At the current pace of innovation in the U.S. rare disease space, developing and approving therapies for just half of the 10,000-plus known rare diseases would take more than 160 years, Bradley Campbell, president and CEO of Amicus Therapeutics Inc., recently told the Senate Committee on Aging.
The BioWorld Biopharmaceutical Index finished 2025 up 24.07%, building slightly on its 23% gain recorded at the end of November. While the increase trailed the Nasdaq Biotechnology Index’s 32.4% rise for the year, it outperformed the broader Dow Jones Industrial Average, which advanced 12.97%.
The BioWorld Biopharmaceutical Index finished 2025 up 24.07%, building slightly on its 23% gain recorded at the end of November. While the increase trailed the Nasdaq Biotechnology Index’s 32.4% rise for the year, it outperformed the broader Dow Jones Industrial Average, which advanced 12.97%.
Biomarin Pharmaceutical Inc. followed up its May takeover of Inozyme Pharma Inc. with a much larger agreement to acquire Amicus Therapeutics Inc. for $14.50 per share in an all-cash transaction, tipping the equity-value scale about $4.8 billion.
Dimerix Ltd. sealed an exclusive license agreement with Amicus Therapeutics Inc. for commercialization of its phase III kidney disease candidate, DMX-200, in a deal valued at AU$940 million (US$601.22 million).
Dimerix Ltd. sealed an exclusive license agreement with Amicus Therapeutics Inc. for commercialization of its phase III kidney disease candidate, DMX-200, in a deal valued at AU$940 million (US$601.22 million).
John Crowley, a longtime industry executive whose story inspired a 2010 Hollywood movie, has been tapped for the role of president and CEO of the Biotechnology Innovation Organization (BIO), effective March 4, 2024, taking over from Rachel King, another longtime executive who has served as BIO’s interim CEO since October 2022.
After a nearly year-long delay pegged to COVID-19 travel restrictions, the U.S. FDA has approved Amicus Therapeutics Inc.’s Pompe disease drug, introducing competition for Sanofi SA’s standard-of-care treatment and anticipating blockbuster sales. The combination of Pombiliti (cipaglucosidase alfa-atga) and Opfolda (miglustat) 65-mg capsules was approved for adults with late-onset Pompe disease, who weigh at least 40 kg and who are not improving on their current enzyme replacement therapy.
The recent start of Neurogene Inc.’s first-in-human trial testing NG-101 in ceroid lipofuscinosis type 5 (CLN5) Batten disease (BD) drew investor attention to the space, where a handful of developers vie for new therapies against the challenging indication.
Amicus Therapeutics Inc.’s plan to spin off its gene therapy work by way of the combination with blank check firm Arya Sciences Acquisition Corp. IV “allows us to be laser-focused on maximizing the opportunity for Galafold [migalastat]” while setting up launch preparations for AT-GAA, said Bradley Campbell, chief operating officer.
