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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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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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Huge inequalities found in access to myeloma clinical trials in Europe, says MPE

pharmaphorum

Led by the MPE CEE Workgroup on Access, the report – Addressing access barriers to myeloma clinical trials in Central and Eastern Europe – evaluated the number of clinical trials held in CEE countries between 1 January 2001 and 28 September 2020. Approximately 50,000 people in Europe are diagnosed with myeloma each year.

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2022 spells change for EU pharmaceutical legislation

European Pharmaceutical Review

This suggests that any of the modelled changes to the regulation could lead to a decrease in the number of OMPs developed… Some of the assessed changes to the regulation could undermine the business model under which OMP?focused One of the key priorities of the EU’s strategy is to encourage innovation to solve unmet medical needs.

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

European Pharmaceutical Review

This can lead to severe health risks for the patient, in which case the pharmacy would be held liable. Proposal for a Directive of the European Parliament and of the Council on the Union code relating to medicinal products for human use, and repealing Directive 2001/83/EC and Directive 2009/35/EC.” Article 63 EMA (29 January 2020).

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Botanical drugs – what is the best way forward for regulatory and market approval?

European Pharmaceutical Review

11 The THR pathway was created with the purpose of establishing a simplified registration procedure for all traditional herbal medicines that do not fulfil the requirements for the MA (strict) pathway under Directive 2001/83/EC. 10,12 The THR scheme is also limited to products used for oral or external use and/or inhalation. 2012/1916).

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Achieving scale up success under economic uncertainty

European Pharmaceutical Review

In 2001 and 2007, through 2009, the economy was hit hard. Therefore, it is best to collaborate with leading experts and scientists you can trust with your molecule. About the author Brandon Fincher is President of Early Stage Development & Testing at Cambrex, a leading global CDMO.