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Viltolarsen: First Approval | SpringerLink




CAS 2055732-84-6
Mol weight6924.8155

APPROVED FDA 2020/8/12, Viltepso


  • NCNP-01
  • NS-065
  • NS-065/NCNP-01
  • WHO 10771
  • WHO-10771
ViltepsoInjection, solution250 mg/1IntravenousNs Pharma, Inc.2020-08-13Not applicableUS flag 

SYNWatanabe N, Nagata T, Satou Y, Masuda S, Saito T, Kitagawa H, Komaki H, Takagaki K, Takeda S: NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy. Mol Ther Nucleic Acids. 2018 Dec 7;13:442-449. doi: 10.1016/j.omtn.2018.09.017.

US9079934No2011-08-312031-08-31US flag


all-P-ambo-[2′,3′-Azanediyl-P,2′,3′-trideoxy-P-(dimethylamino)-2′,3′-seco](2′-N→5′)(CCTCCGGTTC TGAAGGTGTT C)

C244H381N113O88P20 : 6924.82

Viltolarsen, sold under the brand name Viltepso, is a medication used for the treatment of Duchenne muscular dystrophy (DMD).[3][4][2] Viltolarsen is an antisense oligonucleotide.[3][2]

The most common side effects include upper respiratory tract infectioninjection site reactioncough, and pyrexia (fever).[3][4][2]

Viltolarsen was approved for medical use in the United States in August 2020.[3][4] After golodirsen was approved in December 2019, viltolarsen is the second approved targeted treatment for people with this type of mutation in the United States.[3][5] Approximately 8% of people with DMD have a mutation that is amenable to exon 53 skipping.[3]

Buy Viltepso (viltolarsen) • Price & Costs | TheSocialMedwork

Medical uses

Viltolarsen is indicated for the treatment of Duchenne muscular dystrophy (DMD) in people who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.[3][2]

DMD is a rare genetic disorder characterized by progressive muscle deterioration and weakness.[3] It is the most common type of muscular dystrophy.[3] DMD is caused by mutations in the DMD gene that results in an absence of dystrophin, a protein that helps keep muscle cells intact.[3] The first symptoms are usually seen between three and five years of age and worsen over time.[3] DMD occurs in approximately one out of every 3,600 male infants worldwide; in rare cases, it can affect females.[3]

Adverse effects

The most common side effects include upper respiratory tract infection, injection site reaction, cough, and pyrexia (fever).[3][4][2]

Although kidney toxicity was not observed in the clinical studies, the clinical experience is limited, and kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides.[3]


Viltolarsen was evaluated in two clinical studies with a total of 32 participants, all of whom were male and had genetically confirmed DMD.[3] The increase in dystrophin production was established in one of those two studies, a study that included sixteen DMD participants, with eight participants receiving viltolarsen at the recommended dose.[3] In the study, dystrophin levels increased, on average, from 0.6% of normal at baseline to 5.9% of normal at week 25.[3] Trial 1 provided data for evaluation of the benefits of viltolarsen.[4] The combined populations from both trials provided data for evaluation of the side effects of viltolarsen.[4] Trial 1 was conducted at six sites in the United States and Canada and Trial 2 was conducted at five sites in Japan.[4] All participants in both trials were on a stable dose of corticosteroids for at least three months before entering the trials.[4]

The U.S. Food and Drug Administration (FDA) concluded that the applicant’s data demonstrated an increase in dystrophin production that is reasonably likely to predict clinical benefit in people with DMD who have a confirmed mutation of the dystrophin gene amenable to exon 53 skipping.[3] A clinical benefit of the drug has not been established.[3] In making this decision, the FDA considered the potential risks associated with the drug, the life-threatening and debilitating nature of the disease, and the lack of available therapies.[3]

The application for viltolarsen was granted priority review designation and the FDA granted the approval to NS Pharma, Inc.[3]


  1. ^
  2. Jump up to:a b c d e f “Viltepso- viltolarsen injection, solution”DailyMed. 12 August 2020. Retrieved 18 August 2020.
  3. Jump up to:a b c d e f g h i j k l m n o p q r s t u “FDA Approves Targeted Treatment for Rare Duchenne Muscular Dystrophy Mutation”U.S. Food and Drug Administration (FDA) (Press release). 12 August 2020. Retrieved 12 August 2020.  This article incorporates text from this source, which is in the public domain.
  4. Jump up to:a b c d e f g h “Drug Trials Snapshots: Viltepso”U.S. Food and Drug Administration. 12 August 2020. Retrieved 18 August 2020.  This article incorporates text from this source, which is in the public domain.
  5. ^ Anwar S, Yokota T (August 2020). “Golodirsen for Duchenne muscular dystrophy”. Drugs of Today56 (8): 491–504. doi:10.1358/dot.2020.56.8.3159186PMID 33025945.

