SOUTH PLAINFIELD, N.J.,
Feb. 26, 2021 /PRNewswire/ -- PTC
Therapeutics, Inc. (NASDAQ: PTCT), today announced that the
European Medicines Agency (EMA) Committee for Medicinal Products
for Human Use (CHMP) has recommended the approval of Evrysdi™
(risdiplam) for the treatment of 5q spinal muscular atrophy (SMA)
in patients 2 months and older, with a clinical diagnosis of SMA
Type 1, Type 2 or Type 3 with one to four SMN2 copies. The CHMP
review was completed under the accelerated assessment pathway,
which is offered to medicines deemed to be of major interest for
public health and therapeutic innovation.
"Today's CHMP opinion marks another important advancement in
ensuring Evrysdi is available to SMA patients around the globe,"
said Stuart W. Peltz, Ph.D., Chief
Executive Officer, PTC Therapeutics, Inc. "Given its proven
efficacy and strong safety profile, coupled with the convenience of
an at-home administration, we expect Evrysdi to become the
treatment of choice for SMA patients and their families."
The CHMP recommendation is based on data from the FIREFISH study
in infants aged 2 to 7 months with symptomatic Type 1 SMA and the
SUNFISH study in children and young adults with Type 2 or 3 SMA.
The two pivotal studies were designed to represent a broad spectrum
of people living with SMA, and SUNFISH is the first and only
placebo-controlled trial to include adults with Types 2 and 3 SMA.
A final decision regarding approval is expected from the European
Commission in the next two months and will be applicable to all 27
European Union member states, as well as Iceland, Norway, and Liechtenstein.
Evrysdi is designed to treat SMA by increasing and sustaining
the production of the survival motor neuron (SMN) protein. SMN
protein is found throughout the body and is critical for
maintaining healthy motor neurons and movement. More than 2,500
patients have now been treated with Evrysdi in clinical trials,
compassionate use programs and real-world settings, with patients
ranging from birth to over 70 years old including those previously
treated with other SMA therapies.
Evrysdi has been approved in seven countries thus far including
the U.S., Chile, Brazil, Ukraine, South
Korea, Georgia and
Russia. Evrysdi is under review in
a further 30 countries including Japan and China.
Evrysdi is based on PTC science and is commercialized in
the United States by Genentech, a
member of the Roche Group. Roche leads the clinical development of
Evrysdi as part of a collaboration with the SMA Foundation and PTC
Therapeutics.
About Spinal Muscular Atrophy (SMA)
Spinal muscular atrophy (SMA) is a severe, progressive
neuromuscular disease that can be fatal. It affects approximately 1
in 10,000 babies and when untreated is the leading genetic cause of
infant mortality. SMA is caused by a mutation of the survival motor
neuron 1 (SMN1) gene, which leads to a deficiency of SMN protein.
This protein is found throughout the body and is essential to the
function of nerves that control muscles and movement. Without it,
nerve cells cannot function correctly, leading to progressive
muscle weakness over time. 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.
About Evrysdi™ (risdiplam)
Evrysdi™ is a survival motor neuron 2 (SMN2)-directed RNA
splicing modifier designed to treat SMA caused by mutations in
chromosome 5q that lead to SMN protein deficiency. Evrysdi™ is
designed to distribute evenly to all parts of the body, including
the central nervous system (CNS). Evrysdi™ is administered daily at
home in liquid form by mouth or feeding tube. The U.S. Food and
Drug Administration recently approved Evrysdi™ (risdiplam) for the
treatment of spinal muscular atrophy for adults and children 2
months and older. Evrysdi™ (risdiplam) is marketed in the United States by Genentech, a member of
the Roche Group.
About the Risdiplam Clinical Studies
FIREFISH (NCT02913482) is an open-label, two-part
pivotal clinical trial in infants with Type 1 SMA. Part 1 was a
dose-escalation study in 21 infants with the primary objective of
assessing the safety profile of risdiplam in infants and
determining the dose for Part 2. Part 2 is a pivotal, single-arm
study of risdiplam in 41 infants with Type 1 SMA treated for two
years followed by an open-label extension. The primary objective of
Part 2 was 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 five seconds). The study met its
primary endpoint.
SUNFISH (NCT02908685) is a two part, double-blind,
placebo controlled pivotal study in people aged 2 to 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
the Motor Function Measure 32 (MFM-32) scale at 12 months. MFM-32
is a validated scale used to evaluate fine and gross motor function
in people with neurological disorders, including SMA. The study met
its primary endpoint.
Clinical Trial Safety Data
The safety profile of Evrysdi™ was established across FIREFISH
and SUNFISH pivotal trials. The most common adverse reactions in
later-onset SMA (incidence of at least 10 percent of patients
treated with Evrysdi™ and more frequently than control) were fever,
diarrhea, and rash. The most common adverse reactions in
infantile-onset SMA were similar to those observed in later-onset
SMA patients. Additionally, the most common adverse reactions
(incidence of at least 10 percent) were upper respiratory tract
infection, pneumonia, constipation, and vomiting.
In addition to FIREFISH and SUNFISH, Evrysdi™ is being evaluated
in a broad range of people with SMA, including in:
JEWELFISH (NCT03032172) is an open-label exploratory
trial designed to assess the safety, tolerability, pharmacokinetics
(PK) and pharmacodynamics (PD) in people with SMA aged 6 months to
60 years who received other investigational or approved SMA
therapies for at least 90 days prior to receiving Evrysdi™. The
study has completed recruitment (n=174).
RAINBOWFISH (NCT03779334) is an open-label,
single-arm, multi-center study, investigating the efficacy, safety,
pharmacokinetics, and pharmacodynamics of risdiplam in babies
(~n=25), from birth to 6 weeks old (at first dose), with
genetically diagnosed SMA, who are not yet presenting 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 pipeline of transformative medicines and our mission to
provide access to best-in-class treatments for patients who have an
unmet medical need. The Company's strategy is to leverage its
strong scientific expertise and global commercial infrastructure to
maximize value for its patients and other stakeholders. To learn
more about PTC, please visit us on www.ptcbio.com and follow
us on Facebook, on Twitter at @PTCBio, and on LinkedIn.
For More Information:
Investors:
Kylie
O'Keefe
+1 (908) 300-0691
kokeefe@ptcbio.com
Media:
Jane Baj
+1 (908) 912-9167
jbaj@ptcbio.com
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 historical 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 and,
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 outcome of pricing,
coverage and reimbursement negotiations with third party payors for
PTC's products or product candidates that PTC commercializes or may
commercialize in the future, including Evrysdi™; 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 Evrysdi™; the eligible patient base and commercial
potential of Evrysdi™ 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 (Securities and Exchange Commission). 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 or product candidate will receive or maintain regulatory
approval in any territory, or prove to be commercially successful,
including Evrysdi™.
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.
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SOURCE PTC Therapeutics, Inc.