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Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.
For example, transcriptomic processes are showing the potential to identify and track failures in gene expression and gene regulation of amyloid and tau-related biomarkers, understood as precursors to the onset of Alzheimers disease (AD). A biomarker is a measurable indicator of a biological process, disease state, or response to a treatment.
BIIB129 (25) demonstrated efficacy in disease-relevant preclinical in vivo models of B cell proliferation in the CNS, exhibits a favorable safety profile suitable for clinicaldevelopment as an immunomodulating therapy for MS, and has a low projected total human daily dose.
They offer patient-specific outcome predictions, generated using machine learning models trained on real historical clinical data. Unlearn’s early collaboration with regulators has helped it navigate this space effectively. Integration is straightforward,” Herne notes.
Neuropsychiatric treatment is on the verge of a major transformation. Neuropsychiatric disorders, affecting millions worldwide, disrupt the brain’s intricate processes of mood regulation, cognition and behaviour. “Zelquistinel is a positive modulator of NMDA receptors,” Donello explains.
Originally developed in the 1970s to treat diabetes, these drugs—such as Ozempic, Wegovy, and Mounjaro—have become headline-makers for their ability to induce significant weight loss. Understanding GLP-1 drugs GLP-1, or glucagon-like peptide-1 receptor modulators, mimic natural hormones that regulate insulin and appetite.
However, the FDA’s recent decision to remove the Risk Evaluation and Mitigation Strategy (REMS) requirements for certain approved Chimeric Antigen Receptor T-cell (CAR T) therapies could dramatically shift these numbers, making life-saving treatments more accessible to those in need.
Additionally, treatment led to improvements in height-adjusted total kidney volume (htTKV) , an established clinical marker that correlates with disease progression in ADPKD. These data were viewed as both mechanistically and clinically meaningful , laying the groundwork for future mid- and late-stage development.
The results come from a open-label, proof-of-concept, Phase 2 study (NCT04520451) and highlight rilzabrutinib’s potential as a disease-changing treatment option for a condition that currently has limited and non-specific treatment options and involves substantial patient suffering due to its chronic and progressive course.
Developingtreatments for individuals living with rare diseases is critical, but orphan drug development is laden with unique obstacles that necessitate innovative, multifaceted approaches. These conditions pose substantial challenges for patients, families and health care systems.
By integrating AI into the manufacturing process, the developer achieved higher consistency across batches, improved overall product quality, and reduced time-to-market – all critical factors in ensuring patients receive timely and effective treatment.
As the biotech sector races to improve the tolerability of these revolutionary treatments, Poolbeg Pharma has a novel solution: an oral therapy, POLB 001, to block the development of CRS, by limiting inflammation without affecting the anti-cancer immune responses that are vital for effective immunotherapies.
In China, the high incidence of solid tumors is driving an urgent need for advanced therapies, spurring the push for new treatment approaches. Common challenges for certain patient groups include long-distance travel to clinical sites, time away from work and limited access to knowledge about CGT trials.
The study mentions the overlap in genes associated with Fatigue Dominant long COVID and ME/CFS, including those involved in circadian rhythm regulation and insulin regulation. The article mentions that TLR4 antagonists have been identified as potential candidates for repurposing long COVID treatment.
Strategic Expansion with INZ-701 The centerpiece of this acquisition is INZ-701, an investigational enzyme replacement therapy currently in advanced clinicaldevelopment for the treatment of ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) Deficiency.
Drug development faces significant challenges: long timelines, high costs , complex processes and low probabilities of success (PoS), exacerbated by the shift towards more complex molecules, biologics and cell and gene therapies, hindering patient access to vital treatments. Nature Medicine, 28, 1656–1661 (2022) [link]
Sandoz, a Novartis division, today announced progress in the late-stage clinicaldevelopment program for its proposed biosimilar aflibercept. Sandoz will begin enrolling the first patient in MYLIGHT, a clinical Phase III confirmatory efficacy and safety study, shortly 1. Aflibercept is a key treatment in ophthalmology.
– If Approved, Investigational TAK-994 May Provide a Future Treatment Option Targeting the Orexin Deficiency Underlying NT1. TAK-994 is currently being studied for the treatment of excessive daytime sleepiness (EDS) in patients with narcolepsy type 1 (NT1), 2 a chronic neurological disorder that alters the sleep-wake cycle.
The data, presented at the annual European Alliance of Associations for Rheumatology (EULAR 2025) congress in Barcelona, Spain, show that dapirolizumab pegol resulted in significant and clinically meaningful improvements in disease activity while addressing a major symptom that profoundly impacts patients’ daily lives — fatigue.
Additionally, advanced data mining capabilities enable the extraction of real-world evidence and patient stratification, providing deeper insights into patient populations and treatment outcomes. In regulated domains, every step must be documented, auditable and reproducible to meet stringent regulatory requirements.
We will explore how GDF-15 impacts the tumour microenvironment and hinders the infiltration of T cells into the tumour, as well as the implications of neutralising GDF-15 to reverse its inhibitory effects and sensitise tumours to anti-PD-1 treatment. T-cell infiltration is a prerequisite for responses to checkpoint inhibitors.
Additionally, they identified overlaps between the genes associated with fatigue-dominant long COVID and ME/CFS, especially those involved in circadian rhythm regulation and insulin regulation. PrecisionLife’s mission is to find better treatment options for patients with unmet medical needs, particularly in complex chronic diseases.
In September 2021, Wegovy was approved by the UK Medicines and Health products Regulations Agency (MHRA). mg injection is approved for the treatment of adults with obesity or overweight in the US and UK, as an adjunct to diet and exercise. Wegovy was launched in the US in June 2021 following approval by the U.S.
