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    Top FDA official interested in ‘Project Orbis’ for cell and gene therapies

    A top official from the US Food and Drug Administration (FDA) said he wants to see something similar to the agency’s Project Orbis program developed for cell and gene therapies, as the involvement of other “high-income countries” could help attract developers, asserted Peter Marks, director of FDA’s Center for Biologics Evaluation and Research (CBER), at the Biopharma Congress on 13 February.   At the meeting, FDA officials offered their perspectives on other policy ar...
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    FDA reprimands two device firms for multiple violations

    The US Food and Drug Administration’s (FDA) Center for Devices and Radiological Health (CDRH) recently warned two foreign medical device companies – a manufacturer of microcatheters and a laparoscopic instrument maker – for issues including failing to follow through on corrective actions plans, not seeking necessary approval for a new device use and a lax materials testing program.   The targets of the warning letters, which were posted to FDA’s website on 7 February, ...
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    FDA officials offer advice on gene therapy trials

    Officials from the US Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER) Office of Tissues and Advanced Therapies (OTAT) asserted that the duration of clinical trials for gene therapies depends on the nature of the disease being treated, and that diseases that are more progressive and have a rapid onset may involve shorter trials.   This was one of the learnings imparted by officials during a 7 February virtual town hall meeting to...
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    DSCSA: New interoperability ‘blueprint’ released

    With less than a year remaining before the Drug Supply Chain Security Act (DSCSA) takes full effect, an industry consortium has published new chapters of a guide to help drugmakers and their trading partners establish the necessary electronic systems to track their products through the supply chain.   The new chapters complement an earlier guide on understanding the compliance and baseline business requirements on trading of information, which was released in July 20...
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    FDA draft guidance addresses clinical development of drugs to treat early Lyme disease

    The US Food and Drug Administration (FDA) Center for Drug Evaluation Research (CDER) has issued a draft guidance to help sponsors develop new drugs to treat the early stages of Lyme disease.   The draft addresses criteria for clinical trial enrollment, efficacy endpoints, and clinical microbiology considerations for treating early stages of the disease when manifested by erythema migrans (EM), a rash that is often one of the first symptoms.   In North America, Lyme...
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    FDA draft guidance addresses use of external controls to assess effectiveness of new drugs and biologics

    The FDA Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER) have issued draft guidance describing sponsors can leverage external controls, such as use data from registries and electronic health records, in lieu of data from randomized controlled trials (RCTs) to demonstrate the safety and effectiveness of new drugs and biologics.   The draft guidance also describes how sponsors can communicate with FDA on using dat...
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    FDA releases FY 2022 generic drug research summary

    The US Food and Drug Administration’s (FDA) Office of Generic Drugs (OGD) on Tuesday released a report summarizing its research activities in fiscal year 2022 in 13 different scientific areas under the Generic Drug User Fee Amendments (GDUFA III).   The research generated from the program enabled FDA to issue 177 new and revised product-specific guidances (PSGs), 59 of which were for complex products, according to the summary.   “The recommendations in many of th...
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    FDA issues draft guidance on developing drugs for acromegaly

    The US Food and Drug Administration (FDA) has issued a draft guidance to assist sponsors develop acromegaly treatments for patients who have either not responded to surgery or are in poor health.   Acromegaly is a chronic, rare disease in adults caused by excess growth hormone that can cause enlarged hands and feet, among other symptoms. The most common cause stems from a growth hormone that stimulates hepatic overproduction of insulin-like growth factor-1 (IGF-1).  ...
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    FDA announces FY 2023-2027 BsUFA science and research priorities

    The US Food and Drug Administration (FDA) on Thursday released a roadmap on its science and research priorities for fiscal years 2023-2027 to spur the development of biosimilars and interchangeable products.   Under the Biosimilar User Fee Amendments (BsuFA) III program, FDA has offered $5 million in funding for several research proposals under the regulatory science pilot program (RELATED: FDA offers funding for biosimilar regulatory science pilot , Regulatory Foc...
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    FDA officials impart lessons learned from quality management maturity pilots

    The US Food and Drug Administration’s (FDA) should brief pilot participants ahead of the actual assessments and use a simplified rubric for scoring or rating a manufacturers quality management maturity (QMM) program. These were some of the lessons learned as the agency develops a future rating program for evaluating manufacturers on drug quality, according to results reported in a 10 January article published in The AAPS Journal .   These pilots “afforded FDA the opp...
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    FDA details approach for finding optimal dosages for new cancer drugs

    The US Food and Drug Administration (FDA) has issued a draft guidance to help sponsors identify the optimal dosage for cancer drugs in clinical development and in it, the agency recommends a new approach in selecting such dosages for modern, targeted oncology drugs.   Historically, dose-finding trials for cytotoxic chemotherapy drugs have been designed to determine the maximum tolerated dose (MTD). The MTD paradigm applies to drugs that have a steep dose-response, have...
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    Budget law calls for BARDA and FDA to establish “warm base” manufacturing to better prepare for future pandemics

    The federal budget law passed by Congress and signed by the president in late December calls on federal authorities to establish “warm base” domestic manufacturing to respond quickly to demand surges for medical products during a public health emergency.   Section 2401 of the law calls on the Biomedical Advanced Research and Development Authority (BARDA) to work with the US Food and Drug Administration (FDA) to coordinate these efforts.   The law amends Section 319...