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HepaStem: the somatic cell therapy product

 

HepaStem is a cell suspension constituted of Heterologous Human Adult Liver-derived Progenitor Cells (HHALPC) isolated from normal adult human liver tissue.

 

The Technology:

From a normal adult liver, a suspension predominantly constituted of liver parenchymal cells, is isolated by a proprietary process and further expanded in appropriate culture conditions to obtain billions of liver cells. Cryopreserved liver cells are then plated and cultured, progressively resulting in the emergence of a homogeneous progenitor cell population expressing both mesenchymal and hepatocytic markers.

 

These cells can be used to treat a wide variety of liver diseases, from rare inborn metabolic diseases (which can be classified as “orphan diseases” and mainly affect children), to acquired deficiencies such as acute alcoholic hepatitis or nonalcoholic steatohepatits in adults.

 

 

 

 

The Key Regulatory Milestones:

Status:

On July 10, 2007, HepaStem was classified as medicinal product by the European Medicines Agency (EMA), Committee for Medicinal Products for Human Use (CHMP), Innovation Task Force (ITF).

 

On November 29, 2007, an orphan drug designation was granted by the European Commission (EC) for Promethera HepaStem for the treatment of the Crigler-Najjar syndrome.

 

On February 04, 2008, an orphan designation was granted by the European Commission for Promethera HepaStem for the treatment of Ornithine Transcarbamylase Deficiency disease, one enzyme involved in the urea cycle deficiency.

 

On May 17, 2011, HepaStem was classified as advanced therapy medicinal product (somatic cell medicinal product) by the European Medicines Agency's Committee for Advanced Therapies (CAT).

 

On January 13, 2012, an orphan drug designation was granted by the Office of Orphan Products Development (OOPD) of Food and Drug Administration (FDA) for Promethera HepaStem the treatment of Urea Cycle Disorders.

 

On March 9, 2012, an orphan drug designation was granted by the Office of Orphan Products Development (OOPD) of Food and Drug Administration (FDA) for Promethera HepaStem the treatment of Crigler-Najjar syndrome.

 

On July 17, 2013, orphan drug designations were granted by the European Commission (EC) for HepaStem for the treatment of 7 forms of urea cycle disorders: N-acetylglutamate-synthetase deficiency, argininosuccinic aciduria, carbamoyl-phosphate-synthase-1 deficiency, citrullinaemia type 1, citrullinaemia type 2, hyperargininaemia, ornithine-transcarbamylase deficiency. 

 

 

Scientific Advices:

Scientific advices from EMA are received for the overall development of Promethera® HepaStem.

 

Year

EMA

Scientific Advice/Protocol Assistance on :

2009

EMA

Non-clinical development program

2010

EMA

Quality development program

2012

EMA

Non-clinical development program (follow-up)

2013

EMA

Non-clinical development program (follow-up)

2015

EMA/HTA

Clinical development program

 

Consultations with national competent authorities are made in line with the clinical studies.

 

Year

National Competent Authorities

Scientific Advice on :

2010

FAMHP

Non-clinical development program

2011

FAMHP

Quality aspects for approval of Phase I/II

2011

PEI

Clinical development program of Phase I/II

 

MHRA

Clinical development program of Phase I/II

 

FAMHP

Non-clinical development program of Phase I/II

 

 
Clinical trials:

Promethera HepaStem Phase II

 

HepaStem is currently in Phase II clinical development for the treatment of urea cycle disorders. The trial, a prospective, open label, multi-country study is designed to evaluate the efficacy and safety of several infusions of HepaStem in Urea Cycle Disorders pediatric patients. Approval has been obtained from the Belgian, French and Spanish Regulatory Authorities.

 

Promethera HepaStem Phase I/II

 

HepaStem was primarily tested in a European multi-country Phase I/II clinical trial. The trial, a prospective, open label, multicenter study was a dose escalation study designed to evaluate the safety and the preliminary efficacy of HepaStem in Urea cycle disorders and Crigler-Najjar Syndrome pediatric patients. The clinical trial was conducted in Belgium, France, United Kingdom, Italy and Israel. The study is complete.

 

 

Contacts

PROMETHERA Biosciences S.A./N.V.
Watson & Crick Hill
Rue Granbonpré, 11
B-1435 Mont-Saint-Guibert
(Belgium)
Tél.: +32 (0)10 39 43 00
Fax: +32 (0)10 39 43 01

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