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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.
Rare diseases are defined in the Orphan Drug Act as diseases or conditions that affect less than 200,000 people in the United States. Evaluating and developing treatments for rare diseases remains a key priority for FDA and is incentivized through the Orphan Drug Act.
Metabolism of 2022 FDAapproved small molecule drugs – Part 1 Does CYP3A4 still rule? By Julia Shanu-Wilson It won’t come as much surprise to learn that of the 17 small molecules* approved by the FDA in 2022, CYP3A4 was the major player in drug metabolism.
Metabolism of 2022 FDAapproved small molecule drugs part 2 Mixing it Up By Julia Shanu-Wilson In Part 1 of this topic we looked at metabolism of the small molecule drugsapproved by the FDA in 2022 that were mediated by CYP3A4.
Even though AI-designed drugs arent yet a household term for FDA-approved, commercially available therapies, they are a reality in clinicaldevelopment pipelines.
Food and Drug Administration (FDA) has converted this indication from an accelerated to a full (regular) approval. 10), as determined by an FDA-approved test, or in patients who were not eligible for any platinum-containing chemotherapy regardless of PD-L1 status.
We are at the forefront of drugdevelopment in an area of research called cellular rejuvenation, which is an approach that has the potential to address many diseases of ageing by restoring aged and injured cells to a more youthful and resilient state. What evidence is there that we can reverse aging with drugs?
How have advancements in antibody-drug conjugates (ADCs) expanded treatment options for different types of cancer? The advent of monoclonal antibodies paired with improvements in each ADC design component has led to the approval of 11 ADCs with more than 180 ADCs currently in clinicaldevelopment.
Metabolism of 2023 FDAApproved Small Molecules – PART 1 By Julia Shanu-Wilson 2023 was a fruitful year for drugapprovals by the FDA, with a crop of 34 small molecules out of a total of 55 new drugs [1]. Enzymes involved include CYP1A2, CYP2C8, CYP3A4, CYP4F2, and aldehyde oxidase (AOX).
FDAApproves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1. Food and Drug Administration (FDA) approved Oxlumo (lumasiran) injection for subcutaneous use, the first-ever therapy available for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.
Preliminary blinded data on NVX-CoV2373 in older adults needed to proceed to Phase 3 has previously been positively reviewed by the Food and Drug Administration (FDA). Additional clinical data from the Phase 2 trial conducted in the U.S. NVX-CoV2373 (SARS-CoV-2 vaccine) FDAApproval History. Posted: November 2020.
Food and Drug Administration (“FDA”) has approved the Investigation New Drug (IND) application for TG-1000, a novel treatment for influenza A and B. TAIPEI, Taiwan , Nov. 1, 2020 /PRNewswire/ — TaiGen Biotechnology Company, Limited (“TaiGen”) ( Taiwan : 4157) announced today that U.S.
Pfizer’s PAXLOVID™ receives FDAapproval for adult patients at high risk of progression to severe COVID-19 Pfizer Inc. NYSE: PFE) announced today that the U.S. ” COVID-19 continues to cause significant burden in the U.S. Source link: [link]
OS is a rigorous endpoint that the FDA has recognized as a basis for approval of oncology drugs when a statistically significant improvement can be shown relative to a randomized control arm.
related to a completed Phase 1 clinical trial with Berubicin in malignant brain tumors, which Reata conducted in 2006.
The research collaboration is aimed at identifying novel lead compounds and repurposing existing drugs for rheumatoid arthritis and nonalcoholic steatohepatitis, leveraging Standigm’s AI-powered drug discovery platforms : Standigm BEST , Standigm Insight , and Standigm ASK. Standigm is an AI-driven drug discovery company.
7, 2021 /PRNewswire/ — UNION therapeutics A/S ( UNION ) today announces that the US Food and Drug Administration (FDA) has approved an Investigational New Drug program (IND) for oral orismilast; a next generation PDE4-inhibitor for the treatment of plaque psoriasis in adults.
HELLERUP, Denmark , Jan.
Food and Drug Administration (FDA) has approved a label extension for Evrysdi® (risdiplam) to include babies under two months old with spinal muscular atrophy (SMA). ” Evrysdi is approved in 81 countries and the dossier is under review in a further 27 countries. Food and Drug Administration in 2017.
Five Promising Treatment Areas in Early-Phase DrugDevelopment in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drugdevelopment is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues.
Prescription drugs have the ability to transform a patient’s life and provides them the opportunity to rid a devastating illness, making the development and approval of these medications urgent and necessary. However, over the last 10 years the path leading to drugapproval has become more complicated and expensive.
Food and Drug Administration (FDA) has approved an updated label for ADUHELM (aducanumab-avwa) injection 100 mg/mL solution. Drugdevelopment and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product.
The pharmaceutical industry is under huge pressure to address the high attrition rates in drugdevelopment. With around 90% of candidates failing during clinicaldevelopment, 1 the process is not only long and risky, but also expensive for those involved. When imaged, the hiPSC showed a stable beating rate.
The FDA guidance, Evaluating Drug Effects on the Ability to Operate a Motor Vehicle , indicates that testing in early-phase clinicaldevelopment should emphasize sensitivity over specificity in CNS effects.
. (“Harmony”) (Nasdaq: HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced the U.S. Drug Enforcement Administration. Drug Enforcement Administration.
Food and Drug Administration (FDA) has approved the company’s supplemental Biologics License Application for Xolair® (omalizumab) prefilled syringe for self-injection across all approved U.S. with Xolair since its initial approval in 2003. with Xolair since its initial approval in 2003. indications.
