• Regulatory NewsRegulatory News

    FDA issues potency testing guidance for COVID antibody treatments

    Amid the coronavirus pandemic, the US Food and Drug Administration has issued guidance on potency assays for manufacturers of monoclonal antibodies and other therapeutic proteins to treat COVID-19. The guidance is immediately effective for the duration of the public health emergency.   In the ordinary course of business, FDA would have engagement with drug developers to work on potency assays “over a span of years.” However, according to the guidance, “given the compre...
  • Industry, FDA begin to hammer out PDUFA VII details

    Discussions continue between industry and the US Food and Drug Administration (FDA) regarding the upcoming reauthorization of user fees for prescription drugs, biologics and generic drugs. After public hearing and a first round of meetings that gave a peek into priorities for industry and regulator representatives, this next round began to sort out some detail. (RELATED: PDUFA VII: FDA and industry set priorities in first round of negotiations , Regulatory Focus 29 Octo...
  • Regulatory NewsRegulatory News

    'N of 1' therapies addressed in draft FDA guidance

    Recognizing the pace at which drug developers are moving ever further into individualized medicine, the US Food and Drug Administration (FDA) has issued a draft guidance addressing submission processes for some hyper-specialized treatments.   The new document, which gives high-level guidance for investigational new drug (IND) submissions of individualized anti-sense oligonucleotides (ASOs), takes into consideration that these “N of 1” therapies come with “a set of chal...
  • Regulatory NewsRegulatory News

    Groups seek clarity on interchangeability in BsUFA III

    Industry groups and biosimilar manufacturers are seeking explicit guidance from the US Food and Drug Administration (FDA) on interchangeable biosimilar products. The comments were made to the agency as part of the reauthorization process for Biosimilar User Fee Act (BsUFA III) program.   The recommendations for improvements in the third iteration of FDA’s biosimilars review program were made as part of a public docket opened by FDA. The agency also recently held a ...
  • Regulatory NewsRegulatory News

    Draft FDA guidance addresses oncology drug cross labeling

    In response to increasing requests from sponsors applying for oncology drugs intended to be used in combination regimens, the US Food and Drug Administration has released draft guidance for how oncology drugs used in combination regimens can be referenced by means of cross-labeling.   "Oncology drug applications to the FDA often add investigational drugs to current regimens to create new combination regimens with greater efficacy or safety,” said Richard Pazdur, MD, di...
  • Regulatory NewsRegulatory News

    Promising mRNA tech comes with regulatory, CMC headaches

    As the buzz builds around messenger RNA (mRNA) technology’s use for two leading COVID-19 candidates, manufacturers and regulatory professionals are facing facts: This is not simple technology. Complex manufacturing processes, delivery vehicles that must be treated more as drug substances than excipients, and potential immunogenicity headaches are among the challenges industry faces as this promising technology is harnessed to address an increasing number of health condi...
  • Regulatory NewsRegulatory News

    Euro Convergence: Early consultation critical in overcoming gene therapy hurdles

    When it comes to advancing gene therapy products in the United States and Europe, the key is early consultation with regulators and identification of potential problems, experts said at the Euro Convergence 2020 virtual meeting. “It is better to do it earlier and establish a collaborative and educational approach to discussions,” advised Mridula Shukla, director of global regulatory affairs at Arcutis Biotherapeutics in Palo Alto, Calif.   Patrick Celis, PhD, head of ...
  • RoundupsRoundups

    FDA Approvals Roundup: New formulation for Ultomiris

    New formulation Ultomiris gets new formulation for blood-related disorders  Alexion’s Ultomiris (ravulizumab-cwvz injection) has been  approved  as a 100 mg/mL formulation for treating adults with paroxysmal nocturnal hemoglobinuria (PNH) and for atypical hemolytic uremic syndrome (aHUS) for adults and children aged one month or older.  The  new  formulation of the complement inhibitor will cut annual infusion time by about 60%, or six hours, compared with the 10 m...
  • Regulatory NewsRegulatory News

    Stem cell clinic slapped with CBER warning

    A chiropractor who operates a stem cell clinic has received an untitled letter from the US Food and Drug Administration (FDA) for marketing intrathecal and intravenous injection of human stem cell and tissue-based products to treat such serious conditions as Parkinson’s disease and diabetes.   Michael Johnson runs Optimal Health Stem Cell and Wellness Institute, also doing business as OHSTEMCELL, in Appleton, WI. On 1 October, FDA’s Center for Biologics Evaluation and ...
  • Regulatory NewsRegulatory News

    FDA gives draft adjuvant trial guidance for RCC, bladder cancer

    Two new draft guidances from the US Food and Drug Administration give investigators a roadmap for clinical trials of adjuvant drug or biologic therapy for two common cancers. The first of the guidances released on 1 October addresses adjuvant treatment for bladder cancer, and the other addresses adjuvant treatment of renal cell carcinoma (RCC).   “Currently, there is significant variability in the design, conduct, and analysis of clinical trials” that examine adjuvant ...
  • Regulatory NewsRegulatory News

    Drugmakers get return-to-normal guidance from FDA

    A new temporary guidance for drug and biologics manufacturers from the US Food and Drug Administration (FDA) details recommendations for returning to normal production operations during the public health emergency of the COVID-19 pandemic.   “This guidance provides recommendations to help manufacturers prioritize products as they resume normal operations and as they remediate current good manufacturing practice (CGMP) activities that were necessarily delayed, reduce...
  • Feature ArticlesFeature Articles

    An overview of upstream manufacturing process and process controls for biologics drug substance: A cytokine case

    This article provides an overview for writing up the biological drug substance manufacturing process and process controls. The authors provide an example for the manufacturing of biological drugs consisting of a cytokine. They cover several complex manufacturing steps and details expected to be included in Section 3.2.S.2.2 (Description of Manufacturing Process and Process Controls) of the Common Technical Document (CTD) for the Registration of Pharmaceuticals for Human Us...