jan. 9, 2025 /PRNewswire/ -- Johnson & Johnson (NYSE: JNJ) today announced the nipocalimab Biologics License Application (BLA) received Priority Review...Read more
Single agent and combination data from Phase 1b AML/MDS trial presented at ASH 2024 showed potential of LYT-200 to serve broad range of patients across various lines of treatment LYT-200 is currently being evaluated in two Phase 1/2 trials for the potential treatment of AML/MDS and head and neck cancers BOSTON / Jan 09, 2025 / Business Wire / PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) (“PureTech” or the “Company”), a clinical-stage...Read more
Azenosertib Fast Track Designation granted for Cyclin E1 positive patients by U.S. Food and Drug Administration (FDA) Manuscript focused on role of Cyclin E1/CDK2 activation predicting sensitivity to azenosertib published in npj Precision Oncology Corporate event to be held on January 29, 2025 to provide updates on azenosertib clinical data, development and regulatory path SAN DIEGO, Jan. 09, 2025 (GLOBE NEWSWIRE)...Read more
The VENTANA DP 600 slide scanner, part of Roche's Digital Pathology Dx system, is now cleared by the FDA to aid in clinical diagnosis, enabling pathologists to diagnose patients using digital images. This 240-slide scanner produces excellent image quality of stained histology slides from patient tissue samples, while providing ease-of-use and workflow flexibility for the pathology lab. Primary diagnosis in digital pathology helps...Read more
SOUTH SAN FRANCISCO, Calif., Jan. 08, 2025 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (NASDAQ: DNLI), today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for tividenofusp alfa (DNL310) for the treatment of individuals with Hunter syndrome (MPS II). This designation is in addition to Fast Track Designation, Orphan Drug Designation, and Rare Pediatric Disease Designation, all...Read more
Building on decades of the Company's Alzheimer's research, two differentiated investigational therapies aim to slow pathological tau in distinct populations Fast Track designations reinforce J&J's commitment to Alzheimer's disease development and the potential of its precision approach TITUSVILLE, N.J., Jan. 8, 2025 /PRNewswire/ -- Johnson & Johnson (NYSE: JNJ) announced today that the U.S. Food and Drug Administration (FDA)...Read more
CAMBRIDGE, Mass., Jan. 08, 2025 (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 U.S. Food and Drug Administration (FDA) accepted the company’s supplemental New Drug Application (sNDA) for PYRUKYND® (mitapivat) for the treatment of adult patients with non-transfusion-dependent and...Read more
SGT-212 is the only full-length frataxin replacement gene therapy candidate targeting the CNS and cardiac manifestations of Friedreich’s ataxia Dual route of administration enables direct delivery of AAV-based gene therapy to the cerebellum and heart to potentially address the most significant symptoms of the disease Phase 1b clinical trial initiation expected in 2H 2025 Company to hold a conference call tomorrow,...Read more
Breakthrough Therapy Designation granted based on key positive data from the Phase 2b (RIZE) study and current unmet medical need in congenital hyperinsulinism (HI) Ersodetug continues to advance in clinical development as a potential treatment for hypoglycemia caused by all forms of hyperinsulinism; topline sunRIZE data expected second half of this year REDWOOD CITY, Calif., Jan. 07, 2025 (GLOBE NEWSWIRE) -- Rezolute, Inc. (Nasdaq:...Read more
PARSIPPANY, N.J., Jan. 07, 2025 (GLOBE NEWSWIRE) -- Pacira BioSciences, Inc. (Nasdaq: PCRX), the industry leader in the delivery of innovative, non-opioid pain therapies to transform the lives of patients, announced today that it has received clearance from the U.S. Food & Drug Administration (FDA) to market a new Smart Tip designed to access the medial branch nerves to manage chronic low back pain. The ioveraº system is an...Read more
BRISBANE, Calif., Jan. 06, 2025 (GLOBE NEWSWIRE) -- Tempest Therapeutics, Inc. (Nasdaq: TPST), a clinical-stage biotechnology company developing first-in-class1 targeted and immune-mediated therapeutics to fight cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to amezalpat (TPST-1120), an oral, small molecule, selective PPAR⍺ antagonist for the treatment of patients with...Read more
