Regulatory news - November 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 Offers Further Guidance on Clinical Trial Regulation

The EC have updated their Questions & Answers (Q&A) guidance on the Clinical Trials Regulation (EU) No. 536/2014; with the addition of three new Q&A’s on the following:

  • How requests for information (RFI) will be managed throughout the clinical trial application assessment process (question 2.7);
  • Timing of when the assessment report on part I and II will be made publicly available (question 2.8);
  • Sponsor responsibilities regarding changes to a clinical trial which are not considered as substantial modifications but are relevant for the supervision of the trial (question 3.3).

Further updates of this draft Q&A document will be published progressively as more discussions are held with the Expert Group on Clinical Trials prior to application of the Clinical Trials Regulation.

See here for draft Q&A document.

European Medicines Agency (EMA)

Early Dialogue with Regulators Can Make a Difference for Faster Patient Access to Highly Innovative Therapies

The chairs’ of the EMA’s committee for advanced therapies (CAT), human medicines committee (CHMP) and the head of the EMA’s scientific advice office have produced a perspective document discuss how early dialogue and frequent interactions between medicines developers, regulators, health technology assessment bodies (HTAs) and patients play a key role in delivering robust data needed to enable accelerated access to innovative therapies.

The perspective piece was published in Clinical Pharmacology & Therapeutics and draws on experience from the recent Zynteglo (lentiglobin) authorisation. Zynteglo benefitted from the EMA’s platform PRIME, which provides opportunity for early and enhanced dialogue with developers of promising new medicines that address an unmet need and several interactions with the EMA’s scientific advice office with input from patient representatives. These interactions lead to development of a more robust application package demonstrating the medicines benefits and risk, allowing an accelerated assessment.

The article also discusses key clinical and manufacturing process challenges and how these were overcome and emphasises that early accelerated approvals are only possible if a robust post-approval plan is identified at marketing authorisation stage.

For further information see the EMA announcement and the open access article is available here.

Agence Nationale de Sécurité du Médicament et des Produits de Santé (ANSM)

France to Maintain Fast Track Schemes to Accelerate Clinical Trials

The ANSM has confirmed it will maintain their two fast track procedures with the implementation of the new Clinical Trial Regulation, expected to take effect in spring 2020.

The two fast track options were established last year and extended to advanced therapy medicinal products (ATMPs) in February 2019, provide the opportunity for innovative clinical trial applications to be processed in up to 40 days (110 days for ATMPs) and up to 25 days for known drugs (60 days for already known ATMPs).

ATMP trial applications previously took 180 days to be reviewed and accepted and the ANSM have communicated that early tests of the fast track procedures have proven successful.

See more information on the ANSM website here.



US Food and Drug Administration (FDA)

FDA Publishes Instructions for the Use of the Electronic Human Cell and Tissue Establishment Registration System (eHCTERS)

Manufacturers of Human Cells, Tissues and Cellular and Tissue-Based Products (HCT/Ps) are required to register and list their HCT/Ps with the FDA using eHCTERS.

The FDA have published instructions for how to use the eHCTERS to submit an initial registration, update a registration annually, change information and/or inactivate a registration.

See here for instructions.

Regenxbio Sues FDA Over Clinical Hold on Gene Therapy RGX-314 for Wet AMD

The gene therapy company Regenxbio Inc. is taking the FDA to court over a clinical hold on the company’s two INDs. The FDA notified Regenxbio that its two INDs were on a full clinical hold in October without notice or explanation and the company have since learned that the clinical hold is due to “issues associated with their delivery systems”.

Regenxbio has raised a lawsuit against the FDA, which mainly focuses on grievances specific to the handling of the INDs, but it also covers other topics with more significant implications. The complaint refers to sections of the Food, Drug and Cosmetic Act (FDC Act) 21 U.S.C. §355(i)(3)(B)(ii); FDC Act §505(i)(3)(B)(ii), which allow the FDA to issue a clinical hold for a reason established by the regulation that is other than a determination that the drug involved represents an unreasonable risk to subject safety.

For further information, the lawsuit from Regenxbio is available here.

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