Breakthrough therapy designation btd, established by the food and drug administration safety and innovation act fdasia of 2012, is one of several programs available to the u. Robert yarchoan, of the hiv and aids malignancy branch within the center for cancer research of the national cancer institutes nci. Clinical trial evidence supporting fda approval of drugs granted. Breakthrough drug approval process and postmarketing adr. Sponsors may apply for and receive more than one designation for a given product, but sponsors should apply for each designation separately. Clovis oncology receives breakthrough therapy designation. The criteria for breakthrough therapy designation require preliminary clinical evidence. This prized designation comes with significant oversight, communication and operational demands. Use of the accelerated approval pathway was more varied, with threequarters of breakthroughdesignated drugs approved via accelerated approval 75%, 9 of 12 compared with less than onequarter of nondesignated drugs 24%, 4 of 17. Fdas guidance documents, including this guidance, do not establish legally enforceable. 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. These 2 companies are the biggest winners of the fdas. The breakthrough therapy designation is based on data from the galahad study, a phase 2, multicenter, openlabel clinical trial evaluating the efficacy and safety of. Request pdf expediting drug development the fdas new breakthrough therapy designation the fdas new breakthrough therapy designation for.
Expedited programs for serious conditions drugs and. Breakthrough therapy designation btd, established by the food and drug administration safety and innovation act fdasia of 2012, is one of several programs available to. Breakthroughtherapy designation the authors provide a perspective on the rationale and goals of the designation of breakthrough therapy by the food and drug administration. First quarter, 2020 coding cycle for drug and biological. Ibrutinib receives two oncology breakthrough therapy. Next, we identified 3 regulatory dates using public fda documents and patent extension notices. Imaging was used as a surrogate endpoint in 50% of all breakthrough therapy approvals.
Breakthrough therapy designation brookings institution. Breakthrough therapy designation btd, established by the food and drug. Expedited approvals and fast track designation explained. Tk2 deficiency is a type of myopathy caused by a reduction of mitochondrial dna. Drugsdevelopmentapprovalprocessdruginnovationucm430299. If you decide to request preliminary advice, provide the information below, summarized in 1. The recommended dose of adakveo is 5 mgkg of actual body weight administered by intravenous infusion over a period of 30 minutes at week 0, week 2 and every 4 weeks thereafter. Designation system, intended to facilitate rapid commercialization of pharmaceuticals, medical devices, and regenerative medicine products for breakthrough therapies that addressed an unmet medical need and promise excellent efficacy on the basis of clinical trial data. As an oncology product, ibrutinib receiving the breakthrough therapy designation is an example of progress and hope for patients fighting a range of cancers. Previously, breakthrough therapy designation and orphan drug designation were granted. Breakthrough therapy designation for acalabrutinib will help us bring this potential new medicine to appropriate patients as quickly as possible. Qualifying criteria for breakthrough therapy designation. In 2016, breakthrough therapy designation was received in the u. Sponsors can request breakthrough therapy designation at the time of.
The fdas breakthrough therapy designation is not intended to imply that a drug is actually a breakthrough or that there is highquality. Fast track designation, breakthrough therapy designation, and rmat designation are distinct designation programs with different programmatic requirements. These types of diseases are also referred to as mitochondrial dna depletion syndromes. Breakthroughtherapy designation an fda perspective nejm. The mhra offers a scientific advice service in face to face meetings, which can be requested during any stage of. This phase 2 trial assessed the impact of orencia on the prevention of severe acute gvhd, when added to a standard gvhd prophylactic regimen administered to patients with hematologic malignancies receiving a stem. The fda has granted breakthrough designation for janssen pharmaceuticals jnj6372 for treatment of patients with metastatic nonsmall cell lung cancer, which harbor epidermal growth receptor egfr exon 20 mutations. Cber breakthrough therapy designation requests received by fiscal year. Cber breakthrough therapy designation requests received by. These 2 companies are the biggest winners of the fdas breakthrough designation the food and drug administrations breakthrough therapy designation has created a lot of potential biopharmaceutical.