CLEARWATER, Fla., Jan. 06, 2025 (GLOBE NEWSWIRE) -- Apyx® Medical Corporation (NASDAQ:APYX) (“Apyx Medical”; the “Company”), the manufacturer of a proprietary helium plasma and radiofrequency technology marketed and sold as Renuvion®, is pleased to announce it has submitted a 510(k) premarket notification to the U.S. Food and Drug Administration (the “FDA”) for the AYON Body Contouring System. The AYON Body Contouring System was...Read more
Priority review granted for CUTX-101 with PDUFA target action date set for June 30, 2025 Cyprium is eligible to receive royalties and up to $129 million in aggregate development and sales milestones Cyprium also retains ownership over any Priority Review Voucher that may be issued at NDA approval MIAMI, Jan. 06, 2025 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (Nasdaq: FBIO) (“Fortress”) and its majority-owned subsidiary, Cyprium...Read more
Six-month priority review granted for CUTX-101 copper histidinate with PDUFA target action date set for June 30, 2025 CUTX-101 has potential to be the first FDA-approved treatment for Menkes disease, a rare and fatal pediatric disease SOLANA BEACH, Calif. and AHMEDABAD, India and MIAMI, Jan. 6, 2025 /PRNewswire/ -- Sentynl Therapeutics, Inc. ("Sentynl"), a U.S.-based biopharmaceutical company wholly-owned by Zydus Lifesciences,...Read more
If approved, deramiocel would be first approved therapy for Duchenne muscular dystrophy cardiomyopathy BLA submission triggers $10 million milestone payment to Capricor from Nippon Shinyaku SAN DIEGO, Jan. 02, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced the completion of the...Read more
PRINCETON, N.J., Jan. 02, 2025 (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 has launched Prucalopride Tablets. ANI’s Prucalopride Tablets is the generic version of the reference listed drug (RLD) Motegrity®. "We are delighted to build momentum...Read more
REDWOOD CITY, Calif. / Dec 30, 2024 / Business Wire / Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of medications to treat severe endocrinologic, oncologic, metabolic and neurologic disorders by modulating the effects of the hormone cortisol, has submitted a new drug application (NDA) to the U.S. Food and Drug Administration for its proprietary, selective cortisol...Read more
Secures Access of Up To $30 Million to Support Potential Launch Through Silicon Valley Bank Credit Facility DURHAM, N.C., Dec. 30, 2024 (GLOBE NEWSWIRE) -- Chimerix (NASDAQ: CMRX), a biopharmaceutical company whose mission it is to develop medicines that meaningfully improve and extend the lives of patients facing deadly diseases, today confirms that the Company has submitted a New Drug Application (NDA) with the U.S. Food and Drug...Read more
PDUFA target action date is June 30, 2025 If approved, avutometinib in combination with defactinib would be the first-ever FDA-approved treatment specifically for adults with recurrent KRAS mutant LGSOC BOSTON / Dec 30, 2024 / Business Wire / Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with cancer, today announced that the U.S. Food and Drug Administration (FDA) has...Read more
GOLETA, Calif. / Dec 30, 2024 / Business Wire / Inogen, Inc. (Nasdaq: INGN), a medical technology company offering innovative respiratory products for use in the homecare setting, today announced that it has received U.S. Food and Drug Administration (FDA) 510(k) clearance for the SIMEOX 200 Airway Clearance Device, expanding the company’s ability to market and meet the various needs of patients with chronic respiratory diseases in the...Read more
Aiming to be the first FDA-approved medication to treat suicidal depression Designed to help address the needs of the more than 13 million Americans who seriously consider suicide each year (CDC) Completion of NDA filing expected in the first quarter of 2025 Company to participate in 1x1 meetings in San Francisco during the Annual J.P. Morgan Healthcare Conference on January 13-16, 2025, in San Francisco, CA. To schedule...Read more
PRGN-2012 has the potential to be the first FDA-approved therapeutic for the treatment of adults with RRP, a rare and devastating chronic disease for which the current standard-of-care is repeated surgeries PRGN-2012 received Breakthrough Therapy Designation from the FDA and Orphan Drug Designation from the FDA and the European Commission The BLA, under an accelerated approval pathway, is supported by data from the Phase 1/2 pivotal...Read more
MELBOURNE, Australia and INDIANAPOLIS, Dec. 30, 2024 (GLOBE NEWSWIRE) -- Telix Pharmaceuticals Limited (ASX: TLX; Nasdaq: TLX, Telix, the Company) today announces that it has submitted its Biologics License Application (BLA) to the United States (U.S.) Food and Drug Administration (FDA) for TLX250-CDx (Zircaix®1, 89Zr- girentuximab) kidney cancer imaging2. TLX250-CDx is an investigational PET3 drug product for the non-invasive diagnosis...Read more
