Hematology

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Sancilio Pharmaceuticals announced that it has received the European Medicines Agency’s Orphan Designation for its SC411 (which it plans to market as Altemia), a proposed treatment for sickle cell disease in pediatric patients, in the European Union. The FDA granted the proposed drug a similar designation—the Rare Pediatric Disease designation—in 2017.

This week, the top managed care stories included Cigna's deal to buy Express Scripts; Seema Verma, Alex Azar, and Scott Gottlieb, MD, made the rounds at health conferences; an analysis finds FDA's accelerated approval pathway has been a success with hematology and oncology drugs.

Reviewing 25 years of experience with accelerated approvals (AAs) for malignant hematology and oncology drugs and biologics, FDA officials say that the AA program has demonstrated that it can be used successfully to expedite approval of safe, effective cancer therapies that balance uncertainty with the need to provide faster access to promising agents for serious and life-threatening diseases.