WASHINGTON (Reuters) – U.S. drug regulators gave the nod on Tuesday to Protalix Biotherapeutics Inc and Pfizer Inc’s experimental biotech drug for type 1 Gaucher disease.
The Food and Drug Administration approved intravenous taliglucerase alfa (Elelyso) after rejecting it last year and asking the companies for more data.
Unlike most biotech drugs, which use cells from mammals, taliglucerase is the first to be made from plant cells, using genetically modified carrot cells.
A new drug could ease shortage worries in the $2 billion Gaucher market, which has been dogged by supply issues surrounding the leading therapy Cerezyme (imiglucerase), made by Sanofi SA unit Genzyme.
Gaucher disease stems from an enzyme deficiency that prevents the breakdown of certain fats in the body. It can cause organ damage or death and affects about one in 50,000 to 100,000 people. It is particularly prevalent among Ashkenazi Jews, according to the National Institutes of Health.
The FDA said Elelyso had been granted orphan status, meaning it treats a condition or disease that affects fewer than 200,000 people in the United States. Now that the drug is approved, the companies get seven years of marketing exclusivity.
The drug specifically treats Type 1 Gaucher disease, which affects about 6,000 people in the United States, the FDA said.
Pfizer said it would price the drug 25% below Genzyme’s Cerezyme, which sells for about $200,000 a year, according to Genzyme’s website.
Genzyme earlier this year said new U.S.-based manufacturing plants should ease supply constraints over its Gaucher treatment.
But Shire Plc, which makes a rival Gaucher drug called Vpriv (velaglucerase alfa), last week said it had failed to win FDA approval to manufacture its drug at a new facility in Lexington, Massachusetts.
Pfizer will try to minimize supply disruptions for patients by keeping 24 months of inventory on hand to serve all the 1,500 to 2,000 patients currently treated for Gaucher in the United States, the company told a briefing before the FDA’s approval.