Shares in Isreali group Protalix Therapeutics Inc were on the rise yesterday after a rare treatment protocol for its Gaucher disease drug prGCD (glucocerebrosidase) was accepted by US regulators, effectively allowing its pre-approval use in patients under an expanded access programme.
The company was asked to submit a treatment protocol for its experimental drug to the US Food and Drug Administration and did so last month, seeking approval to allow doctors to prescribe the therapy to patients with the disease in a multicentre, open-label trial throughout the expected drought of Genzyme’s Cerezyme (imiglucerase) and thereafter.
Shortages of Cerezyme on both sides of the Atlantic are looming from next month after viral contamination of a bioreactor forced the closure of Genzyme’s production plant in Boston in June, also affecting the manufacture of the group’s Fabry disease therapy Fabrazyme (agalsidase beta).
NDA by year end?
Protalix chief executive Dr David Aviezer said the company is “working closely with physicians and patient advocacy groups” to allow Gaucher disease patients access to its drug, and that the firm expects to conclude the Phase III pivotal study next month with top-line results in October, and an NDA filing by the end of the year.
UK drugmaker Shire also filed a treatment protocol for its enzyme replacement therapy velaglucerase alfa at the request of US regulators last month, which, if approved, will also enable doctors to prescribe this therapy before it becomes commercially available.