Opdivo Qvantig is the first and only subcutaneously administered PD-1 inhibitor1 Given over three- to five-minutes, Opdivo Qvantig demonstrated consistent efficacy and showed a comparable safety profile to intravenous Opdivo in the Phase 3 CheckMate-67T trial1,3 PRINCETON, N.J. / Dec 27, 2024 / Business Wire / Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) granted approval for Opdivo...Read more
New indication based on results from a global Phase 3 trial demonstrating TEVIMBRA plus chemotherapy significantly improved overall survival for patients with advanced gastric cancers Second FDA approval for TEVIMBRA in 2024 SAN MATEO, Calif. / Dec 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 Ltd., today announced the...Read more
VALENCIA, Calif., Dec. 23, 2024 (GLOBE NEWSWIRE) -- AVITA Medical, Inc. (NASDAQ: RCEL, ASX: AVH), a commercial-stage regenerative medicine company focused on first-in-class devices for wound care management and skin restoration, today announced that the U.S. Food and Drug Administration (FDA) has approved its premarket approval (PMA) supplement for RECELL GO® mini. As a line extension of the RECELL GO system, the RECELL GO mini disposable...Read more
New Drug Application (NDA) is based on pooled data from the pivotal Phase 2 TRUST-I and TRUST-II studies of taletrectinib that demonstrated durable responses and prolonged progression-free survival in patients with advanced ROS1-positive (ROS1+) non-small cell lung cancer (NSCLC) If approved, taletrectinib represents a potential best-in-class treatment option for patients with advanced ROS1+ NSCLC U.S. Food and Drug Administration (FDA)...Read more
ALISO VIEJO, Calif. / Dec 23, 2024 / Business Wire / Glaukos Corporation (NYSE: GKOS), an ophthalmic pharmaceutical and medical technology company focused on novel therapies for the treatment of glaucoma, corneal disorders and retinal diseases, today announced the submission of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for Epioxa™ (Epi-on), its next-generation corneal cross-linking iLink therapy for the...Read more
BRAFTOVI in combination with cetuximab and mFOLFOX6 is the first and only combination regimen with targeted therapy approved for use as early as first-line for patients with metastatic colorectal cancer with a BRAF V600E mutation Accelerated approval is based on 61% overall response rate compared to 40% in control arm in the Phase 3 BREAKWATER trial NEW YORK / Dec 20, 2024 / Business Wire / Pfizer Inc. (NYSE: PFE) today announced that...Read more
Averaging up to 20% of weight loss, adults taking Zepbound had at least 25 fewer breathing interruptions each hour as they slept Up to 50% of adults taking Zepbound no longer had symptoms associated with OSA after one year INDIANAPOLIS, Dec. 20, 2024 /PRNewswire/ -- Eli Lilly and Company (NYSE: LLY) today announced the U.S. Food and Drug Administration (FDA) approved Zepbound® (tirzepatide) as the first and only prescription...Read more
ALYFTREK™ is approved for patients 6 years and older with at least one responsive mutation, including 31 additional mutations not responsive to other CFTR modulator therapies In head-to-head clinical trials, ALYFTREK was non-inferior on ppFEV1 and further decreased sweat chloride compared to TRIKAFTA® BOSTON / Dec 20, 2024 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug...Read more
Approximately 300 more people with cystic fibrosis in the U.S. are now eligible for a medicine that treats the underlying cause of their disease for the first time BOSTON / Dec 20, 2024 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) has approved the expanded use of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for the treatment of people with...Read more
BOSTON, Dec. 20, 2024 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients living with rare neuroendocrine diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved an expanded indication for IMCIVREE® (setmelanotide) to include children as young as 2 years old. IMCIVREE is indicated to reduce excess...Read more
WASHINGTON, Dec. 20, 2024 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for VGT-1849A, a selective antisense oligonucleotide (ASO)-based JAK2 inhibitor for the treatment of polycythemia vera (PV), a form of a rare hematologic malignancy that is estimated to affect 1 in 2000 Americans.1 PV is a chronic myeloproliferative...Read more
