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BioMarin prepares for haemophilia A gene therapy data readout, filing

Key long-term data expected in July

BioMarin has recorded strong growth for its full 2018 results, as it prepares to file a first-in-class gene therapy for haemophilia A later this year.

The San Rafael, California-based rare disease company recorded an overall loss last year of $77m, but is confident of breaking into profit within a few years as its portfolio grows.

Its late-stage haemophilia A therapy, valoctocogene roxaparvovec (valrox) could be a multi-billion dollar success, if it can prove that it can effectively cure many patients of the bleeding disorder.

The key test will come this year when the company releases phase 2 data which it hopes will confirm the AAV gene therapy's long term durability and safety.

The company has rivals close behind it – Spark and Pfizer and Sangamo, but BioMarin could gain the upper hand this year if its data is robust. Spark suffered a safety setback in August last year, handing an advantage to BioMarin, with early data suggesting its therapy is superior at increasing levels of blood-clotting Factor VIII.

BioMarin already has six rare disease drugs already on the market, including its biggest earner Morquio syndrome treatment Vimizim, which reached $482m in revenues in 2018, up 17% and powering a 14% in total revenues of $1.5bn.

The company expects to hit revenues of $2bn by 2020, helped on by another new product, phenylketonuria therapy Palynziq, which is also forecast to eventually hit peak annual sales of up to $1bn.  The product was launched in the US last year and BioMarin expect a recommendation from Europe’s CHMP imminently.

However Valrox is undoubtedly generating the most anticipation, and the company says it now expects to submit it to the FDA via a potential accelerated approval, which it must apply for by the end of this quarter.


Chief executive Jean-Jacques Bienaimé said:

"In May, we provided two years of clinical data from the Phase 2 study with the 6e13 vg/kg dose of valoctocogene roxaparvovec gene therapy for severe haemophilia A that demonstrated the elimination of need for prophylaxis and no spontaneous bleeds. In addition, we amended the protocol of the global GENEr8-1 (phase 3) pivotal study by increasing the number of participants from 40 to 130 in order to evaluate superiority compared to the current standard of care. We now anticipate completing enrollment during the third quarter of 2019.”

Bienaimé says the company will decide in the second half of 2019 whether or not it will pursue an accelerated approval path.

This could see Valrox reach the market by 2020, which should put it significantly ahead of rival products from Spark and Pfizer/Sangamo.

The company plans to present detailed three-year Phase 2 valrox data at the International Society on Thrombosis and Haemostasis (ISTH) congress in July.

Based on the life time cost of treating haemophilia in the US being easily in excess of $1bn, analysts at Credit Suisse last year forecast that that a haemophilia gene therapy could command a US net price of $1.5m or higher.

However as there are many existing treatments which manage the condition, analysts believe not all patients will opt for gene therapy.

There is also a significant proportion of patients who have a pre-existing antibodies which will neutralise the AAV gene vector that BioMarin’s product relies on.

Article by
Andrew McConaghie

22nd February 2019

From: Sales



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