Sentences with phrase «clinical safety and efficacy»

Exemptions under Article 8 (1)(c) based on establishing clinical superiority of a second product over an approved orphan product would be wholly in conflict with the basis on which generic products obtain approval, viz. relying on the reference product's clinical safety and efficacy.

Not exact matches

Actual results and the timing of events could differ materially from those anticipated in the forward - looking statements due to these risks and uncertainties as well as other factors, which include, without limitation: the uncertain timing of, and risks relating to, the executive search process; risks related to the potential failure of eptinezumab to demonstrate safety and efficacy in clinical testing; Alder's ability to conduct clinical trials and studies of eptinezumab sufficient to achieve a positive completion; the availability of data at the expected times; the clinical, therapeutic and commercial value of eptinezumab; risks and uncertainties related to regulatory application, review and approval processes and Alder's compliance with applicable legal and regulatory requirements; risks and uncertainties relating to the manufacture of eptinezumab; Alder's ability to obtain and protect intellectual property rights, and operate without infringing on the intellectual property rights of others; the uncertain timing and level of expenses associated with Alder's development and commercialization activities; the sufficiency of Alder's capital and other resources; market competition; changes in economic and business conditions; and other factors discussed under the caption «Risk Factors» in Alder's Annual Report on Form 10 - K for the fiscal year ended December 31, 2017, which was filed with the Securities and Exchange Commission (SEC) on February 26, 2018, and is available on the SEC's website at www.sec.gov.

These risks and uncertainties include: Gilead's ability to achieve its anticipated full year 2018 financial results; Gilead's ability to sustain growth in revenues for its antiviral and other programs; the risk that private and public payers may be reluctant to provide, or continue to provide, coverage or reimbursement for new products, including Vosevi, Yescarta, Epclusa, Harvoni, Genvoya, Odefsey, Descovy, Biktarvy and Vemlidy ®; austerity measures in European countries that may increase the amount of discount required on Gilead's products; an increase in discounts, chargebacks and rebates due to ongoing contracts and future negotiations with commercial and government payers; a larger than anticipated shift in payer mix to more highly discounted payer segments and geographic regions and decreases in treatment duration; availability of funding for state AIDS Drug Assistance Programs (ADAPs); continued fluctuations in ADAP purchases driven by federal and state grant cycles which may not mirror patient demand and may cause fluctuations in Gilead's earnings; market share and price erosion caused by the introduction of generic versions of Viread and Truvada, an uncertain global macroeconomic environment; and potential amendments to the Affordable Care Act or other government action that could have the effect of lowering prices or reducing the number of insured patients; the possibility of unfavorable results from clinical trials involving investigational compounds; Gilead's ability to initiate clinical trials in its currently anticipated timeframes; the levels of inventory held by wholesalers and retailers which may cause fluctuations in Gilead's earnings; Kite's ability to develop and commercialize cell therapies utilizing the zinc finger nuclease technology platform and realize the benefits of the Sangamo partnership; Gilead's ability to submit new drug applications for new product candidates in the timelines currently anticipated; Gilead's ability to receive regulatory approvals in a timely manner or at all, for new and current products, including Biktarvy; Gilead's ability to successfully commercialize its products, including Biktarvy; the risk that physicians and patients may not see advantages of these products over other therapies and may therefore be reluctant to prescribe the products; Gilead's ability to successfully develop its hematology / oncology and inflammation / respiratory programs; safety and efficacy data from clinical studies may not warrant further development of Gilead's product candidates, including GS - 9620 and Yescarta in combination with Pfizer's utomilumab; Gilead's ability to pay dividends or complete its share repurchase program due to changes in its stock price, corporate or other market conditions; fluctuations in the foreign exchange rate of the U.S. dollar that may cause an unfavorable foreign currency exchange impact on Gilead's future revenues and pre-tax earnings; and other risks identified from time to time in Gilead's reports filed with the U.S. Securities and Exchange Commission (the SEC).

Announced a clinical trial collaboration with Pfizer, Inc. (Pfizer) to evaluate the safety and efficacy of the investigational combination of Yescarta and Pfizer's utomilumab, a fully humanized 4 - 1BB agonist monoclonal antibody, in patients with refractory large B - cell lymphoma.

We do this by conducting clinical trials in which we collect safety and efficacy data about our experimental drugs with the goal of submitting those data to regulatory authorities, like the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), so that these experimental drugs can be approved for use by patients.

