The Sumitomo Dainippon-Roivant Alliance encompasses up to 11 biopharmaceutical Vants with more than 25 innovative clinical programs and multiple potential product launches from 2020 to 2022, and access to key elements of Roivant’s proprietary technology platforms including DrugOme and Digital Innovation.
Zogenix, with headquarters in Emeryville, California, announced the acquisition of Oakland, California-based Modis. Both companies focus on rare diseases.
Mallinckrodt Announces Positive Top-Line Results from Pivotal Phase 3 Study of Terlipressin in Patients with HRS-1
Mallinckrodt plc announced positive top-line results from the company’s pivotal Phase 3 CONFIRM clinical study evaluating the efficacy and safety of terlipressin in 300 adults with hepatorenal syndrome type 1 (HRS-1).
The U.S. FDA issued a new draft guidance for drugmakers aiming their resources at Fabry disease, a rare genetic disease.
The U.S. Food and Drug Administration accepted Alnylam Pharmaceuticals Inc.’s New Drug Application for givosiran – an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 in development for the treatment of acute hepatic porphyria – and granted Priority Review for the NDA.
The U.S. Food and Drug Administration approved Pfizer Inc.’s Ruxience (rituximab-pvvr) – a biosimilar to Rituxan (rituximab) – for the treatment of adult patients with non-Hodgkin’s lymphoma, chronic lymphocytic leukemia, and granulomatosis with polyangiitis and microscopic polyangiitis.
Rare diseases specialist Swedish Orphan Biovitrum (Sobi) agreed to buy the drug emapalumab and is reorganizing to increase focus on late-stage development in hematology and immunology.
Catalyst Pharmaceuticals Inc., under fire for a high price tag on a rare disease drug, sued the U.S. Food and Drug Administration to challenge the recent approval of a lower-cost rival medicine.
Pharma companies may not like their products being the subject of ICER reports, but they can provide a jumping-off point for manufacturers to expand the conversation of the value of new medicines in the rare disease area.
The first rule of rare: There are no rules. There are over 7,000 different rare diseases – each with its own symptomology, history, and clinical and political landscape. Most are virgin territory for marketers; only 5 percent have an approved treatment.