Unique cell-impermeable1 design extends life-saving treatment for dialysis patients Merit to host a WRAPSODY informational call on January 28, 2025 SOUTH JORDAN, Utah, Dec. 20, 2024 (GLOBE NEWSWIRE) -- Merit Medical Systems, Inc. (NASDAQ: MMSI), a leading global manufacturer and marketer of healthcare technology, announced today that the WRAPSODY® Cell-Impermeable Endoprosthesis has received premarket approval from the US Food and...Read more
THE WOODLANDS, Texas, Dec. 20, 2024 (GLOBE NEWSWIRE) -- Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX) today announced it has received a complete response letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding the New Drug Application (NDA) for Zynquista™ (sotagliflozin) as an adjunct to insulin therapy for glycemic control in adults with type 1 diabetes and chronic kidney disease (CKD). This expected communication...Read more
SYMVESS is a first-in-class bioengineered human tissue designed to be a universally implantable vascular conduit for use in arterial replacement and repair In clinical testing SYMVESS was observed to have high rates of patency, or blood flow, and low rates of amputation and infection Highly experienced sales team already recruited and trained in preparation for commercial launch DURHAM, N.C., Dec. 19, 2024 (GLOBE NEWSWIRE) --...Read more
TRYNGOLZA shown to significantly reduce triglycerides and substantially reduce acute pancreatitis events in adults with FCS; a rare, highly debilitating and life-threatening disease Indicated for adults with FCS regardless of genetically or clinically confirmed diagnosis TRYNGOLZA is the first of four independent launches planned over the next three years, pending approvals Ionis to host webcast today at 6:45pm ET...Read more
New collagen-based dermal matrix designed for tissue generation, complementary to RECELL and PermeaDerm Cohealyx expected to triple AVITA Medical’s addressable market in burns VALENCIA, Calif., Dec. 19, 2024 (GLOBE NEWSWIRE) -- AVITA Medical, Inc. (NASDAQ: RCEL, ASX: AVH), a commercial-stage regenerative medicine company focused on first-in-class devices for wound care management and skin restoration, today announced that the U.S....Read more
MIAMI, FLORIDA / ACCESSWIRE / December 19, 2024 / MIRA Pharmaceuticals, Inc. (NASDAQ:MIRA), a preclinical-stage pharmaceutical company focused on developing therapies for neurological and neuropsychiatric disorders, today announced that it has submitted an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for Ketamir-2, its novel oral ketamine analog for the treatment of neuropathic pain. The IND...Read more
If approved, vatiquinone would be the first and only authorized therapy for children with FA PTC's fourth approval application submitted to FDA in 2024 WARREN, N.J., Dec. 19, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the submission of the vatiquinone New Drug Application (NDA) for the treatment of children and adults living with Friedreich ataxia (FA) to the U.S. Food and Drug...Read more
WARREN, N.J., Dec. 19, 2024 (GLOBE NEWSWIRE) -- Aquestive Therapeutics, Inc. (NASDAQ: AQST) (“Aquestive” or the “Company”), a pharmaceutical company advancing medicines to bring meaningful improvement to patients' lives through innovative science and delivery technologies, today announced the U.S. Food and Drug Administration (FDA) has granted seven years of orphan drug exclusivity (ODE) to Libervant® (diazepam) Buccal Film for the acute...Read more
NOVATO, Calif., Dec. 19, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA or the Agency) seeking accelerated approval for UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA). “The path to get a treatment to the point of a BLA filing has been long...Read more
SAN FRANCISCO, Dec. 19, 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 diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for NX-5948, a highly selective degrader of Bruton’s tyrosine kinase (BTK), for the treatment of...Read more
RYONCIL (remestemcel-L) is the first MSC product approved by FDA for any indication. RYONCIL is the first FDA-approved therapy for children aged 2 months and older, including adolescents and teenagers, with steroid-refractory acute graft versus host disease (SR-aGvHD), a life-threatening condition with high mortality rates. In a single-arm, multi-center, Phase 3 trial of children with SR-aGvHD, 89% of whom had high severity Grade C or...Read more
