• RoundupsRoundups

    FDA Approvals Roundup: Nulibry, Amondys 45, Pepaxto

    A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).   New approvals Nulibry okayed to cut mortality risk in babies with rare genetic disorder Origin Biosciences’ Nulibry (fosdenopterin injection) has been approved for reducing the risk of death from molybdenum cofactor deficiency (MoCD) type A, a rare, genetic metabolic disorder marked by severe seizures. The condition presents soon after birth and most patients...
  • Regulatory NewsRegulatory News

    FDA warns COVID trial sponsor for failing to submit IND

    The US Food and Drug Administration (FDA) last month warned a clinical trial sponsor for failing to submit an investigational new drug (IND) application for a study of a product to treat COVID-19.   In its warning letter to KDunn and Associates, P.A. dba HealthQuilt, FDA said it observed “objectionable conditions” during an August inspection related to a trial the company sponsored titled “An Open-Label Pilot Study to Assess the Efficacy and Safety of [redacted] in Sub...
  • Feature ArticlesFeature Articles

    February's Regulatory Focus: Global and regional harmonization and alignment

    Feature articles during February included a range of topics focused on harmonization and alignment across global and regional regulatory entities, including electronic submissions through the European Medicines Agency (EMA), tools for updating the EU In Vitro Diagnostic Regulation (EU IVDR) template, China’s new PAC regulation, Project Orbis and improving patients access to new therapies, as well as a report on medtech companies’ preparation for EU Medical Device Regulatio...
  • Regulatory NewsRegulatory News

    FDA on Rare Disease Day: New initiatives and an upcoming conference

    On Monday, the US Food and Drug Administration (FDA) announced a series of efforts and a day-long conference centered on addressing the unmet needs of patients with rare diseases. FDA leaders announced the initiatives in recognition of Rare Disease Day, observed on February 28.   A new request for applications (RFA) issued by FDA augments the agency’s Orphan Products Grants Program. The RFA is seeking natural history studies to address unmet needs in rare diseases, l...
  • Feature ArticlesFeature Articles

    Project Orbis: Maximizing patient access to new medicines

    To ensure equitable access to new medicines for all patients, drug manufacturers aim to register new products in as many countries as possible. Project Orbis, an initiative of the FDA Oncology Center of Excellence (OCE), is a pilot program that provides applicants with a framework for concurrent submission and review of oncology products among international partners to allow earlier registration in countries outside of the US. This article summarizes Genentech’s experience...
  • Regulatory NewsRegulatory News

    Updated: FDA issues EUA for J&J's one-shot COVID vaccine

    The US Food and Drug Administration (FDA) has issued an emergency use authorization for the COVID-19 vaccine developed by Janssen, a division of Johnson & Johnson. “The authorization of this vaccine expands the availability of vaccines, the best medical prevention method for COVID-19, to help us in the fight against this pandemic, which has claimed over half a million lives in the United States,” said Acting FDA Commissioner Janet Woodcock, MD in a statement announcing ...
  • Regulatory NewsRegulatory News

    FDA eases storage, transportation conditions for Pfizer-BioNTech vaccine

    The US Food and Drug Administration (FDA) on Thursday updated the emergency use authorization (EUA) for Pfizer and BioNTech’s COVID-19 vaccine to allow the vaccine to be transported and stored at temperatures found in conventional pharmaceutical freezers for up to two weeks.   The announcement comes one week after Pfizer and BioNTech disclosed they had submitted data demonstrating their vaccine’s stability at higher temperatures based on manufacturing data from the p...
  • Regulatory NewsRegulatory News

    England and Canada diverge from US on cancer drug decisions

    Regulatory and health technology assessment agencies in Canada and England are diverging from the US when it comes to oncology drugs. But they often rely on the same surrogate endpoints to make their decisions, according to two new analyses published in JAMA Internal Medicine .   The first study , led by Avi Cherla, MSc, of the London School of Economics and Political Science, revealed that among 68 cancer indications with accelerated approval in the US that also rec...
  • RoundupsRoundups

    FDA Approvals Roundup: Libtayo

    A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).   New indications Libtayo gets extended indication for NSCLC with high PD-L1 expression Regeneron’s Libtayo (cemiplimab-rwlc injection) has been approved as a first-line therapy for locally advanced non‒small cell lung cancer (NSCLC) in patients whose tumors have high programmed death-ligand 1 expression established by an FDA-approved test. The patients should ...
  • Regulatory NewsRegulatory News

    FDA targets remdesivir, thymosin in compounding concerns

    The US Food and Drug Administration has updated information on compounding drugs during the COVID-19 pandemic, focusing on some specific compounded drugs related to COVID-19.   “FDA has concerns about the use of certain drugs prepared by compounders to treat COVID-19,” wrote the agency on its web page addressing compounding during the pandemic.   The first drug called into question by FDA is thymosin-alpha 1. “The agency is aware of drug products containing thymo...
  • Regulatory NewsRegulatory News

    FDA's COVID therapeutics, mAbs guidance accounts for variants

    As the world rolls into year 2 of the COVID-19 pandemic, regulators and drug developers are grappling with how to address variant strains of SARS-CoV-2 as they bud and expand globally.   The inevitable path for RNA viruses is to mutate, a fact acknowledged by Janet Woodcock, MD, acting commissioner US Food and Drug Administration, in a Monday press conference announcing the availability of new and updated guidance for developers of therapeutics, vaccines, and tests for...
  • Regulatory NewsRegulatory News

    OPDP warns Paragard maker over promotional video

    A sponsored video that aired on NBC’s Boston affiliate in October has landed CooperSurgical a warning letter from the US Food and Drug Administration’s (FDA) Office of Prescription Drug Promotion (OPDP) for failing to include risk information about the company’s Paragard intrauterine device (IUD).   The warning letter is the second sent by OPDP in 2021 and is the second enforcement letter sent to CooperSurgical concerning Paragard. In 2019, OPDP sent the company an unt...