XenpozymeTM (olipudase alfa-rpcp) approved by
FDA as first disease-specific treatment for ASMD (non-CNS
manifestations)
Paris,
August 31, 2022. The U.S. Food
and Drug Administration (FDA) has approved XenpozymeTM (olipudase
alfa-rpcp) for the treatment of non-central nervous system
(non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD)
in adult and pediatric patients. Xenpozyme is the first therapy
indicated specifically for the treatment of ASMD, and is currently
the only approved treatment for this disease.
Bill SiboldExecutive Vice
President, Head, Specialty Care at Sanofi “Sanofi teams have been
dedicated to bringing hope to patients living with ASMD and their
families. This is a devastating and extremely rare disease that
affects both children and adults. The approval of Xenpozyme
represents the culmination of bold work done in research and
development, and our unwavering commitment to this historically
overlooked community.”
ASMD, historically known as Niemann-Pick disease
types A, A/B, and B, is an extremely rare, progressive genetic
disease with significant morbidity and mortality. It has been
estimated that there are fewer than 120 patients diagnosed with
ASMD in the U.S. Approximately two-thirds of patients with ASMD in
the U.S. are pediatric. Signs and symptoms of ASMD can present in
infancy, childhood, or adulthood, and may include enlarged spleen
or liver, difficulty breathing, lung infections, and unusual
bruising or bleeding, among other disease manifestations. Until
now, management of ASMD included supportive care to address the
impact of individual symptoms and careful monitoring to detect
potential disease complications.
David GuyParent to Kaila, age
16, living with ASMD“As young parents, it was initially devastating
to me and my wife when our daughter, Kaila, received her diagnosis
of ASMD. We faced so many unknowns when we first heard the
diagnosis: what does this mean, how will this affect her, and most
importantly what hope is there for a treatment option? We were
grateful to find hope when we enrolled Kaila in the clinical trials
for olipudase alfa.”
In the U.S., Xenpozyme received Breakthrough
Therapy designation, which expedites the development and review of
drugs intended to treat serious or life-threatening diseases and
conditions. The FDA evaluated Xenpozyme under Priority Review,
which is reserved for medicines that represent potentially
significant improvements in efficacy or safety in treating serious
conditions.. In March 2022, Xenpozyme was approved in Japan under
the SAKIGAKE (or “pioneer”) designation, marking the first approval
for olipudase alfa anywhere in the world. In June 2022, the
European Commission (EC) approved Xenpozyme for use in Europe.
ASMD represents a spectrum of disease, with two
types that may represent opposite ends of a continuum referred to
as ASMD type A and ASMD type B. ASMD type A/B is an intermediate
form that includes varying degrees of central nervous system (CNS)
involvement.
ASCEND and ASCEND-Peds clinical trials showed
that Xenpozyme improved lung function and reduced spleen and liver
volumes in adults and children
The approval is based on positive data from the
ASCEND and ASCEND-Peds clinical trials, in which Xenpozyme showed
clinically relevant improvement in lung function (as measured by
diffusing capacity of the lung for carbon monoxide, or DLco) and
platelet count, and reduction of spleen and liver volumes, with a
demonstrated safety profile.
Melissa WassersteinMD, Pediatric
Genetic Medicine, Albert Einstein College of Medicine and the
Children’s Hospital at Montefiore“ASMD is an extremely rare,
progressive, and potentially fatal genetic disease that impacts
children and adults around the world. Until now, those living with
ASMD have had no FDA-approved treatment to combat this devastating
condition. I’m proud of the work that has been done and look
forward to witnessing the impact that this treatment may have on
those living with ASMD.”
The ASCEND trial evaluated the efficacy and
safety of Xenpozyme; 31 adult patients with ASMD type A/B or type B
were randomized to receive Xenpozyme or placebo for 52 weeks
(primary analysis). In the trial, Xenpozyme improved lung function,
assessed as the percent change from baseline to Week 52 in
predicted diffusing capacity of the lung for carbon monoxide
(DLco), and reduced spleen volume, evaluated as percent change from
baseline in multiples of normal (MN).
- Twelve (12) patients treated with
Xenpozyme had a mean change in percent predicted DLco from baseline
(49.1%) to Week 52 (59.4%). This change represents a 23.9% relative
improvement compared to a 3% improvement in DLco from baseline in
the 17 patients from the placebo group (48.5%) to Week 52 (49.9%).
The difference between the two arms (20.9%) was nominally
statistically significant (p=0.0003).
- Thirteen (13) patients treated with
Xenpozyme had a mean reduction in spleen volume by 38.9% from
baseline (11.5 MN) to Week 52 (7.2 MN) compared to a mean increase
by 0.5% for the 17 patients in the placebo group from baseline
(11.2 MN) to Week 52 (11.2 MN). The difference between the two arms
(39.4%) was nominally statistically significant (p<0.0001).
