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) has assigned a target action date... 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 treatment of keratoconus, a progressive,... 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 submission marks a major step in advancing... 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 Administration (FDA).
"We are excited to have... 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 fibromyalgia and the first new drug for treating... 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, Inc. (Nasdaq: ARQT), a co... Read more
Phase 1a Trial of STAR-0310 in Healthy Volunteers Expected to Initiate in Q1 2025
Early Proof-of-Concept Results Expected in Q3 2025 BOSTON / Dec 10, 2024 / Business Wire / Astria Therapeutics, Inc. (Nasdaq:ATXS), a biopharmaceutical company focused on developing life-changing therapies for allergic and immunologic diseases, today announced the U.S. Food and Drug Administration (FDA) clearance of its Investigational New Drug (IND) application for STAR-0310,... Read more
BELTSVILLE, Md., Dec. 10, 2024 (GLOBE NEWSWIRE) -- NextCure, Inc. (Nasdaq: NXTC), a clinical-stage biopharmaceutical company committed to discovering and developing novel, first-in-class, and best-in-class therapies to treat cancer, today announced that the U.S. Food and Drug Administration (FDA) accepted an Investigational New Drug (IND) application for initiation of a Phase 1 clinical trial to evaluate LNCB74, a B7-H4-targeting antibody-drug conjugate (ADC) as a therapeutic... Read more
New Dosing Regimen Simplifies Administration NASHVILLE, Tenn., Dec. 9, 2024 /PRNewswire/ -- Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company focused on delivering high-quality products to improve patient care, announced today the FDA has approved a supplemental New Drug Application (sNDA) for its Acetadote® (N-acetylcysteine for injection) product. Acetadote is an intravenous (IV) formulation of N-acetylcysteine (NAC) indicated to... Read more
Potential Approval in Q3 2025 in Recurrent H3 K27M-Mutant Diffuse Glioma
Submission Plan Follows Productive and Collaborative Pre-NDA Interactions with FDA
Company to Host Conference Call on Tuesday, December 10 at 8:30 AM ET DURHAM, N.C., Dec. 09, 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 announced... Read more
SAN DIEGO, Dec. 09, 2024 (GLOBE NEWSWIRE) -- Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) today announced the U.S. Food and Drug Administration (FDA) accepted its New Drug Application (NDA) for investigational candidate paltusotine for the treatment and long-term maintenance therapy of acromegaly in adults. If approved, paltusotine will be the first and only once-daily, oral, selective somatostatin receptor type 2 nonpeptide agonist available for adults living with a... Read more
Phase 1 clinical trial for OP-3136 to initiate in early 2025 SAN FRANCISCO, Dec. 09, 2024 (GLOBE NEWSWIRE) -- Olema Pharmaceuticals, Inc. (“Olema” or “Olema Oncology”, Nasdaq: OLMA), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of targeted therapies for breast cancer and beyond, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application... Read more
The sNDA submission is based on positive results from Studies 501 and 502 demonstrating CAPLYTA’s robust antidepressant efficacy and favorable safety and tolerability profile
CAPLYTA, if approved as an adjunctive therapy in MDD, would be indicated for the treatment of three different major psychiatry indications affecting over 30 million adult patients in the US BEDMINSTER, N.J., Dec. 03, 2024 (GLOBE NEWSWIRE) -- Intra-Cellular Therapies, Inc. (Nasdaq: ITCI),... Read more
QUEENSBURY, N.Y. / Dec 02, 2024 / Business Wire / Delcath Systems, Inc. (NASDAQ: DCTH), an interventional oncology company focused on the treatment of primary and metastatic liver cancers, today announced that the U.S. Food and Drug Administration (FDA) has completed its 30-day review of the Company's Investigational New Drug (IND) application for a Phase 2 clinical trial evaluating HEPZATO™ in combination with standard of care (SOC) for liver-dominant metastatic colorectal... Read more
PDUFA Target Action Date Set for September 26, 2025 SOUTH SAN FRANCISCO, Calif., Dec. 02, 2024 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) today announced that the U.S. Food & Drug Administration (FDA) has accepted the company’s New Drug Application (NDA) for aficamten, a next-in-class cardiac myosin inhibitor, for the treatment of obstructive hypertrophic cardiomyopathy (HCM).
