Publications/White Papers

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P4A experts regularly share their unique perspectives that inform and challenge the norm through articles and white papers.


Generation Bio pioneers revolutionary gene therapy delivery system

The biotech venture capital firm Atlas Ventures has announced the creation of Generation Bio, a company that has developed a system by which the dosage of a gene therapy can be varied to maximise the potential benefit. The company has started on the back of a $25 million Series A funding round from Atlas. The […]

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Spark Therapeutics Announces $850 000 price tag of gene therapy treatment Luxturna

Spark Therapeutics has announced its recently approved Luxturna (voretigene neparvovec-rzyl), a gene therapy treatment for biallelic RPE65 mutation-associated retinal dystrophy will cost $425 000 per eye, or $850 000 in total, in the U.S. This is below the $1 million price tag that was originally speculated. Spark Therapeutics is also working with Harvard Pilgrim, a […]

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FDA approves gene therapy for rare form of vision loss

The U.S. Food and Drug Administration (FDA) has approved the use of Spark Therapeutics’ gene therapy Luxturna (voretigene neparvovec-rzyl) for the treatment of a rare inherited form of vision loss and blindness. This marks the first approval by the FDA of a directly administered gene therapy for the treatment of a disease caused by mutations […]

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FDA issues new draft guidance for Orphan Drug development

The U.S. Food and Drug Administration (FDA) published its recommendation for a new approach to drug development for rare pediatric diseases. The new approach supports the FDA’s commitment to increase the efficiency and speed of developing drugs, as well as supporting the sponsors of the research. The draft guidance, ‘Pediatric Rare Diseases: Collaborative Approach for […]

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The EMA has updated its practical guidance for pharmaceutical companies for procedures related to Brexit

From 30th March 2019, when the United Kingdom’s (U.K.) is expected to withdraw from the European Union (EU), all EU law will no longer apply to the U.K. The European Medicines Agency (EMA) and the European Commission have therefore published additional practical guidance to help pharmaceutical companies legally prepare for this transition. This guidance will […]

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Amsterdam will be the new host of the EMA

Amsterdam has been selected as the new post-Brexit home for the European Medicines Agency (EMA). It was chosen in a draw of lots between it and Milan, after three rounds of voting had failed to pick a favourite. The EMA has been located in London since its formation in 1995, where it employs around 900 […]

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EMA and EUnetHTA publish a 3 year collaboration plan to improve efficiency and quality

The European Medicines Agency (EMA) and the European Network for Health Technology Assessment (EUnetHTA) have finalised “a joint work plan outlining key areas of collaboration for the next three years”. The main areas of collaboration are information exchange at market entry, data generation post approval and early dialogue. These areas are expected to also provide […]

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