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  • Regulatory NewsRegulatory News

    Generic drug approvals continued to fall in 2021

    The US Food and Drug Administration (FDA) approved or tentatively approved 776 Abbreviated New Drug Applications (ANDAs) for generic drugs in 2021, continuing a steady decline in generic approvals in recent years.   In calendar year 2020 , FDA approved or tentatively approved 948 ANDAs for generic drugs, which was down from 1,014 in 2019 . The latest figures are part of the FDA’s Office of Generic Drugs (OGD) 2021 Annual Report .   But the agency approved 93 “fi...
  • Feature ArticlesFeature Articles

    January’s Regulatory Focus: Leadership in RA, expanding diversity, and more

    Feature articles during January focused on regulatory leadership, a novel approach to hiring regulatory professionals, expedited programs for reducing time to approval for certain drugs, and the Cuban biotechnology industry and its response to COVID-19. Keywords – assessment aid, breakthrough therapy, Cuba, diversity, ILAP, leadership, PRIME   Leadership, hiring, and expanding diversity Effective leadership in regulatory affairs is rooted in an understanding a...
  • Feature ArticlesFeature Articles

    Expedited pathways: Breakthrough therapy designation, PRIME, and innovative licensing & access

    Abstract Over the last decade, regulators have introduced initiatives to reduce time to approval for medicines that address an unmet medical need. This article compares the US Food and Drug Administration’s (FDA’s) breakthrough therapy designation (BTD), the European Medicine’s Agency’s (EMA’s) PRIority MEdicines (PRIME) scheme, and the innovative licensing and access pathway (ILAP) from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) and reviews th...
  • Regulatory NewsRegulatory News

    FDA rolls out more guidance on 'N of 1' gene therapies

    Developers of individualized investigational antisense oligonucleotide (ASO) therapies for ultra-rare diseases received additional guidance from the US Food and Drug Administration (FDA). In two draft documents, the agency has provided new information for sponsor-investigators and for those overseeing manufacture of these so-called “N of 1” therapies for people with severely debilitating or life-threatening genetic disease.   "Progress in individualized medicines provi...
  • Feature ArticlesFeature Articles

    October’s Regulatory Focus: Linguistic review, advanced therapies in Asia-Pacific, and more

    Feature articles during October included an in-depth look at the linguistic review process in Europe and  reviews of the regulatory pathways for herbal medicinal products, medical devices, and supplements in the EU and for advanced therapies in the Asian-Pacific region. Also included was an article on the challenges of data integrity remediation in cGMP facilities.   Linguistic review and herbal product regulation The linguistic review process for centrally authori...
  • Regulatory NewsRegulatory News

    New public-private consortium will tackle gene therapies for rare diseases

    Two federal agencies are partnering with pharmaceutical companies and non-profit organizations to form a consortium aimed at boosting the development of gene therapies for rare diseases.   In a Wednesday announcement, the US Food and Drug Administration (FDA) and the National Institutes of Health (NIH) revealed the launch of the Bespoke Gene Therapy Consortium (BGTC). The consortium’s work will be managed by the Foundation for the NIH and sit within the NIH Acceleratin...
  • Regulatory NewsRegulatory News

    For gene therapies, FDA drafts trial guidance, finalizes "sameness" for orphan exclusivity

      A new draft guidance from the US Food and Drug Administration (FDA) gives cell and gene therapy developers a detailed framework for the conduct of umbrella trials, offering the potential for enhanced speed and efficiency in early-stage clinical trials.   In the umbrella trials envisioned under the draft guidance, two or more versions of a cellular or gene therapy product would be studied for one specific disease using just one trial design, shared infrastructure, a...
  • Regulatory NewsRegulatory News

    Convergence: Regulatory considerations in advancing gene and cellular therapies

    When developing a regulatory submission for a gene or cellular therapy, it’s essential to keep the basics in mind, such as how to demonstrate the product’s identity, purity, and potency, Haroon Hashmi, PhD, told attendees at RAPS Convergence 2021.   “Even when we think about these complex technologies -- new emerging technologies -- certain regulatory principles always stay in place,” said Hashmi, senior vice president at Ziopharm Oncology, which has developed the Slee...
  • Regulatory NewsRegulatory News

    USP, NIST and NIIMBL collaborate to boost viral vector quality standards

    A new public-private collaborative among the United States Pharmacopeia (USP), the US Department of Commerce’s National Institute of Standards and Technology (NIST) and the National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL) pools the expertise or the organizations to develop better measurement tools to assess the quality of adeno-associated virus (AAV), an increasingly important vector for gene therapy delivery.   According to a 27 June ann...
  • Regulatory NewsRegulatory News

    EMA's PRAC weighs in on more vaccine side effects

    The European Medicines Agency (EMA) has weighed in on several rare adverse events for COVID-19 vaccines and one gene therapy for treating thalassemia in their latest Pharmacovigilance Risk Assessment Committee (PRAC) meeting.   At its July meeting, EMA’s Pharmacovigilance Risk Assessment Committee (PRAC) found mRNA-based COVID-19 vaccines, like Pfizer’s Comirnaty and the Moderna vaccine marketed in the EU as Spikevax, carry a small risk of myocarditis and pericarditis....
  • Regulatory NewsRegulatory News

    EMA consults on ICH S12 guideline, shares comments on PFDD reflection paper

    The European Medicines Agency (EMA) on Thursday opened a public consultation on the International Council for Harmonisation’s (ICH) S12 guideline on nonclinical biodistribution (BD) considerations for gene therapies.   The S12 guideline recently reached Step 2b of the ICH process after being endorsed at the ICH Assembly virtual meeting earlier this month. (RELATED: ICH reports ‘significant milestones’ reached on guidelines ranging from impurity testing to eCTD standa...
  • ReconRecon

    Recon: ASCO 21 updates; Moderna goes for teen EUA

    Welcome to Regulatory Reconnaissance, your daily regulatory news and intelligence briefing.   In Focus: US Moderna says that its vaccine is effective for 12- to 17-year-olds and that it will seek F.D.A. authorization ( NYT ) ( NPR ) President Biden still hasn’t appointed a permanent FDA commissioner. That has big implications for upcoming drug approvals ( Fortune ) Bipartisan Legislation Introduced to Advance Biomedical Research Stalled by Pandemic ( Global Genes ) ...