Novartis received the U.S. Food and Drug Administration’s Breakthrough Therapy tag for the company’s experimental medicine iptacopan, which the Swiss drugmaker hopes wins approval to treat the rare blood disorder paroxysmal nocturnal hemoglobinuria.

The FDA approved San Diego-based Retrophin Inc.’s 100 mg and 300 mg tablets of Thiola EC (tiopronin) to treat cystinuria.