A stroke drug invented at The Scripps Research Institute has shown signs of safety and efficacy in a mid-stage clinical trial.

Ultimately, AA147 — or some version of it — would need to undergo clinical trials for safety and efficacy before it could potentially be used to treat patients.

Although gene therapy research has made great strides in recent years, it has yet to be widely deployed, and no CRISPR - edited genes have yet been tested for safety or efficacy in human clinical trials.

«If nanoparticles are carrying a drug to deliver, different uptake could mean different therapeutic efficacy and other important differences, such as safety, in clinical data.»

Clinical trials that specifically test ADAR1 - targeted therapeutics for their safety and efficacy against multiple myeloma are still necessary before this approach could become available to patients.

«The study results elucidate the molecular mechanisms underlying disease progression in multiple sclerosis models, providing a basis for future clinical trials to determine safety and efficacy of these chemical agents in humans with demyelinating disorders,» says Patrizia Casaccia, MD, PhD, Professor of Neuroscience, Genetics and Genomic Sciences at Mount Sinai and senior author of the study.

Following the success of this preliminary safety and feasibility study, more patients are being recruited for a larger clinical trial of the procedure to test the efficacy and durability of the procedure.

The main objective of this study was to verify the safety of the drug and to confirm its efficacy on the clinical manifestations of the disease.

«So we bought the rights to the drug and started working with Professor Gabay to assess its safety and efficacy in a clinical trial,» explains Andrew Sleight, CEO of AB2 Bio Ltd., the Lake Geneva startup that has the World Wide license of the drug.

Three studies presented at the American Epilepsy Society's 69th Annual Meeting in Philadelphia highlight emerging efficacy and safety data of Epidiolex, a pharmaceutical liquid formulation of cannabidiol, which is currently undergoing U.S. Food and Drug Administration (FDA) authorized Phase 3 pivotal clinical trials in the United States and across the globe by GW Pharmaceuticals.

In broad terms, a regulatory writer assists in the production of the clinical documentation required by these national regulatory agencies when assessing the safety and efficacy of drugs.

Despite a lack of rigorous clinical trials to test safety or efficacy, a ban of the treatment by health authorities, and a lack of peer review by the scientific community, the privately funded Stamina Foundation has been using cultured mesenchymal stem cells that have been exposed to putative conditions that favour neuronal differentiation to treat different diseases in severely or terminally ill patients.

With the initial contract of $ 24.9 million over the next 18 months, Mapp Pharmaceutical will manufacture a small amount of the drug for early - stage clinical studies to demonstrate its safety and efficacy in people.

The documentation ranges across the whole spectrum of drug development: Investigators» brochures provide information on all that is currently known about the medicine and so need periodic updating; accurate and concise protocols are required to ensure that trials are performed effectively; clinical trial reports (generally from phase II and III studies) present the information gathered from the trials; higher level documents provide summaries of efficacy and safety data from clinical trial programmes; expert reports provide critical interpretation of the results; and response documents clarify any points that are not clear to the regulatory agencies or provide additional analyses or supporting data for any items of concern.

Previous clinical trials have evaluated the efficacy, safety and tolerability of CIP on human patients.

Researchers conducted a double - blind, randomized, placebo - controlled clinical trial to evaluate the safety and efficacy of pinaverium.

We look forward to future collaboration in additional clinical trials to confirm the safety and efficacy of ravidasvir,» said Dr. Sherine Helmy, CEO, Pharco.

In a phase 1b clinical trial with 21 patients, researchers tested the safety and efficacy of combining the immunotherapy drug pembrolizumab with an oncolytic virus called T - VEC.

The clinical trials will test the safety and efficacy of the procedure.

A clinical trial investigating a treatment for blindness is under way this winter to evaluate the safety and efficacy of replacing diseased eye cells with stem cells.

The authors call for more uniform documentation of the clinical efficacy and safety of fecal microbiota transplantation.

Further research will be needed to adapt the technique for humans, but the approach could offer improved safety and efficacy control for human clinical trials, which are now underway in Canada.

The safety and efficacy of these methods have been well established through long - term clinical outcomes studies in the field, with the nasal lining protecting the brain from infection just as the blood brain barrier has done.

To get through the FDA's demanding approval process, drug companies must demonstrate their product's safety and efficacy in costly randomized clinical trials.

