The U.S. Food and Drug Administration approved Chiesi Global Rare Diseases’ Ferriprox (deferiprone) for the treatment of transfusional iron overload caused by sickle cell disease or other anemias in adults and children ages 3 years and older.
Shares of Avrobio were up in trading after the company posted positive clinical data from gene therapy trials in three different rare lysosomal diseases: Fabry, Gaucher type 1 and cystinosis.
Shares of Protalix Biotherapeutics were up in trading after the company announced the Phase III BRIDGE study met key objectives in treating Fabry Disease.
London-based Freeline announced the closing of a $120 million extended Series C financing to advance the company’s gene therapy programs, and is considering additional capital fundraising options during 2020.
Over the course of 2018, the FDA approved 59 different novel drugs that range for the treatment of various cancers, chronic obstructive pulmonary disease (COPD), traveler’s diarrhea, migraine headaches and more.
Shares of Cambridge, Mass.-based Avrobio plunged more than 50 percent after the company provided an updated look at a gene therapy for Fabry disease that showed promise for the treatment, but also raised some concern for investors.
Amicus Therapeutics inked a definitive agreement for 10 gene therapy programs licensed from Nationwide Children’s Hospital through the acquisition of Celenex.
Amicus Therapeutics set an average price of $315,000 per year for the company’s newly approved Fabry disease treatment.