Regulatory news - December 2019

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


European Commission (EC)

EC Provides Clarity on Clinical Trial Authorisation with Conditions

The EC has updated its questions and answers (Q&A) document on the Clinical Trial Regulation to explain what is means to receive a clinical trial authorisation, subject to conditions.

The Q&A clarifies that an initial clinical trial application, substantial amendment or addition of a Member State concerned can be authorised, authorised subject to conditions or rejected.

Where an application is authorised subject to conditions, the EC explains that such authorisations are “restricted to conditions which by their nature cannot be fulfilled at the time of that authorisation”, and setting a condition is “only possible in case of an application with a positive benefit/risk balance”. For instance, such conditions may include a “request for additional data not available at the time of the authorisation, e.g. data needed for later trial parts, but not preventing the start of the trial”.

To read the updated Q&A document, see here.

European Medicines Agency (EMA)

December 2019 EMA Management Board Meeting Highlights

The EMA Management Board meeting held in December was hosted by the Dutch government and covered defining the work programme for 2020 and beyond.

A key topic for discussion was the EU IT system required by the Clinical Trial Regulation. An update on the progress was provided, including the outcome of the ‘audit readiness assessment’ conducted by nominated EU Member State authorities, the EC and sponsors, as well as the EMA and IT supplier to identify critical items requiring further development prior to the formal audit. The Board endorsed the proposal to commence the audit in December 2020.

For full details of the discussions held at the December meeting, see here.



US Food and Drug Administration (FDA)

FDA Finalises Adaptive Trial Designs Guidance

The FDA finalised guidance on adaptive clinical trial designs for drugs and biologics, which sets out FDA recommendations on adaptive trial design principles and the information to be submitted for review as part of an investigational new drug application (IND), new drug application (NDA), biologics license application (BLA) and other supplemental applications.

The final version has not been drastically changed since the draft version released for consultation in September 2019; the main changes being additional clarity on recommendations for Bayesian adaptive trial designs, and the extent of pre-specification required for governing adaptations to studies.

For further information, see here. The final guidance is available here.

FDA Debates Creating a Pilot to Evaluate Novel Excipients

The FDA are proposing to create a voluntary pilot program for the evaluation of toxicology and quality of novel excipients intended for use in human medicines. The Agency have requested feedback from industry on aspects of the program prior to deciding whether to continue to development.

The FDA currently review novel excipients as part of an investigational new drug application (IND) or marketing application (NDA or BLA), but stakeholders have encouraged the agency to establish a program for the submission and review of toxicological and quality data for novel excipients outside of the context of an IND, NDA or BLA.

For more information, see here.

FDA Warns Companies for Offering Unapproved Stem Cell Products

Two California-based companies (Liveyon Labs Inc. and Liveyon LLC) have been warned by the FDA for processing and distributing unapproved stem cell products derived from umbilical cord blood. The companies have also been warned for “deviations from current good tissue practice (cGTP) and current good manufacturing practice (cGMP) requirements, including deficient donor eligibility practices, inadequate aseptic practices to prevent contamination and deficient environmental monitoring”.

Earlier in the month, two Florida-based companies (RichSource Stem Cells and Chara Biologics) also received warning letters for offering unapproved stem cell products to patients.

To read the full press release, see here.

FDA Drafts Guidance on Bridging Data for Combination Product Applications

The FDA issued a draft guidance aimed at sponsors of drug-device and biologic-device combination products, explaining use of bridging data from earlier stages of development or other programmes to support an application. The guidance includes three example scenarios:

  • Bridging within an IND from a drug developed in a prefilled syringe to a drug developed in an autoinjector;
  • Bridging from one autoinjector (prototype 1) to another autoinjector (prototype 2) for the same drug; after Phase 3 studies have been completed but before NDA submission;
  • Bridging of data from combination product that employs the same device combined with a different drug.

The draft guidance is out for consultation until 18th February 2020. For more information and to download the draft guidance, see here.

Japan’s Astellas to buy Gene Therapy Developer Audentes for $3 billion

Astellas Pharma Inc. is buying US drug maker Audentes Therapeutics Inc. for approximately $3 billion, in a push to make genetic medicines a key area of growth. The acquisition will result in Astellas obtaining Audentes gene therapy programmes, in addition to their proprietary manufacturing knowhow in gene therapy.

Genetic drugs will become a fifth primary focus for Astellas, joining their existing business lines in regenerative, immuno-oncology, immunotherapy and neuromuscular medicine.

For more information, see here.

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