Two new phase III clinical trials investigating the efficacy and safety of bitopertin, a glycine uptake inhibitor considered to be a promising new add - on therapy for treating negative symptoms in schizophrenia, failed to show a benefit of the drug over placebo.

If these preclinical studies are successful, the researchers plan to further develop their CAR T cell therapy and test its safety and efficacy for different types of metastatic cancer in upcoming clinical trials.

A total of 347 patients (155 treated with vitamin E, 192 not treated with vitamin E) were included in the analysis which compared data from three clinical trials that investigated the efficacy and safety of vitamin E as a treatment for NASH: the PIVENS, TONIC and FLINT trials.

Promising outcomes of preliminary studies in patients in Southeast Asia and parts of Western and Central Africa led the World Health Organization to make the recommendation, but safety and efficacy have not yet been proven in large - scale clinical trials.

At Merck Frosst Canada, I have been an integral part of the discovery of a new molecule, the testing of its mechanism of action, the validation of its safety and efficacy in Phase IIb and III clinical trials, the market approval of the resulting product, and the subsequent management of patients and their disease.

It costs millions of dollars to develop drugs and get them tested in animals before they can ever be used in clinical trials for safety and efficacy in humans.

This prospective, single - arm, phase II clinical trial was conducted to evaluate the safety, feasibility, and efficacy of induction chemotherapy followed by SABR in 34 patients who had biopsy - proven PDA.

For very rare diseases such as WAS, multicenter clinical trials are the only effective way of proving the safety and efficacy of gene therapy and having it rapidly approuved and made available to all patients.

On a more pragmatic level, the researchers hope to soon be able to translate the findings into clinical trials to test safety and efficacy in people.

As an aid to clinical decision making, they conducted an in - depth review of the safety and efficacy of proposed treatments for PMDD.

Adam R. Glassman, M.S., of the Jaeb Center for Health Research, Tampa, Fla., and colleagues examined the incremental cost - effectiveness ratios (ICERs) of aflibercept, bevacizumab, and ranibizumab for the treatment of DME with an analysis of efficacy, safety, and resource utilization data at 1 - year follow - up from the Diabetic Retinopathy Clinical Research (DRCR) Network Comparative Effectiveness Trial.

Many factors may cause differences between current expectations and actual results including unexpected safety or efficacy data observed during preclinical or clinical studies, clinical trial site activation or enrollment rates that are lower than expected, changes in expected or existing competition, changes in the regulatory environment, failure of Syndax's collaborators to support or advance collaborations or product candidates and unexpected litigation or other disputes.

If their phase I clinical trial to test the safety and preliminary efficacy of this therapy gains approval, University of Pittsburgh researchers will test the therapy on 10 patients who suffer from chronic ischemic stroke — the most common form, in which clots block blood flow.

Further studies of ALN - TTR02 are needed to examine the clinical efficacy and safety of long - term transthyretin knockdown in patients with familial amyloidotic polyneuropathy.

To address these issues, «the committee recommends that any initial MRT clinical investigations focus on minimizing the future child's exposure to risk while ascertaining the safety and efficacy of the techniques.»

«To preserve patient access to this treatment option, and given the scale that we've reached, demonstrating the safety and efficacy of fecal transplantation through adequate and well - controlled clinical trials is the right thing to do,» explained Carolyn Edelstein, OpenBiome's director of Outreach and Public Affairs, in an op - ed published earlier today with the Fecal Transplant Foundation.

ViaCyte is proceeding to initiate an open - label clinical trial to evaluate the PEC - Direct product candidate for safety and definitive evidence of efficacy.

Dogs will be a crucial non clinical model to evaluate safety and efficacy of new therapeutics.

SEATTLE, WA and DURHAM, NC — The HIV Vaccine Trials Network (HVTN) and the HIV Prevention Trials Network (HPTN) today announced the initiation of HVTN 704 / HPTN 085, also known as Antibody Mediated Prevention (AMP) or the AMP Study, a Phase 2b clinical trial to evaluate the safety and efficacy of VRC01, a broadly neutralizing monoclonal antibody (bnAb).

Finch expects to begin enrolling for a Phase II clinical trial to evaluate the safety and efficacy of FIN - 403 for the prevention of recurrence in recurrent C. difficile patients in the second half of 2017.

Medical professional societies and clinical researchers advocated for the policy based on the available literature on FMT's safety and efficacy and the lack of treatment alternatives for this patient population.