SRP-001 targets the central nervous system (CNS) by producing N-arachidonoylaminophenol (AM404) in the midbrain’s periaqueductal grey (PAG) region, crucial for pain sensation and regulation. He is spearheading the scientific and clinicaldevelopment and fundraising of South Rampart Pharma and technology development efforts.
So far, GDF-15 has mainly been known as an important factor in feto-maternal tolerance and a regulator of nausea, anorexia, and food aversion in different diseases, including cancer and the pregnancy complication hyperemesis gravidarum. Visugromab is a monoclonal antibody – a treatment modality that has been extensively studied and de-risked.
The companies expect to complete submission of the NDA for treatment of MDD in the second half of 2022; associated filing for postpartum depression anticipated in the first half of 2023. Food and Drug Administration (FDA) for zuranolone in the treatment of major depressive disorder (MDD). Sage Therapeutics, Inc.
In this IO arena, we continue to search for additional treatments that can further benefit patients. Immune checkpoint inhibitors, a well-established class of IO treatments, are designed to improve the ability of T-cells to fight cancer by removing inhibitory or suppressive mechanisms that may dampen the anti-cancer functions of T cells.
Holzkirchen, May 3, 2021 – Sandoz, a Novartis division, today announced progress in the late-stage clinicaldevelopment program for its proposed biosimilar aflibercept. Sandoz will begin enrolling the first patient in MYLIGHT, a clinical Phase III confirmatory efficacy and safety study, shortly 1.
Reaching the end goal of delivering transformational new treatment options for patients can take a decade or more even when companies execute successfully and are well capitalized. It could also encourage more investment in the space and make us better partners to regulators and payors alike.
The clinical trials sector prompted a recent modernization of ICH E8 and a subsequent renovation of ICH E6 to provide updated guidance that is appropriate and flexible enough to address the increasing diversity of clinical trial designs and data sources employed to support regulatory and other health policy decisions.
With radiopharmaceuticals emerging as a diagnostic and therapeutic (theranostics) procedure, many are in the clinicaldevelopment pipeline and are expected to play a crucial role in the future of healthcare. The goal is to deliver a precise radiation dose to the diseased tissues while minimizing damage to surrounding healthy tissue.
(Janssen) has announced it received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommending the expanded use of Tremfya (R) (guselkumab) for the treatment of adult patients with active psoriatic arthritis (PsA) in the European Union (EU). In July 2020, the U.S.
Food and Drug Administration (FDA) made public a potentially game-changing proposal concerning the regulatory framework for laboratory-developed tests (LDTs). Understanding the nuances and implications of these changes is paramount for specialists in regulatory affairs and the clinicaldevelopment arena.
Xolair for self-injection offers healthcare providers and appr opriate patients another administration option for more flexibility in managing their treatment. Roche’s Chief Medical Officer and Head of Global Product Development. today announced that the U.S. indications. indications. with Xolair since its initial approval in 2003.
14, 2021 (GLOBE NEWSWIRE) — Innovation Pharma (Formerly known as Cellceutix) (OTCQB:IPIX) (“the Company”), a clinical stage biopharmaceutical company, is pleased to announce that the U.S. A molecular screening study of 11,552 compounds also supports Brilacidin as a promising novel coronavirus treatment. WAKEFIELD, Mass.,
To spur more frequent engagement between patients, sponsors, and regulators, Richie Kahn and Jenn McNary co-founded Canary Advisors , a patient engagement consultancy that works to better align development programs with patient needs.
(NYSE American:PLX) (TASE:PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx ® plant cell-based protein expression system, and Chiesi Global Rare Diseases , a business unit of Chiesi Farmaceutici S.p.A.,
Tobolowsky & Véronique Li, Senior Medical Device Regulation Expert & David B. Moreover, DMCs are being used in trials of modest size and in the context of increased globalization of medical product development. The 2006 guidance noted that these would be “rare cases” limited to “specific issues of urgent concern.”
As much of the content of this draft guidance for cellular and gene therapy (CGT) products is articulated elsewhere, this document serves as a one-stop shop or Cliffs Notes for the numerous guidance documents now covering CGT product development. Next, sponsors should consider no-treatment control.
In this article, we review two case studies involving the successful use of RWD or RWE in advancing the clinicaldevelopment of treatments for rare diseases. During that meeting, the sponsor presented data by endpoint, which complemented the data package and indicated the benefits of treatment outweighed the risks.
Here we explore the evolution and impact of market exclusivity policies in the EU and US, highlighting their role in fostering innovation and accessibility in rare disease treatment. This was a turning point, igniting a wave of research and development into treatments for rare diseases that had previously been neglected.
[link] Immunity is complex and can be dangerous when exploited clinically, as demonstrated by the lethal administration of TNF, or anti-CD40L antibody (Biogen) or CAR-T cells expressing the CD16 Fc-receptor (Unum), among many other examples. This is where things get interesting due to the complexity of the immune system and its regulation.
Food and Drug Administration (“FDA”) for the treatment of agitation associated with delirium. Treatment choices are limited, and commonly used off-label therapies are not always effective or may result in prolonged, deep sedation. The Company plans to initiate a Phase 2 trial within the next several months. “We
mg, 5 mg, and 10 mg is approved for the treatment of patients with chronic heart failure who are receiving standard treatment for chronic heart failure. Bayer is proud of the approval of vericiguat in Japan and the contribution it has shown in clinical trials.”. Verquvo (vericiguat) 2.5 About Vericiguat Vericiguat 2.5
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