Data were from a final four-year analysis (up to Week 216) of
the open-label extension trial of a VILTEPSO Phase 2 study
NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka), is a wholly owned subsidiary of
Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President, Dr.
Toru Nakai)
PARAMUS,
N.J., March 19, 2023 /PRNewswire/ -- NS Pharma
is pleased to announce participation in the Muscular Dystrophy
Association's (MDA) Clinical & Scientific Congress 2023 being
held in Dallas, Texas.
The company will be presenting previously reported long-term
efficacy and safety data (final analysis up to Week 216) from the
open-label extension of a Phase 2 study of VILTEPSO®
(viltolarsen).
The presentation will be given by Edward
Smith, MD, Duke University
School of Medicine. For more information, please visit the MDA
Congress website to view the abstract:
https://www.mdaconference.org/abstract-library/results-of-a-4-year-viltolarsen-extension-study-of-functional-and-safety-outcomes/
"There have been amazing scientific advances in the treatment of
Duchenne, which is why it is important to keep the community
informed about existing therapeutic options and the long-term
evidence these treatments have generated," said Dr. Smith. "The
Muscular Dystrophy Association is one of the premier advocacy
groups supporting the Duchenne community and I am pleased to take
part in this year's knowledge sharing at the organization's
Clinical & Scientific Congress on behalf of NS Pharma."
About VILTEPSO® (viltolarsen) injection
Prior to its
approval in the U.S. in August 2020,
VILTEPSO was granted Priority Review as well as Rare Pediatric
Disease, Orphan Drug and Fast Track Designations. In March 2020, VILTEPSO was approved in Japan for the treatment of patients with DMD
who are amenable to exon 53 skipping therapy. Prior to its approval
in Japan, VILTEPSO was granted
with the SAKIGAKE designation, Orphan Drug designation, and
designation of Conditional Early Approval System.
Indication
VILTEPSO is indicated for the treatment of
Duchenne muscular dystrophy (DMD) in patients who have a confirmed
mutation of the DMD gene that is amenable to exon 53 skipping. This
indication is approved under accelerated approval based on an
increase in dystrophin production in skeletal muscle observed in
patients treated with VILTEPSO. Continued approval for this
indication may be contingent upon verification and description of
clinical benefit in a confirmatory trial.
Important Safety Information
Warnings and Precautions:
Kidney toxicity was observed in animals who received viltolarsen.
Although kidney toxicity was not observed in the clinical studies
with VILTEPSO, the clinical experience with VILTEPSO is limited,
and kidney toxicity, including potentially fatal
glomerulonephritis, has been observed after administration of some
antisense oligonucleotides. Kidney function should be monitored in
patients taking VILTEPSO. Serum creatinine may not be a reliable
measure of kidney function in DMD patients.
Serum cystatin C, urine dipstick, and urine
protein-to-creatinine ratio should be measured before starting
VILTEPSO. Consider also measuring glomerular filtration rate before
starting VILTEPSO. During treatment, monitor urine dipstick every
month, and serum cystatin C and urine protein-to-creatinine ratio
every three months.
Urine should be free of excreted VILTEPSO for monitoring of
urine protein. Obtain urine either prior to VILTEPSO infusion, or
at least 48 hours after the most recent infusion. Alternatively,
use a laboratory test that does not use the reagent pyrogallol red,
which has the potential to generate a false positive result due to
cross reaction with any VILTEPSO in the urine. If a persistent
increase in serum cystatin C or proteinuria is detected, refer to a
pediatric nephrologist for further evaluation.
Adverse Reactions: The most common adverse reactions include
upper respiratory tract infection, injection site reaction, cough,
and pyrexia.
To report an adverse event, or for general inquiries, please
call NS Pharma Medical Information at 1-866-NSPHARM
(1-866-677-4276)
For more information about VILTEPSO, see full Prescribing
Information.
About NS Pharma, Inc.
NS Pharma, Inc., is a wholly
owned subsidiary of Nippon Shinyaku Co., Ltd. For more information,
please visit http://www.nspharma.com. NS Pharma is a registered
trademark of the Nippon Shinyaku group of companies.
Contact
U.S. Media Contact:
media@nspharma.com
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content:https://www.prnewswire.com/news-releases/viltepso-viltolarsen-injection-open-label-extension-clinical-trial-data-scheduled-for-presentation-at-the-mda-clinical--scientific-conference-2023-301775371.html
SOURCE NS Pharma