GlaxoSmithKline’s Nucala has been approved by the US Food and Drug Administration (FDA) for the treatment of hypereosinophilic syndrome (HES), making it the first biologic approved in this indication.
Treatment becomes first and only biologic for HES in the US.
(NASDAQ: HARP), a clinical-stage immunotherapy company developing a novel class of T cell engagers, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for HPN217 for the treatment of multiple myeloma. President and Chief Executive of Harpoon Therapeutics. “I
As a first step, Manguso set his sights on treatments called PD-1 checkpoint inhibitors, which at the time had shown tremendous promise in clinical trials and would receive their first FDAapproval in 2014. Along the way, TIDE is showing how careful, systematic science at scale can quickly home in on promising drug targets.
If approved, Actemra/RoActemra would be the first U.S. FDAapproval is expected in the second half of this year. Chief Medical Officer and Head of Global Product Development, Roche. today announced that the U.S. A decision on U.S. Actemra/RoActemra is not U.S.
Several years ago, the Food and Drug Administration (FDA) recommended that drugdevelopers take special care to show that potential drugs to treat diabetes don’t adversely affect the cardiovascular system [1]. In fact, the evidence suggests that such drugs might even offer some protection against heart disease.
The Swiss company’s antibody-drug conjugates are highly potent to deliver the payload only once bound to the tumor cells, giving them a leg up on traditional cancer therapies that can’t distinguish between tumor cells and healthy cells. It has shown remarkably high safety and tolerability measure during non-clinicaldevelopment. .
Metabolism of de novo-designed macrocyclic drugsapproved by the FDA By Julia Shanu-Wilson To date, only 4% (67) of FDAapproveddrugs are macrocycles [1]. Current macrocycles in clinical use generally focus on treatment of infectious diseases, cancer and auto-immune disorders.
Metabolism of de novo-designed macrocyclic drugsapproved by the FDA By Julia Shanu-Wilson To date, only 4% (67) of FDAapproveddrugs are macrocycles [1]. Current macrocycles in clinical use generally focus on treatment of infectious diseases, cancer and auto-immune disorders.
of new cancer drugs tested in Phase I were likely to receive Food and Drug Administration (FDA) approval. Additionally, in 2021, a literature review estimated the average capitalized research and development (R&D) costs per new cancer medicine at between $944 million and $4.54
Clinical trials for ultra-rare diseases can be particularly challenging to mount due to small, geographically-dispersed patient populations. For such trials, the US Food and Drug Administration (FDA) may allow the use of credible real-world data (RWD) and real-world evidence (RWE) in lieu of data collected in a Phase 3 trial.
The Panel has accepted the addition of a permanent Category I CPT procedure code to replace the currently available Category III CPT code (0356T) for the administration of drug-eluting intracanalicular inserts, including DEXTENZA® (dexamethasone ophthalmic insert) 0.4 mg, effective January 1, 2022. About DEXTENZA. Ocular Therapeutix, Inc.
A significant challenge in developing novel therapies is the limited understanding of how the target gene influences the underlying disease biology, which contributes to the considerable attrition observed in investigative therapies. Leveraging genetic evidence to develop new therapeutics has been successful in the past.
Cerecor intends to use the net proceeds of the offering for general corporate purposes and working capital, primarily to support the ongoing clinicaldevelopment of key assets within its pipeline and for general and administrative expenses. The company is also developing two monoclonal antibodies, CERC-002, and CERC-007.
Adagrasib Formula C32H35ClFN7O2 cas 2326521-71-3 Mol weight 604.1174 Antineoplastic Disease Non-small cell lung cancer 2022/12/12 FDAAPPROVED, KRAZATI (Mirati Therapeutics) MRTX-849 MRTX849 KRAS G12C inhibitor MRTX849 Adagrasib , sold under the brand name Krazati , is an anticancer medication used to treat non-small cell lung cancer. [1]
Food and Drug Administration (FDA) has a busy end of November planned, with numerous PDUFA dates to address. The FDAapproved it under the brand name Gavreto on September 4. The drug is a once-daily oral precision therapy designed for highly potent and selective targeting of oncogenic RET alterations.
9, 2020 /PRNewswire/ — United Therapeutics Corporation (Nasdaq: UTHR) announced today that the United States Food and Drug Administration (FDA) has granted orphan drug designation to treprostinil for the treatment of patients with idiopathic pulmonary fibrosis (IPF). SILVER SPRING, Md. and RESEARCH TRIANGLE PARK, N.C. ,
18 December 2020 — The US Food and Drug Administration (FDA) has requested further clarifying analyses of clinical data, to complete its review of the New Drug Application (NDA) for roxadustat, an oral hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor for patients with anemia of chronic kidney disease (CKD).
a biopharmaceutical company engaged in the discovery and development of RNAi therapeutics against cancer and fibrotic diseases, today announced dose administration for the first patient in a Phase 2a clinical study of the company’s lead drug candidate, STP705, for the treatment of cutaneous basal cell carcinoma.
(Nasdaq: NVAX), a late-stage biotechnology company developing next-generation vaccines for serious infectious diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for NVX-CoV2373, the Company’s COVID-19 vaccine candidate. About Fast Track Designation. Source link.
Based on the positive Phase 1 results reported and the unprecedented need for COVID-19 vaccines, Clover and its partners are confident to enter late-stage clinicaldevelopment for both adjuvanted vaccines. The Phase 1 clinical trial was funded by the Coalition for Epidemic Preparedness Innovations (CEPI).
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