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Background on the Grants Program Launched as part of the Orphan Drug Act of 1983, this program aims to encourage research and development of drugs, biologics, medical devices, and medical foods for rare diseases, defined as conditions that affect fewer than 200,000 people in the U.S. Relative to other areas of medicine (e.g.,
Currently Available Pharmacotherapies in the ALS Treatment Armory The battle against ALS has seen the introduction of a limited number of approved pharmacological treatments combined with other essential areas of multidisciplinary care.
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 drugs approved by the FDA in 2022 that were mediated by CYP3A4. Oxidation of adagrasib occurs on the methylpyrrolidine group 4. link] [5] Rahbaek et al.,
Jerry has over 30 years of experience in the biopharmaceutical industry and has been involved in the discovery, clinicaldevelopment, and global commercialisation of more than a dozen FDA-approved drugs with multiple successful exits. What’s next for Life Bioscience’s preclinical studies?
First and only FDA-approved subcutaneous treatment option for anti-aquaporin-4 antibody positive NMOSD that can be self-administered by a person with NMOSD or a caregiver every four weeks. First and only approved therapy for NMOSD designed to target and inhibit interleukin-6 receptor activity, using novel recycling antibody technology.
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.
and Shionogi Limited as shareholders, today announced the positive findings of a pooled analysis of COVID-19-related impacts across the investigational long-acting cabotegravir and rilpivirine clinicaldevelopment programme. These findings were presented today at the 2020 Infectious Diseases Society of America (IDSA) IDWeek.
Metabolism of 2023 FDAApproved Small Molecules – PART 1 By Julia Shanu-Wilson 2023 was a fruitful year for drug approvals by the FDA, with a crop of 34 small molecules out of a total of 55 new drugs [1]. link] [10] FDA prescribing information for gepirone. link] [11] Zheng et al.,
Pfizer’s PAXLOVID™ receives FDAapproval for adult patients at high risk of progression to severe COVID-19 Pfizer Inc. 1) “Great advancements have been made in the fight against COVID-19, yet the virus remains a present and unpredictable concern. NYSE: PFE) announced today that the U.S. More than 11.6
executive vice president of Research and Development at Amgen. “We are pleased to present additional analyses for LUMAKRAS, our newly approved KRAS G12C inhibitor, as well as a trial-in-progress poster for acapatamab, our investigational BiTE molecule being studied in NSCLC and other solid tumors. Reese, M.D., In the U.S.,
Initiation of Phase III clinical trial programme for fenebrutinib, an investigational medicine designed to be a highly selective and reversible Bruton’s tyrosine kinase (BTK) inhibitor, which may offer novel approach to suppress disease activity and slow disease progression in MS.
Results in early stage clinical trials may not be indicative of full results or results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements or the scientific data presented.
The clinical data from FIGARO-DKD are scheduled for presentation at the European Society of Cardiology (ESC) Congress 2021 on August 28th. The cardiology franchise at Bayer already includes a number of products and several other compounds in various stages of preclinical and clinicaldevelopment.
With this FDA blessing, pembrolizumab may address a critical unmet treatment need and has the implicit to come a new standard of care in the adjuvant setting for meetly named cases.”. Merck has the assiduity’s largest immuno-oncology clinical exploration program.
Real-world effectiveness data on Oxbryta ® (voxelotor) in the treatment of sickle cell disease featured as oral presentation. Two abstracts have been accepted for presentation, including real-world effectiveness data of Oxbryta ® (voxelotor) tablets in the treatment of sickle cell disease (SCD). FDAapproval. 26-31, 2020.
WAKIX is the first and only treatment approved by the FDA for people with excessive daytime sleepiness or cataplexy associated with narcolepsy that is not scheduled as a controlled substance by the U.S. WAKIX received FDAapproval for the treatment of excessive daytime sleepiness in adult patients with narcolepsy in August 2019.
His mother had a presentation of the disease that suggested her immune system was already on the job. But immunotherapy was not yet widely used and had not been applied clinically to Merkel cell carcinoma, so she received traditional chemotherapy and radiation therapy, suffering life-threatening complications along the way.
However, acceptance of that data depends on a sponsor’s understanding of the FDA criteria for historical control groups, study data standards, and clinical meaningfulness. 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.
Harpoon has four drug product candidates in clinicaldevelopment for the treatment of solid and hematologic malignancies based on its proprietary TriTAC platform. In April 2020, Harpoon announced dosing of the first patient with HPN217 (BCMA TriTAC) in a Phase 1/2 clinical trial focused on relapsed/refractory multiple myeloma (RRMM).
Additional presentations on investigational programmes, including Alzheimer’s disease and Huntington’s disease, help advance scientific understanding of neurological disorders. Roche will present data from five studies from the EVRYSDI clinicaldevelopment programme, which was designed to represent a broad spectrum of people living with SMA.
Amgen (NASDAQ: AMGN) today announced the presentation of long-term efficacy and safety data from the CodeBreaK 100 Phase 1/2 trial in patients with KRAS G12C-mutated advanced non-small cell lung cancer (NSCLC) who received LUMAKRAS® (sotorasib)*. Longest Follow-Up of Patients Treated With a KRAS G12C Inhibitor.
Oral presentation at the International Association for the Study of Lung Cancer’s (IASLC) 2021 World Conference on Lung Cancer (WCLC) shows evidence that the bispecific mechanism of action for RYBREVANT TM can provide anti-tumor activity against either EGFR-mutated or MET-mutated non-small cell lung cancer. 4] , [5] , [6].
