Cardiovascular disease
In its first commercial quarter for ATTR-cardiomyopathy, Alnylam’s Amvuttra reached roughly 1,400 patients and made more than $490 million.
The FDA greenlit multiple new drugs this month and issued some notable label expansions, including for Eli Lilly’s Kisunla. Meanwhile, the regulator turned away a cell therapy for Duchenne muscular dystrophy and a gene therapy for the rare disease Sanfilippo syndrome.
While Eli Lilly’s diabetes blockbuster was non-inferior to its older incretin therapy Trulicity in a highly anticipated Phase III cardiovascular outcomes trial, analysts had hoped for statistical significance in reducing major events such as cardiovascular death, heart attack and stroke.
Against steep odds and well-established paradigms, these four companies have successfully been commercializing their products on their own.
The strategic reprioritization comes after the company hit two major hurdles in the past year, including a clinical hold for an investigational gene therapy and an FDA rejection for its lead asset.
The drug came to AstraZeneca through its acquisition of CinCor Pharma in 2023, with the hopes of beefing up its cardiovascular and kidney disease pipelines.
Ekterly’s road to approval was not a smooth one. Last month, the FDA informed KalVista it would not meet its PDUFA date due to resource constraints and reports surfaced that Commissioner Marty Makary tried to have the application rejected.
Flagship Pioneering’s ProFound Therapeutics will use its proprietary technology to mine the expanded proteome for novel cardiovascular therapeutics. Novartis has promised to pay up to $750 million per target, though it has not specified how many targets it will go after.
Andembry is the first monthly Factor XIIa inhibitor for the prevention of HAE attacks and will be available to patients “before the end of June,” CSL announced.
The pending deal was rumored overnight after a report from the Financial Times, spurring analysts to speculate that if true, the entire gene editing space would see a boost at the markets.
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