* Approval based on NETTER-P trial in which Lutathera demonstrated a

    consistent safety profile and comparable drug exposure between pediatric
    (ages 12-17) and adult patients
  * Gastroenteropancreatic neuroendocrine tumors (GEP-NETs) are a rare cancer
    that is often unresectable and commonly diagnosed in the late stages of
    disease
  * Novartis, a leader in radioligand therapy (RLT), is investigating a
    portfolio of RLTs to treat a broad range of cancers, including GEP-NETs,
    lung, prostate, breast, colon, brain and pancreatic cancers

Basel, April 23, 2024 - Novartis today announced that the U.S. Food and Drug
Administration (FDA) approved Lutathera(®) (USAN: lutetium Lu 177 dotatate /
INN: lutetium (177Lu) oxodotreotide) for the treatment of pediatric patients 12
years and older with somatostatin receptor-positive (SSTR+)
gastroenteropancreatic neuroendocrine tumors (GEP-NETs), including foregut,
midgut, and hindgut NETs. This approval makes Lutathera the first therapy
specifically reviewed and approved for use in pediatric patients with GEP-NETs.
"Lutathera is now the very first therapy approved specifically for children with
GEP-NETs, offering new hope to young patients living with this rare cancer,"
said Tina Deignan, Therapeutic Area Head, Oncology US. "Radioligand therapies
have extraordinary potential to shape the future of cancer care. With this
approval, we have taken another vital step toward fulfilling that vision,
strengthening our commitment to researching and developing the RLT platform
across multiple cancer types and treatment settings."
NETs are a type of cancer that originates in neuroendocrine cells throughout the
body and are commonly considered slow-growing malignancies(1). The diagnosis of
NETs is often delayed due to the inactive nature of the disease, and
approximately 10% to 20% of pediatric patients are diagnosed with metastatic
disease(2,3). Even though NETs are an orphan disease, their incidence has
increased over the past several decades(1,4-6).
"While GEP-NETs in children and adolescents are rare, the impact can be
devastating. Today's approval addresses a critical need for new treatment
options for these vulnerable patients," said Dr. Theodore Laetsch, trial
investigator and Director, Developmental Therapeutics Program, Children's
Hospital of Philadelphia (CHOP), a NETTER-P clinical trial site. "The
introduction of radioligand therapy significantly advanced how we treat GEP-
NETs, and I'm encouraged that younger patients now have the potential to benefit
from this innovation."
The approval was based on the NETTER-P trial, which evaluated Lutathera in
patients aged 12 to Â