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First Haemophilia Gene Therapy Licensed by EMA

First Haemophilia Gene Therapy licensed by European Medicines Agency

On Wednesday of this week, August 24th, it was announced that the European Medicines Agency ( EMA) have granted a conditional marketing authorisation to the company Biomarin for their FVIII Gene Therapy Valoctocogene Roxaparvovec (brand name: Roctavian).

Image of Biomarin press release

A conditional marketing authorisation (CMA) recognises that the medicine fulfills an unmet need based on a positive benefit-risk assesment. The CMA will also require the company to provide further data from ongoing studies within defined timelines to confirm that the benefits continue to outweigh the risks.

Here is a link to the press release announcement:,-BioMarins-ROCTAVIAN-TM-valoctocogene-roxaparvovec-,-Approved-by-European-Commission-EC

The conditional marketing authorisation was granted based on the results of the clinical trial. The Phase 3 (final phase) clinical trial data was published in March (on St. Patrick’s day!) in the New England Journal of Medicine.



In the Phase 3 clinical trial, data was reviewed on 132 people with haemophilia who had been treated with the Gene Therapy vector. One year after infusion, the average (mean) FVIII level was 42.9 IU/dl (42%) and the median (mid point) where half the people are above this level and half below) was 23.9 IU/dl. Looking at the FVIII  expression level achieved in more detail:

·         50 of the 132  (37.9%) people had a FVIII level of more than or equal to 40 IU/dl which put them in the normal range effectively.

·          66 people ( 50%) had a FVIII level between 5U/dl and 40 iu/dl which put them in the mild haemophilia range

·          16 people (12.1%) had a FVIII level of less than 5 IU/dl which put them in the moderate to severe range

The  mean number of bleeds per year decreased from 4.8 to 0.8 (the median annual bleed rate decreased from 2.8-0). This constitutes an 83.8% decrease in bleeds. FVIII use decreased by 98.6%.

It is now expected that the Gene Therapy will go on to be considered by national payers and Health Technology Assessment bodies in Europe. In Ireland, this will include the Haemophilia Product Selection and Monitoring Advisory Board.

-Brian O’Mahony