BridgeBio Pharma And Affiliate Origin Biosciences Announces FDA Acceptance Of Its New Drug Application For Fosdenopterin For The Treatment Of MoCD Type A
Application accepted under Priority Review designation with Breakthrough Therapy Designation and Rare Pediatric Disease Designation previously granted
There are currently no approved therapies for the treatment of MoCD Type A, which results in severe and irreversible neurological injury for infants and children.
This is BridgeBio’s first NDA acceptance
SAN FRANCISCO, September 29, 2020 – BridgeBio Pharma, Inc. (Nasdaq: BBIO) and affiliate Origin Biosciences today announced the US Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for fosdenopterin (previously BBP-870/ORGN001), a cyclic pyranopterin monophosphate (cPMP) substrate replacement therapy, for the treatment of patients with molybdenum cofactor deficiency (MoCD) Type A.
The NDA has been granted Priority Review designation. Fosdenopterin has previously been granted Breakthrough Therapy Designation and Rare Pediatric Disease Designation in the US and may be eligible for a priority review voucher if approved. It received Orphan Drug Designation in the US and Europe. This is BridgeBio’s first NDA acceptance.
“We want to thank the patients, families, scientists, physicians
and all others involved who helped us reach this critical
milestone,” said BridgeBio CEO and founder Neil Kumar, Ph.D.
“MoCD Type A is a devastating disease with a median survival of
less than four years and we are eager for our investigational
therapy to be available to patients, who currently have no
approved treatment options. BridgeBio exists to help as many
patients as possible afflicted with genetic diseases, no matter
how rare. We are grateful that the FDA has accepted our first
NDA for priority review and we look forward to submitting our
second NDA later this year for infigratinib for second line
treatment of cholangiocarcinoma.”
About Fosdenopterin
Fosdenopterin is being developed for the treatment of patients
with MoCD Type A. Currently, there are no approved therapies for
the treatment of MoCD Type A, which results in severe and
irreversible neurological injury with a median survival between
3 to 4 years. Fosdenopterin is a first-in-class cPMP
hydrobromide dihydrate and is designed to treat MoCD Type A by
replacing cPMP and permitting the two remaining MoCo synthesis
steps to proceed, with activation of MoCo-dependent enzymes and
elimination of sulfites.
About Molybdenum Cofactor Deficiency (MoCD) Type A
MoCD Type A is an ultra-rare, autosomal recessive, inborn
error of metabolism caused by disruption in molybdenum cofactor
(MoCo) synthesis which is vital to prevent buildup of
s-sulfocysteine, a neurotoxic metabolite of sulfite. Patients
are often infants with severe encephalopathy and intractable
seizures. Disease progression is rapid with a high infant
mortality rate.Those who survive beyond the first few month’s
experience profuse developmental delays and suffer the effects
of irreversible neurological damage, including brain atrophy
with white matter necrosis, dysmorphic facial features, and
spastic paraplegia. Clinical presentation that can be similar to
hypoxic-ischemic encephalopathy (HIE) or other neonatal seizure
disorders may lead to misdiagnosis and underdiagnosis. Immediate
testing for elevated sulfite levels and S-sulfocysteine in the
urine and very low serum uric acid may help with suspicion of
MoCD.
About Origin Biosciences
Origin
Biosciences, an affiliate of BridgeBio Pharma, is a
biotechnology company focused on developing and commercializing
a treatment for Molybdenum Cofactor Deficiency (MoCD) Type A.
Origin is led by a team of veteran biotechnology executives.
Together with patients and physicians, the company aims to bring
a safe, effective treatment for MoCD Type A to market as quickly
as possible. For more information on Origin Biosciences, please
visit the company’s website at www.origintx.com.
About BridgeBio Pharma
BridgeBio is a team of experienced drug discoverers, developers and
innovators working to create life-altering medicines that target
well-characterized genetic diseases at their source. BridgeBio
was founded in 2015 to identify and advance transformative
medicines to treat patients who suffer from Mendelian diseases,
which are diseases that arise from defects in a single gene, and
cancers with clear genetic drivers. BridgeBio’s pipeline of over
20 development programs includes product candidates ranging from
early discovery to late-stage development. For more information
visit bridgebiodev.wpengine.com.
BridgeBio Pharma Forward Looking Statements
This press release contains forward-looking statements.
Statements we make in this press release may include statements
which are not historical facts and are considered
forward-looking within the meaning of Section 27A of the
Securities Act of 1933, as amended, and Section 21E of the
Securities Exchange Act of 1934, as amended, 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 Securities Exchange Act
and are making this statement for purposes of complying with
those safe harbor provisions. These forward-looking statements,
including statements relating to Origin Biosciences’ clinical
development plans, clinical trial results, timing and completion
of clinical trials and regulatory submissions, competitive
environment and clinical and therapeutic potential of BBP-870,
reflect our current views about our plans, intentions,
expectations, strategies and prospects, which are based on the
information currently available to us and on assumptions we have
made. Although we believe that our plans, intentions,
expectations, strategies and prospects as reflected in or
suggested by those 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 variety of
risks and factors that are beyond our control including, without
limitation, Origin Biosciences’ ability to continue its planned
clinical development and regulatory submissions for BBP-870 and
the timing and success of any such continued clinical
development and planned regulatory submissions, as well as those
set forth in the Risk Factors section of BridgeBio Pharma Inc.’s
most recent Quarterly Report on Form 10-Q and our other SEC
filings. Except as required by law, we assume no obligation to
update publicly any forward-looking statements, whether as a
result of new information, future events or otherwise.
Contact:
Grace Rauh
[email protected]
(917) 232-5478