The FDA assigned the NDA a standard review with a Prescription... Read more
Phase 1b Study in CKD Patients to begin early 2025
SAN DIEGO / Dec 02, 2024 / Business Wire / Revelation Biosciences, Inc. (NASDAQ: REVB) (the “Company” or “Revelation”), a clinical-stage life sciences company that is focused on harnessing the power of trained immunity for the treatment of disease, announced today that the United States Food and Drug Administration (FDA) has accepted its investigational new drug (IND) application for Gemini. This game changing milestone... Read more
Prescription Drug User Fee Act (PDUFA) Date Set for March 23, 2025 CAMBRIDGE, Mass. / Nov 25, 2024 / Business Wire / Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the Company’s supplemental New Drug Application (sNDA) for vutrisiran, an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis with cardiomyopathy... Read more
Sangamo plans to initiate enrollment of patients in the Phase 1/2 study for ST-503 in mid-2025 RICHMOND, Calif. / Nov 19, 2024 / Business Wire / Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced that the U.S. Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for its ST-503 program, an investigational epigenetic regulator for the treatment of intractable pain due to idiopathic small fiber... Read more
Phase 1b study for the potential treatment of osteoarthritis and low back pain expected to commence in the first quarter of 2025
Initial data readout planned for early 2026
Neuropathic component of chronic pain linked to LPA1 activation SAN DIEGO / Nov 18, 2024 / Business Wire / Contineum Therapeutics, Inc. (NASDAQ: CTNM) (Contineum or the Company), a clinical stage biopharmaceutical company focused on discovering and developing novel, oral small molecule therapies... 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
There are currently no FDA approved therapies to treat familial chylomicronemia syndrome PASADENA, Calif. / Nov 18, 2024 / Business Wire / Arrowhead Phar... Read more
PDUFA Date is April 2, 2025 LEXINGTON, Mass. / Nov 18, 2024 / Business Wire / Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra) today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the resubmitted New Drug Application (NDA) for topical ocular reproxalap, a first-in-class investigational new drug candidate, for the treatment of the signs and symptoms of dry eye disease. The FDA assigned a Prescription Drug User Fee Act (PDUFA) date... Read more
Northvale, New Jersey--(Newsfile Corp. - November 18, 2024) - Elite Pharmaceuticals, Inc. (OTCQB: ELTP) ("Elite" or the "Company"), a specialty pharmaceutical company engaged in the development, manufacture, and distribution of niche generic products, today announced that it received approval from the US Food and Drug Administration (FDA) for an Abbreviated New Drug Application (ANDA) for a generic version of Vyvanse® (Lisdexamfetamine Dimesylate) with strengths of 10... Read more
VAX-31 Infant Indication: Investigational New Drug Application Cleared by FDA; Company Expects to Initiate VAX-31 Infant Phase 2 Study by the End of January 2025
VAX-31 Adult Indication: Breakthrough Therapy Designation Granted by FDA; Company Plans to Initiate Adult Phase 3 Pivotal, Non-Inferiority Study by Mid-2025
VAX-31, Designed to Cover Currently Circulating and Historically Prevalent Strains, is Being Studied for the Prevention of Invasive Pneumococcal... Read more
FDA sets PDUFA Action Date of June 28, 2025
Company Preparing for 2025 Commercial Launch LOS ALTOS, Calif., Nov. 11, 2024 (GLOBE NEWSWIRE) -- Unicycive Therapeutics, Inc. (Nasdaq: UNCY), a clinical-stage biotechnology company developing therapies for patients with kidney disease (the “Company” or “Unicycive”), today announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for Oxylanthanum Carbonate (OLC)... Read more
Donidalorsen will be a first-in-class RNA-targeted medicine for hereditary angioedema, assuming approval
Donidalorsen PDUFA date set for August 21, 2025
Donidalorsen has the potential to be Ionis' second independent commercial launch CARLSBAD, Calif., Nov. 4, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for donidalorsen,... Read more
Recurrent low-grade serous ovarian cancer is a rare cancer with no FDA-approved treatments
Company seeking accelerated approval and priority review of its NDA submission in patients with KRAS mutant low-grade serous ovarian cancer; FDA filing decision expected before the end of 2024 with potential for FDA approval decision by mid-2025 BOSTON / Oct 31, 2024 / Business Wire / Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines... Read more
WARREN, N.J., Oct. 30, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the U.S. Food and Drug Administration (FDA) has accepted for review the resubmission of the New Drug Application (NDA) for Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).
"The NDA acceptance for review is a significant milestone that brings us one step closer to providing this important treatment to boys and young men living... Read more
Encinitas, California--(Newsfile Corp. - October 29, 2024) - Kiora Pharmaceuticals, Inc. (NASDAQ: KPRX) ("Kiora" or the "Company") today announced it received regulatory approval to initiate a Phase 2 clinical trial to investigate KIO-301 for vision restoration in patients with retinitis pigmentosa. The ABACUS-2 trial will be a 36 patient, multi-center, double-masked, randomized, controlled, multiple dose study enrolling patients with ultra-low vision or no light perception... Read more
TYRA-300 is the first oral FGFR-3 selective inhibitor to be well-tolerated in clinical studies
First child with achondroplasia expected to be dosed in Q1 2025 CARLSBAD, Calif., Oct. 28, 2024 /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 today that the U.S.... Read more
IDE275 (GSK959) development is progressing into First-in-Human Phase 1 clinical trial(s) for the treatment of MSI-High solid tumors, representing IDEAYA's 5th potential first-in-class clinical program
MSI-High prevalence in endometrial, colorectal, and gastric cancers is ~31%, 20%, and 19%, respectively, highlighting the market potential of IDE275 (GSK959)
IDEAYA to receive a $7 million payment for IND acceptance, and potential future aggregate milestones of up to $950... Read more
