SOUTH PLAINFIELD, N.J.,
April 7, 2020 /PRNewswire/
-- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that
the U.S. Food and Drug Administration (FDA) has extended the
Prescription Drug User Fee Act (PDUFA) date for its review of the
New Drug Application (NDA) of risdiplam to August 24, 2020. Roche recently submitted
additional data including comprehensive data from SUNFISH part 2 to
help provide access to risdiplam for a broad range of people living
with spinal muscular atrophy (SMA), triggering this extension. The
FDA has informed Roche that the review team is working
expeditiously to complete their review of the application as
quickly as possible. The FDA has also indicated to Roche that no
substantive review issues have been identified to date.
"We are encouraged that the FDA has no substantive review
issues. Their interest in the additional results from the clinical
studies demonstrating risdiplam's activity supports our goal of
enabling access to this important therapy for all SMA patients,"
stated Stuart W. Peltz, Ph.D., Chief
Executive Officer, PTC Therapeutics. "Enabling access to a
home-administered oral therapy for a broad patient population is
critically important and we look forward to the FDA living up to
its commitment to review the application as quickly as
possible."
In order to further support broad, global access to risdiplam
for people living with SMA, Roche has submitted applications for
approval in Indonesia,
Taiwan, Chile, Brazil, South
Korea and Russia and the
submission of the filing in China
is imminent. The submission of a Marketing Authorization
Application (MAA) to the EMA and filings in other international
markets remain on track for mid-2020.
In November 2019, the FDA granted
Priority Review for risdiplam. Risdiplam has the broadest clinical
trial program being evaluated to treat SMA, with patients ranging
from birth to 60 years old, and includes patients previously
treated with SMA-targeting therapies, including approved
treatments. The clinical trial population represents the diverse,
real-world spectrum of people living with this disease with the aim
of ensuring access for all appropriate patients. To date, more than
400 patients have been treated with risdiplam across all studies to
date, with no treatment-related safety findings leading to study
withdrawal in any risdiplam trial. The SMA program is a
collaboration between PTC, the SMA Foundation, and Roche.
About Spinal Muscular Atrophy (SMA)
Spinal muscular
atrophy (SMA) is a severe, inherited, progressive neuromuscular
disease that causes devastating muscle atrophy and disease-related
complications. It is the most common genetic cause of infant
mortality and one of the most common rare diseases, affecting
approximately one in 11,000 babies. SMA leads to the progressive
loss of nerve cells in the spinal cord that control muscle
movement. Depending on the type of SMA, an individual's physical
strength and their ability to walk, eat or breathe can be
significantly diminished or lost.
SMA is caused by a mutation in the survival motor neuron 1
(SMN1) gene that results in a deficiency of SMN protein. SMN
protein is found throughout the body and increasing evidence
suggests SMA is a multi-system disorder and the loss of SMN protein
may affect many tissues and cells, which can stop the body from
functioning.
About risdiplam
Risdiplam is an investigational
survival motor neuron2 (SMN2) splicing modifier for SMA and is an
orally administered liquid. It is designed to durably increase and
sustain SMN protein levels both throughout the central nervous
system and in peripheral tissues of the body. Risdiplam is being
studied in a broad clinical trial program in SMA, with patients
ranging from birth to 60 years old, and includes patients
previously treated with other SMA-targeting therapies. The clinical
trial population represents the broad, real-world spectrum of
people living with this disease. The risdiplam clinical development
program was designed with the aim of enabling access for all
appropriate patients.
Risdiplam is currently being evaluated in four multicenter
trials in people with SMA:
- SUNFISH (NCT02908685) – SUNFISH is a two-part, double-blind,
placebo-controlled pivotal study in people aged 2-25 years with
types 2 or 3 SMA. Part 1 (n=51) determined the dose for the
confirmatory Part 2. Part 2 (n=180) evaluated motor function using
total score of Motor Function Measure 32 (MFM-32) at 12 months.
MFM-32 is a validated scale used to evaluate fine and gross motor
function in people with neurological disorders, including SMA.
