Oct 08, 2019 great news for glioblastoma today i came across these news ref. Lilly receives fda breakthrough therapy designation for. In the phase 2 portion of the braveaa1 study up to week 36, there were no new safety signals with no serious adverse events reported. The breakthrough therapy designation, established in 2012 by the us congress to expedite the development of drugs that show. List of drugs granted breakthrough therapy designation. The investor relations website contains information about acceleron pharma inc. Seattle genetics and astellas receive fda breakthrough. Breakthrough therapy designation for acalabrutinib for the treatment of patients with mantle cell lymphoma mcl who have received at least one prior therapy. Jun 19, 2014 this is easily recognized within the first year that section 902 was introduced, with 94 total requests for breakthrough therapy designation being received by the fda 32 of which were granted. Breakthrough therapy sakigake first nda in the world intent to develop and file nda.
Esketamine receives breakthrough therapy designation from u. People with lifethreatening diseases often need new medicines. Appendix 1 describes the process for breakthrough therapy designation. Cytodyn files for breakthrough therapy designation with. The breakthrough therapy designation, established in 2012 by the us congress to expedite the development of drugs that show promising early clinical evidence of benefit over.
Overview of fda expedited programs with a focus on. The fda breakthrough therapy designation is based on the positive phase 2 results of lilly s adaptive phase 23 study braveaa1, which evaluated treatment with baricitinib versus placebo in adult patients with aa. Breakthrough therapy designation for silver diamine fluoride. Glioblastoma drug gets fda breakthrough therapy designation. Aprea therapeutics receives fda breakthrough therapy. Facilitating expedited development of advanced therapy. Not all products designated as breakthrough therapies ultimately will be shown to have. Expediting drug development the fdas new breakthrough. The breakthrough therapy designation is granted based on the fda criteria, which is all about unique technology providing a benefit thats not seen with therapies on the market.
Acalabrutinib is a highlyselective, potent bruton tyrosine kinase btk inhibitor in development for the treatment of multiple bcell cancers. The breakthrough therapy designation reaffirms that debio 1143 has the potential to offer a significant benefit over the current standard of care. Expedited programs for serious conditions drugs and. Acceleron receives fda breakthrough therapy designation. This breakthrough therapy designation will allow us to maximize the potential of debio 1143 to become an innovative radiochemo enhancing treatment for lascchn patients, commented angela zubel. In this section, we propose criteria for breakthrough therapy designation, apply these criteria to different categories of potential breakthrough therapies, and discuss the process by which fda will make a breakthrough therapy designation. Breakthrough therapy designation for silver diamine. Designation is based upon preliminary clinical evidence of the potential for substantial improvement over existing therapies on one or more clinically significant endpoints. A new drug may be designated as a breakthrough therapy if it is intended to treat a serious or lifethreatening disease and preliminary clinical evidence suggests it provides a substantial improvement over existing therapies. Cytodyn files for breakthrough therapy designation with the.
Fdas breakthrough therapy designation btd program has been, by all accounts, a popular program. This is easily recognized within the first year that section 902 was introduced, with 94 total requests for breakthrough therapy designation being received by the fda 32 of which were granted. Adenoassociated virus vectorbased gene therapy for treatment of patients with confirmed biallelic rpe65 mutationassociated. With the breakthrough therapy designation program adding to the tools that the u. Overview of fda expedited programs with a focus on breakthrough. This is a list of drugs which granted breakthrough therapy designation by years. Fda grants breakthrough therapy designation for debiopharms. Authorized by the fda safety and innovation act in 2012, the breakthrough therapy designation btd program is a development tool that fda uses for transformative therapies that show great promise in early clinical trials, according to baumgartner. Overview of fda expedited programs with a focus on breakthrough therapy miranda raggio, rn, bsn, ma cder breakthrough therapy program manager regulatory affairs team. Feb 19, 2020 the breakthrough therapy designation was granted based on results from the doseescalation cohort and expansion cohort a of the phase 1b 2 trial, ev103 nct03288545, evaluating patients with locally advanced or metastatic urothelial cancer who are unable to receive cisplatinbased chemotherapy treated in the firstline setting with padcev in. Breakthrough therapy designation the authors provide a perspective on the rationale and goals of the designation of breakthrough therapy by the food and drug administration. Drugs may be listed more than once, as breakthrough designation is awarded individually for each indication.
