FDA OKs Testing of OPMD Drug in US

Israel-based BioBlast Pharma says a California site will be added soon to locations already testing Cabaletta in oculopharyngeal muscular dystrophy in Israel and Canada

Article Highlights:
  • BioBlast Pharma is developing and testing Cabaletta, a chemical chaperone designed to prevent abnormal protein clumping in cells, as a potential treatment for oculopharyngeal muscular dystrophy (OPMD).
  • A trial is underway in Israel and Canada, and a site in California will open this spring, following a recent green light from the U.S. Food and Drug Administration.
by Margaret Wahl on March 17, 2015 - 2:31pm

BioBlast Pharma, based in Tel Aviv, Israel, has been given clearance from the U.S. Food and Drug Administration (FDA) to test its experimental drug Cabaletta in the U.S. in people with oculopharyngeal muscular dystrophy (OPMD). In a March 12, 2015, press release, the company announced that it plans to add a California site during the second quarter of this year to the Cabaletta in OPMD trial sites already up and running in Canada and Israel.

Cabaletta is a "chemical chaperone," designed to prevent abnormal aggregation (clumping) of proteins inside cells, a phenomenon thought to contribute to muscle weakness in OPMD patients. The ultimate goal of Cabaletta treatment is to sustain or improve function, particularly in the muscles around the eyes and the swallowing muscles, which are the most affected in OPMD.

"The FDA's clearance to conduct a phase 2-3 clinical trial for OPMD is a critical step in advancing our development plan," said Colin Foster, president and CEO of BioBlast, adding, "We believe the Cabaletta platform can be an effective treatment for thi devastating disease that would bring hope to the patients and their families."

For more information

For details and contact information about the OPMD trial, see Safety, Tolerability and Efficacy Study of Cabaletta to Treat OPMD, or enter NCT02015481 in the search box at ClinicalTrials.gov. See also See BioBlast is Developing an OPMD Drug.


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