Priority review for Gaucher drug
pharmafile | December 12, 2013 | News story | Sales and Marketing | EMA, FDA, Gaucher, Genzyme, Sanofi
The FDA is to conduct a speedy investigation into Sanofi’s twice-daily oral candidate for Gaucher disease, which means that Cerdelga is now being looked at in both the US and Europe as a possible alternative to enzyme replacement therapy.
The US regulator has given Cerdelga (eliglustat) a six-month priority review designation – cutting the standard review time by four months – while the EMA accepted a marketing authorisation application for the drug in October.
Gaucher disease is a rare inherited disorder affecting fewer than 10,000 people worldwide, in which patients do not have enough of an enzyme called glucocerebrosidase, which normally breaks down a fat called glucocerebroside.
Without the enzyme glucocerebroside builds up in the body, typically in the liver, spleen, bone and some other tissues, causing a wide range of problems such as anaemia, tiredness, easy bruising and a tendency to bleed, an enlarged spleen and liver, and bone pain and fractures.
Type 1 is the most common form of Gaucher disease and usually does not affect the brain.
Developed by Sanofi subsidiary Genzyme – itself a veteran of the Gaucher market – Cerdelga is a novel, oral glucosylceramide analogue, which is designed to partially inhibit the enzyme glucosylceramide synthase, resulting in reduced production of glucosylceramide.
“The acceptance of our applications for Cerdelga represents another important milestone in our commitment to understand and respond to the needs in the Gaucher community, providing more choice for the treatment of patients,” said Genzyme’s chief executive David Meeker.
The applications are based on two Phase III studies for the new pill, ENGAGE (which included patients new to therapy) and ENCORE (including those switching from enzyme replacement therapy).
Sanofi’s case for Cerdelga also includes four years’ worth of safety and efficacy data from Phase II study.
Pfizer’s Elelyso (taliglucerase alfa) is currently approved by the FDA to treat the disease in the US.
Market exclusivity for orphan medicines is given as an incentive for companies to develop medicines for rare diseases, which may otherwise not be developed due to the high costs and small patient populations – although treatment in this field is expensive, making it a potentially lucrative therapy area for successful manufacturers.
Elelyso was refused marketing authorisation by a European committee because a competitor drug, Shire’s Vpriv (velaglucerase alfa), was authorised in 2010 for the same condition.
Both are enzyme replacement therapies that work in the same way – and Sanofi hopes Cerdelga, with its novel action, will offer another option for doctors.
Adam Hill
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