Company Expects to Complete BLA Submission by End of 1Q 2025 LANGHORNE, Pa. / Dec 18, 2024 / Business Wire / Savara Inc. (Nasdaq: SVRA) (the Company), a clinical stage biopharmaceutical company focused on rare respiratory diseases, initiated a rolling submission of a BLA to the FDA for MOLBREEVI for the potential treatment of aPAP, a chronic and debilitating rare lung disease characterized by the abnormal build-up of surfactant...Read more
MONTREAL, Dec. 18, 2024 (GLOBE NEWSWIRE) -- Theratechnologies Inc. (“Theratechnologies” or the “Company”) (TSX: TH) (NASDAQ: THTX), a biopharmaceutical company focused on the development and commercialization of innovative therapies, today announced that the Company has submitted a Prior Approval Supplement (PAS) to the U.S. Food and Drug Administration (FDA) describing the changes made to the manufacturing environment of the facility...Read more
Dravet syndrome is a genetic developmental and epileptic encephalopathy (DEE) often caused by a mutation in SCN1A This is the third Rare Pediatric Disease Designation for relutrigine, adding to those granted for SCN2A and SCN8A DEEs Praxis plans to initiate an all-DEE trial (EMERALD), inclusive of Dravet syndrome, in 1H2025 BOSTON, Dec. 18, 2024 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage...Read more
FOSTER CITY, Calif. / Dec 17, 2024 / Business Wire / Gilead Sciences, Inc. (Nasdaq: GILD) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to Trodelvy® (sacituzumab govitecan-hziy) for the treatment of adult patients with extensive-stage small cell lung cancer (ES-SCLC) whose disease has progressed on or after platinum-based chemotherapy. The Breakthrough Therapy Designation is...Read more
World Health Organization (WHO) has classified the global resurgence of cholera at the highest internal level for emergencies; 1.3 to 4 million cholera cases and 21,000 to 143,000 cholera-related deaths occur each year worldwide Cholera is an acute diarrheal illness caused by infection of the intestine with the bacterium Vibrio cholerae Crofelemer previously granted orphan-drug designation by the FDA and the European Medicines Agency...Read more
SALT LAKE CITY, Dec. 17, 2024 /PRNewswire/ -- Lipocine Inc. (NASDAQ: LPCN), a biopharmaceutical company leveraging its proprietary technology platform to augment therapeutics through effective oral delivery, today announced that the U.S. Food and Drug Administration ("FDA") has granted Fast Track Designation to LPCN 1148 as a treatment for sarcopenia in patients with decompensated cirrhosis. LPCN 1148, an oral prodrug of bioidentical...Read more
FDA is expected to assign a Prescription Drug User Fee Act (PDUFA) target action date and announce whether Priority Review has been granted in the Day 74 Letter TNX-102 SL is a non-opioid, centrally acting analgesic, granted Fast Track designation by FDA Fibromyalgia affects more than 10 million adults in the U.S. who are mostly women TNX-102 SL has the potential to be the first member of a new class of analgesic drugs for...Read more
If approved, clesrovimab has the potential to be available to help address the burden of RSV disease in the U.S. in time for the 2025-26 season RAHWAY, N.J. / Dec 17, 2024 / Business Wire / Merck (NYSE: MRK), known as MSD outside of the United States and Canada, today announced the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for clesrovimab (MK-1654), the company’s investigational...Read more
Smith+Nephew (LSE:SN, NYSE:SNN), the global medical technology company, today announces it has received 510(k) clearance from the United States Food & Drug Administration for a stemless anatomic total shoulder for the AETOS Shoulder System (AETOS Stemless). AETOS Stemless addresses the growing demand for anatomic total shoulder replacement with a small operating room footprint allowing for an efficient procedure.1 It is...Read more
CHICAGO / Dec 16, 2024 / Business Wire / MAIA Biotechnology, Inc., (NYSE American: MAIA) (“MAIA”, the “Company”), a clinical-stage biopharmaceutical company developing targeted immunotherapies for cancer, today announced that the FDA has designated THIO for the treatment of pediatric-type diffuse high-grade gliomas (PDHGG) as a drug for a “rare pediatric disease.” “THIO is a versatile anti-cancer agent that has demonstrated positive...Read more