Regulatory watch impact of breakthrough therapy designation on cancer drug development of 17. The most significant difference in these two programs is related to the type of data needed to substantiate the request. Comparison of breakthrough therapy designation and mhra. Fda pulls breakthrough status from trevenas pain drug. Contact us today to arrange a 30minute introductory call. Janssen and pharmacyclics are working with the fda to determine any potential implications of the breakthrough therapy designations to the ongoing and planned development activities. Expedited programs for regenerative medicine therapies for. The standard for breakthrough therapy designation is not the same as the standard for drug approval and not all drugs receiving breakthrough therapy designation will receive approval for marketing. This document will be used as a basis for the division to comment on whether a request for a breakthrough therapy designation btd is appropriate, at this time, may be too preliminary, or does not currently meet the btd criteria. The fda breakthroughdrug designation department of. Acalabrutinib granted breakthrough therapy designation by. Fast track and breakthrough therapy are the most similar programs designed to expedite the development of drugs for serious conditions. Til therapy conducted by steven rosenbergnci published results showing.
The fda granted breakthrough therapy designation based on the totality of clinical data from the acalabrutinib development programme, including data from the phase ii acely004 clinical trial in. It is both rewarding and validating to receive the fdas breakthrough therapy designation for vts270, which we believe may provide the first effective treatment for. The newest category to address this problem is the breakthrough designation. The breakthrough therapy designation is based on findings from an investigatorinitiated study supported by bristolmyers squibb. Trevena has lost its breakthrough designation for pain treatment oliceridine, just a few months after the fda rejected its marketing application for the drug. Fda, july 30, 2018 poteligeo intravenous injection for nonhodgkin lymphoma the fda has approved poteligeo mogamulizumabkpkc, kyowa kirin, inc. Started in 1995 cms to give fda authorization to categorize medical devices to a category a or b product for purposes of reimbursement. Incyte announces fda approval of tabrecta capmatinib.
Breakthrough therapy designation for acalabrutinib for the treatment of patients with mantle cell lymphoma mcl who have received at least one prior therapy. Breakthrough therapy sakigake first nda in the world intent to develop and file nda. Fda breakthrough therapy designation program european. Cytodyn files for breakthrough therapy designation with. 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. 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. Acalabrutinib is a highlyselective, potent bruton tyrosine kinase btk inhibitor in development for the treatment of multiple b.
Can be submitted with ind, but need for clinical evidence precludes this in most cases. Developing standards for breakthrough therapy designation in. Cytodyn files request with fda for preliminary meeting for. Novartis announces fda approval of met inhibitor tabrecta. Therapy designation to mt1621, modis therapeutics investigational therapy for thymidine kinase 2 tk2 deficiency. Accelerating breakthrough molecules to breakthrough medicines. Drugs may be listed more than once, as breakthrough designation is awarded individually for each indication. Indications of breakthrough therapies at initial approval. The companys pursuit of a breakthrough therapy designation is further strengthened by continued strong clinical indications from mtnbc and mbc patient data. If you are a drug developer seeking regulatory support for orphan drug designation, fast track designation, breakthrough therapy designation, other fdaema expedited programs, type a, b preind, eops, or c meeting assistance, or ind filings, the biopharma global team can help. Developing standards for breakthrough therapy designation.
Fda grants breakthrough therapy designation for mdma. According to fda guidelines, treatments that receive breakthrough therapy designation must target a serious or life. Janssen bags fda breakthrough therapy designation for lung. Cytodyn files request with fda for preliminary meeting for breakthrough therapy designation for leronlimab in the treatment of cancer. Cytodyn files for breakthrough therapy designation with the fda for the use of leronlimab for the treatment of metastatic triplenegative breast cancer. Breakthrough therapy designation includes all the features of the fast track designation, as well as more intensive guidance from the fda on a drug s clinical development program. List of drugs granted breakthrough therapy designation.
The breakthrough designation is designed to expedite the development and drug approval process for drugs that are intended for the treatment of serious and lifethreatening medical conditions. Rucaparib is an oral, potent inhibitor of parp1 and parp2 being developed for the treatment of platinumsensitive. Of all of the expedited pathways, earning breakthrough therapy designation is the most coveted. Cytodyn files request with fda for preliminary meeting for breakthrough therapy designation for leronlimab in the treatment of cancer the companys pursuit of a breakthrough therapy designation is further strengthened by continued strong clinical indications from mtnbc and mbc patient data. In this report, we will address these issues as they pertain to the oncology field. The criteria for breakthrough therapy designation require. Breakthrough therapy designation breakthrough therapy designation applies to the drug either alone or in combination with other drugs and the specific use for which it is being studied.
The breakthrough therapy designation was granted by the fda on the basis of the results of a clinical study performed under a cooperative research and development agreement crada by a team led by dr. Fast track can be granted based on preliminary data, such as activity in a nonclinical model or pharmacological. Preliminary breakthrough therapy designation request. 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. Data reported at asco 2019 annual meeting on june 1, 2019.
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