Clementia Granted Rare Pediatric Disease Designation by FDA for Palovarotene for Fibrodysplasia Ossificans Progressiva
11 Février 2019 - 12:30PM
Clementia Pharmaceuticals Inc. (Nasdaq: CMTA), a clinical-stage
biopharmaceutical company innovating treatments for people with
ultra-rare bone disorders and other diseases, today announced that
the U.S. Food and Drug Administration (FDA) has granted Rare
Pediatric Disease designation to palovarotene for the treatment of
fibrodysplasia ossificans progressiva (FOP). Palovarotene, an
investigational therapy for FOP, has previously been granted Orphan
Drug, Fast Track and Breakthrough designations by the FDA for FOP.
“FOP is a rare, devastating disease that begins to irreversibly
impact individuals in childhood,” said Clarissa Desjardins, Ph.D.,
founder and chief executive officer of Clementia. “The receipt of
this designation highlights the urgent need for a treatment for
people with FOP. The entire Clementia team is working towards
an NDA submission in the second half of 2019 in hopes of achieving
our first regulatory approval, an important step toward achieving
our mission of bringing innovative and effective treatments to
individuals who currently have none.”
In October 2018, based on a meeting with the FDA, Clementia
announced that the Agency was supportive of the company’s plan to
submit a New Drug Application (NDA) for palovarotene for the
prevention of heterotopic ossification (HO) associated with flare
up symptoms in patients with FOP. The FDA based its assessment on
the efficacy and safety data generated in the completed Phase 2
clinical program. The company’s NDA preparations are underway, with
a submission targeted for the second half of 2019. If approved,
Clementia expects a first commercial launch for palovarotene in
2020.
The Rare Pediatric Disease Designation qualifies investigational
palovarotene for a Rare Pediatric Disease Priority Review Voucher
(PRV), granted at the time of NDA approval. The PRV Program is
intended to encourage development of therapies to prevent and treat
rare pediatric diseases. This PRV can be redeemed to receive a
priority review of a subsequent marketing application or sold to a
third party.
About PalovarotenePalovarotene is an RARγ
agonist being developed as a potential treatment for patients with
ultra-rare and debilitating bone diseases, including fibrodysplasia
ossificans progressiva (FOP) and multiple osteochondromas (MO), as
well as other diseases. Palovarotene was in-licensed from Roche
Pharmaceuticals, where it was previously evaluated in more than 800
subjects, including 450 patients treated for up to two years.
Palovarotene has received Orphan Drug status for FOP and MO from
the U.S. Food and Drug Administration (FDA) and the European
Medicines Agency (EMA). In addition, palovarotene has been granted
Fast Track, Breakthrough Therapy and Pediatric Rare Disease
designations for FOP from the FDA.
About Fibrodysplasia Ossificans Progressiva
(FOP)FOP is a rare, severely disabling disorder
characterized by heterotopic ossification (HO), or bone that forms
outside the normal skeleton, in muscles, tendons or soft tissue. In
FOP, HO progressively restricts movement by locking joints, leading
to a cumulative loss of function, progressive disability, and
increased risk of early death. FOP is caused by a mutation in the
ACVR1 gene, resulting in excess signaling in the bone morphogenetic
pathway, a key pathway controlling bone growth and development, by
way of both ligand-dependent and independent mechanisms. There are
currently no approved treatments for FOP.
About Clementia Pharmaceuticals
Inc.Clementia is a clinical-stage company innovating
treatments for people with ultra-rare bone disorders and other
diseases with high medical need. The company is preparing to submit
an NDA in the second half of 2019 to seek approval of its lead
product candidate, palovarotene, a novel RARγ agonist, for
fibrodysplasia ossificans progressiva (FOP). The ongoing Phase 3
MOVE Trial is evaluating an additional dosing regimen of
investigational palovarotene for FOP. Palovarotene is also in a
Phase 2 trial, the MO-Ped Trial, for the potential treatment of
multiple osteochondromas (MO, also known as multiple hereditary
exostoses, or MHE). In addition, Clementia has commenced a Phase 1
trial for an eye drop formulation of palovarotene for the potential
treatment of dry eye disease and is also investigating other
conditions that may benefit from RARγ therapy. For more
information, please visit www.clementiapharma.com and connect with
us on Twitter @ClementiaPharma.
Cautionary Note Regarding
Forward-Looking StatementsThis press release may include
“forward-looking statements” within the meaning of the applicable
securities laws, including with respect to the proposed timing of
submission of the NDA for palovarotene. Each forward-looking
statement contained in this press release is subject to known and
unknown risks and uncertainties and other unknown factors that
could cause actual results to differ materially from historical
results and those expressed or implied by such statement. In
addition to statements which explicitly describe such risks and
uncertainties, readers are urged to consider statements labeled
with the terms “believes,” “belief,” “expects,” “intends,”
“anticipates,” “will,” or “plans” to be uncertain and
forward-looking. Applicable risks and uncertainties include, among
others, the Company’s ability to successfully complete in a timely
manner the studies required to be completed in order to submit the
NDA, the Company’s ability to generate revenue and become
profitable; the risks related to its heavy reliance on
palovarotene, its only current product candidate; the risks
associated with the development of palovarotene and any future
product candidate, including the demonstration of efficacy and
safety; its dependence on licensed intellectual property, including
the ability to source and maintain licenses from third-party
owners; as well as the risks identified under the heading “Risk
Factors” in the Company’s Annual Report on Form 20-F filed with the
Securities and Exchange Commission (“SEC”), as well as the other
information its file with the SEC or on SEDAR. Clementia cautions
investors not to rely on the forward-looking statements contained
in this press release when making an investment decision in its
securities. Investors are encouraged to read the Company’s filings
with the SEC or on SEDAR, available at www.sec.gov or
www.sedar.com, for a discussion of these and other risks and
uncertainties. The forward-looking statements in this press release
speak only as of the date of this press release, and the Company
undertakes no obligation to update or revise any of these
statements, whether as a result of new information, future events
or otherwise, except as required by law.
Investor/Media Contact:Joseph WalewiczClementia
Pharmaceuticals Inc.+1-514-940-1080
Alicia Davis THRUST Strategic Communications +1-910-620-3302
alicia@thrustsc.com
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