First cable-free, ambulatory ECG that captures the heart’s electrical signals from three distinct directions for high-fidelity data collection and advanced diagnostics Patients can have the credit card-sized device with them at all times, ready to record an ECG whenever they feel symptoms and reduce delays in care Company to initiate Early Access Program to gain important patient and physician feedback on the use of the system in...Read more
ZORYVE cream 0.05% provided meaningful disease clearance and rapid reduction in itch in pivotal trials Roflumilast cream was well tolerated and demonstrated a favorable safety and tolerability profile for up to 56 weeks of treatment Approximately 1.8 million children with atopic dermatitis (AD) aged 2 to 5 are topically treated in the United States WESTLAKE VILLAGE, Calif., Dec. 16, 2024 (GLOBE NEWSWIRE) -- Arcutis Biotherapeutics,...Read more
EFFICACY: In the pivotal studies, ADORING 1 and ADORING 2, up to 46% of patients on VTAMA cream achieved vIGA-AD™ treatment success at Week 8 versus 18% of patients on vehicle. POWERFUL SKIN CLEARANCE: The majority of patients entered with or achieved complete disease clearance (vIGA-AD=0) at least once in the 48-week open-label ADORING long-term extension (LTE) study and remained treatment-free (remittive effect) for an average of ~80...Read more
CRENESSITY, the first new treatment available in 70 years to the classic congenital adrenal hyperplasia (CAH) community, offers a paradigm-shifting treatment approach FDA approval supported by data from the largest-ever clinical trial program in pediatric and adult patients with classic CAH CRENESSITY is expected to be commercially available in approximately one week Rare Pediatric Disease Priority Review Voucher granted in...Read more
WALTHAM, Mass., Dec. 13, 2024 (GLOBE NEWSWIRE) -- Checkpoint Therapeutics, Inc. (“Checkpoint”) (Nasdaq: CKPT), today announced that the U.S. Food and Drug Administration (“FDA”) has approved UNLOXCYT™ (cosibelimab-ipdl) for the treatment of adults with metastatic cutaneous squamous cell carcinoma (“cSCC”) or locally advanced cSCC who are not candidates for curative surgery or curative radiation. UNLOXCYT is the first and only...Read more
New Anatomically Shaped Asymmetric Stemless Design for Total Shoulder Replacement WARSAW, Ind., Dec. 13, 2024 /PRNewswire/ -- Zimmer Biomet Holdings, Inc. (NYSE and SIX: ZBH), a global medical technology leader, today announced U.S. Food and Drug Administration (FDA) 510(k) clearance for the OsseoFit™ Stemless Shoulder System for total shoulder replacement. This innovative implant is designed to match the natural...Read more
Designations aim to expedite the development and review of promising therapies for serious conditions with unmet medical needs Phase 3 ECLIPSE registrational program in chronic hepatitis delta to begin in the first half of 2025 SAN FRANCISCO / Dec 12, 2024 / Business Wire / Vir Biotechnology, Inc. (Nasdaq: VIR) today announced that tobevibart and elebsiran have received U.S. Food and Drug Administration (FDA) Breakthrough...Read more
COPENHAGEN, Denmark, Dec. 12, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that the U.S. Food & Drug Administration (FDA) has accepted for review its supplemental Biologics License Application (sBLA) in adult growth hormone deficiency (GHD) for TransCon hGH (lonapegsomatropin-tcgd; marketed as SKYTROFA® for pediatric GHD). The FDA set a Prescription Drug User Fee Act (PDUFA) goal date of July 27,...Read more
IMM-1-104 has the potential to benefit melanoma patients who have progressed on or are intolerant to immune checkpoint inhibitors IMM-1-104 was observed to be uniquely well tolerated in Phase 1 data shared at ESMO 2024, relative to MEK inhibitors currently used to treat melanoma Melanoma patients actively enrolling in one of five arms in the company’s ongoing Phase 2a clinical study of IMM-1-104 CAMBRIDGE, Mass.,...Read more
TORONTO and PHILADELPHIA, Dec. 12, 2024 (GLOBE NEWSWIRE) -- Medicus Pharma Ltd. (NASDAQ: MDCX) (TSXV: MDCX) ("Medicus" or the "Company") is pleased to announce that its Investigational New Animal Drug (INAD File No.013880) has received Minor Use in Major Species Designation (“MUMS”) from the U.S. Food and Drug Administration (“FDA”) for its dissolvable Doxorubin-containing microneedle array (D-MNA) to treat external squamous cell...Read more
Chimerix is on a mission to develop medicines that meaningfully improve and extend the lives of patients facing deadly diseases. The company is devoted to filling gaps in the treatment paradigm. Chimerix’s most advanced clinical-stage program is in development for H3 K27M-mutant glioma....
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