Viltolarsen Available Through Expanded Access Program


Eligible patients with Duchenne muscular dystrophy may receive the drug while it is under priority review by the FDA.

Nippon Shinyaku has announced that eligible patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping therapy may receive viltolarsen through the launch of the viltolarsen Expanded Access Program.

Viltolarsen, an exon-skipping therapy injected directly into the bloodstream, had its new drug application (NDA) accepted in early February 2020. It had previously been granted fast track, orphan drug, and rare disease designations in addition to priority review. The designations were based off results from the company’s phase 2 clinical trial (NCT02740972), where researchers observed both drug-induced dystrophin and exon-skipping efficiency in both dose groups of viltolarsen-treated patients.

The launch of the Expanded Access Program allows eligible patients to receive viltolarsen while it remains an investigational drug under FDA review. Though the drug has not been FDA approved, Nippon Shinyaku will continue to evaluate the safety and efficacy of viltolarsen in patients with DMD amenable to exon 53 skipping therapy.

Expanded access may be appropriate to patients if they meet all 5 points of criteria, which include having a serious disease or condition; those whose life is immediately threatened by their disease or condition with no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition; there is no option to enroll in a clinical trial; potential patient benefit justifies the potential risks of treatment; and providing the investigational medical product will not interfere with investigational trials that could support a medical product’s development or marketing approval for the treatment indication.

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A patient’s healthcare provider must make a request to NS Pharma, a subsidiary of Nippon Shinyaku, if they are interested in applying for the program. NS Pharma is asking physicians to inquire via email, at The program will also allow physicians to prescribe the drug at no cost.

The company is currently still recruiting patients for its ongoing phase 3 RACER53 trial (NCT04060199), which will randomize ambulatory boys with DMD with 80 mg/kg of viltolarsen or placebo for up to 48 weeks. The double-blind, multi-center study is expected to enroll 74 patients amenable to exon 53 skipping and will include functional tests such as Time to Stand Test (TTSTAND), Time to Run/Walk 10 Meters Test (TTRW), Six-minute Walk Test (6MWT), North Star Ambulatory Assessment (NSAA), Time to Climb 4 Steps Test (TTCLIMB) and Hand-held dynameter.

Viltolarsen aims to become the second drug approved, behind Sarepta Therapeutics’ Vyondys 53 (golodirsen), for DMD patients with mutations amenable for exon 53 skipping. Viltolarsen’s Precription Drug User Fee Act (PDUFA) date is set sometime within the third quarter of 2020.


Expanded access program launched for viltolarsen, an investigational exon 53 skipping antisense oligonucleotide [news release] Paramus, NJ: NS Pharma; Published March 9, 2020. Accessed March 23, 2020.

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