news
NICE recommends first licenced treatment for AL amyloidosis
Having been available in Scotland and Northern Ireland since 2022, NICE’s positive opinion of the combination treatment means it is now accessible on the NHS.
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Regs & Legs
article
Rare disease spotlight: first EU-approved treatment for Friedreich’s ataxia
In this exclusive article, Biogen’s Vice President and Head of the Neuromuscular Development Unit, Dr Toby Ferguson, discusses the current rare disease landscape and recent developments in treating Friedreich’s ataxia (FA).
news
Implications of AI regulation for data integrity
According to research, organisations should invest in training for those working with artificial intelligence (AI) to support data integrity assurance in AI applications.
article
Making ATMPs a reality for rare disease patients
Advanced therapy medicinal products (ATMPs) will be among the first to undergo joint clinical assessments at EU level in 2025 and these will form the basis for national value assessments and pricing negotiations. Here, Paolo Morgese from the Alliance for Regenerative Medicine discusses how methodologies for joint clinical assessments (JCAs)…
news
MHRA Chief Executive to step down
During Dame June Raine’s five-year tenure as Chief Executive, MHRA delivered regulation facilitating key innovations in gene therapy and the world’s first COVID vaccine.
news
NICE recommends first medicine for severe alopecia areata
Clinical studies of Pfizer’s oral medicine for alopecia areata have demonstrated response rates continued to improve for up to two years.
news
Novel immunotherapy approved for melanoma
The first non-CAR-T adoptive cell therapy to reach the market has been approved by the US Food and Drug Administration (FDA).
news
USP recommends revisions to proposed RMM chapters
Revisions for two proposed USP chapters recommend changes for rapid tests required for releasing sterile short-life products, as well as for rapid microbiological methods (RMM) for detecting contamination in these products.
news
Vertex secures European approval for CRISPR cell therapy
Now conditionally approved in Europe for sickle cell disease and transfusion-dependent beta thalassemia, the CRISPR therapy offers eligible patients a functional cure.
news
EMA Annex 1 Q&A discusses bioburden considerations
In its recent responses to FAQs relating to Annex 1, the European Medicines Agency (EMA) discusses bioburden guidelines for good manufacturing practice (CGMP) and good distribution practice (GDP).
news
US legislation to benefit small molecule drug innovation
Passing of two key legislations in the US is set to support innovation for small molecule drugs and the biotech sector, Biotechnology Innovation Organization (BIO) asserts.
news
NICE recommends injectable bispecific antibody for aggressive blood cancer
Recommendation of AbbVie’s bispecific antibody for NHS use is based on a Phase I/II trial, which observed a 62 percent overall response rate in diffuse large B-cell lymphoma (DLBCL) patients.
podcasts
EPR Podcast 22 – Pharmaceutical packaging – Manu Kittanakere, Gilead
In this podcast, Manu Kittanakere, Associate Director of Packaging Engineering at Gilead discusses the future of pharmaceutical packaging, including the growing importance of AI, automation and sustainability in packaging engineering.
news
Medicines for Europe calls for simpler SPC manufacturing waiver
To ensure timely market entry of generics and biosimilars in the EU, the Supplementary Protection Certificate (SPC) Manufacturing Waiver must be clearer, asserts Medicines for Europe.
article
Pharmaceutical industry: 2023 in retrospect
In this article, Dave Elder delves deeper into some ongoing developments in topic areas highlighted in European Pharmaceutical Review (EPR) during 2023.