- Twelve (12) patients treated with
Xenpozyme had a mean reduction in liver volume by 26.5% from
baseline (1.4 MN) to Week 52 (1.0 MN) compared to a mean decrease
of 1.8% for the 17 patients in the placebo group from baseline (1.6
MN) to Week 52 (1.6 MN). The difference between the two arms
(24.7%) was nominally statistically significant (p<0.0001).
- Thirteen (13) patients treated with
Xenpozyme had a mean improvement in platelet count by 18.3% from
baseline (109.3x109/L) to Week 52 (126.4x109/L) compared to
increase by 2.7% for the 16 patients in the placebo group from
baseline (115.6x109/L) to Week 52 (120.2x109/L). The difference
between the two arms (15.6%) was nominally statistically
significant (p=0.0280).
- All ASCEND patients treated with
Xenpozyme showed improvement in key endpoints (DLco and spleen and
liver volume).
- Most frequently
reported adverse drug reactions in adults (incidence ≥10%) were
headache, cough, diarrhea, hypotension, and ocular hyperemia.
The single-arm ASCEND-Peds trial studied 8
pediatric patients younger than 12 years of age with ASMD type A/B
or type B who all received Xenpozyme, with a primary objective of
evaluating the safety and tolerability of Xenpozyme for 64 weeks.
All patients completed the study and continued in an extension
trial. The ASCEND-Peds trial also explored efficacy endpoints of
progressive lung disease, spleen and liver enlargement, and
platelet count. After one year of treatment (52 weeks):
- Three (3)
patients who were able to perform the test at baseline treated with
Xenpozyme had a mean relative improvement of 45.9% in percent
predicted DLco from baseline (48.5%) to Week 52 (70.9%) (children
over the age of five were assessed if they were able to perform the
test).
- Eight (8)
patients treated with Xenpozyme had mean reduction in spleen volume
by 46.7% from baseline (18.3 MN) to Week 52 (9.5 MN).
- Eight (8)
patients treated with Xenpozyme had a mean reduction in liver
volume by 38.1% from baseline (2.5 MN) to Week 52 (1.6 MN).
- Seven (7)
patients treated with Xenpozyme had a mean improvement in platelet
count by 37.6% from baseline (136.7x109/L; n=8) to Week 52
(184.5x109/L).
- Serious adverse
reactions of anaphylactic reaction were reported in 2 (25%)
Xenpozyme-treated pediatric patients.
-
Treatment-related serious adverse reactions, hypersensitivity
reactions including anaphylaxis, and infusion associated reactions
occurred within 24 hours of infusion and were observed in a higher
percentage of pediatric patients than in adult patients.
- Most frequently
reported adverse drug reactions in pediatric patients (incidence
≥20%) were pyrexia, cough, diarrhea, rhinitis, abdominal pain,
vomiting, headache, urticaria, nausea, rash, arthralgia, pruritus,
fatigue, and pharyngitis.
A scientific innovation for patients living with
ASMD
Xenpozyme, a hydrolytic lysosomal
sphingomyelin-specific enzyme replacement therapy, is designed to
replace deficient or defective acid sphingomyelinase (ASM), an
enzyme that allows for the breakdown of the lipid sphingomyelin. In
individuals with ASMD, the deficiency in the ASM enzyme leads to
sphingomyelin accumulation in various tissues. Xenpozyme is not
expected to cross the blood-brain barrier or modulate CNS
manifestations of ASMD. Xenpozyme has not been studied in patients
with ASMD type A.
Xenpozyme is adminstered intravenously every two
weeks, and its administration requires a dose escalation phase
followed by a maintenance phase.
Xenpozyme is expected to be available in the
U.S. in the coming weeks. The U.S. list price, or wholesale
acquisition cost, of Xenpozyme is $7,142.00 per vial. Actual
patient out-of-pocket costs may be lower, as the list price does
not reflect insurance coverage, co-pay support for eligible
patients, or financial assistance from patient support
programs.
Sanofi is committed to helping eligible U.S.
patients access the support they need and to help reduce barriers
throughout their treatment journey. As part of its commitment to
treatment access and affordability for innovative therapies, Sanofi
provides disease education, financial and co-pay assistance
programs, and other support services to eligible patients. For more
information, patients can call 1-800-745-4447 and select Option 3,
contact Info@CareConnectPSS.com, or visit www.Xenpozyme.com.
About Sanofi We are an innovative
global healthcare company, driven by one purpose: we chase the
miracles of science to improve people’s lives. Our team, across
some 100 countries, is dedicated to transforming the practice of
medicine by working to turn the impossible into the possible. We
provide potentially life-changing treatment options and life-saving
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