NS Pharma
- Company typePrivate
- Founded1999
- HeadquartersParamus, United States
- Headcount51–100
- GTM typeB2B
- OfferingHardware or Manufacturing
What NS Pharma does
NS Pharma, Inc. is a U.S.-based, wholly-owned subsidiary of Nippon Shinyaku Co., Ltd., a Japanese pharmaceutical company headquartered in Kyoto with over a century of history. The company is headquartered at 140 East Ridgewood Avenue in Paramus, New Jersey, with a secondary Innovation Research Partnering office in Cambridge, Massachusetts, and operates with 51-100 employees. NS Pharma focuses exclusively on rare diseases with high unmet medical need, spanning Duchenne muscular dystrophy (DMD), Hunter syndrome (MPS II), and eosinophilic granulomatosis with polyangiitis (EGPA).
The company's portfolio rests on three proprietary technology platforms: exon-skipping antisense oligonucleotides (producing dystrophin proteins 84-97% as long as the full-length protein for common DMD mutations), gene therapy (via NAVSUNLI/RGX-121 for MPS II and ATSN-101 for LCA1), and selective JAK1 inhibition (NS-229 for EGPA). Its single commercial product is VILTEPSO (viltolarsen), an exon 53-skipping therapy that received FDA accelerated approval in August 2020 for DMD. The pipeline includes deramiocel (CAP-1002) for DMD cardiomyopathy with a PDUFA target date of August 22, 2026; NAVSUNLI (clemidsogene lanparvovec) for MPS II under BLA review; brogidirsen (NS-089/NCNP-02) for exon 44 skipping with 4.5-year clinical data; NS-229 in Phase 2 for EGPA; and earlier-stage programs including NS-050/NCNP-03, NS-051/NCNP-04, RGX-111, EXG-7001, and RNAa programs with MiNA Therapeutics.
NS Pharma generates revenue primarily through direct U.S. commercialization of VILTEPSO via its own specialty field sales force targeting neurologists and rare disease treatment centers, supplemented by licensing and royalty arrangements on partnered products. The company holds exclusive U.S. commercialization rights for deramiocel (under a 2022 agreement with Capricor Therapeutics), NAVSUNLI (under a January 2025 agreement with REGENXBIO), and ATSN-101 (with Atsena Therapeutics). It also maintains patient support services (NS Support), market access infrastructure, and a network of patient advocacy partnerships (CureDuchenne, PPMD, MDA, Jett Foundation, etc.). Revenue figures are not publicly disclosed.
NS Pharma firmographics
Firmographics- Name
- NS Pharma
- Legal name
- NS Pharma, Inc.
- Website
- https://nspharma.com
- Company type
- Private
- Founded year
- 1999
- Operating status
- Operating
- Headcount range
- 51–100 employees
- Ownership category
- akta.pro rank
NS Pharma industry classification
Industry- Product category
- Rare Disease Biopharmaceuticals
- NAICS
- Pharmaceutical Preparation Manufacturing (325412), Biological Product (except Diagnostic) Manufacturing (325414), Research and Development in Biotechnology (except Nanobiotechnology) (541714)
- SIC
- Pharmaceutical Preparations (2834), Biological Products, (No Disgnostic Substances) (2836)
- akta.pro primary industry
- Neurology & CNS Specialty Pharmaceuticals (HLAIACAC)
- akta.pro secondary industries
- Neurology & Psychiatry (CNS) Pharmaceuticals (HLAIAAAF), Immunology & Inflammation Pharmaceuticals (HLAIAAAD), Specialty Injectable & Infusion Therapies (Cross-therapeutic) (HLAIACAO), Cardiometabolic Pharmaceuticals (Cardiovascular/Diabetes/Obesity) (HLAIAAAE)
Keywords
Where NS Pharma is headquartered
LocationHeadquarters
- HQ city
- Paramus
- HQ country
- United States
- HQ region
- North America
Offices2 records
Markets served
NS Pharma business model
Business model- GTM type
- B2B
- Offering type
- Hardware or Manufacturing
- Cost components
- Technology or R&D, Personnel, Operations, Marketing or Sales, Supply Chain
Revenue model
- Pharmaceutical Product Sales: NS Pharma generates revenue from the sale of Viltepso (viltolarsen), an FDA-approved exon-skipping therapy for DMD. The company commercializes the product directly in the U.S. market through its commercial infrastructure.
- