• 2 A clinical trial to test the efficacy of combining the epigenetic cancer drug with pembrolizumab, a humanized antibody that inhibits PD-1, is currently underway. (genengnews.com)
  • As of Sept. 30, 2013, 29 investigational drugs have received Breakthrough Therapy designation. (pharmamanufacturing.com)
  • LEVADEX is an investigational acute drug for migraine that has completed Phase 3 clinical development. (prnewswire.com)
  • NOVATO, Calif.- Ultragenyx Pharmaceutical Inc. , a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, has reported positive interim data on the acute effects of the investigational treatment UX007 (triheptanoin) at the end of the initial 24-week treatment period of the Phase 2 study in long-chain fatty acid oxidation disorder (LC-FAOD) patients. (drugdiscoverynews.com)
  • Congratulations on receiving a Breakthrough Therapy designation for Volasertib, Boehringer Ingelheim's investigational drug for treating acute myeloid leukemia. (pharmamanufacturing.com)
  • Volasertib is under evaluation for the treatment of patients aged 65 or older with previously untreated acute myeloid leukemia (AML), who are ineligible for intensive remission induction therapy. (pharmamanufacturing.com)
  • This research study is a Phase I clinical trial, which tests the safety of an investigational drug and also tries to define the appropriate dose of the investigational drug to use for further studies. (childrenshospital.org)
  • If current drugs or other types of cancer treatment no longer benefit you, you may wish to explore joining a clinical trial. (cancercare.org)
  • It was conducted by the HIV Prevention Trials Network (HPTN), a global clinical trials organization that tests the safety and effectiveness of HIV therapies. (wraltechwire.com)
  • The Investigational Drug Service (IDS) at Roswell Park Comprehensive Cancer Center is responsible for pharmacy support of clinical research studies. (roswellpark.org)
  • Investigational Drug Service (IDS) provides clinical and operational pharmacy related services for investigators at Roswell Park Comprehensive Cancer Center. (roswellpark.org)
  • The Breakthrough Therapy designation for Volasertib is based on Phase II clinical findings observed to date. (pharmamanufacturing.com)
  • A Breakthrough Therapy program is for a drug that treats a serious or life-threatening condition, and for which preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on a clinically significant endpoint(s) over available therapies. (pharmamanufacturing.com)
  • By contrast, a Fast Track program is for a drug that treats a serious/life-threatening condition, but where nonclinical or clinical data demonstrate the potential to address unmet medical need. (pharmamanufacturing.com)
  • The Phase III trial, known as POLO-AML-2 (Clinical Trial Identifier: NCT01721876), is a randomized, double-blind study designed to assess the efficacy and safety of Volasertib in combination with low-dose cytarabine (LDAC), compared to placebo in combination with LDAC, as a potential treatment for patients aged 65 or older with previously untreated AML who are ineligible for intensive remission induction therapy. (pharmamanufacturing.com)
  • According to Rocket Pharmaceuticals, the study will assess the impact of the gene therapy on PKP2 myocardial protein expression, cardiac biomarkers, clinical predictors of life-threatening ventricular arrhythmias, and sudden cardiac death. (empr.com)
  • But Konig stressed that we are still in the early days of clinical trials, and more research is necessary to understand the safety and efficacy of these therapies. (medscape.com)
  • Breakthrough Therapy Designation is intended to expedite development and review timelines when preliminary clinical evidence indicates the drug may demonstrate substantial improvement on one or more clinically significant endpoints over available therapies for serious or life-threatening conditions. (jnj.com)
  • The esketamine Phase 2 clinical trial data presented by Janssen in May 2016 at the Society of Biological Psychiatry 71st Annual Scientific Meeting in Atlanta, Georgia, provided preliminary clinical evidence to support the Breakthrough Therapy Designation for major depressive disorder with imminent risk for suicide. (jnj.com)
  • We are currently conducting clinical trials to further evaluate the clinical benefit of esketamine and look forward to working closely with the FDA throughout the development and review process to bring this important potential new therapy to patients in critical need. (jnj.com)
  • INTRODUCTION: Despite clinical trials, there are still no approved specific therapies or any vaccine against COVID-19. (bvsalud.org)
  • There are a host of challenges in translation of investigational new drug agents from animals into human clinical testing, however. (cdc.gov)
  • Initial articles in this special issue describe common pre-clinical (animal) testing paradigms used to assess potential otoprotective drug agents and design-related factors that impact translation of promising agents into human clinical trials. (cdc.gov)
  • While otoprotective drugs will ultimately be developed for use by specific noise -exposed populations, there has been little effort to develop pre-clinical (animal) models that accurately model exposure hazards across diverse human populations. (cdc.gov)
  • Rhythm Pharmaceuticals Inc. has presented preclinical data on its new drug candidate, RM-718, a next-generation melanocortin MC4 receptor (MC4R) agonist for weekly administration with a potential development path for hypothalamic obesity. (bioworld.com)
  • The FDA's designations are supported by preclinical studies indicating that the gene therapy candidate has the potential to decrease arrhythmias and increase survival. (empr.com)
  • I think the future of oncology, in terms of treating solid tumors , will be these combination therapies," Dr. Hermann says. (genengnews.com)
  • We characterized the proportion of applicable US biopharmaceutical companies, with an oncology related drug, implementing Cures Act requirements for expanded access policies and whether available policies contain the information described in the Act. (bvsalud.org)
  • Our results suggest the Cures Act may be having a limited impact on its goals of supporting timely medical decisions and closing informational gaps for patients and doctors around expanded access to investigational oncology therapies, especially for products sponsored by smaller and newer companies. (bvsalud.org)
  • Investigational trials of anticancer drugs: establishing safeguards for experimentation. (cdc.gov)
  • It belongs to a group of HIV drugs called integrase inhibitors that block the HIV enzyme integrase. (wraltechwire.com)
  • Volasertib is an investigational inhibitor of polo-like kinase, an enzyme in the Plk family that regulates cell division. (pharmamanufacturing.com)
  • This therapy may be used if an illness has no vaccine or treatment. (mayoclinic.org)
  • Pfizer Inc. (NYSE:PFE) today announced that the U.S. Food and Drug Administration (FDA) accepted for priority review a Biologics License Application (BLA) for its 20-valent pneumococcal conjugate vaccine (20vPnC) candidate, as submitted for the prevention of invasive disease and pneumonia caused by Streptococcus pneumoniae serotypes in the vaccine in adults ages 18 years and older. (pfizer.com)
  • Immunosuppressive drugs may reduce the immune response to influenza vaccine. (medscape.com)
  • Stamford, Conn. - April 24, 2019 - Purdue Pharma L.P. today announced that the U.S. Food & Drug Administration ( FDA )'s Office of Generic Drugs has granted Competitive Generic Therapy ( CGT ) designation to nalmefene hydrochloride (HCl) injection, the company's investigational opioid antagonist for the emergency treatment of known or suspected opioid overdose. (purduepharma.com)
  • The FDA 's CGT designation allows for expedited review of generic versions of medications with inadequate generic competition, and is intended to help facilitate the timely market entry of generic drugs. (purduepharma.com)
  • Cara Pardon discusses Breakthrough Therapy Designation. (fda.gov)
  • Breakdown for us please how the Breakthrough Therapy designation was realized. (pharmamanufacturing.com)
  • At this time, Boehringer Ingelheim is unaware of any other Breakthrough Therapy designation in this AML treatment setting. (pharmamanufacturing.com)
  • The innovation provisions of FDA's Safety and Innovation Act (FDASIA) and Breakthrough Therapy designation allow the FDA to work collectively with pharma companies to expedite the development, review and availability of important new treatments. (pharmamanufacturing.com)
  • The designation status has begun to change the way the FDA and the industry prioritize drug development, but understanding the significance of its impact on timing and influence in the health system will take more time. (pharmamanufacturing.com)
  • Breakthrough Therapy and Fast Track designation programs are similar because they both are intended to expedite the development and review of drugs for serious or life-threatening conditions. (pharmamanufacturing.com)
  • Traditionally, drug reviews occur through a series of scheduled meetings, but with the Breakthrough Therapy designation the company and FDA speak more frequently. (pharmamanufacturing.com)
  • TITUSVILLE, N.J., Aug. 16, 2016 - Janssen Research & Development, LLC, one of the Janssen Pharmaceutical Companies of Johnson & Johnson, announced today that the U.S. Food and Drug Administration (FDA) has granted a Breakthrough Therapy Designation for esketamine, an investigational antidepressant medication, for the indication of major depressive disorder with imminent risk for suicide. (jnj.com)
  • This also marks the second time esketamine has received a Breakthrough Therapy Designation from the U.S. regulatory authority. (jnj.com)
  • More than 50 drugs of different types are now being used singularly or in combination to treat blood cancers. (lls.org)
  • Some blood cancers are treated effectively with a single drug. (lls.org)
  • Chimeric antigen receptor (CAR) T-cell therapy has offered a novel approach to treating hematologic cancers since 2017, but there are early signs that these cellular immunotherapies could be repurposed for B-cell mediated autoimmune diseases. (medscape.com)
  • June and his research team led the development of CAR T-cell therapies for blood cancers. (medscape.com)
  • B-cell targeted therapies have been around since the early 2000s with drugs like rituximab , a monoclonal antibody medication that targets CD20, an antigen expressed on the surface of B cells. (medscape.com)
  • The US Food and Drug Administration (FDA) expanded access pathway allows patients with life-threatening or serious conditions to access investigational drugs outside of trials, under certain conditions. (bvsalud.org)
  • In the past decade alone, new drugs and new uses for existing drugs have greatly improved rates of cure or remission for patients of all ages. (lls.org)
  • In September of last year, researchers in Germany reported that five patients with refractory systemic lupus erythematosus (SLE) treated with CAR T-cell therapy all achieved drug-free remission. (medscape.com)
  • That helps to eradicate the cells that are at the root of the leukemia, something conventional therapies can't do, Dr. Lee says. (genengnews.com)
  • However, many conventional and investigational drug therapies combine drugs that attack cancer cells at different points in their growth cycles. (lls.org)
  • This release discusses investigational uses of an agent in development and is not intended to convey conclusions about efficacy or safety. (purduepharma.com)
  • This research study is evaluating the safety, tolerability and preliminary efficacy of the drugs marizomib and panobinostat in pediatric patients with diffuse intrinsic pontine glioma (DIPG). (childrenshospital.org)
  • The only option available is using investigational drugs for compassionate use. (bvsalud.org)
  • AIM: The present short communication aimed to highlight the need for early and expanded access to investigational drugs for compassionate use as well as a call for an update of the existing regulation in Bulgaria concerning compassionate use in the era of COVID-19. (bvsalud.org)
  • CONCLUSION: The global pandemic mandates Bulgarian Drug Agency for a reasonable update of the existing national regulation concerning compassionate use and off-label therapies. (bvsalud.org)
  • In the era of COVID-19, it is important for Bulgarian patients to have early and expanded access to investigational drugs for compassionate use. (bvsalud.org)
  • Treatment of monkeypox with tecovirimat as a first-line agent is available through CDC for compassionate use through an investigational drug protocol. (cdc.gov)
  • The use of olfactory progenitors as a cell-based therapy for Parkinson's disease (PD) would allow harvest without invasive surgery, provide an autologous cell population, eliminate need for immunosuppression and avoid the ethical concerns associated with embryonic tissues. (scirp.org)
  • Candace Jarvis provides a description of the timelines and requirements for requesting a meeting with CBER's Office of Tissues and Advanced Therapies consistent with the agreements set in place in the PDUFA V negotiations. (fda.gov)
  • Both are effective at depleting B cells in blood, but these engineered CD19-targeted T cells can reach B cells sitting in tissues in a way that antibody therapies cannot, Konig explained. (medscape.com)
  • The Food and Drug Administration (FDA) has granted Fast Track and Orphan Drug designations to RP-A601 for the treatment of plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM), an inherited heart disease caused by mutations in the PKP2 gene. (empr.com)
  • Rocket Pharmaceuticals receives FDA Fast Track and Orphan Drug designations for RP-A601 gene therapy for PKP2 arrhythmogenic cardiomyopathy (ACM). (empr.com)
  • Since then, two biotechnology companies - Cabaletta Bio in Philadelphia and Kyverna Therapeutics in Emeryville, California - have already been granted fast-track designations from the US Food and Drug Administration for CAR T-cell therapy for SLE and lupus nephritis . (medscape.com)
  • The talks highlighted a promising trend-epigenetic modification could be the next wave in cancer therapy, especially when exploited in tandem with the administration of other medications, 4SC AG's Chief Development Officer Frank Hermann, M.D., told GEN . (genengnews.com)
  • Celltrion Healthcare is committed to delivering innovative and affordable medications to promote patients' access to advanced therapies. (pipelinereview.com)
  • There is no guarantee that nalmefene HCl injection, an investigational agent, will successfully complete development or gain FDA approval. (purduepharma.com)
  • Voyager Therapeutics Inc. has selected a lead development candidate for its superoxide dismutase 1 (SOD1)-mutated amyotrophic lateral sclerosis (ALS) gene therapy program. (bioworld.com)
  • Google's Scott Penberthy joins the podcast for a visionary discussion that scans the horizon for startling changes artificial intelligence will bring to drug development in the relatively near future. (bioworld.com)
  • This report provides comprehensive information on the therapeutic development for Nausea, complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. (prnewswire.com)
  • and aceneuramic acid, an investigational therapy in development for GNE Myopathy, from Nobelpharma, AAIPharma and the HIBM Research Group. (drugdiscoverynews.com)
  • Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency. (drugdiscoverynews.com)
  • We have accelerated development of our inhaled COVID-19 antibody cocktail therapy, leveraging our antibody platform to address the ongoing challenges of the pandemic" said Dr. HoUng Kim, Ph.D., Head of Medical and Marketing Division at Celltrion Healthcare. (pipelinereview.com)
  • Esketamine for intranasal administration is an investigational compound being studied by Janssen as part of a global development program. (jnj.com)
  • Substrate reduction therapy offers an approach to treatment of certain metabolic disorders, especially glycogen storage diseases and lysosomal storage disorders. (wikipedia.org)
  • If a safe pharmacological treatment can be developed, one that causes the increased expression of lysosomal sialidase in neurons, a new form of therapy, essentially curing the disease, could be on the horizon. (wikipedia.org)
  • Metabolic therapies under investigation for Late-Onset TSD include treatment with the drug OGT 918 (Zavesca). (wikipedia.org)
  • If you have COVID-19 and your immune system is weakened by treatment or disease, convalescent plasma therapy may be an option. (mayoclinic.org)
  • Marizomib has not been approved by the U.S. Food and Drug Administration (FDA) as a treatment for any disease. (childrenshospital.org)
  • Panobinostat has not approved by the U.S. Food and Drug Administration (FDA) for the treatment of diffuse intrinsic pontine glioma but it has been approved for other uses. (childrenshospital.org)
  • Patients must have received their last fraction of radiation therapy at least 2 weeks prior to treatment initiation. (childrenshospital.org)
  • Founded in 2010, Ultragenyx has rapidly built a diverse portfolio of product candidates with the potential to address diseases for which the unmet medical need is high, the biology for treatment is clear and for which there are no approved therapies. (drugdiscoverynews.com)
  • Prior to initiating treatment with UX007, 27 of the 29 patients were on the standard-of-care MCT oil therapy. (drugdiscoverynews.com)
  • RP-A601 is an adeno-associated virus (AAV.rh74)-based gene therapy that is being investigated as a one-time curative treatment for these patients. (empr.com)
  • Schistosoma haematobium or S. mansoni and large-scale control efforts include providing regular treatment to at-risk groups and supporting drug delivery through schools. (who.int)
  • Risk of additive immune system effects with etrasimod has not been studied in combination with antineoplastic, immune-modulating, or noncorticosteroid immunosuppressive therapies. (medscape.com)
  • Active, chronic disease of the immune system (including stable disease treated with immune therapy (e.g. (who.int)
  • At the 15th annual Discovery on Target conference held in Boston, MA in September 2017, scientists described developments in designing epigenetic cancer drugs. (genengnews.com)
  • Together, the drugs appear to prevent cancer cells from evading detection. (genengnews.com)
  • Trinity, Florida (March 22, 2016) - Florida Cancer Affiliates ( FCA ) is helping to pioneer a new tool aimed to protect prostate cancer patients from the negative effects of radiation therapy. (mckesson.com)
  • Recently a patient, one of the first in Florida, was injected at FCA with SpaceOAR® hydrogel , the first FDA cleared spacing device to protect the rectum in men undergoing radiation therapy for prostate cancer. (mckesson.com)
  • Men facing prostate cancer may have some difficult decisions to make, but utilizing SpaceOAR hydrogel during radiation therapy should not be one of them. (mckesson.com)
  • Drug therapy can speed up cancer cell death. (lls.org)
  • This approach often makes therapy more effective and reduces the chance that the cancer cells will become resistant to a particular drug. (lls.org)
  • Sometimes cancer cells may be resistant to the initial drugs used or can become resistant to the drugs after a period. (lls.org)
  • [ 6 ] These patients may benefit from standard allergic rhinitis medical therapy, and early data suggest they may also benefit from immunotherapy. (medscape.com)
  • Are Cellular Therapies the Future of Autoimmune Disease? (medscape.com)
  • But this is only the tip of the iceberg regarding cellular therapies for autoimmune disease, said Max Konig, MD, PhD , an assistant professor of medicine in the division of rheumatology at Johns Hopkins University School of Medicine in Baltimore. (medscape.com)
  • Live-attenuated vaccines should be avoided for at least 3mo after cessation of immunosuppressive therapy. (medscape.com)
  • A few days before these modified T cells are infused back into the patient, the patients are given a low-dose chemotherapy (lymphodepletion) regimen to help increase the effectiveness of the therapy. (medscape.com)
  • Effective prevention of NIHL with drug agents would reduce the prevalence of NIHL. (cdc.gov)
  • Newer "targeted therapies" and "risk-adapted therapies" have resulted in higher overall response rates and decreased side effects. (lls.org)
  • Given the poor prognosis of all patients with DIPG, patients may enroll at any point in their disease course provided they have received standard radiation therapy (also defined below) and meet all other eligibility requirements. (childrenshospital.org)
  • Patients who receive standard radiation therapy with concurrent chemotherapy may be eligible as long as other criteria are met. (childrenshospital.org)
  • The study began with a four-week run-in period to assess baseline data while on the standard of care therapy including medium-chain triglyceride (MCT) oil, if applicable. (drugdiscoverynews.com)
  • MAPP ) today announced that the Company will present new data from two safety studies of LEVADEX® orally inhaled migraine drug. (prnewswire.com)
  • The study showed CT-P63 to be safe and well tolerated, with no significant drug-related adverse events (AEs). (pipelinereview.com)
  • Therapy for allergic rhinitis is focused on desensitization and use of decongestants, antihistamines, and mast-cell mediators. (medscape.com)
  • It is NOT recommended for use in combination with other JAK inhibitors, biologic disease-modifying antirheumatic drugs (DMARDs), or with potent immunosuppressants such as azathioprine and cyclosporine. (drugs.com)
  • Standard radiation therapy is defined, for this study, as 54-60 Gy standard focal (photon or proton) radiation therapy, administered over 6 weeks (+/- 10 days). (childrenshospital.org)
  • By manipulating the brain's metabolism of GM2 gangliosides, an effective therapy could potentially be developed. (wikipedia.org)
  • If the generated blood ROS concentration is too low, then fungi, bacteria or fibrin might threaten the life of the patient, and it could be of great medical interest to stimulate PMN by physiologic drugs. (scirp.org)
  • Additional early-stage research and drug discovery news in brief. (bioworld.com)
  • Like the developers of LSD1 combination therapies, 4SC AG researchers are pairing an epigenetic drug, in this case an HDAC inhibitor, with immunotherapies. (genengnews.com)
  • The Prescription Drug User Fee Act (PDUFA) goal date for a decision by the FDA on the 20vPnC application is in June 2021. (pfizer.com)
  • FCA has a long history of bringing new technological advances for radiation therapy to the local communities in Florida. (mckesson.com)
  • Because of the close proximity of the prostate to the rectum, prostate radiation therapy typically results in some radiation hitting the rectum, which can sometimes cause side effects. (mckesson.com)
  • The hydrogel spacer maintains this space until radiation therapy is complete. (mckesson.com)
  • The U.S. Food and Drug Administration gave emergency authorization for COVID-19 convalescent plasma to be used, and it is still an option for people who have weakened immune systems. (mayoclinic.org)
  • The 21st Century Cures Act ("Cures Act") requires certain drug companies to publicly disclose their expanded access policies. (bvsalud.org)
  • Miglustat and eliglustat are substrate reduction therapies for Gaucher's disease, inhibiting the synthesis of glucosylceramide. (wikipedia.org)
  • In 2020, doctors used convalescent plasma therapy to treat coronavirus disease 2019 (COVID-19). (mayoclinic.org)
  • Callaway, E. (2011) Gene therapy offers hope for Parkinson's disease. (scirp.org)
  • The type of drug your doctor chooses to treat you can depend on your age, the type and stage of disease, your response to previous treatments and other factors. (lls.org)
  • Quality of life is assessed at baseline, every 4 weeks while on therapy, and then every 8 weeks until disease progression. (knowcancer.com)