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2] Fitusiran was approved for medical use in the United States in March 2025. [2] 1] [2] Adverse effects The US Food and Drug Administration prescription label for fitusiran contains a boxed warning for thrombotic events (blood clotting) and gallbladder disease (with some recipients requiring gallbladder removal). [2] 26 March 2025.
Taletrectinib CAS 1505514-27-1 as salt: 1505515-69-4 , Taletrectinib adipate FDA 6/11/2025, Ibtrozi, To treat locally advanced or metastatic ROS1-positive non-small cell lung cancer AB-106, DS-6051a 405.5 1] Taletrectinib was approved for medical use in the United States in June 2025. [3] April 2025. Retrieved 14 June 2025.
NGS has revolutionised genomic analysis, enabling the identification of disease-related genetic variants. This ability to correlate seemingly disparate data is crucial for tackling complex targets and discovering new treatments for diseases considered difficult to treat with conventional approaches.
Retrieved 17 April 2025. ^ “Qalsody- tofersen injection” DailyMed. “FDA grants accelerated approval for Biogen ALS drug that treats rare form of the disease” CNBC. “FDA advisors vote against effectiveness of Biogen’s ALS drug for rare and aggressive form of the disease” CNBC.
Proteinuria reduction is a well-established surrogate marker for the progression of kidney disease, and this level of decline is considered both statistically significant and clinically meaningful in slowing disease progression toward end-stage kidney disease (ESKD). in the group treated with sibeprenlimab relative to placebo.
The data, which encompass up to 3 years of follow-up after a single infusion of the treatment, were shared in an oral presentation at the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2025, held from June 13–16 in Glasgow, United Kingdom. President and Chief Executive Officer of Intellia Therapeutics.
Based on Nobel Prize-winning research, IRLAB has grown rapidly to become recognised and respected as a world-leader in understanding the complex neuropharmacology of CNS disorders, especially Parkinson’s disease (PD). Top-line data from the Phase IIb study is expected at the beginning of 2025.
Acoltremon AR-15512 CAS 68489-09-8 WeightAverage: 289.419 Monoisotopic: 289.204179113 Chemical FormulaC 18 H 27 NO 2 FDA 2025, 5/28/2025, To treat the signs and symptoms of dry eye disease Tryptyr WS-12 WS 12 (1R,2S,5R)-N-(4-methoxyphenyl)-5-methyl-2-(propan-2-yl)cyclohexane-1-carboxamide Fema No. 29 May 2025.
2017, 8, 9, 975980 [link] The predominant expression of phosphoinositide 3-kinase (PI3K) in leukocytes and its critical role in B and T cell functions led to the hypothesis that selective inhibitors of this isoform would have potential as therapeutics for the treatment of allergic and inflammatory disease. 16 April 2025.
recent announcements from Merck and Sanofi for obesity and broader metabolic disease). Rise of the best-in-class biologics plays: Are you looking to in-license or invest in “de-risked” biology that is already in the clinic? Join the club. Additional trials (e.g.,
Genmab Announces Leadership Transition as Chief Legal Officer Birgitte Stephensen Retires, Greg Mueller Appointed Successor Genmab A/S (Nasdaq: GMAB), a leading international biotechnology company focused on antibody-based therapeutics for cancer and other serious diseases, today announced a major leadership transition within its executive team.
1] [2] [3] Levacetylleucine is the second medication approved by the US Food and Drug Administration (FDA) for the treatment of Niemann-Pick disease type C. [2] 2] The US Food and Drug Administration (FDA) granted the application for levacetylleucine priority review , fast track , orphan drug , and rare pediatric disease designations. [2]
Rahway NJ USA) research facility in the Longwood medical area of Boston, Massachusetts, on 24 June 2025. Understanding the role of neuroimmune interactions has become increasingly important in tackling complex neurological diseases. The event is held in partnership with Drug Discovery News. Matt received his B.Sc.
