Regulatory news - September 2019

Keep up to date with regulatory news from around the world with the Cell and Gene Therapy Catapult regulatory newsletter.


European Medicines Agency

EMA Consults on Paediatric Study Preparedness Framework

The European Network of Paediatric Research at the European Medicines Agency (EnPR-EMA) opened a public consultation on a draft framework for paediatric clinical trial preparedness. Trial preparedness is defined here as “a structured assessment of the key factors that could increase the likelihood of a smooth and timely course of a paediatric clinical trial, integrating information from multiple stakeholders on what is possible within a development plan and/or individual studies” (including study feasibility).

The recommendations in this document are targeted at both sponsors and Principal Investigators; advising that trial preparation should be initiated before and conducted in parallel to designing the paediatric development plan and the individual studies, and in parallel to sponsor readiness.

To read the full consultation, see here.


NICE Recommends Novel Gene Therapy Treatment for Rare Inherited Eye Disorder

Voretigene neparvovec (Luxturna), manufactured by Novartis Pharmaceuticals UK, is a novel gene therapy treatment for people with vision loss caused by inherited retinal dystrophy from confirmed RPE65 gene mutations. This is the first licenced gene therapy for vision loss caused by RPE65-mediated inherited retinal dystrophy and has been recommended by NICE for use on the NHS.

The evaluation at NICE has taken 20 weeks instead of the average of 38 weeks within the Highly Specialised Technologies programme, which NICE state is thanks to “the company’s willingness to work with us early and constructively”.

Inherited retinal dystrophies are a group of eye diseases caused by genetic mutations which result in gradual degeneration of photoreceptor cells in the retina. The RPE65 gene is required for making a protein essential for normal vision. RPE65-mediated inherited retinal dystrophies are rare and serious, ultimately leading to near-total blindness in those diagnosed with the disease.

Voretigene neparvovec is injected directly into the retina and works by introducing a healthy copy of the defective RPE65 gene into retinal cells, enabling people with the condition to produce functional RPE65 protein.

Evidence from the clinical trials shows that voretigene neparvoec improves vision and prevents the condition from getting worse. There is no long-term clinical evidence, but the committee considered it is biologically plausible that the treatment effect is likely to continue for decades. It is estimated that 86 people would be eligible for treatment with voretigene neparvovec in England.

The draft guidance is now with consultees, including Novartis Pharmaceuticals UK, healthcare professionals and patient/carer organisations. If no appeals against the draft are received, NICE expect to publish the final guidance next month.

To read the full article, see here.


Medicines and Healthcare products Regulatory Agency (MHRA)

Additional Guidance from the MHRA for a no-deal Brexit

The MHRA has added a new publication to their guidance on a possible no-deal Brexit scenario. The regulatory submissions and vigilance activities section has been updated with the guidance ‘exceptions and modifications to the EU guidance on good pharmacovigilance practices that will apply in a no-deal Brexit’. The guidance describes aspects of the EU guidance on good pharmacovigilance practice that will no longer apply to the MHRA and UK MAHs or are to be read subject to modification in a no-deal Brexit.

The Agency has also updated their guidance on the process of submitting regulatory notifications to the UK to include a User Reference Guidance for gaining access to MHRA submissions.

For further guidance from the MHRA on the regulation of medicines and medical devices in a possible no-deal Brexit, see here.


International Commission on the Clinical Use of Human Germline Genome Editing

National Academies of Science, Engineering and Medicine and Royal Society Consult on Human Genome Editing Framework

The US National Academies of Science, Engineering and Medicine (NASEM) and the UK’s Royal Society have convened to host an international commission to develop a framework for scientists, clinicians, and regulatory authorities to consider when assessing potential clinical applications of human genome editing. The framework will identify scientific, medical, regulatory and ethical requirements necessary for such applications.

The commission has issued a call for evidence including twelve questions pertinent to diseases and conditions appropriate to consider for applications of human germline genome editing, and what evidence would be necessary to support first‑in‑human use of the technology. The commission will consider submissions to the consultation ahead of a two-day workshop in London in November.

For further information on the first commission meeting held on 13 August 2019, see here. Further information on the Commission is available here.

Public Consultations