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Regulatory-first for gene editing therapy

European Pharmaceutical Review

The Investigational New Drug (IND) application for Intellia Therapeutics’ in vivo CRISPR-based candidate NTLA-2001, has been cleared as a gene editing therapy for transthyretin (ATTR) amyloidosis with cardiomyopathy (ATTR-CM) in the US. This supports NTLA-2001’s potential as a single-administration therapeutic.

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Psychedelic medicines: are they gaining traction in Europe?

European Pharmaceutical Review

For example, the relaxed attitude towards cannabis in the Netherlands is well-known and Portugal has decriminalised the possession and consumption of all illicit substances since 2001. 6 Therefore, the quality of data provided to regulators will be key in obtaining MAs and subsequently marketing of psychedelic medicines.

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Bridging the healthcare digital divide: a gradual approach to adopting ePI

European Pharmaceutical Review

They are vital to ensuring the safe and effective use of medicines, making them an integral part of healthcare in Europe. The European Medicines Agency (EMA) underscores the role of digital platforms in disseminating product information electronically. percent as EU average with some Member States as high as 25 percent).

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2022 spells change for EU pharmaceutical legislation

European Pharmaceutical Review

These events led to the publication of the EU Pharmaceuticals Strategy in 2020, 1 which, in addition to a range of other activities, aims to ensure the regulatory system for human medicines is future?proof To this end, the strategy lays out a timeline for review of the EU’s general legislation on medicines for human use.

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Heart failure: clinical trials update 2022

European Pharmaceutical Review

NTLA-2001, a novel CRISPR/Cas9-based therapy in the first-ever human gene editing trial ( NCT04601051 ) in vivo (in the body), reduced transthyretin (TTR) proteins by at least 90 percent in amyloidogenic transthyretin (ATTR) cardiomyopathy patients, 28 days after a single IV infusion, with benefits lasting four to six months.

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Interchangeability of biosimilars in the EU – the industry impact

European Pharmaceutical Review

A biosimilar is a biological medicine ‘similar’ to another biological medicine already approved in the EU (the ‘reference medicine’). 4 However, in some cases, comparative data may not be required across all therapeutic indications and may be extrapolated to other indications already approved for the reference medicine.

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EU pharmaceutical legislation revisions: what are the implications for biopharma?

European Pharmaceutical Review

The proposed changes to EU pharmaceutical legislation broadly aim to: Support innovation and boost attractiveness of EU market Ensure timely and equitable access to medicines for patients across the EU Address other ongoing issues, including antimicrobial resistance and environmental impact of medicines. analysis (Source: L.E.K)