- Positive opinion from CHMP based on Phase 3 MARCH study with
highly statistically significant (p<0.0001) reduction in
pruritus severity between LIVMARLI vs. placebo across all PFIC
types studied.
- CHMP assessment concluded that LIVMARLI in PFIC brings
significant clinical benefit over the existing approved
therapy.
- Additionally, LIVMARLI received favorable COMP opinion
recommending maintenance of Orphan Drug Designation in PFIC.
- Following CHMP and COMP opinions, a decision by the European
Commission is expected in the third quarter of 2024.
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that
the European Committee for Medicinal Products for Human Use (CHMP)
has adopted a positive opinion of LIVMARLI® (maralixibat) oral
solution for the treatment of Progressive Familial Intrahepatic
Cholestasis (PFIC) in patients three months of age and older. The
CHMP also concluded that LIVMARLI in PFIC brings significant
clinical benefit based on improved efficacy and a major
contribution to patient care compared to the existing approved
treatment for PFIC. The CHMP positive opinion is the scientific
recommendation to the European Commission for marketing
authorization in Europe.
In addition, the Committee for Orphan Medicinal Products (COMP),
has also recommended maintenance of Orphan Drug Designation for
LIVMARLI in PFIC. The European Commission’s endorsement of the CHMP
and COMP opinions is expected in the third quarter of 2024.
“The positive and favorable opinions by both CHMP and COMP
recognizes the potential of LIVMARLI in treating patients with PFIC
in Europe, underscoring the compelling long-term data collected in
the largest PFIC study across a broad range of genetic types,” said
Chris Peetz, chief executive officer at Mirum. “Further, we are
thrilled that, if approved by the European Commission, the PFIC
community will have a treatment option that has been shown to
significantly improve key liver health parameters starting from
three months of age.”
The CHMP and COMP opinions are 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 is currently approved in Europe for the treatment of
cholestatic pruritus in patients with Alagille syndrome (ALGS) two
months of age and older. In the United States, LIVMARLI is approved
for the treatment of cholestatic pruritus in patients with ALGS
three months of age and older and for cholestatic pruritus in
patients with PFIC five years of age and older. LIVMARLI is also
approved in other regions globally. Mirum has submitted a
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 in the
United States later this year.
About Progressive Familial Intrahepatic Cholestasis
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 approved
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 for 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.
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. 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 (X).
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 benefits of LIVMARLI
and perceived superiority against other products, the marketing
authorisation application pending with the European Commission and
planned regulatory submissions in other countries. 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,” “could,” “would,”
“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 the COVID-19 pandemic, and the other risks described in
Mirum’s filings with the Securities and Exchange Commission. 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. A further
description of risks and uncertainties can be found in Mirum’s
Annual Report on Form 10-K for the fiscal year ended December 31,
2023 and in its subsequent reports on Form 10-Q, including in the
sections thereof captioned “Risk Factors,” as well as in its
subsequent reports on Form 8-K, all of which are filed with the
U.S. Securities and Exchange Commission and available at
www.sec.gov.
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version on businesswire.com: https://www.businesswire.com/news/home/20240531358508/en/
Media Contact: Erin Murphy media@mirumpharma.com
Investor Contact: Andrew McKibben investors@mirumphama.com
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