In-licensing and development of so-called "bio-betters" will play critical roles in Pfizer's move, announced June 14, into the rare diseases space. The Rare Diseases Research Unit, to be overseen by Jose Carlos Gutierrez-Ramos, senior VP, Biotherapeutics Research and Development, will be part of Pfizer's worldwide R&D unit devoted to discovering novel medicines for orphan diseases, those affecting fewer than 200,000 patients. Initially it will target hemophilia and muscular dystrophy, both caused by genetic mutations
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Advanz Pharma would have had to show that the European Commission’s decision to revoke Ocaliva’s conditional marketing approval risked causing serious and irreparable harm, according to lawyers from Van Bael & Bellis.
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A new pilot aims to take Brazil closer to ‘digital transformation.’
While treatments for scleroderma are in development, none are approved for the condition that is associated with high morbidity and mortality. The European Medicines Agency says guidance on how to design development programs for the disorder is needed.
Madrigal Pharmaceuticals’ Rezdiffra is on track to become the first medicine approved in the EU for non-cirrhotic metabolic dysfunction-associated steatohepatitis after the European Medicines Agency recommended that it be granted conditional marketing authorization.
Highlights from Day Four of the BIO International Convention include policy concerns helping constrain dealmaking, Novartis discussing its approach to partnering, and Generate looking for funding to move into Phase III.
