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
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
First Breakthrough Therapy Designation for AstraZeneca and Daiichi Sankyo’s datopotamab deruxtecan Based on TROPION-Lung05 Phase II trial and supported by data from TROPION-Lung01 Phase III trial WILMINGTON, Del. / Dec 09, 2024 / Business Wire / Datopotamab deruxtecan (Dato-DXd) has been granted Breakthrough Therapy Designation (BTD) in the US for the treatment of adult patients with locally advanced or metastatic epidermal growth...Read more
Designation based on encouraging Phase 1b clinical data, including that SER-155 resulted in a 77% relative risk reduction in bacterial bloodstream infections versus placebo Breakthrough Therapy meeting with FDA on next study of SER-155 in allo-HSCT expected in Q1 2025 Seres seeking SER-155 strategic partnership to accelerate next study in allo-HSCT and expand to multiple target populations CAMBRIDGE, Mass., Dec. 09, 2024 (GLOBE...Read more
Supported by evidence from clinical studies that indicate that zorevunersen may demonstrate substantial improvement over available therapies Update on the company’s plans for a global, randomized, controlled Phase 3 registrational study anticipated by year-end BEDFORD, Mass. / Dec 04, 2024 / Business Wire / Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to restoring protein expression by...Read more
First breakthrough therapy designation for investigational sac-TMT in the U.S. RAHWAY, N.J. / Dec 03, 2024 / Business Wire / Merck (NYSE: MRK), known as MSD outside of the United States and Canada, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to sacituzumab tirumotecan (sac-TMT) for the treatment of patients with advanced or metastatic nonsquamous non-small cell lung...Read more
WOBURN, Mass., Nov. 21, 2024 (GLOBE NEWSWIRE) -- Replimune Group, Inc. (NASDAQ: REPL), a clinical stage biotechnology company pioneering the development of novel oncolytic immunotherapies, today announced that it has submitted a biologics license application (BLA) to the FDA for RP1 (vusolimogene oderparepvec) in combination with nivolumab for the treatment of adult patients with advanced melanoma who have previously received an anti-PD1...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...Read more
The Breakthrough Therapy Designation (BTD) for investigational nipocalimab in Sjögren's disease, a prevalent autoantibody disease with no approved advanced therapies, is supported by results from the Phase 2 DAHLIAS study A greater than 70 percent relative improvement in systemic disease activity at Week 24 was demonstrated in study participants on average who received nipocalimab 15 mg/kg compared to participants who received...Read more
Designation based on positive interim analysis of Phase 2b VANTAGE study FOSTER CITY, Calif. / Oct 10, 2024 / Business Wire / Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to volixibat as a potential treatment for cholestatic pruritus in patients with primary biliary cholangitis (PBC). The regulatory designation is based on the...Read more
Designation is based on data from the clinical development program which demonstrated clinical proof of concept Felzartamab, an investigational anti-CD38 monoclonal antibody, is a potential first-in-class therapeutic candidate for a range of rare immune-mediated indications with planning underway for Phase 3 development FDA Breakthrough Therapy Designation is intended to expedite the development and review of drugs for serious or...Read more
NOVATO, Calif., Oct. 07, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that it has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for setrusumab (UX143) as a treatment to reduce the risk of fracture associated with osteogenesis imperfecta (OI) Type...Read more
Supported by positive data from Phase 2b FASCINATE-2 trial of denifanstat in patients with MASH Preparations are ongoing to initiate Phase 3 program for denifanstat by the end of 2024 SAN MATEO, Calif., Oct. 01, 2024 (GLOBE NEWSWIRE) -- Sagimet Biosciences Inc. (Sagimet, Nasdaq: SGMT), a clinical-stage biopharmaceutical company developing novel therapeutics targeting dysfunctional metabolic and fibrotic pathways, today...Read more
U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation and Rare Pediatric Disease designation for bexicaserin for the treatment of Dravet syndrome LA JOLLA, Calif. / Sep 19, 2024 / Business Wire / Longboard Pharmaceuticals, Inc. (Nasdaq: LBPH), a clinical-stage biopharmaceutical company focused on developing novel, transformative medicines for neurological diseases, today announced that the FDA has granted Rare...Read more
Breakthrough Therapy Designation was granted based on preliminary clinical evidence from the PROPEL 2 clinical trial, meeting the FDA’s requirement of potentially demonstrating substantial improvement in efficacy over available therapies on clinically significant endpoint(s) BridgeBio will leverage the benefits of Breakthrough Therapy Designation to expedite the development and regulatory review of infigratinib in the United States and...Read more
PALISADE Phase 3 results demonstrate plozasiran reduced triglycerides by 80% from baseline and reduced the risk of developing acute pancreatitis by 83% in patients with genetically confirmed and clinically diagnosed FCS1 PASADENA, Calif. / Sep 10, 2024 / Business Wire / Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that the United States Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to...Read more
