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Under its newly agreed acquisition, Sanofi is to acquire Inhibrx, Inc., adding INBRX-101 to its portfolio, a therapy that could improve the treatment options available for alpha-1 antitrypsin deficiency (AATD).
In this exclusive Q&A with European Pharmaceutical Review, Sheila Ann Mikhail, co-founder of AskBio and keynote speaker at CPHI Barcelona delves into the current landscape and future potential of the gene therapy sector. She explores strategies for making gene therapies more affordable and accessible, innovations in drug delivery, as well…
When CTI BioPharma is acquired by Swedish Orphan Biovitrum AB (Sobi), the companies intend to advance an oral kinase inhibitor treatment for a rare haematological disease.
Takeda’s largest manufacturing capacity expansion investment in Japan will fund the build of a new facility for plasma-derived therapies.
A PEGylated enzyme replacement therapy for Fabry disease has received recommendation for a marketing authorisation from the European Medicines Agency (EMA)'s human medicines committee.
Driven by the approval of pipeline agents, mRNA non-vaccine therapeutics are set to reach $2 billion in sales by 2028, says GlobalData.
According to a new report, the orphan drug market is growing more than twice as fast as the non-orphan market and, by 2026, orphan drug sales will account for 20 percent of all prescription drug sales.
The BIA is committed ensuring patient access to treatments for rare and very rare conditions, sometimes referred to as orphan and ultra-orphan medicines...