Pharming
Group N.V. (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM/Nasdaq:
PHAR) announces that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has granted an accelerated assessment for
the Marketing Authorization Application (MAA) for leniolisib. Leniolisib has
been studied for the treatment of activated PI3K delta syndrome (APDS), a rare
primary immunodeficiency, in adults and adolescents age 12 or older in the
European Economic Area (EEA). Pharming is on track and plans to submit its MAA
for leniolisib to the EMA in October 2022.
Accelerated assessment reduces the
timeframe for the CHMP to review an MAA from 210 days to 150
days. The EMA will grant, upon request, accelerated assessment of an MAA
if they decide the product is of major interest for public health and
therapeutic innovation.
The
clinical development for leniolisib includes positive data from a Phase II/III
study of the product, which met both its co-primary endpoints in the target
patient population of evaluated reduction in lymph node size and correction of
immunodeficiency. The primary efficacy results demonstrated clinical efficacy
of leniolisib over placebo with a statistically significant reduction from
baseline in the log10 transformed sum of product of diameters (SPD) in the
index lymphadenopathy lesions (p=0.0012) and normalization of immune
dysfunction, as evidenced by increased proportion of naïve B cells from
baseline (p<0.0001). The shrinking of lymphadenopathy lesions and increased
proportion of naïve B cells are important in patients as they indicate a reduction
in APDS disease markers.
In
the study, leniolisib was generally well-tolerated, with the majority of
reported adverse events in both treatment groups classified as mild. There were
no adverse events that led to discontinuation of study treatment, there were no
deaths, and the incidence of serious adverse events (SAEs) was lower in the
leniolisib group than the placebo group. None of the SAEs were suspected to be
related to study treatment.
Anurag Relan, Chief Medical Officer of
Pharming, commented:
“The
acceptance of an accelerated regulatory review for leniolisib underlines the
high unmet need for patients with APDS, with the product potentially being the
first approved treatment for this rare disease. This is an important milestone
for the APDS community and for Pharming and is built on the successful Phase
II/III data, which we first reported in February 2022. We remain focused on
progressing leniolisib through the regulatory review process, with our MAA on
track for submission in October of this year, as we seek to make this important
new product available to immunologists, hematologists, and their patients in
Europe.”
No comments:
Post a Comment