BridgeBio Pharma, Sentynl Therapeutics and Medison Pharma Announce Approval in Israel for NULIBRY® (fosdenopterin) for the Treatment of MoCD Type A
– NULIBRY is the first and only approved therapy in the United States (U.S.) and in Israel to treat patients with MoCD Type A, an ultra-rare, life-threatening genetic disorder that often progresses rapidly in infants with a median overall survival age of about four years
– NULIBRY received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) in July 2022 recommending approval by the European Commission (EC) in the European Union (EU)
– NULIBRY was approved by the U.S. Food and Drug Administration (FDA) in February 2021 and was BridgeBio’s first approved therapy; Medison acquired commercialization rights to NULIBRY in Israel in December 2019; Sentynl acquired global rights to NULIBRY in March 2022
PALO ALTO and SOLANA BEACH, Calif. and PETACH TIKVA, Israel, Aug. 10, 2022 (GLOBE NEWSWIRE) — BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio), a commercial-stage biopharmaceutical company that focuses on genetic diseases and cancers, and Sentynl Therapeutics, Inc. (Sentynl), a U.S.-based biopharmaceutical company focused on bringing innovative therapies to patients living with rare diseases owned by Zydus Lifesciences Ltd. (formerly known as Cadila Healthcare Ltd.), along with partner Medison Pharma, a global pharmaceutical company focused on providing access to highly innovative therapies to patients in international markets, today announced that the State of Israel Ministry of Health has approved NULIBRY® (fosdenopterin) for Injection as the first therapy in Israel to treat MoCD Type A with the indication to reduce the risk of mortality for patients with molybdenum cofactor deficiency (MoCD) Type A. MoCD Type A is an ultra-rare and progressive condition, known to impact less than 150 patients globally with a median survival of four years.
“At BridgeBio, we believe that no disease is too rare to address. We are grateful that NULIBRY is now an approved therapy in Israel, and we are focused on ensuring that even more children around the world who suffer with MoCD Type A have access to this treatment,” said BridgeBio founder and CEO Neil Kumar, Ph.D.
NULIBRY is a cPMP substrate replacement therapy that was approved by the U.S. FDA in February 2021 to reduce the risk of mortality in patients with MoCD Type A. In July 2022 the CHMP recommended that NULIBRY be approved in the EU as a treatment for patients with MoCD Type A. Based on the CHMP recommendation, a decision by the European Commission (EC), which authorizes marketing applications in the EU, is expected on the NULIBRY application later this year.
“The approval of NULIBRY in Israel is a promising development for children with MoCD Type A. At Zydus and Sentynl we are driven by the purpose of empowering people with the freedom to live healthier and more fulfilled lives. NULIBRY will bring us closer to realizing our purpose and making a meaningful difference in the lives of children with this rare disorder,” said Dr. Sharvil Patel, Managing Director of Zydus Lifesciences, the parent company of Sentynl Therapeutics.
“We are very pleased to share the news of the Israeli approval on the heels of receiving the CHMP’s recommendation in favor of NULIBRY. We are confident this is only the beginning for patients living with MoCD Type A and we intend to continue advancing our efforts to broaden access to this therapy throughout the world for children in need, while increasing awareness of a disease that has historically been underdiagnosed,” said Matt Heck, CEO of Sentynl.
The approval by the State of Israel Ministry of Health is supported by data from three clinical trials that demonstrated efficacy of NULIBRY for the treatment of patients with MoCD Type A compared to data from a natural history study. These studies showed that NULIBRY reduced the risk of death by 82% and increased the probability of survival to 84% at three years compared to 55% in the untreated, genotype-matched, historical control group in the natural history study.
Animal studies have identified that NULIBRY has phototoxic potential. In the clinical trials, the most common adverse reactions reported in two or more NULIBRY-treated patients with MoCD Type A were catheter-related complications (89%), pyrexia (fever; 78%), viral infection (56%), pneumonia (44%), otitis media (ear infection; 44%), vomiting (44%) and cough/sneezing (44%). Adverse reactions for the rcPMP-treated patients were similar to the NULIBRY-treated patients.
“We are proud that our partnership with BridgeBio Pharma and Sentynl enables us to provide this highly innovative therapy to babies born with MoCD Type A, a severely progressive genetic disease that results in irreversible neurological damage and eventual death,” said Meir Jakobsohn, Founder and CEO of Medison Pharma. “The Israeli Ministry of Health’s decision brings us one step closer to accelerating access of this cutting-edge therapy to those in need.”
In March 2022, Sentynl acquired the global rights to NULIBRY and is responsible for the ongoing development and commercialization of NULIBRY in the U.S. and developing, manufacturing, and commercializing fosdenopterin globally. Sentynl and BridgeBio share development responsibilities through the approval of the marketing authorization application under accelerated assessment with the European Medicines Agency.
About Molybdenum Cofactor Deficiency (MoCD) Type A
MoCD Type A is an autosomal recessive, inborn error of
metabolism caused by mutations in the molybdenum cofactor
synthesis 1 gene and characterized by a deficiency in molybdenum
cofactor production, leading to a lack of molybdenum-dependent
enzyme activity.1,2 The lack of activity leads to
decreased sulfite oxidase activity with buildup of sulfite and
secondary metabolites (such as S-sulfocysteine) in the brain,
which causes irreversible neurological damage.2
The most common presenting symptoms of MoCD Type A are seizures, feeding difficulties and encephalopathy. Patients with MoCD Type A who survive beyond infancy typically suffer from progressive brain damage, which presents in characteristic patterns on magnetic resonance imaging (MRI). This damage leads to severe psychomotor impairment and an inability to make coordinated movements or communicate with their environment.
