Regulatory news - May 2020

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

UK

Medicines and Healthcare products Regulatory Agency (MHRA)

MHRA Regulatory Guidance on COVID-19

The MHRA continue to update their guidance for industry on the regulatory approaches being taken during the COVID-19 outbreak. Updates made to guidance include additional information to the following sections:

  • MHRA regulatory flexibilities resulting from coronavirus (COVID-19)
  • Managing clinical trials during Coronavirus (COVID-19)

See the MHRA website for the current guidance.

 

EU

European Medicines Agency (EMA)

EMA Approval Granted for Zolgensma

The gene therapy Zolgensma for the hereditary disease spinal muscular atrophy (SMA) developed by Swiss drugmaker Novartis has been granted European approval by the EMA.

The gene therapy has been approved for treatment of young children with SMA up to 21 kilograms and has also been approved in Japan.

Further information on Zolgensma is available here.

 

INTERNATIONAL

USA

US Food and Drug Administration (FDA)

FDA Extends Review of Bristol-Myers Squibb’s BLA for Lisocabtagene Maraleucel (liso-cel) for Adult Patients with Relapsed or Refractory Large B-Cell Lymphoma

Bristol-Myers Squibb have announced the FDA has extended the review of their CAR‑T treatment, called liso-cel, by three months, delaying the decision date to November 16, 2020. The extension of review timeline comes as a result of additional information requested by the FDA, which was deemed to constitute a substantial amendment to the initial BLA application. Bristol-Myers Squibb will work closely with the FDA to support the continued review of the BLA.

The investigational CAR T-cell therapy, lisocabtagene maraleucel (liso-cel) is used for treatment of adults with relapsed or refractory large B-cell lymphoma after at least two prior therapies.

To read the press release, see here.

 

FDA Refuses BLA Idecabtagene Vicleucel (ide-cel, bb2121) for the Treatment of Patients with Multiple Myeloma

The FDA issued a Refusal to File letter to Bristol-Myers Squibb and bluebird bio regarding the BLA for their cell therapy ide-cel, for patients with heavily pre-treated relapsed and refractory multiple myeloma.

Ide-cel is a B-cell maturation antigen (BCMA)-directed genetically modified autologous chimeric antigen receptor (CAR) T cell immunotherapy and was granted Breakthrough Therapy Designation (BTD) by the FDA. It has also been granted PRIority Medicines (PRIME) designation and Accelerated Assessment status by the EMA.

The Co-Developers of the cell therapy submitted the BLA in March 2020, and the FDA preliminary review determined that the Chemistry, Manufacturing and Control (CMC) module of the dossier requires further information. Bristol-Myers Squibb intend to resubmit the BLA by the end of July 2020.

To read the full press release, see here.

 

Thermo Fisher Invest in New Gene Therapy Plant

Thermo Fisher plans to invest $180 million in building a new site that will double the capacity for viral vector manufacture for gene therapies. The new facility is planned to complete in 2022 and reflects the growing demand for gene therapies and vaccines.

For more information, see here.

 

Public Consultations

EUROPEAN COMMISSION (EC)

 

Title

Consultation

Period

Category

1

Targeted stakeholders' consultation on Annex 21: Importation of medicinal products, of the Eudralex volume 4

20 Mar

To

20 June 2020

Draft Guidance

 

FOOD AND DRUG ADMINISTRATION (FDA)

 

Title

Consultation

Period

Category

1

Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations

28 Jan 2020

To

28 July 2020

Draft Guidance

 

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