Agios Anticipates PK Deficiency Diagnosis Ramp-Up After Pyrukynd Approval
The company hopes to grow the current diagnostic rate from 30% to 70% over two to five years. “Optimal reimbursement” for mitapivat will likely take a year, with Medicare/Medicaid lagging.
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Public Company Edition: PTC will get $350m up front as part of an initial $500m commitment. Also, while Biohaven and Vaxcyte launched traditional follow-on offerings, Recursion and Agios took the private placement and royalty sale routes, while others revealed job cuts and other strategic updates.
Novo Nordisk’s Forma Buy Builds On Foothold In Rare Blood Disorders
Novo's head of rare disease Ludovic Helfgott talked to Scrip about the $1.1bn acquisition and expansion into sickle cell disease.
Fueled By Connections: Making It Happen In Rare Disease
Agios’s Sarah Gheuens talks to In Vivo about the responsibility of providing new therapies to rare disease populations, building trust with historically underserved communities, and why she loves working with small molecules.