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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.,
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?
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.,
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.
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.
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.
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.
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.
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
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.
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.
. “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.
Until recently, the FDA relied on a monograph process through which firms could bring OTC drugs to market without FDAapproval so long as it adhered to pre-set terms under the monograph. FDA’sclinical pharmacology analysis challenged previous in vitro estimates of bioavailability for orally administered phenylephrine.
The data were presented at the 2021 American Association for Cancer Research (AACR) Annual Meeting, held virtually April 10-15, 2021. FDA-approved oral prescription medicine, 120 mg or 160 mg dependent on weight (<50 kg or ?50 chief medical officer, oncology at Lilly. Retevmo is an U.S. About Lilly Oncology.
As a reminder, there currently is no FDAapproval pathway to develop a generic or biosimilar copy of a cell therapy product, which provides a significant barrier-to-entry from generic competition. An additional objective this year will be to build greater awareness for our past achievements and our future plans.
The results were shared in an oral presentation at the virtual 2020 American Society of Hematology (ASH) Annual Meeting. REGN5458 and odronextamab are currently under clinicaldevelopment, and their safety and efficacy have not been evaluated by any regulatory authority.
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.
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.,
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.
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.
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.
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.
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.
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.
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.
Basel, 10 September 2020 – Roche (SIX: RO, ROG; OTCQX: RHHBY) will present new ENSPRYNG® (satralizumab) data on reducing relapse severity in the treatment of neuromyelitis optica spectrum disorder (NMOSD), a rare disease of the central nervous system. Roche’s Chief Medical Officer and Head of Global Product Development.
FDA accepted for priority review Libtayo ® (cemiplimab-rwlc) for both advanced non-small cell lung cancer and basal cell carcinoma. FDAapproved Inmazeb for Ebola ( Zaire ebolavirus). Updates from the clinical pipeline include: Dupixent ® (dupilumab). Regeneron Pharmaceuticals, Inc. Business Highlights.
Orphan drug designation is the first step in receiving orphan drug exclusivity following approval, which confers seven years of market exclusivity for the relevant indication. Chairman and Chief Executive Officer of United Therapeutics.
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).
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.
Food and Drug Administration (FDA) has granted Orphan Drug and Rare Pediatric Disease (RPD) designations to PBKR03 for the treatment of Krabbe disease (Globoid Cell Leukodystrophy). Passage Bio expects to initiate a Phase 1/2 trial for PBKR03 in the first half of 2021. president and chief executive officer of Passage Bio.
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.
Opdivo ’s leading global development program is based on Bristol Myers Squibb’s scientific expertise in the field of Immuno-Oncology, and includes a broad range of clinical trials across all phases, including Phase 3, in a variety of tumor types. To date, the Opdivo clinicaldevelopment program has treated more than 35,000 patients.
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.
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