Regulatory news - July 2020

INTERNATIONAL

USA

US Food and Drug Administration (FDA)

FDA Approves First Cell-Based Gene Therapy for Adult Patients with Relapsed or Refractory MCL

The FDA has approved Tecartus (brexucabtagene autoleucel), a cell-based gene therapy for treatment of adult patients diagnosed with mantle cell lymphoma (MCL), who have not responded to or relapsed following other treatment regimes. Tecartus is a chimeric antigen receptor (CAR) T cell therapy and comes as the first cell-based gene therapy to be approved by the FDA for treatment of MCL.

Tecartus was approved under the Accelerated Approval pathway, receiving Priority Review and Breakthrough Therapy designations, in addition to Orphan Drug Designation.

For further information, see here.

FDA Extends Enforcement Discretion Policy for Certain Regenerative Medicine Products

The FDA has extended the enforcement discretion policy for certain human cell, tissue, and cellular and tissue-based products (HCT/Ps). Due to the current challenges faced by the COVID-19 pandemic, the enforcement discretion policy extension will allow manufactures and potential sponsors an additional six months to determine whether they need to submit an Investigational New Drug (IND) or marketing application, and to prepare such an application if needed.

The FDA have revised its guidance to reflect that FDA intend to exercise enforcement discretion with respect to IND and premarket approval requirements for certain HCT/Ps through to 31^st May 2021. The policy does not apply to products associated with reported safety concerns or that have the potential to cause significant safety concerns to patients.

For further information, see here. The updated guidance for industry can be found here.

Public Consultations

EUROPEAN COMMISSION (EC)