- FIREFISH (NCT02913482) – an open-label, two-part pivotal
clinical trial in infants with type 1 SMA. Part 1 was a
dose-escalation study in 21 infants. The primary objective of Part
1 was to assess the safety profile of risdiplam in infants and
determine the dose for Part 2. Part 2 is a pivotal, single-arm
study of risdiplam in 41 infants with type 1 SMA treated for 24
months, followed by an open-label extension. Enrollment for Part 2
was completed in November 2018. The
primary objective of Part 2 is to assess efficacy as measured by
the proportion of infants sitting without support after 12 months
of treatment, as assessed in the Gross Motor Scale of the Bayley
Scales of Infant and Toddler Development – Third Edition (BSID-III)
(defined as sitting without support for 5 seconds).
- JEWELFISH (NCT03032172) – an open-label exploratory trial in
people with SMA aged 6 months–60 years who have been previously
treated with SMA-directed therapies. The study has completed
recruitment.
- RAINBOWFISH (NCT03779334) – an open-label, single-arm,
multicenter study, investigating the efficacy, safety,
pharmacokinetics and pharmacodynamics of risdiplam in babies
(~n=25), from birth to six weeks of age (at first dose) with
genetically diagnosed SMA who are not yet presenting with symptoms.
The study is currently recruiting.
About PTC Therapeutics, Inc.
PTC is a science-driven,
global biopharmaceutical company focused on the discovery,
development and commercialization of clinically differentiated
medicines that provide benefits to patients with rare disorders.
PTC's ability to globally commercialize products is the foundation
that drives investment in a robust and diversified pipeline of
transformative medicines and our mission to provide access to
best-in-class treatments for patients who have an unmet medical
need. To learn more about PTC, please visit us
at www.ptcbio.com and follow us on Facebook,
on Twitter at @PTCBio, and on LinkedIn.
Forward-Looking Statements:
This press release
contains forward-looking statements within the meaning of The
Private Securities Litigation Reform Act of 1995. All statements
contained in this release, other than statements of historic fact,
are forward-looking statements, including statements regarding: the
future expectations, plans and prospects for PTC; advancement of
PTC's joint collaboration program in SMA, including any potential
regulatory submissions, regulatory approvals or commercial
prospects; PTC's strategy, future operations, future financial
position, future revenues, projected costs; and the objectives of
management. Other forward-looking statements may be identified by
the words "guidance", "plan," "anticipate," "believe," "estimate,"
"expect," "intend," "may," "target," "potential," "will," "would,"
"could," "should," "continue," and similar expressions.
PTC's actual results, performance or achievements could differ
materially from those expressed or implied by forward-looking
statements it makes as a result of a variety of risks and
uncertainties, including those related to: the enrollment, conduct,
and results of studies under the SMA collaboration and events
during, or as a result of, the studies that could delay or prevent
further development under the program, including any potential
regulatory submissions and potential commercialization with regards
to risdiplam; the eligible patient base and commercial potential of
risdiplam or any of PTC's other product candidates; and the factors
discussed in the "Risk Factors" section of PTC's most recent Annual
Report on Form 10-K, as well as any updates to these risk factors
filed from time to time in PTC's other filings with the SEC. You
are urged to carefully consider all such factors.
As with any pharmaceutical under development, there are
significant risks in the development, regulatory approval and
commercialization of new products. There are no guarantees that any
product will receive or maintain regulatory approval in any
territory, or prove to be commercially successful, including
risdiplam.
The forward-looking statements contained herein represent PTC's
views only as of the date of this press release and PTC does not
undertake or plan to update or revise any such forward-looking
statements to reflect actual results or changes in plans,
prospects, assumptions, estimates or projections, or other
circumstances occurring after the date of this press release except
as required by law.
For More Information:
Investors:
Alex
Kane
+1 (908) 912- 9643
akane@ptcbio.com
Media:
Jane Baj
+1 (908) 912-9167
jbaj@ptcbio.com
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SOURCE PTC Therapeutics, Inc.