The fda has responded with a new initiative called breakthrough therapy designation that is intended to streamline and speed up the approval of new medicines for serious disease. But the driver behind that is prolonged overall survival and the ability to downstage unresectable advanced pancreatic cancer status. The breakthrough therapy bt designation created an additional regulatory process for the fda to expedite the development and commercial approval. Breakthrough therapy designation for silver diamine fluoride granted by fda o advantage arrest silver diamine fluoride 38% is the first drug to receive breakthrough therapy designation for the arrest of tooth decay o it is the only breakthrough therapy designation ever received for an oral care medicine. On july 9, 2012, the breakthrough therapy designation was signed into law. A breakthrough therapy designation is intended to facilitate and expedite. A breakthrough year for unmet diseases evaluatepharma. Breakthrough therapy designation btd, established by the food and drug. Cmc considerations when a drug development project is. Despite todays current standard of care, highrisk locallyadvanced head and neck cancer remains an area of unmet medical need.
The term drug refers to the combination of two or more drugs if the combination is the subject of the breakthrough therapy designation or request. Developing standards for breakthrough therapy designation in. Preliminary breakthrough therapy designation request btdr. Expedited programs for serious conditionsdrugs and. Regulatory watch impact of breakthrough therapy designation on cancer drug development of 17. Breakthrough therapy designation was established as an additional regulatory tool that would allow the fda and sponsors to be more responsive to the current state of science, establish a more. The breakthrough therapy designation is supported by data from a phase 1, firstinhuman, openlabel, multicenter study nct02609776. Food and drug administration for major depressive disorder with imminent risk for suicide fda action marks second breakthrough therapy designation for intranasal esketamine, highlighting its potential as treatment for patients with major depressive disorder who are at imminent risk. Breakthrough therapy is a united states food and drug administration designation that expedites drug development that was created by congress under section 902 of the 9 july 2012 food and drug administration safety and innovation act.
Breakthrough therapy designation, as well as the processes fda will take to. A microbiota restoration therapy mrt for the treatment. About breakthrough therapies friends of cancer research. Exploring the qualifying criteria park hyatt hotel washington, dc friday, april 24, 2015. Esketamine receives breakthrough therapy designation from. Oncolytic virus therapy is a very relevant approach to address cancer where viruses are converted into therapeutic agents as they are reprogrammed to treat diseases. Use of the accelerated approval pathway was more varied, with threequarters of breakthrough designated drugs approved via accelerated approval 75%, 9 of 12 compared with less than onequarter of nondesignated drugs 24%, 4 of 17. Started in 1995 cms to give fda authorization to categorize medical devices to a category a or b product for purposes of reimbursement. Breakthroughtherapy designation an fda perspective nejm. This breakthrough therapy designation will allow us to maximize the potential of debio 1143 to become an innovative radiochemo enhancing treatment for lascchn patients. Comparison of breakthrough therapy designation and mhra processes. The fdas breakthrough therapy designation is not intended to imply that a drug is actually a breakthrough or that there is highquality evidence of.
Astellas and seattle genetics receive fda breakthrough. Impact of breakthrough therapy designation on cancer drug. Acceleron receives fda breakthrough therapy designation for. The mhra offers a scientific advice service in face to face meetings, which can be requested during any stage of. Roche fda grants breakthrough therapy designation for. Breakthrough therapy designation was established as an additional regulatory tool that would allow the fda and sponsors to be more responsive to the current state of. Developing standards for breakthrough therapy designation.
Food and drug administration grants breakthrough therapy designation for jnj68284528, an investigational bcma cart cell therapy designation based on the ongoing u. The food and drug administration has granted breakthrough therapy designation for the investigational, oncedaily oral janus kinase 1 jak1selective inhibitor upadacitinib abt494 in adult patients with moderate to severe atopic dermatitis who are candidates for systemic therapy. Associate executive director pharmaceuticals and medical devices agency. Breakthrough therapy designation brookings institution. Designation is based upon preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over. The purpose of this guidance for industry is to provide a single resource for information on fdas policies and procedures for these four programs as well as threshold criteria generally. Fda grants breakthrough therapy designation for debiopharm. Breakthrough therapy designation btd, established by the food and. Fda grants breakthrough therapy designation for roches. Food and drug administration fda has for expediting drug development, the fda reassessed the endpoints needed. A request for breakthrough therapy designation should describe what category of breakthrough therapy the investigational agent would fit into by including a summary of the disease and setting the therapy aims to treat, expected outcomes for that patient population, and the existing if applicable therapies available to treat the disease. Breakthrough therapy designation program was initiated in 2012 by the us fda to expedite development of treatments for serious or life threatening illness that demonstrate substantial improvement over existing therapies sponsor and fda collaborate in a dynamic process to ensure smooth progression towards supporting clinical trials. Fda reserves the right to rescind the designation if subsequent evidence demonstrates that the therapy no longer meets the criteria or the program is no longer being pursued. However, the question remains whether btd translates into faster approval not only in the us but to other regulatory agencies, quicker decision from health technology assessments hta and ultimately global availability of medicines.
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