The European Medicines Agency (EMA)'s Committee for Medicinal Products for Human Use (CHMP), has recommended against issuing marketing authorization for Taliglucerase Alfa, an enzyme replacement treatment for Gaucher disease.

Gaucher disease is estimated to affect some 1 in 50,000 to 1 in 100,000 people in the general population. People from Eastern and Central Europe (Ashkenazi) of Jewish heritage, are at highest risk. In short, it is caused by dysfunctional metabolism of sphingolipids. The disorder is characterized by bruising, fatigue, anemia, low blood platelets, and enlargement of the liver and spleen. It is caused by a hereditary deficiency of the enzyme glucosylceramidase There are three main subtypes of Gaucher disease:
 

• Type 1 disease is most common. It involves bone disease, anemia, an enlarged spleen and thrombocytopenia. Type I affects both children and adults. It is most common in the Ashkenazi Jewish population.
• Type 2 disease usually begins in infancy with severe neurologic involvement. This form can lead to rapid, early death.
• Type 3 disease may cause liver, spleen, and brain problems. Patients may live into adulthood.

The disease is caused by a recessive mutation in a gene located on chromosome 1 and affects both males and females. It is an autosomal recessive disease. This means that the mother and father must both pass one abnormal copy of the gene to the child in order for the child to develop the disease. A parent who silently carries an abnormal copy of the gene is called a carrier.

The CHMP did however give a positive risk-benefit assessment for taliglucerase alfa saying that the benefits of the medicine outweighed its risks in the treatment of Type 1 Gaucher disease. The Committee said it could not give Marketing Authorization at this time, because another company, Shire, has a drug called velaglucerase alfa, a similar treatment, and already received prior Marketing Authorization with orphan drug designation for the same condition. Pfizer made a request for derogation from Shire's orphan market exclusivity based on a number of factors, however the EMA denied their application.

Diem Nguyen, General Manager Biosimilars said:

 

"While we are disappointed by the CHMP's recommendation, we are encouraged that the Committee gave a positive risk-benefit assessment. The recommendation was based solely on orphan market exclusivity and not the safety and efficacy profile of taliglucerase alfa ... Pfizer will continue to work with relevant stakeholders to determine appropriate next steps."

Pfizer and Protalix said they are dedicated to the treatment of Gaucher disease worldwide and will continue to move forward with other global regulatory filings for taliglucerase alfa. Taliglucerase alfa, known under the brand name ELELYSO, was approved by the U.S. Food and Drug Administration (FDA) on May 1, 2012 for the long term enzyme replacement therapy (ERT) of adults with a confirmed diagnosis of Type 1 Gaucher disease. The drug therefore works, it's just a question of Pfizer being pipped to the post by Shire in the EU's domain.

Pfizer and Protalix BioTherapeutics, Inc. have been working together since 2009, to develop and commercialize taliglucerase alfa. Under the agreement, Pfizer received exclusive worldwide licensing rights for the commercialization of taliglucerase alfa, while Protalix retained the exclusive commercialization rights in Israel.

 

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