- FDA approval based on MARCH Phase 3 study
with highly statistically significant (p<0.0001) reduction in
pruritus severity between LIVMARLI vs. placebo
- Label covers broad range of PFIC subtypes
- Immediate launch and availability for
eligible patients in the U.S.
Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that
the U.S. Food and Drug Administration (FDA) has approved LIVMARLI®
(maralixibat) oral solution for the treatment of cholestatic
pruritus in patients five years of age and older with progressive
familial intrahepatic cholestasis (PFIC). Mirum has also submitted
an additional supplemental new drug application (sNDA) to introduce
a higher concentration formulation of LIVMARLI, used during the
MARCH study, to enable label expansion for younger patients with
PFIC, later this year.
This press release features multimedia. View
the full release here:
https://www.businesswire.com/news/home/20240313135184/en/
LIVMARLI® (maralixibat) oral solution is
now available for prescribing in the US for cholestatic pruritus in
patients with PFIC. (Photo: Business Wire)
LIVMARLI is also approved for the treatment of cholestatic
pruritus in patients with Alagille syndrome (ALGS) in the U.S.
(three months and older), Europe (two months and older), in Canada
and other regions globally.
“LIVMARLI has the potential to have a transformational impact
for patients with cholestatic pruritus associated with PFIC, and
importantly, offers an option for those patients with the rarest of
subtypes,” said Chris Peetz, chief executive officer at Mirum.
“Thank you to the patients, families, and clinicians whose study
participation made this approval possible.”
The approval is based on data from the Phase 3 MARCH study, the
largest randomized trial conducted in PFIC, with 93 patients across
a range of genetic PFIC types, including PFIC1, PFIC2, PFIC3,
PFIC4, PFIC6, and unidentified mutational status.
“LIVMARLI’s approval in cholestatic pruritus for patients with
PFIC is a result of years of investigation and a collection of a
strong body of clinical evidence showing meaningful improvements
across a number of important parameters, including pruritus,
affecting children with PFIC,” said Richard Thompson, professor of
molecular hepatology at King’s College London and an investigator
in the MARCH study. “I am pleased that we will have a well-studied
and efficacious option to offer patients whose life has been
disrupted by itch.”
“PFIC is a difficult disease for both the patient and family and
significantly impedes the quality of life for all,” said Emily
Ventura, executive director of the PFIC Network and mom to a
daughter with PFIC. “These young and fragile patients endure an
itch so severe that they experience deficits in their sleep,
nutrition, growth, and, in the past, some have turned to transplant
to resolve their itch. We are hopeful that LIVMARLI will have a
measurable impact for patients, potentially offering a new normal
and relief from the sleepless nights and disruption to their lives
caused by cholestatic pruritus.”
About PFIC
Progressive familial intrahepatic cholestasis (PFIC) is a rare
genetic disorder that causes progressive liver disease typically
leading to liver failure. In people with PFIC, liver cells are less
able to secrete bile. The resulting buildup of bile causes liver
disease in affected individuals. Signs and symptoms of PFIC
typically begin in infancy. Patients experience severe itching,
jaundice, failure to grow at the expected rate (failure to thrive),
and an increasing inability of the liver to function (liver
failure). The disease is estimated to affect one in every 50,000 to
100,000 births in the United States and Europe. More than six types
of PFIC have been genetically identified, all of which are
similarly characterized by impaired bile flow and progressive liver
disease.
About LIVMARLI® (maralixibat) oral solution
LIVMARLI® (maralixibat) oral solution is an orally administered,
once-daily, ileal bile acid transporter (IBAT) inhibitor and the
only approved medication by the U.S. Food and Drug Administration
for the treatment of cholestatic pruritus in patients with Alagille
syndrome (ALGS) three months of age and older and progressive
familial intrahepatic cholestasis (PFIC) five years of age and
older.
LIVMARLI is also the only approved IBAT inhibitor approved by
the European Commission for the treatment of cholestatic pruritus
in patients with ALGS two months and older, and by Health Canada
for the treatment of cholestatic pruritus in ALGS. For more
information for U.S. residents, please visit LIVMARLI.com.
Mirum has also submitted LIVMARLI for approval in Europe in PFIC
for patients two months of age and older.
LIVMARLI has received Breakthrough Therapy designation for ALGS
and PFIC type 2 and orphan designation for ALGS and PFIC. To learn
more about ongoing clinical trials with LIVMARLI, please visit
Mirum’s clinical trials section on the company’s website.
IMPORTANT SAFETY INFORMATION
Limitation of Use: LIVMARLI is not for use in PFIC type 2
patients who have a severe defect in the bile salt export pump
(BSEP) protein.