Retrieved 17 April 2025. January 2025. Archived from the original on 21 January 2025. Retrieved 21 January 2025. 3] Structure, reactivity, and synthesis Inavolisib is a synthetic, organic, small compound (the full structure can be seen here ). [8] 3] Names Inavolisib is the international nonproprietary name. [22]
A new roadmap for cannabis and cannabis policy research mfleming Fri, 01/03/2025 - 11:38 Nora's Blog January 14, 2025 Image Getty Images/ Fiordaliso The greatly increased availability of cannabis over the last two decades has outpaced our understanding of the public-health impacts of the drug.
Published June 26, 2025 Gwendolyn Wu Senior reporter post share post print email license B cells, like those pictured above, malfunction in autoimmune disease. Vor Biopharma licensed a drug in June 2025 that targets proteins essential to B cell survival. and European drugmakers. His research, writing offer clues.
4] A medication used to reduce the amount of steroid replacement required in patients with a genetic disease that causes, amongst other symptoms, a steroid deficiency. Retrieved 25 January 2025. January 2025. Archived from the original on 21 January 2025. . : 321839-75-2 Molecular Weight 519.50 1] It is taken by mouth. [1]
The $400,000 prize, to be shared among the four winners, recognizes their role in creating and advancing a technology that has reshaped how physicians treat leukemia, lymphoma, and multiple myeloma, and is now showing promise in treating autoimmune and infectious diseases. Their work puts us on the doorstep of finding cures to help millions.
Dedicated rare disease unit to be headquartered in Boston. Double-digit revenue growth through 2025; acquisition strengthens AstraZeneca’s broad-based revenue and the company will further globalise Alexion’s portfolio . Geographical presence to be enhanced with broad coverage across primary, speciality and highly specialised care.
Published June 24, 2025 Gwendolyn Wu Senior reporter post share post print email license Lexeo Therapeutics and two life sciences investors are working together to launch a startup to develop cardiac RNA therapies. Earlier this year, it laid off 15% of its workforce.
Published July 8, 2025 Gwendolyn Wu Senior reporter post share post print email license Proteasomes are cellular machines for breaking down proteins. Love Employee via Getty Images For decades now, scientists have been experimenting with a new way to target and destroy proteins linked to disease.
In 2019, we introduced Vision 3×3, the company’s strategic roadmap through 2025, to build a leading biopharma company by achieving sustainable growth through multiple approaches.
The company submitted its Biologic License Application to the FDA which has set a Prescription Drug User Fee Act (PDUFA) date for June 25, 2021.
AbbVie announced positive top-line results from the Phase 3 ADVANCE and MOTIVATE studies, which evaluated the efficacy and safety of Skyrizi (risankizumab) for induction therapy in adult patients with moderate to severe Crohn’s disease (CD). AbbVie and Frontier Medicines, Corp.,
12/29/2023 FDORA, Section 3202 Rare Diseases: GAO to release a report to Congress assessing the policies, practices, and programs of the FDA with respect to the review of applications for approval of drugs and biologics intended to treat rare disease, with a focus on the effectiveness of FDA’s policies and challenges encountered by sponsors.
Published June 27, 2025 By BioPharma Dive staff post share post print email license Daniel Tadevosyan via Getty Images Today, a brief rundown of news involving Biocryst and Peter Marks, as well as updates from UCB and Altimmune that you may have missed. By Delilah Alvarado • May 13, 2025 Keep up with the story.
a), which specifies that IVDs are “those reagents, instruments, and systems intended for use in the diagnosis of disease or other conditions, including a determination of the state of health, in order to cure, mitigate, treat, or prevent disease or its sequelae” and that they are medical devices. IVDs are defined at 21 CFR 809.3(a),
Published June 26, 2025 Ned Pagliarulo Lead Editor post share post print email license An Incyte researcher works in a laboratory. On June 26, 2025, the company announced the replacement of longtime CEO Hervé Hoppenot with Bill Meury. Last year, Jakafi brought in nearly $2.8 billion in sales. Track funding rounds here.
A post-ASCO update on tumor targets DLL3, B7H3 and HER3 July 2025 Paul D Rennert, SugarCone Biotech LLC Introduction: DLL3, B7H3 and HER3 are compelling tumor antigens to target with cancer therapeutics. However, HER3-DXd missed achieving a statistically significant improvement in overall survival, leading to the withdrawal of its U.S.
