An analyst wrote that the deal would create value for Sarepta, which has a gene therapy in Phase II development for Duchenne muscular dystrophy, SRP-9001, but lacks the resources to commercialize it abroad.
The FDA had previously rejected Sarepta's application for Vyondys 53 in August, citing infections and kidney toxicity in preclinical studies. However, some analysts saw the move as a 'slap on the wrist' for the controversial approval of Sarepta's first DMD drug.
Munck Wilson Mandala Partner Greg Howison shared his perspective on some of the legal ramifications around AI, IP, connected devices and the data they generate, in response to emailed questions.
In a note to investors, SVB Leerink analyst Joseph Schwartz suggested that the 'questionable' approval of Sarepta's Exondys 51 may have played a role in the FDA's decision to turn down golodirsen.
The decision stood in contrast with the agency's highly controversial approval of Sarepta's previous DMD drug, Exondys 51. The company's shares fell sharply in after-hours trading.
Data from the drugmaker's Phase Ib study showed patients responding, but an analyst wrote that it appears less potent and to have higher toxicity than that of its competitor, Sarepta Therapeutics.
An analyst wrote that the therapy's combination of AAV delivery with a validated means of treating DMD could distinguish it from other gene therapies in what is already a crowded space.
At the Abarca Forward conference earlier this year, George Van Antwerp, managing director at Deloitte, discussed how social determinants of health and a personalized member experience can improve medication adherence and health outcomes.
After two painfully incremental drug approvals, the Duchenne muscular dystrophy field is still searching for a therapy that can substantially improve patients’ lives and lifespan. Sounds like a job for CRISPR/Cas9.
Emflaza, the brand name for deflazacort, has never been approved for sale in the United States, but the steroid has been sold for decades in other countries — at much lower prices
Marathon Pharmaceuticals is doing some backtracking after the public, the media and now Bernie Sanders objected to the list price of its upcycled corticosteroid for Duchenne muscular dystrophy.
The FDA has given the green light to Emflaza (deflazacort), a new drug for Duchenne muscular dystrophy. Announced Thursday, the news is bittersweet. Deflazacort is a corticosteroid, a class of drugs that doctors have used off-label for many years.
Gabby Everett, the site director for BioLabs Pegasus Park, offered a tour of the space and shared some examples of why early-stage life science companies should choose North Texas.
Exondys 51 doesn’t cure Duchenne muscular dystrophy and will only help a minority of patients.
Plus, health insurance startup Oscar to build concierge clinic in Arizona, AIDS Healthcare Foundation wants the FDA and Congress to investigate Gilead and eHealth hires its new CEO from Playboy.
As a whole host of new Duchenne Muscular Dystrophy therapies mature down the regulatory pathway, the global market for the degenerative disease is expected to expand at an incredible rate – from $8.2 million in 2014 to $990 million in 2019, or so says market research firm GlobalData. The present market’s got only paltry offerings to treat this […]
Multiple biological pathways go awry to cause disease. Cambridge startup Catabasis is developing a platform to simultaneously modulate multiple targets in a disease pathway, with a focus on diseases like Duchenne muscular dystrophy and ALS The startup’s in the midst of a $20.4 million fundraise, according to a recent SEC filing. So far, it’s raised about […]