Further reading

External links

Clinical data
Trade namesViltepso
Other namesNS-065/NCNP-01
License dataUS DailyMedViltolarsen
US: N (Not classified yet)[1]
Routes of
Drug classAntisense oligonucleotide
ATC codeNone
Legal status
Legal statusUS: ℞-only [2]In general: ℞ (Prescription only)
CAS Number2055732-84-6
Chemical and physical data
Molar mass6924.910 g·mol−1

//////////Viltolarsen, Viltepso, 维托拉生  , FDA 2020, EU 2020, APPROVALS 2020, NCNP-01, NS-065, NS-065/NCNP-01, WHO 10771, WHO-10771, ビルトラルセン

1 Comment

  1. Sunil says:

    Is this medicine helpful for BMD Or any other if yes please advise

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DR ANTHONY MELVIN CRASTO, Born in Mumbai in 1964 and graduated from Mumbai University, Completed his Ph.D from ICT, 1991,Matunga, Mumbai, India, in Organic Chemistry, The thesis topic was Synthesis of Novel Pyrethroid Analogues, Currently he is working with AFRICURE PHARMA, ROW2TECH, NIPER-G, Department of Pharmaceuticals, Ministry of Chemicals and Fertilizers, Govt. of India as ADVISOR, earlier assignment was with GLENMARK LIFE SCIENCES LTD, as CONSUlTANT, Retired from GLENMARK in Jan2022 Research Centre as Principal Scientist, Process Research (bulk actives) at Mahape, Navi Mumbai, India. Total Industry exp 32 plus yrs, Prior to joining Glenmark, he has worked with major multinationals like Hoechst Marion Roussel, now Sanofi, Searle India Ltd, now RPG lifesciences, etc. He has worked with notable scientists like Dr K Nagarajan, Dr Ralph Stapel, Prof S Seshadri, etc, He did custom synthesis for major multinationals in his career like BASF, Novartis, Sanofi, etc., He has worked in Discovery, Natural products, Bulk drugs, Generics, Intermediates, Fine chemicals, Neutraceuticals, GMP, Scaleups, etc, he is now helping millions, has 9 million plus hits on Google on all Organic chemistry websites. His friends call him Open superstar worlddrugtracker. His New Drug Approvals, Green Chemistry International, All about drugs, Eurekamoments, Organic spectroscopy international, etc in organic chemistry are some most read blogs He has hands on experience in initiation and developing novel routes for drug molecules and implementation them on commercial scale over a 32 PLUS year tenure till date Feb 2023, Around 35 plus products in his career. He has good knowledge of IPM, GMP, Regulatory aspects, he has several International patents published worldwide . He has good proficiency in Technology transfer, Spectroscopy, Stereochemistry, Synthesis, Polymorphism etc., He suffered a paralytic stroke/ Acute Transverse mylitis in Dec 2007 and is 90 %Paralysed, He is bound to a wheelchair, this seems to have injected feul in him to help chemists all around the world, he is more active than before and is pushing boundaries, He has 100 million plus hits on Google, 2.5 lakh plus connections on all networking sites, 100 Lakh plus views on dozen plus blogs, 227 countries, 7 continents, He makes himself available to all, contact him on +91 9323115463, email, Twitter, @amcrasto , He lives and will die for his family, 90% paralysis cannot kill his soul., Notably he has 38 lakh plus views on New Drug Approvals Blog in 227 countries...... , He appreciates the help he gets from one and all, Friends, Family, Glenmark, Readers, Wellwishers, Doctors, Drug authorities, His Contacts, Physiotherapist, etc He has total of 32 International and Indian awards

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