The FDA approved San Diego-based Retrophin Inc.’s 100 mg and 300 mg tablets of Thiola EC (tiopronin) to treat cystinuria.
Roche is buying U.S.-based gene therapy specialist Spark Therapeutics for $4.3 billion after developments in this area convinced the Swiss drugmaker to “step up”, Chief Executive Severin Schwan said.
Spark Therapeutics licensed rights to its breakthrough gene therapy Luxturna outside the United States to Switzerland-based Novartis.
The FDA approved Spark Therapeutics’ treatment for a rare form of blindness, marking the first time the U.S. regulatory agency approved a gene therapy for an inherited disease.
Spark Therapeutics Inc.’s experimental gene therapy for a rare form of blindness improves vision and should be approved, advisers to the Food and Drug Administration concluded, paving the way for the first U.S. gene therapy for an inherited disease.