About NULIBRY® (fosdenopterin) for
Injection
NULIBRY®(fosdenopterin) for Injection is a substrate
replacement therapy that provides an exogenous source of cPMP,
which is converted to molybdopterin. Molybdopterin is then
converted to molybdenum cofactor, which is needed for the
activation of molybdenum-dependent enzymes, including sulfite
oxidase, an enzyme that reduces levels of neurotoxic sulfites.
It is the first and only FDA-approved therapy indicated to
reduce the risk of mortality in patients with MoCD Type A, and
clinical trials have demonstrated that patients treated with
NULIBRY or rcPMP had an improvement in overall survival compared
to the untreated, genotype-matched, historical control group.
References
1 Mechler K et al. Genet Med. 2015;17(12):965-970.
2 Schwarz G. Cur Op in Che Bio. 2016;31:179-187.
About BridgeBio Pharma, Inc.
BridgeBio Pharma, Inc. (BridgeBio) is a commercial-stage
biopharmaceutical company founded to discover, create, test and
deliver transformative medicines to treat patients who suffer
from genetic diseases and cancers with clear genetic drivers.
BridgeBio’s pipeline of development programs ranges from early
science to advanced clinical trials. BridgeBio was founded in
2015 and its team of experienced drug discoverers, developers
and innovators are committed to applying advances in genetic
medicine to help patients as quickly as possible. For more
information visit bridgebiodev.wpengine.com and follow us on LinkedIn and Twitter.
About Sentynl Therapeutics
Sentynl Therapeutics is a U.S.-based biopharmaceutical company
focused on bringing innovative therapies to patients living with
rare diseases. The company was acquired by the Zydus Group in
2017. Sentynl’s experienced management team has previously built
multiple successful pharmaceutical companies. With a focus on
commercialization, Sentynl looks to source effective and highly
differentiated products across a broad spectrum of therapeutic
areas to address unmet needs. Sentynl is committed to the
highest ethical standards and compliance with all applicable
laws, regulations, and industry guidelines. For more
information, visit www.sentynl.com and follow us on LinkedIn.
About Zydus
The Zydus Group, with an overarching purpose of empowering
people with freedom to live healthier and more fulfilled lives,
is an innovative, global pharmaceutical company that discovers,
develops, manufactures, and markets a broad range of healthcare
therapies. The group employs over 23000 people worldwide and is
driven by its mission to unlock new possibilities in
life-sciences through quality healthcare solutions that impact
lives. The group aspires to become a global life-sciences
company transforming lives through pathbreaking discoveries.
For more information, visit
https://www.zyduslife.com/zyduslife/.
About Medison Pharma
Medison is a global pharma company focused on providing access
to highly innovative therapies to patients in international
markets.
Medison is the first to create an international commercialization platform for highly innovative therapies, helping to save and improve lives by making the best available novel treatments accessible to patients in international markets. Medison has a track record of multi-territorial partnerships with leading pharmaceutical and biotech companies seeking to expand their global reach.
Medison is also an active investor in disruptive healthcare technologies and provides its partners with exposure to innovation in biotech and digital health. To learn more visit www.medisonpharma.com.
BridgeBio Pharma, Inc. Forward-Looking Statements
This press release contains forward-looking statements.
Statements we make in this press release may include statements
that are not historical facts and are considered forward-looking
within the meaning of Section 27A of the Securities Act of 1933,
as amended (the “Securities Act”), and Section 21E of the
Securities Exchange Act of 1934, as amended (the “Exchange
Act”), which are usually identified by the use of words such as
“anticipates,” “believes,” “estimates,” “expects,” “intends,”
“may,” “plans,” “projects,” “seeks,” “should,” “will,” and
variations of such words or similar expressions. We intend
these forward-looking statements to be covered by the safe
harbor provisions for forward-looking statements contained in
Section 27A of the Securities Act and Section 21E of the
Exchange Act and are making this statement for purposes of
complying with those safe harbor provisions. These
forward-looking statements, including statements relating to,
the timing and outcome of the EC’s decision on NULIBRY, the
potential ability for NULIBRY to be the first and only approved
therapy in the EU to treat patients with MoCD type A, and the
potential ability to provide treatment options to MoCD Type A
patients in Europe, Israel and around the world, reflect our
current views about our plans, intentions, expectations,
strategies and prospects, and are based on the information
currently available to us and on assumptions we have made and
are not forecasts, promises nor guarantees. Although we believe
that our plans, intentions, expectations, strategies and
prospects as reflected in or suggested by these forward-looking
statements are reasonable, we can give no assurance that the
plans, intentions, expectations or strategies will be attained
or achieved. Furthermore, actual results may differ materially
from those described in the forward-looking statements and will
be affected by a number of risks, uncertainties and assumptions,
including, but not limited to, the success of our ongoing
collaboration with Sentynl, including our co-development
responsibilities through approval of the marketing authorization
application under accelerated assessment with the EMA and
through approval of NULIBRY’s regulatory submission with the
Israeli Ministry of Health, Sentynl’s ability to successfully
develop and commercialize NULIBRY in the U.S. and to develop,
manufacture, and commercialize fosdenopterin globally, Medison
Pharma’s ability to successfully provide access to NULIBRY in
Israel, as well as those risks set forth in the Risk Factors
section of our most recent Annual Report on Form 10-K and
BridgeBio Pharma’s other SEC filings. Moreover, we operate in a
very competitive and rapidly changing environment in which new
risks emerge from time to time. Except as required by applicable
law, we assume no obligation to update publicly any
forward-looking statements, whether as a result of new
information, future events or otherwise.
BridgeBio Contact:
Grace Rauh
[email protected]
(917) 232-5478
Sentynl Therapeutics Contact:
Michael Hercz
[email protected]
(888) 507-5296
Medison Pharma Contact:
Margo Corn-Ladani
[email protected]
(972) 52-660-6710