Vertex/CRISPR's gene editing therapy cost effective at $1.9 million - pricing review group (updated)
12/04/2023 23:10
(Adds bluebird bio's reference in the analysis, adds name of
Vertex and CRISPR Therapeutics' therapy)
April 12 (Reuters) - Vertex Pharmaceuticals Inc VRTX.O
and CRISPR Therapeutics AG's CRSP.BN one-dose gene editing
therapy for sickle cell disease would be cost effective if
priced at up to $1.9 million, an influential U.S. drug pricing
group said on Wednesday.
The two companies are hoping to get approval for the world's
first therapy based on Nobel prize-winning CRISPR technology,
for two types of blood disorders - sickle cell disease (SCD) and
transfusion-dependent beta thalassemia.
They expect to potentially cure SCD through a single dose of
the therapy, exa-cel. SCD is a genetic disorder that causes
strokes, organ damage, severe pain and early death and affects
roughly 100,000 people in the United States.
In its draft report, the Institute for Clinical and Economic
Review (ICER) said a price range between $1.2 million and $1.7
million annually would meet some commonly used thresholds for
cost effectiveness, in the health care system perspective.
The analysis also refers to bluebird bio Inc's BLUE.O
sickle cell experimental gene therapy lovo-cel.
ICER is not a government agency and has no say on the drug
prices that manufacturers set, but many large health insurers
take its reports into account when they negotiate prices with
drug manufacturers and determine patient access.
Taking into account broader societal benefits of curing the
disease, ICER said a price range between $1.3 million and $1.9
million would be cost effective.
Gene therapies have been pricey and their list prices have
crossed the million-dollar mark in recent years.
Last year, Australian drugmaker CSL Ltd CSL.AX set the
list price of its gene therapy for blood disorder hemophilia B
at a record $3.5 million, while bluebird bio priced its gene
therapy for beta thalassemia at $2.8 million.
More than half a dozen companies are developing cutting edge
gene therapies for sickle cell disease after ignoring it for
decades, despite calls from patient advocates to do more.
SCD occurs among about 1 out of every 365 Black or
African-American births, according to the U.S. Centers for
Disease Control and Prevention estimates.
Novartis AG NOVN.S , Sangamo Therapeutics SGMO.O and
CRISPR Therapeutics CRSP.O and Vertex Pharmaceuticals VRTX.O
are some of the drugmakers working on a gene therapy for SCD.
(Reporting by Manas Mishra, Raghav Mahobe and Sriparna Roy in
Bengaluru; Editing by Shinjini Ganguli, Sonali Paul and Krishna
Chandra Eluri)
((Manas.Mishra@thomsonreuters.com;
www.twitter.com/Manaswrites15;))