- European Commission grants LIVMARLI marketing authorization for
treatment of PFIC in patients three months and older.
- Approval follows positive opinion from CHMP concluding
LIVMARLI’s clinical benefit over existing therapy in PFIC.
- LIVMARLI also received positive COMP opinion recommending
maintenance of Orphan Drug Designation in PFIC.
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that
the European Commission has granted marketing authorization for
LIVMARLI® (maralixibat) oral solution for the treatment of
progressive familial intrahepatic cholestasis (PFIC) in patients
three months of age and older. The approval follows a positive
opinion by the CHMP which 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. Further, evaluation by COMP recommended
maintenance of Orphan Drug Designation for LIVMARLI in PFIC.
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. Data from MARCH
showed statistically significant reduction in pruritus
(p<0.0001) and serum bile acids (p<0.0001) between LIVMARLI
versus placebo in the All-PFIC cohort (n=64). Significant
improvements were also observed in total bilirubin and growth
versus placebo. The most common treatment emergent adverse event
was diarrhea, which was predominantly mild, with no severe cases,
and transient.
“We are thrilled that the European Commission has granted
marketing authorization for LIVMARLI in PFIC, acknowledging the
strength of data collected and recognizing the important treatment
opportunity for patients living with this rare liver disease,” said
Chris Peetz, chief executive officer at Mirum. “Our hope is that
LIVMARLI helps to improve key liver parameters and brings healthier
days ahead to young patients diagnosed with PFIC in Europe. We are
grateful to the researchers, patients, and families who made this
approval possible.”
“LIVMARLI’s approval provides a treatment backed by years of
clinical research and meaningful data that demonstrate a reduction
in cholestatic itch and prognostic markers of improved liver
health, including a reduction of serum bile acids, all important
signals for positive long-term outcomes,” said Professor Richard
Thompson, King’s College London. “It is encouraging to know that
physicians in Europe will have a new option that has the potential
to improve the liver health and quality of life for patients and
their families.”
“The patient community in Europe will greatly benefit from
LIVMARLI’s approval, supported by years of impressive data showing
improvements in the most burdensome aspects of disease,” said Emily
Ventura, executive director, PFIC Network. “PFIC can be
life-altering and can have a devastating impact on patients. We are
encouraged to see that young patients will have a new medication
and hope for a life less burdened by cholestasis.”
LIVMARLI is also approved by the U.S. Food and Drug
Administration for the treatment of cholestatic pruritus in
patients with PFIC five years of age and older. Mirum has submitted
a supplemental new drug application to introduce a higher
concentration formulation of LIVMARLI, used during the MARCH study,
to enable label expansion for younger patients with PFIC, and
expects to receive FDA feedback this year.
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, chenodiol, has been evaluated in a Phase 3
clinical study, RESTORE, to treat patients with CTX, with positive
topline results reported in 2023. Mirum has filed a new drug
application with the FDA for the approval chenodiol to treat CTX in
the U.S.
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 commercial success of LIVMARLI
in Europe for PFIC, pricing reimbursement for LIVMARLI in any
specific country, and the real world impact of LIVMARLI in 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,”
“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/20240708474225/en/
Media Contact: Erin Murphy media@mirumpharma.com
Investor Contact: Andrew McKibben investors@mirumpharma.com
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