Rye
Brook, N.Y. (February 26, 2021) –
The U.S. Food and Drug Administration (FDA) today announced approval of
melphalan flufenamide, also known as melflufen (Pepaxto), in combination with
dexamethasone for the treatment of adults with multiple myeloma whose
disease has returned or worsened following prior treatment with four different
classes of drugs.
Today’s
approval of melflufen was based on results from HORIZON, a pivotal phase 2
study. Melflufen demonstrated a 23.7% overall response rate (ORR) in patients
with so-called “triple-class refractory multiple myeloma” or RRMM. The
responses in melflufen-treated patients were durable and often deepened with
prolonged treatment, suggesting that patients may benefit from staying on
treatment for as long as possible.
Despite the
availability of new treatments, multiple myeloma will progress in most
patients. In recent years, a new subset of patients with RRMM has been identified.
The current approaches to the treatment of RRMM are limited and generally have
short-lived effectiveness.
Multiple
myeloma is among The Leukemia & Lymphoma Society’s
primary research initiatives. LLS currently has over 30 active projects (over a
$40M commitment) in multiple myeloma focused on laboratory and clinical
research. They include world-class researchers at Dana Farber Cancer Institute,
MD Anderson Cancer Center and the University of Pennsylvania who are looking at
ways to provide safe and effective targeted therapies and to adapt CAR
T-cell immunotherapy for patients with multiple myeloma.
Today’s
approval of melflufen is an encouraging advancement for a patient population
with significant unmet medical need who have already been treated with the
three major classes of myeloma drugs.
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