Food and Drug Administration (FDA) has accepted for review a new supplemental Biologics License Application (sBLA) and granted it priority review status, seeking to update the U.S. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of October 25, 2025. If approved, the updated U.S. Source link
“Ziihera offers a targeted monotherapy with a favorable safety profile and compelling efficacy, representing a crucial advance in the fight against this challenging disease.” May 2025 : China’s National Medical Products Administration (NMPA) issued conditional approval for the same indication. formerly BeiGene, Ltd.)
The Agenda also includes several long-term actions expected to take place in 2024 and 2025 – included as part of FDA’s long-term regulatory agenda. The anticipated date of publication is June 2023, meaning we should see this regulation imminently. Read our analysis of that rule here and here. ]
All products are received in one of two forms: A New Drug Application (or NDA, for pharmaceuticals) or a Biologics License Application (BLA, for biologics). In this document, the agency describes how to design a DAP, which should set enrollment goals using data about the incidence and/or prevalence of a disease in the U.S. population.
flu vaccines Yesterday, FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) weighed in on strains for seasonal influenza vaccines for the Northern hemisphere 2024-2025 season. Vaccines advisory panel endorses move to trivalent formulation for U.S. This means that each hemisphere has its own influenza season.
In the pharmaceutical sector, new technologies could help us not only to treat but probably cure or even prevent diseases,” Baumann said. Last year alone, the company concluded more than 25 collaboration and licensing agreements and acquisitions in its Pharmaceuticals Division. New opportunities are also arising in agriculture.
First approval of a conjugate vaccine that helps protect against 20 serotypes responsible for the majority of invasive pneumococcal disease and pneumonia, 1,2,3,4,5,6,7 including seven responsible for 40% of pneumococcal disease cases and deaths in the U.S. Following today’s FDA approval, the U.S. Jansen, Ph.D.,
WUSF / AgencyIQ November 1 Initial deadline for NDSRIs Under a 2023 guidance document, the FDA has recommended that pharmaceutical companies assess Nitrosamine Drug-Related Substance Impurities for their products by November 1, 2023, with confirmatory testing due by August 1, 2025.
CBER’s Peter Marks offers insights on a new Rare Disease Hub, upcoming accelerated approval guidance This week, PETER MARKS, director of FDA’s Center for Biologics Evaluation and Research (CBER), spoke at several sessions of the Drug Information Association (DIA) annual meeting.
If you’re looking for something specific, we recommend using the search function in your browser (Ctrl-F) to look for keywords of interest. Priority B List.
Most research to date has provided doses in a highly controlled, positive environment, often with a licensed mental health practitioner present to help guide the participant through the experience. funded a planned large psychedelic study for patients with incurable diseases, information also reported by our colleagues at POLITICO.
Modeled after a similar pathway for pharmaceuticals – the Innovative Licensing Access Pathway (ILAP) – the new pilot program intends to ensure end-to-end assistance for developers of innovative devices addressing unmet medical needs. cancer vaccines, cardiovascular disease). Read AgencyIQ’s complete analysis of the IDAP pilot here.]
The earliest ones relied on simple linear regression and attempted to correlate genetic variations with observable traits or disease risks — such as drug metabolization rates or cancer susceptibility. The 2025 genetic network paper fit that bill exactly. 2024 Statistical models of organisms have existed for decades.
27 key programs highlighted, including assets that could potentially contribute revenue by 2025 and others in the 2026-2028 time frame. Major revenue contributions through 2025 anticipated from Oncology, Vaccines, Rare Disease and Inflammation and Immunology. BNT162 mRNA-based Vaccine Program.
Published June 23, 2025 Delilah Alvarado Staff Reporter post share post print email license A micrograph image of influenza A virus. By Ben Fidler and Gwendolyn Wu • Updated June 19, 2025 Retrieved from Vinay Prasad on May 08, 2025 Biotech is guessing how Vinay Prasad might change the FDA. Influenza A virus" [Micrograph].
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