MeiraGTx Announces AAV-CNGA3 Granted Rare Pediatric Disease Designation by the U.S. FDA for the Treatment of Achromatopsia
August 27 2018 - 08:00AM
MeiraGTx Holdings Plc (NASDAQ:MGTX), a vertically integrated,
clinical stage gene therapy company, today announced that the
Offices of Orphan Products Development and Pediatric Therapeutics
of the U.S. Food and Drug Administration (FDA) have granted rare
pediatric disease designation to the Company’s gene therapy product
candidate AAV-CNGA3 for the treatment of patients with
achromatopsia (ACHM) due to mutations in the CNGA3 gene.
ACHM is an inherited retinal disease that severely limits a
person’s sight by preventing cone photoreceptors in the eye from
functioning. Individuals with ACHM are often legally blind from
birth, have extreme sensitivity to light, and experience
involuntary eye movements. AAV-CNGA3 is an investigational gene
therapy treatment designed to restore cone function, delivered to
the cone receptors at the back of the eye via subretinal
injection.
“We are very pleased that the FDA and EMA continue to recognize
the important research MeiraGTx is doing for people living with
rare inherited disorders such as ACHM,” said Zandy Forbes, Ph.D.,
chief executive officer of MeiraGTx. “Our focus remains on patient
benefit and developing products that offer a cure to people living
with serious diseases. Working closely with expert clinicians and
the FDA, we look forward to bringing impactful therapies to those
in need.”
In 2018, AAV-CNGA3 was granted orphan drug designation (ODD) by
the FDA and received a positive opinion from the European Medicines
Agency’s (EMA) Committee for Orphan Medicinal
Products recommending orphan medicinal product
designation. Both the ODD and EMA positive opinion for
AAV-CNGA3 are indicated for the treatment of ACHM caused by
mutations in the CNGA3 gene.
A rare pediatric disease designation may be granted by the FDA
to drugs and biologics intended to treat certain orphan diseases
affecting fewer than 200,000 patients in the U.S., the serious
or life-threatening manifestations of which primarily affect
individuals aged 18 years or younger. Under the FDA’s Rare
Pediatric Disease Priority Review Voucher program, a sponsor that
receives approval for a biologics license application for a rare
pediatric disease may be eligible to receive a voucher for a
priority review of a subsequent marketing application for a
different product.
About Achromatopsia Achromatopsia is an
inherited retinal disorder that specifically prevents cone
photoreceptors from functioning. ACHM is characterized by severely
reduced visual acuity of 20/200 or worse, disabling light
sensitivity (photoaversion) and involuntary back and forth eye
movements (nystagmus). ACHM occurs in approximately one in 30,000
people in the United States, with 92 percent of cases caused by
mutations
in CNGB3 and CNGA3 genes. Currently, there
are no effective treatments for this disease.
About MeiraGTx MeiraGTx (NASDAQ:MGTX)
is a vertically integrated, clinical stage gene therapy company
with four ongoing clinical programs and a broad pipeline of
preclinical and research programs. MeiraGTx has core
capabilities in viral vector design and optimization and gene
therapy manufacturing, as well as a potentially transformative gene
regulation technology. Led by an experienced management
team, MeiraGTx has taken a portfolio approach by
licensing, acquiring and developing technologies that give depth
across both product candidates and indications. MeiraGTx’s initial
focus is on three distinct areas of unmet medical need: inherited
retinal diseases, severe forms of xerostomia and neurodegenerative
diseases. Though initially focusing on the eye, salivary gland and
central nervous system, MeiraGTx intends to expand its
focus in the future to develop additional gene therapy treatments
for patients suffering from a range of serious diseases.
For more information, please visit www.meiragtx.com.
Forward-Looking StatementsThis press release
contains forward-looking statements within the meaning of the
Private Securities Litigation Reform Act of 1995. All statements
contained in this press release that do not relate to matters of
historical fact should be considered forward-looking statements,
including, without limitation, statements regarding product
pipeline, anticipated product benefits, goals and strategic
priorities, product candidate development, growth expectations or
targets and pre-clinical and clinical data, as well as statements
that include the words “expect,” “intend,” “plan,” “believe,”
“project,” “forecast,” “estimate,” “may,” “should,” “anticipate”
and similar statements of a future or forward-looking nature. These
forward-looking statements are based on management’s current
expectations. These statements are neither promises nor guarantees,
but involve known and unknown risks, uncertainties and other
important factors that may cause actual results, performance or
achievements to be materially different from any future results,
performance or achievements expressed or implied by the
forward-looking statements, including, but not limited to, our
incurrence of significant losses; any inability to achieve or
maintain profitability, acquire additional capital, identify
additional and develop existing product candidates, continue
operating as a going concern, successfully execute strategic
priorities, bring product candidates to market, build-out the
manufacturing facility and processes, successfully enroll patients
in and complete clinical trials, accurately predict growth
assumptions, recognize benefits of any orphan drug designations,
retain key personnel or attract qualified employees, or incur
expected levels of operating expenses; failure of early data to
predict eventual outcomes; failure to obtain FDA or other
regulatory approval for product candidates within expected time
frames or at all; the novel nature and impact of negative public
opinion of gene therapy; failure to comply with ongoing regulatory
obligations; contamination or shortage of raw materials; changes in
healthcare laws; risks associated with our international
operations; significant competition in the pharmaceutical and
biotechnology industries; dependence on third parties; risks
related to intellectual property; litigation risks; and the other
important factors discussed under the caption “Risk Factors” in our
Quarterly Report on Form 10-Q for the quarterly period ended June
30, 2018 as such factors may be updated from time to time in our
other filings with the SEC, which are accessible on the SEC’s
website at www.sec.gov. These and other important factors could
cause actual results to differ materially from those indicated by
the forward-looking statements made in this press release. Any such
forward-looking statements represent management’s estimates as of
the date of this press release. While we may elect to update such
forward-looking statements at some point in the future, unless
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subsequent events cause our views to change. Thus, one should not
assume that our silence over time means that actual events are
bearing out as expressed or implied in such forward-looking
statements. These forward-looking statements should not be relied
upon as representing our views as of any date subsequent to the
date of this press release.
Contacts Investors: MeiraGTx
Investors@meiragtx.com
or
Media: W2O pure Kelly Boothe, Ph.D. (415)
946-1076kboothe@w2ogroup.com
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