logo
  

BBIO : FDA Grants Fast Track Designation For Therapy To Treat Limb-girdle Muscular Dystrophy Type 2i

BridgeBio Pharma Inc. (BBIO) said Wednesday that the U.S. Food and Drug Administration granted Fast Track designation for the investigation of BBP-418 as a treatment option for Limb-girdle Muscular Dystrophy Type 2i or LGMD2i.

BridgeBio's LGMD2i investigational therapy is one of the company's 14 programs that are in the clinic or commercial setting for patients living with genetic diseases and genetically-driven cancers.

LGMD2i is an inherited recessive muscular dystrophy caused by mutation of fukutin-related protein (FKRP). FKRP is a critical enzyme that adds a specific sugar molecule to a muscle cell structural protein called alpha-dystroglycan (aDG).

Due to defective FKRP enzyme function, muscle cells of patients affected by LGMD2i lack a robust cushioning system that is provided by fully glycosylated aDG proteins.

Pediatric and adult patients with LGMD2i most commonly present with upper and lower extremity ("limb") and thoracic ("girdle") dysfunction , and without treatment often develop additional severe clinical manifestations, including loss of independent ambulation, severe breathing issues which can require mechanical ventilation, cardiomyopathy and premature death.

For comments and feedback contact: editorial@rttnews.com

Business News

Editors Pick
CVS Health is set to recruit qualified candidates to fill 25,000 clinical and retail jobs during a one-day national career event on Friday, September 24. This will enable CVS to meet the high demand for pharmacists, pharmacy technicians, nurses, and retail store associates to support flu season, COVID-19 vaccinations and testing. Twitter, Inc. (TWTR) announced Monday that it has entered into a binding agreement to settle the consolidated class action lawsuit commenced in 2016 in the United States District Court for the Northern District of California. Under the terms of the proposed settlement, Twitter will pay $809.5 million... Drug major Pfizer has again expanded the earlier two recalls of its anti-smoking Drug Chantix (Varenicline) in the form of tablets, to now include all the lots, for the potential presence of nitrosamine impurity, N-Nitroso- varenicline, at or above the FDA interim acceptable intake limit (ADI), the U.S. Food and Drug Administration or FDA said in a statement.
Follow RTT