Regulatory news - August 2020

EU

European Commission (EC)

European Parliament Release a Temporary Derogation for Medicinal Products Containing or Consisting of GMOs for Treatment of COVID-19

The EC has adopted the Regulation (EU) 2020/1043 on the conduct of clinical trials with and supply of medicinal products for human use containing or consisting of genetically modified organisms (GMOs) intended to treat or prevent coronavirus disease (COVID-19).

Clinical trials involving investigational medicinal products (IMPs) that contain or consist of GMOs fall under the GMO legislation (Contained Use Directive (2009/41/EC) and Deliberate Release Directive (2001/18/EC)) However, this regulation provides a temporary, and strictly COVID-19 related derogation from certain rules for clinical trials involving GMOs. The main objective of this temporary derogation is to support the development of safe and effective medicinal products for the treatment or prevention of COVID-19, facilitating the possibility to conduct clinical trials on GMO-IMPs as soon as possible.

For clinical trials involving GMO-IMPs, the following aspects will apply:

• All operations related to the conduct of clinical trials including packaging and labelling, storage, transport, destruction, disposal, distribution, supply, administration or use of investigational medicinal products (IMPs) containing or consisting of GMOs, with the exception of the manufacturing of the IMP, shall not require a prior environmental risk assessment, when these operations relate to the conduct of a clinical trial authorised in accordance with the Clinical Trials Directive 2001/20/EC;
• Sponsors are required to implement appropriate measures to minimise foreseeable negative environmental impacts resulting from the intended or unintended release of the IMP into the environment.

The derogation will apply for as long as WHO has declared COVID-19 to be a pandemic, or as long as an implementing act by which the commission recognises a situation of public health emergency due to COVID-19.

See here to read the adopted regulation.

INTERNATIONAL

USA

US Food and Drug Administration (FDA)

FDA Release Guidance on Endotoxin Limits for the Development of Oncology Drugs and Biological Products

The FDA have published draft guidelines for establishing endotoxin limits for the development of anticancer drugs and biological products. The guidance describes the FDA’s recommendations to Sponsors for setting endotoxin limits for the development of IMPs intended for use in combination with other approved drugs or for the co-development of two or more IMPs in the treatment of advanced cancer, when evaluated in early clinical trials as part of a multidrug regimen. A risk-based approach is recommended, weighing the potential risks of possible exposure to increased levels of endotoxin across all components of a multidrug regimen against the potential benefits to patients with serious and life-threatening cancers.

The draft guidance is available here.

FDA Reject BioMarin’s Gene Therapy for Severe Haemophilia A

The FDA issued a complete response letter to BioMarin for their gene therapy valoctogene roxaparvovec, which has been developed for the treatment of haemophilia A.

BioMarin released a statement to explain that ‘having previously agreed with the Agency on the extent of data necessary to support the BLA, the FDA introduced a new recommendation for two years of data from the Company's ongoing 270-301 study (Phase 3) to provide substantial evidence of a durable effect using Annualized Bleeding Rate (ABR) as the primary endpoint’. The Agency have recommended that BioMarin complete the current ongoing Phase 3 Study and submit two-year follow-up safety and efficacy data on all study participants.

To read the full press release, see here.

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EUROPEAN COMMISSION (EC)