SEATTLE and VANCOUVER, British Columbia, July 31, 2024 (GLOBE NEWSWIRE) -- Achieve Life Sciences, Inc. (Nasdaq: ACHV), a late-stage pharmaceutical company committed to the global development and commercialization of cytisinicline for smoking cessation and nicotine dependence, announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for cytisinicline for nicotine e-cigarette, or vaping,...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
BRISBANE, Calif., May 28, 2024 (GLOBE NEWSWIRE) -- Vera Therapeutics, Inc. (Nasdaq: VERA), a late clinical-stage biotechnology company focused on developing and commercializing transformative treatments for patients with serious immunological diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to atacicept for the treatment of IgAN. The designation reflects the FDA’s...Read more
DURECT plans to confirm the efficacy and safety of larsucosterol in a registrational Phase 3 clinical trial CUPERTINO, Calif., May 21, 2024 /PRNewswire/ -- DURECT Corporation (Nasdaq: DRRX), a late-stage biopharmaceutical company pioneering the development of epigenetic therapies to transform the treatment of serious and life-threatening conditions such as acute organ injury and cancer, today announced that the U.S. Food and...Read more
The designation is based on Phase III INAVO120 results, showing the inavolisib-based regimen more than doubled progression-free survival compared with palbociclib and fulvestrant alone in the first-line setting Approximately 40% of people with HR-positive breast cancer have a PIK3CA mutation and often face poorer prognosis and resistance to endocrine treatment This is the 29th Breakthrough Therapy Designation for Genentech’s...Read more
CAMBRIDGE, Mass., May 16, 2024 /PRNewswire/ -- Nuvalent, Inc. (Nasdaq: NUVL), a clinical-stage biopharmaceutical company focused on creating precisely targeted therapies for clinically proven kinase targets in cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation (BTD) to NVL-655 for the treatment of patients with locally advanced or metastatic ALK-positive non-small cell...Read more
Petosemtamab granted BTD for the treatment of previously treated HNSCC UTRECHT, The Netherlands and CAMBRIDGE, Mass., May 13, 2024 (GLOBE NEWSWIRE) -- Merus N.V. (Nasdaq: MRUS), a clinical-stage oncology company developing innovative, full-length multispecific antibodies (Biclonics® and Triclonics®) for cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for...Read more
Avidity initiating global Phase 3 HARBOR™ study for delpacibart etedesiran this quarter Delpacibart etedesiran data from MARINA-OLE™ showed reversal of disease progression in multiple functional measures in DM1 compared to END-DM1 natural history data SAN DIEGO, May 8, 2024 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA...Read more
First Ever Breakthrough Designation for a Drug Being Developed for PWS Designation is Based on Data from the Phase 3 Program for DCCR Planned Submission of a New Drug Application (NDA) for DCCR Remains on Track for Mid-2024 REDWOOD CITY, Calif., April 29, 2024 (GLOBE NEWSWIRE) -- Soleno Therapeutics, Inc. (Soleno) (NASDAQ: SLNO), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare...Read more
Ziftomenib is the first investigational treatment to be granted Breakthrough Therapy Designation for NPM1-mutant AML Registration-directed trial of ziftomenib in NPM1-mutant AML on track to complete enrollment by mid-2024 SAN DIEGO, April 22, 2024 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment...Read more
Breakthrough Therapy Designation (“BTD”) provides an expedited review pathway, as well as increased access to U.S. Food and Drug Administration (“FDA”) guidance on trial design, with the potential to significantly reduce drug development timelines First known BTD granted by the FDA for an adjunctive psychedelic based therapy for the treatment of Major Depressive Disorder (“MDD”) Robust, sustained and statistically...Read more
A single oral administration of MM120 100 µg met its key secondary endpoint and maintained a clinically and statistically significant HAM-A reductions compared to placebo at 12 weeks with a 65% clinical response rate and 48% clinical remission rate MindMed plans to hold an End-of-Phase 2 meeting with the U.S. Food & Drug Administration (FDA) in the first half of 2024 and initiate its Phase 3 clinical program in the second half of...Read more
CAMBRIDGE, Mass., Feb. 27, 2024 /PRNewswire/ -- Nuvalent, Inc. (Nasdaq: NUVL), a clinical-stage biopharmaceutical company focused on creating precisely targeted therapies for clinically proven kinase targets in cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation (BTD) to NVL-520 for the treatment of patients with ROS1-positive metastatic non-small cell lung cancer (NSCLC)...Read more
Latozinemab is the most advanced progranulin-elevating candidate in development for FTD-GRN and has now become the first investigational medicine to receive a Breakthrough Therapy Designation for the treatment of FTD-GRN SOUTH SAN FRANCISCO, Calif., Feb. 07, 2024 (GLOBE NEWSWIRE) -- Alector, Inc. (Nasdaq: ALEC) and GSK plc (LSE/NYSE: GSK) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough...Read more
C4 Therapeutics is pioneering a new class of small-molecule drugs that selectively destroy disease-causing proteins via degradation using the innate machinery of the cell. This targeted protein degradation approach offers advantages over traditional drugs, including the potential to treat a wider range of diseases...
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