Regulatory news - August 2017

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

United Kingdom

UK government to provide new funding to support SMEs developing innovative medicines

As part of the UK government’s Accelerated Access Review (AAR) which aims to speed up access to innovative drugs, devices and diagnostics for NHS patients, £86 million funding for new medicine and technology has been recently announced to allow small and medium sized enterprises (SMEs) to develop and test new technologies.

In particular, the announcment by the Department of Health and the Department for Business, Energy and Industrial Strategy includes:

Up to £6 million over the next 3 years to help SMEs with innovative medicines and devices get the evidence they need by testing in the real world, building on existing opportunities such as the Early Access to Medicine Scheme (EAMS).

£80 million will also be allocated across Academic Health Science Networks (ASHNs), the Digital Health Technology Catalyst and the Pathway Transformation Fund aimed at helping NHS organisations to integrate new technologies into everyday practices.

Click here to view the government’s press release.


MHRA Offers Interactive Guide on New EU Device, IVD Regulations

In the last week, the UK MHRA has released a new interactive guide to support developers in understanding their obligations under the new EU regulations for medical devices (MDR) and in vitro diagnostics (IVDR) adopted by the European Commission on 5th April 2017.

These new requirements will transition over a 3 year period by 26th May 2020 for Medical Devices, and 26th May 2022 for In Vitro Diagnostic Medical Devices. MHRA notes that before this time, devices can be placed on the market under the current EU directives or the new regulations, if they comply.

In the guide, the MHRA provides guidance on classifying against the new risk classification criteria, requirements for conformity assessment, the new Unique Device Identifier (UDI) system for manufacturer tracing in high risk devices (e.g. heart valves and pacemakers) and new requirments for vigilance reporting.

Click here to view the interactive guide.

United States


FDA approves first gene therapy in the United States

The FDA has issued an early approval in the United States to Novartis for the first CAR-T cell therapy, marking the first approval of it’s kind globally. On the 30th August 2017, Kymriah (tisagenlecleucel) was approved for the treatment of patients up to 25 years of age with B-cell precursor ALL that is refractory or in second or later relapse.

Novartis was granted Priority Review and Breakthrough Therapy designations for Kymriah (originally CTL019) which it began to develop in 2012 through collaboration with the University of Pennsylvania. The Biologics License Application (BLA) was reviewed using a coordinated, cross-agency approach with the clinical review coordinated by the FDA's Oncology Center of Excellence. CBER conducted all other aspects of review and made the final product approval determination.

“Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research (CBER). “Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials.”

The FDA approval has been issued with a Risk Evaluation and Mitigation Strategy (REMS) by Novartis for handling of Cytokine Release Syndrome (CRS) and neurological events:

Because of the risk of CRS and neurological events, Kymriah is being approved with a risk evaluation and mitigation strategy (REMS), which includes elements to assure safe use (ETASU). The FDA is requiring that hospitals and their associated clinics that dispense Kymriah be specially certified. As part of that certification, staff involved in the prescribing, dispensing, or administering of Kymriah are required to be trained to recognize and manage CRS and neurological events.

Click here to view the press release from the FDA.

Click here to view the press release from Novartis.

Statement from FDA Commissioner outlines FDA perspective on regenerative medicines and stem cell therapy regulation

Scott Gottlieb recently released a statement outlining the FDA’s new policy steps and enforcement efforts to ensure proper oversight of stem cell therapies and regenerative medicines.

In his statement, the recently appointed FDA Comissioner writes:

“With all of the medical potential, also comes novelty and uncertainty as this field matures. There are a small number of unscrupulous actors who have seized on the clinical promise of regenerative medicine, while exploiting the uncertainty, in order to make deceptive, and sometimes corrupt, assurances to patients based on unproven and, in some cases, dangerously dubious products […] This puts the entire field at risk.”

In an effort to protect public health and accommodate the complexity of developing these therapies whilst continuing to facilitate innovation, the FDA has pledged to step up enforcement of regulatory compliance and develop the regulatory framework.

“The FDA will advance the new framework this fall […] The policies will be set forth in a series of guidance documents that are the result of a public process we have held in recent years. The new policy will build upon the agency’s current risk-based, flexible regulatory framework. It will also serve to implement provisions of the 21st Century Cures Act related to regenerative medicine.”

Click here to view the full press release.

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