PTC Therapeutics Inc. (NASDAQ: PTCT) is experiencing significant momentum within the biotechnology sector following a surge in its stock price to a 52-week high of $40.69. This increase reflects heightened investor confidence driven by the positive clinical outcomes of its Friedreich ataxia (FA) investigational treatment, vatiquinone. Over the past year, the company’s share value has […]
Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company at the forefront of developing cell and exosome-based therapies, has shared encouraging three-year safety and efficacy results from its ongoing HOPE-2 open label extension (OLE) study. The study investigates the effects of deramiocel (CAP-1002), Capricor’s leading therapeutic candidate, on Duchenne muscular dystrophy (DMD), particularly focusing on cardiac aspects […]
In a significant advancement for the treatment of Duchenne muscular dystrophy (DMD), Italfarmaco S.p.A. has announced the U.S. Food and Drug Administration (FDA) approval of Duvyzat (givinostat), a novel histone deacetylase (HDAC) inhibitor, for patients aged 6 and older. This approval signifies a major milestone in the management of DMD, a rare and progressive neuromuscular […]
In a promising development for the treatment of rare diseases, Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a frontrunner in precision genetic medicine, announced that the U.S. Food and Drug Administration (FDA) has accepted and filed the company’s efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS (delandistrogene moxeparvovec-rokl), marking a pivotal moment in the battle against […]
IPS HEART, a private cell therapy company developing treatments for Duchenne muscular dystrophy and heart failure, announced that it has received a third Rare Pediatric Drug Designation (RPDD) from the US Food and Drug Administration (FDA). The designation is for their product candidate, ISX9-CPC, which is being developed to treat cardiomyopathy associated with Danon disease, […]
Sarepta Therapeutics’ SRP-9001 (delandistrogene moxeparvovec) has received the backing of the Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) of the US Food and Drug Administration (FDA) for accelerated approval for Duchenne muscular dystrophy (DMD). The FDA committee voted 8 to 6 in favor of the investigational gene therapy for the treatment of ambulatory patients […]
Sarepta Therapeutics said that it plans to file a biologics license application (BLA) with the US Food and Drug Administration (FDA) to seek accelerated approval for SRP-9001 (delandistrogene moxeparvovec) for the treatment of ambulant individuals with Duchenne muscular dystrophy (DMD). The investigational DMD gene therapy is being co-developed with Roche under a $2.85bn deal signed […]
ReveraGen Biopharma and Santhera Pharmaceuticals have secured a $1.2 million grant for funding their clinical trial of vamorolone in Becker muscular dystrophy (BMD) from the US Food and Drug Administration (FDA). The orphan grant funding has been issued under Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01) grants program. It adds […]
US-based PTC Therapeutics has secured marketing approval in Russia for Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). The approval for Translarna has been given by the Ministry of Health of the Russian Federation for Duchenne muscular dystrophy caused by a nonsense mutation in patients, aged two years and older. Stuart […]
Roche has signed a licensing deal worth up to $2.85 billion with Sarepta Therapeutics for the exclusive ex-US commercial rights to the latter’s SRP-9001 (AAVrh74.MHCK7.micro-dystrophin), an investigational gene therapy for Duchenne muscular dystrophy (DMD). As per the agreement terms, Sarepta Therapeutics will get an upfront payment made up of $750 million in cash and $400 […]