2019-11-11_Bloomberg_Businessweek

(Steven Felgate) #1

B U S I N E S S


1


18


Edited by
James E. Ellis

● A costly targeted gene
therapy shows the promise and
pitfalls of medical advances

A $1.8 Million Drug


To Treat Sicily’s Curse


On a sweltering summer morning, Daniela Miccichè
headed from her home in the center of Sicily to a hos-
pital in the ancient city of Palermo on the coast. It’s a
trip she repeats every month. A travel agent from the
town of Caltanissetta, Miccichè has no choice but to
make the two-hour journey to the Franco and Piera
Cutino clinic. Scores of others pass through its front
doors, all with the same disease. She’s among an
estimated 7,000 Italians, many of them Sicilian, who

dependontransfusionstotreatbetathalassemia,a
geneticdisorderthathampersredbloodcells’ability
to carry oxygen.
Miccichè has waited a long time for the day she
can be free from these procedures, a constant in
her life since she was 3 years old. “I grew up think-
ing that one day we would reach this goal,” she says.
PatientssuchasMiccichèareclosetohaving
theirwishgranted,butit’sgoingtocosttheItalian
medicalsystema hugesum.A novelgenetherapy
from U.S.-based Bluebird Bio Inc., approved in
EuropeinJune,issettoberolledoutnextyear
following reimbursement negotiations with
government health systems. The treatment, called
Zynteglo, has a sophisticated mechanism and a

ILLUSTRATION BY REBEKKA DUNLAP

Bloomberg Businessweek November 11, 2019
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