BioMarin Pharmaceutical’s acquisition of Amicus Therapeutics is a marriage of two rare disease biotech companies. Amicus’s two commercialized products, for the enzyme deficiencies Fabry disease and Pompe disease, are each projected to become blockbuster sellers.
BIO’s new president and CEO is a familiar face. John Crowley, a longtime BIO board member, was named the life sciences industry trade group’s new leader, starting in March.
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Sanofi dominates the market for therapies that treat Pompe disease, a rare enzyme deficiency. The FDA has approved an Amicus Therapeutics combination treatment for patients inadequately served by the Sanofi products.
The SPAC merger that Amicus Therapeutics planned for its gene therapy assets has been called off due to market conditions. The rare disease company is instead keeping those assets but streamlining its portfolio in order to save $400 million—the amount that the gene therapy spinout was in line to receive from the merger deal.
Amicus Therapeutics is spinning its gene therapy pipeline into a separate entity that will go public via a SPAC merger. Executives say the transaction puts Amicus on a path to profitability while simultaneously securing the cash needed to develop the gene therapy assets.
Amicus Therapeutics CEO John Crowley said the totality of data support the Pompe disease therapy’s submission to the FDA. That rolling application is on track for completion in the second quarter.
The company acquired 10 AAV-based gene therapies in development for lysosomal storage disorders, including two already in early clinical trials.
Renowned neurologist and author Oliver Sacks has died of cancer at the age of 82, Amicus Therapeutics has acquired a drug to treat a rare skin disorder through its purchase of Scioderm in a $229 million deal.
Amicus Therapeutics (NASDAQ:FOLD) has hired a biopharmaceutical industry veteran as chief financial officer of its business to develop treatments for lysosomal storage diseases and neurodegenerative diseases. William D. Baird III previously worked at PTC Therapeutics where he served for 10 years, seven as CFO, according to a company statement. Amicus’ lead drug undergoing phase 3 […]