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BioMarin receives FDA nod for its would-be blockbuster

Published on 25/05/18 at 10:10am

BioMarin is confident that its newly approved treatment for phenylketonuria (PKU) is set to become a blockbuster and it’s priced the drug accordingly, at $488 per unit.

This might seem like a huge amount but when patients will eventually be using one or two units of pegvaliase per day, every day, that price starts to add up – meaning that cost per patient could end up between $170,000 and $340,000 each year.

PKU is a rare disease that sees patients experience phenylalanine deficiency, a condition that can cause brain damage if not managed by treatment. Around 50,000 people are diagnosed with the condition each year and pegvaliase will be available now to treat the more serious forms of the illness.

“BioMarin is thrilled to be able to offer this important new therapy to adults with PKU who are unable to control their Phe levels with existing options. The approval of Palynziq is the culmination of more than a decade of perseverance by BioMarin employees dedicated to bringing treatments to PKU adult patients,” said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin.  “We are proud of this medical achievement and appreciate the FDA's thoughtful review of our application.  We also are grateful to the PKU patients and medical communities for their continued partnership and participation in the clinical program that led to the approval of this effective therapy.”

BioMarin plans to be able to deliver the medication to patients in the US beginning in June, whilst it waits on the EMA’s decision on approval to reach patients in Europe.

Besides this approval, the biotech is also pushing through the development of much-hyped valrox, a treatment for haemophilia A – though recent Phase 1/2 data for the drug were not roundly well-received, after it displayed a lack of consistency in Factor VIII levels after one or two years of treatment.

The Phase 3 data will be watched closely to see whether BioMarin has another blockbuster on its hands.

Ben Hargreaves

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