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FDA Approves Test to Detect Leukemia and Lymphoma The FDA approved the marketing of ClearLLab Reagents ( T1 , T2 , B1 , B2 , M ), the first FDA-authorized test that can be used with flow cytometry to detect chronic leukemia , acute leukemia , non-Hodgkin lymphoma , myeloma , myelodysplastic syndromes , and myeloproliferative neoplasms .
The ClearLLab test can detect cancerous cells in the blood , bone marrow , and lymph nodes , and provides information on the type of leukemia or lymphoma . The test marks proteins found on the surface of cells with fluorescent dyes for further analysis via a flow cytometer .
The FDA granted the test marketing authorization through the agency ’ s de novo premarket review pathway . The decision was based on findings from a study comparing test results on 279 samples between the ClearLLab system and other detection methods used at four independent clinical sites . ClearLLab results aligned with the study sites ’ final diagnoses 93.4 percent of the time , and the new system correctly detected cancer presence 84.2 percent of the time .
The FDA is establishing special controls for the test to ensure its accuracy , reliability , and clinical relevance .
The agency advises that trained professionals review results from the ClearLLab test .
Source : U . S . Food and Drug Administration news release , June 29 , 2017 .
FDA Unveils Plan to Eliminate Backlog of Orphan Drug Designation Applications The FDA has announced a plan to eliminate the agency ’ s backlog of orphan drug designation requests within 90 days , as well as respond to any new requests for the designation within 90 days of receipt . The FDA has approximately 200 pending orphan drug designation requests , and the number of requests has been increasing each year .
As part of its Medical Innovation Development Plan ( MIDP ), the agency will create a team of senior reviewers with significant expertise in orphan drug designation to focus solely on the backlogged applications , starting with the oldest requests . The agency will also use a streamlined Designation Review Template to increase consistency and efficiency of drug reviews .
To ensure that all future requests receive a response within 90 days of receipt , the agency said it will reorganize its staffing to maximize expertise ,
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improve workload efficiencies , and better leverage expertise across the FDA ’ s medical product centers . The FDA also plans to establish a new Orphan Products Council to address scientific and regulatory issues to guarantee that the agency is applying a consistent approach to regulating orphan drug products and reviewing designation requests .
The MIDP aims to ensure that the FDA ’ s regulatory tools and policies are modern , risk-based , and efficient under the new direction of Commissioner Scott Gottlieb , MD .
Source : U . S . Food and Drug Administration news release , June 29 , 2017 .
President Declares State of Emergency After Urged by Federal Opioid Commission On August 10 , President Trump declared the opioid crisis a national emergency in response to a report from the Commission on Combating Drug Addiction and the Opioid Crisis . This designation is typically reserved for natural disasters .
President Trump did not offer specific details on what the state of emergency would involve , but the authors of the report suggested that issuing this declaration would temporarily waive many of the regulations on federal funding and allow agencies to access federal disaster funds to pay for more resources to fight the opioid epidemic , including limited drug reimbursement from Medicaid .
Other recommendations from the interim report provided by the Commission included :
• increase treatment capacity by empowering the U . S . Department of Health and Human Services to grant waivers allowing states to spend federal money on treatment services
• mandate prescriber education initiatives within medical and dental schools to enhance prevention efforts
• provide federal funding and technical support to states to enhance interstate data sharing among statebased prescription drug monitoring programs to better track patientspecific prescription data
• enforce laws requiring insurance companies to cover treatment for mental health and substance abuse disorders
A finalized report from the commission will be published later this year .
Sources : CNN , August 11 , 2017 ; The Washington Post , August 10 , 2017 ; Congressional Quarterly , August 1 , 2017 .
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Researchers Carry Out First Gene Editing of Embryos in the United States |
A team of researchers at Oregon Health and Science University successfully used gene-editing technology to correct defective genes in a human embryo . This is the first report of safe , successful gene editing in the United States .
Led by Shoukhrat Mitalipov , PhD , researchers used the CRISPR gene-editing technique , which allows scientists to remove segments of the genome and replace them with new segments of DNA . The edited embryos were not allowed to develop past a few days .
CRISPR and gene editing are controversial topics in the medical research world . The National Institutes of Health announced that it will not fund any use of gene-editing technologies in human embryos , and some countries have already signed a convention prohibiting the practice of editing embryonic genes over ethical concerns .
A peer-reviewed journal will publish the results soon .
Sources : Reuters , July 27 , 2017 ; The Oregonian , July 27 , 2017 .
FDA Approves Combination Daunorubicin and Cytarabine for High-Risk AML The FDA approved CPX-351 , a fixed combination of cytarabine and daunorubicin , for the treatment of adults with newly diagnosed therapy-related acute myeloid leukemia ( t-AML ) or AML with myelodysplasia-related changes ( AML- MRC ). Both are considered high-risk AML subtypes .
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The drug was approved based on results from a study of 309 patients with newly diagnosed t-AML or AML-MRC who were randomized to receive daunorubicin and cytarabine either in the fixedcombination formulation or as separate treatments . Patients who received CPX- 351 had a longer median overall survival compared with those who received separate treatments ( 9.56 months vs . 5.95 months ).
Common adverse events associated with CPX-351 included hemorrhage , febrile neutropenia , rash , edema , nausea , mucositis , diarrhea , constipation , musculoskeletal pain , and fatigue .
Patients who have a history of serious hypersensitivity to daunorubicin , cytarabine , or any component of the formulation should not use CPX-351 , and the prescribing information includes a boxed warning not to interchange CPX-351 with other daunorubicin- and / or cytarabine-containing products .
Source : U . S . Food and Drug Administration news release , August 3 , 2017 .
Venetoclax Gets Breakthrough-Therapy Designation for AML The FDA granted breakthrough-therapy designation to the BCL2 inhibitor venetoclax , in combination with cytarabine , for the treatment of older patients with treatment-naïve AML who are ineligible to receive intensive chemotherapy .
The decision was supported by data from an ongoing , open-label , phase Ib trial of venetoclax plus low-dose cytarabine in 20 older patients (> 65 years ). As of last follow-up ( October 1 , 2015 ), 18 patients ( median age = 74 years ) had received the venetoclax combination : Eight patients received venetoclax 600 mg , and 10 received venetoclax 800 mg . The median time on study was 127.5 days ( range = 30-272 days ).
At 12-month follow-up , the overall response rate was 44 percent ( complete remission , n = 4 ; complete remission without complete marrow recovery , n = 4 ; resistant disease , n = 8 ; death before evaluation , n = 2 ).
Two patients in the venetoclax 800 mg group experienced a dose-limiting toxicity ( grade 4 thrombocytopenia lasting > 42 days ). The most common adverse event ( AE ) was febrile neutropenia ( 33.3 %). AEs occurring in ≥30 percent of patients included nausea ( 77.8 %), anemia ( 55.6 %), febrile neutropenia ( 38.9 %), neutropenia ( 38.9 %), fatigue ( 38.9 %), vomiting ( 33.3 %), diarrhea ( 33.3 %), and hypokalemia ( 33.3 %). ●
Sources : Roche press release , July 28 , 2017 ; Lin T , Strickland S , Fiedler W , et al . Phase 1b / 2 study of venetoclax with low-dose cytarabine in treatment-naïve patients aged ≥65 years with acute myelogenous leukemia . Abstract # E911 . Presented at the European Hematology Association Annual Congress , June 2017 ; Madrid , Spain .
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