LIVMARLI can cause side effects, including:
Liver injury. Changes in certain liver tests are common
in patients with Alagille syndrome and PFIC but can worsen during
treatment. These changes may be a sign of liver injury. In PFIC,
this can be serious or may lead to liver transplant or death. Your
healthcare provider should do blood tests and physical exams before
starting and during treatment to check your liver function. Tell
your healthcare provider right away if you get any signs or
symptoms of liver problems, including nausea or vomiting, skin or
the white part of the eye turns yellow, dark or brown urine, pain
on the right side of the stomach (abdomen), bloating in your
stomach area, loss of appetite or bleeding or bruising more easily
than normal.
Stomach and intestinal (gastrointestinal) problems.
LIVMARLI can cause stomach and intestinal problems, including
diarrhea and stomach pain. Your healthcare provider may advise you
to monitor for new or worsening stomach problems including stomach
pain, diarrhea, blood in your stool or vomiting. Tell your
healthcare provider right away if you have any of these symptoms
more often or more severely than normal for you.
A condition called Fat Soluble Vitamin (FSV) Deficiency
caused by low levels of certain vitamins (vitamin A, D, E, and K)
stored in body fat is common in patients with Alagille syndrome and
PFIC but may worsen during treatment. Your healthcare provider
should do blood tests before starting and during treatment and may
monitor for bone fractures and bleeding which have been reported as
common side effects.
US Prescribing Information EU SmPC Canadian Product
Monograph
About Mirum Pharmaceuticals, Inc.
Mirum Pharmaceuticals, Inc. is a biopharmaceutical company
dedicated to transforming the treatment of rare diseases affecting
children and adults. Mirum has three approved medications:
LIVMARLI® (maralixibat) oral solution, CHOLBAM® (cholic acid)
capsules, and CHENODAL® (chenodiol) tablets.
LIVMARLI, an IBAT inhibitor, is approved for the treatment of
two rare liver diseases affecting children and adults. It is
approved for the treatment of cholestatic pruritus in patients with
Alagille syndrome in the U.S. (three months and older), in Europe
(two months and older), and in other regions globally. It is also
approved in the U.S. in cholestatic pruritus in PFIC patients five
years of age and older. Mirum has submitted for approval in Europe
for the treatment of PFIC in patients two months of age and older.
CHOLBAM is FDA-approved for the treatment of bile acid synthesis
disorders due to single enzyme deficiencies and adjunctive
treatment of peroxisomal disorders in patients who show signs or
symptoms or liver disease. CHENODAL has received medical necessity
recognition by the FDA to treat patients with cerebrotendinous
xanthomatosis (CTX).
Mirum’s late-stage pipeline includes two investigational
treatments for debilitating liver diseases. Volixibat, an IBAT
inhibitor, is being evaluated in two potentially registrational
studies including the Phase 2b VISTAS study for primary sclerosing
cholangitis and Phase 2b VANTAGE study for primary biliary
cholangitis. Lastly, CHENODAL, has been evaluated in a Phase 3
clinical study, RESTORE, to treat patients with CTX, with positive
topline results reported in 2023.
To learn more about Mirum, visit mirumpharma.com and follow
Mirum on Facebook, LinkedIn, Instagram and Twitter.
Forward-Looking Statements
Statements contained in this press release regarding matters
that are not historical facts are “forward-looking statements”
within the meaning of the Private Securities Litigation Reform Act
of 1995. Such forward-looking statements include statements
regarding, among other things, the potential impacts of LIVMARLI in
PFIC and for any future label expansion, the additional
improvements in disease beyond pruritus, and the ability of
LIVMARLI to modify PFIC patient disease experience and the
continued commercial success of LIVMARLI across ALGS and PFIC.
Because such statements are subject to risks and uncertainties,
actual results may differ materially from those expressed or
implied by such forward-looking statements. Words such as “will,”
“intended,” “potential” and similar expressions are intended to
identify forward-looking statements. These forward-looking
statements are based upon Mirum’s current expectations and involve
assumptions that may never materialize or may prove to be
incorrect. Actual results could differ materially from those
anticipated in such forward-looking statements as a result of
various risks and uncertainties, which include, without limitation,
risks and uncertainties associated with Mirum’s business in
general, the impact of geopolitical and macroeconomic events , and
the other risks described in Mirum’s filings with the Securities
and Exchange Commission, including its most recent Annual Report on
Form 10-K, as updated by any subsequent Quarter Reports on Form
10-Q. All forward-looking statements contained in this press
release speak only as of the date on which they were made and are
based on management’s assumptions and estimates as of such date.
Mirum undertakes no obligation to update such statements to reflect
events that occur or circumstances that exist after the date on
which they were made, except as required by law.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240313135184/en/
Media: Erin Murphy 510-508-6521 media@mirumpharma.com
Investors: Andrew McKibben ir@mirumpharma.com
Sam Martin Argot Partners ir@mirumpharma.com
Mirum Pharmaceuticals (NASDAQ:MIRM)
Historical Stock Chart
From Apr 2024 to May 2024
Mirum Pharmaceuticals (NASDAQ:MIRM)
Historical Stock Chart
From May 2023 to May 2024