The FDAapproved the first gene therapy in 2017 for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL), and as of December 2023, there are over 30 approved cell and gene therapies on the market in the U.S.A. Asia, and Europe.
Data from this study will be presented at an upcoming scientific congress. Merck has an extensive clinicaldevelopment program in prostate cancer evaluating KEYTRUDA as monotherapy and in combination with LYNPARZA and other anti-cancer therapies. Merck’s Focus on Cancer.
The data will be presented in due course. Results from the CALAVI Phase II trials do not impact approved indications or pending approvals for Calquence in patients with blood cancers. Related Articles: Calquence (acalabrutinib) FDAApproval History. J Hematol Oncol. 2016;9(21).
Source: AstraZeneca .
DEXTENZA is FDAapproved for the treatment of ocular inflammation and pain following ophthalmic surgery. is a biopharmaceutical company focused on the formulation, development, and commercialization of innovative therapies for diseases and conditions of the eye using its proprietary bioresorbable hydrogel-based formulation technology.
The company will complete a full evaluation of the IDHENTIFY data and work with investigators to present detailed results at a future medical meeting. senior vice president, Global ClinicalDevelopment, Hematology, Bristol Myers Squibb. senior vice president, Global ClinicalDevelopment, Hematology, Bristol Myers Squibb.
Eliav Barr, senior vice president, head of global clinicaldevelopment and chief medical officer, Merck Research Laboratories. Of the patients who developed pneumonitis, 54% interrupted KEYTRUDA, 63% discontinued KEYTRUDA, and 71% had resolution. Thyroiditis can present with or without endocrinopathy.
This classification aims to expedite the development and review of drugs that are intended to treat a serious condition when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over already available therapies on a clinically significant endpoint(s).
Strikingly, a strong genetic link between target and disease biology is emerging as a predictor for success in clinical trials. 2,3 This is exemplified by drugs that target disease-specific genes or genetically distinct patient subsets which are more likely to succeed in demonstrating efficacy in clinicaldevelopment.
executive vice president, chief medical officer, Global Drug Development, Bristol Myers Squibb. “Our Summary of Presentations. Presentation. To date, the Opdivo clinicaldevelopment program has treated more than 35,000 patients. All times noted are Central European Summer Time (CEST). Abstract Title.
Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee (ODAC), by a vote of 11 to 1 with one abstention, supported FDAapproval of LYNPARZA plus abiraterone and prednisone or prednisolone (abi/pred) for the first-line treatment of adult patients with BRCA -mutated ( BRCA m) metastatic castration-resistant prostate cancer (mCRPC).
ImmunityBio’s hAd5 vaccine generated robust neutralizing antibody activity that was present 14 days post-final vaccination. Risks and uncertainties related to this endeavor include, but are not limited to, the company’s beliefs regarding the success, cost, and timing of its development activities and clinical trials.
Results from CheckMate -648 were presented in an oral session during the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting and were selected for the official ASCO press program. To date, the Opdivo clinicaldevelopment program has treated more than 35,000 patients. On March 25, 2011, the U.S.
30, 2020 /PRNewswire/ — Eli Lilly and Company (NYSE: LLY) and Incyte (NASDAQ: INCY) announced today new data for baricitinib (marketed as OLUMIANT ® ) will be presented at the annual Fall Clinical Dermatology meeting taking place virtually October 29-November 1, 2020. INDIANAPOLIS , Oct.
Data selected for an oral presentation in a Clinical Trials Plenary Session at the American Association for Cancer Research Annual Meeting 2021. To date, the Opdivo clinicaldevelopment program has treated more than 35,000 patients.
All three doses of tirzepatide achieved superior A1C and weight reductions compared to semaglutide in data simultaneously presented at the American Diabetes Association’s® 81st Scientific Sessions®. Participants in the semaglutide treatment arm started the study at a dose of semaglutide 0.25
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Metabolism of de novo-designed macrocyclic drugs approved by the FDA By Julia Shanu-Wilson To date, only 4% (67) of FDAapproved drugs are macrocycles [1]. Current macrocycles in clinical use generally focus on treatment of infectious diseases, cancer and auto-immune disorders.
Fast Track designation facilitates the development and expedites the review of drugs that are intended to treat serious and life-threatening conditions and show the potential to fill an unmet medical need. Brilacidin for UP/UPS was licensed to Alfasigma S.p.A. in July 2019, who have recently initiated a Phase 1 study with their formulation.
These results are being presented today during a European Society for Medical Oncology (ESMO) Virtual Plenary. 0.82]; p=0.00031) versus the chemotherapy-placebo regimen – a statistically significant and clinically meaningful EFS result. KEYTRUDA is currently approved under accelerated approval in the U.S.
Results from EMPEROR-Preserved were presented at the European Society of Cardiology Congress 2021 on August 27 and published in The New England Journal of Medicine. vice president, ClinicalDevelopment & Medical Affairs, Cardio-Metabolism & Respiratory Medicine, Boehringer Ingelheim Pharmaceuticals, Inc.
Launch activities continue following the full FDAapproval for second-line metastatic triple-negative breast cancer (“mTNBC”) and accelerated approval for metastatic urothelial cancer. Full data will be presented later this year and discussions are underway with global regulatory authorities. Viral Diseases.
. “We look forward to providing an important new medicine and helping patients find the relief they so desperately seek from the varied and debilitating symptoms of this disease, contingent upon FDAapproval.” Almirall also plans to submit these results this year to the European Medicines Agency (EMA) for authorization.
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