BioBlast gets FDA fast track nod for muscle disease drug

Israeli pharmaceutical company BioBlast Pharma Ltd.(Nasdaq:ORPN) has announced that it has received US Food and Drug Administration (FDA) approval for fast track clinical trials of Cabaletta, its leading product, for treatment of oculopharyngeal muscular dystrophy (OPMD), a rare muscular disease.

The fast track provides a company with better access to FDA staff in devising the trial procedures for products, with the aim of attaining more rapid approval when the trials results are submitted. Such approval is usually given only for diseases for which there is currently no treatment.

Founded by Dr. Dalia Megiddo and Udi Gilboa, BioBlast has acquired a number of technologies for treating genetically caused diseases. All the diseases that BioBlast is attempting to treat are rare diseases, but all the technologies it has acquired are designed for treatment of several such diseases, so the company's technological potential is designed to suit the general market. Its leading product is for OPMD, a deadly disease with several dozen patients in Israel and several hundred in the US. A Phase II/III trials is currently taking place in Israel and China, with the US scheduled for inclusion soon.

BioBlast's current market cap is $99 million. The company share has plummeted 37% since its IPO last July, when it raised $35 million. Several months ago, a new CEO, Colin Foster, was appointed for the company, replacing Megiddo, who had managed the company since it was founded.

Published by Globes [online], Israel business news - http://www.globes-online.com - on April 12, 2015

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BioBlast gets FDA fast track nod for muscle disease drug