New drug for Duchy muscular dystrophy (DMD)! FDA approves second 53 exon jump therapy Viltepso!

!-- NS Pharma, a subsidiary of Nippon Shinyaku, Japan, announced recently that the U.S. Food and Drug Administration (FDA) Viltepso (Viltolarsen) has been approved for use in patients with Duchymy muscular dystrophy (DMD) who have been genetically tested for mutations that are suitable for treatment with exon 53 skipping, which accounts for about 8% of the total number of DMD patients.
in March, Viltepso was approved in Japan.
medication, Viltepso is administered intravenously, once a week at a dose of 80 mg/kg, at home/hospital/treatment centres and administered by trained health care professionals.
Viltepso is the FDA-approved second treatment for DMD, exon skip therapy No. 53.
end of 2019, Sarepta's drug Vyondys 53 (golodirsen) was approved by the FDA.
2 drugs are antisant oligonucleotide drugs, the mechanism of action is the same, by shielding (jumping) anti-muscular dystrophy protein (anti-muscular atrophy protein, Dys) gene exon 53 to promote the production of functional anti-muscular dystrophy protein.
based on increased levels of antimyostrophy protein, a key protein that supports muscle health, Viltepso received accelerated FDA approval.
the lack of antimyostrophy protein is the root cause of DMD, increasing antimyostrophy protein levels as early as possible is a key goal in treating DMD.
it's worth noting that Viltepso is the first and only exon skip therapy that has been shown to increase antimyostrogen levels in children as young as 4 years of age.
approval of Viltepso will depend on whether there are clinical benefits in Phase III validated clinical trials.
Viltepso was evaluated in two clinical studies involving 32 male DMD patients.
One was a two-cycle Phase II study in North America on DMD boys between the ages of 4 and 10 (study 1, n- 16) and the other was a multi-center open label study (study 2, n-16) in Japan for DMD boys between the ages of 5 and 18.
of antimyostrophy protein levels was confirmed in study 1.
the study, 100% of patients (8/8) who received the recommended dose of 80 mg/kg/wk (N=8) showed an increase in antimyostrophy protein levels after treatment with Viltepso, and 88% (7/8) had antimyostrogen levels of 3% or higher than normal.
Overall, patients with antimyostrophy protein levels were 0.6% of normal during baseline examinations, while patients were observed to increase their antimyostrophy protein levels to 6% of normal levels after 20-24 weeks of treatment with Virtepso (80 mg/kg/wk).
safety, DMD patients treated weekly with Viltepso (80 mg/kg/wk) (combined with 2 studies), the most common side effects include upper respiratory tract infections, injection site reactions, cough and fever.
FDA concluded that the applicant's data showed an increase in the production of antimyostrophy protein in DMD patients, which could reasonably predict the clinical benefits of Virtepso in DMD patients who were genetically tested to have mutations that were proven to be suitable for treatment with the skipping exon No. 53.
clinical efficacy of the drug has not been proven.
decision, the FDA considered the potential risks associated with the drug, the lethality and debilitating nature of the disease, and the lack of available treatments.
as part of an accelerated approval process, the FDA requires NS Pharma to conduct clinical trials to confirm the clinical benefits of the drug.
the ongoing study was designed to assess whether Viltepso improved standing time in DMD patients.
if the trial fails to verify clinical efficacy, the FDA may initiate procedures to revoke approval of the drug.
Although nephrotoxicity has not been observed in Viltepso clinical studies, Viltepso has limited clinical experience and has been observed after the drug was given some antisemical oligonucleotides, including potentially fatal cytocytopheritis.
taking Viltepso should monitor kidney function.
children with DMD (Photo: University of Florida) Duchy muscular dystrophy (DMD) is a rare and fatal neuromuscular genetic disease characterized by aggressive muscle degeneration and weakness, the most common form of muscular dystrophy.
DMD is caused by mutations in the DMD gene that lead to the loss of muscular dystrophy proteins.
symptoms of the disease usually appear between the ages of 3 and 5 and worsen over time.
dmD patients will experience symptoms of recessive and irreversible muscle loss by the age of 2.
heart and respiratory muscle problems begin in ad age and can lead to serious life-threatening complications.
the disease is widespread and fatal, and patients usually die in their 20s.
worldwide, about one in every 3,600 boys has DMD;
Sarepta is an industry leader in treating DMD with "exon skip therapy."
so far, the company has received FDA approval for two DMD skip therapies: (1) In 2016, Exondys 51 (eteplirsen) was approved for the treatment of DMD patients who were tested to be suitable for treatment with exon 51 skipping, which accounts for about 12% of all DMD patients.
(2) In 2019, Vyondys 53 (golodirsen) was approved for the treatment of DMD patients who have been tested to be suitable for treatment with exon 53 skipping, which accounts for about 8% of the total number of DMD patients.
currently, Sarepta's third DMD skip therapy, Casimersen, is also under FDA scrutiny for treating DMD patients who are suitable for skipping exon 45, which accounts for about 8 percent of all DMD patients.
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