The Pharma Data

AUGUST 15, 2021

Food and Drug Administration (FDA) has granted Fast Track designation to VTX-801, Vivet’s clinical-stage gene therapy for the treatment of Wilson Disease – a rare, genetic disorder that reduces the ability of the liver and other tissues to regulate copper levels, causing severe hepatic damage, neurological symptoms, and potentially death.

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Drug Discovery World

MAY 6, 2024

A more established Amsterdam-based enterprise is gene therapy company uniQure, developer of etranacogene dezaparvovec, the first gene therapy for haemophilia B, which was approved for use in the US in November 2022 and in the EU in February 2023. The CDD will sponsor and manage KWF-supported trials.

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Drug Discovery World

SEPTEMBER 14, 2023

joined Pfizer’s Executive Leadership Team as Chief Oncology Research and Development Officer and Executive Vice President reporting to Chairman and Chief Executive Officer, Albert Bourla. In July 2023, AstraZeneca and Ionis Pharmaceuticals expanded their existing collaboration on eplontersen to also include Latin America.

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Camargo

AUGUST 11, 2021

As regulatory requirements become increasingly harmonized across the globe, the development and marketing of pharmaceutical products worldwide are also becoming more streamlined. As of January 1, 2021 , EU pharmaceutical regulations do not apply to the United Kingdom, which formally left the EU in January 2020.

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FDA Law Blog

JANUARY 4, 2023

The purpose of these town halls are to discuss topics related to OTAT-regulated products, engage with product development stakeholders, and to provide information to help stakeholders to help advance drug development. Manufacturers who hold a biological license should report manufacturing deviations to the FDA per 21 CFR 600.14.

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The Pharma Data

DECEMBER 9, 2020

The Chinese biopharmaceutical industry is growing in leaps and bounds, but there is still a huge unmet need when it comes to getting patients access to the breakthrough therapeutic modalities and platforms like RNAi, cell and gene therapy and others. A solution could be at hand with Overland Pharmaceuticals.

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Drug Discovery World

MARCH 29, 2023

Peter O’Donnell, Applied Clinical Trials’ Brussels Correspondent, summarises this as the EMA being tasked with creating a European network of patient representatives, academics, drug developers, investigators, and centres with expertise in the performance of studies in the paediatric population.

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FDA Law Blog

SEPTEMBER 20, 2021

We have seen first-hand the difficult task faced by CBER review staff to keep up with the flood of innovative cell and gene therapy products, as well as the taxing influx of COVID-19 pandemic related applications requiring review resources in both Centers. Notable Dates and Timelines – Applications open, Q4 of FY 2023.

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XTalks

AUGUST 2, 2023

Meanwhile, BioMarin’s gene therapy Roctavian is approved by the FDA for the treatment of hemophilia A. In September 2022, Roche acquired Good Therapeutics for an upfront payment of $250 million, and has access to their PD-1-regulated IL-2 program. But all three drugs have new competitors. Both are anti-CD20 agents.

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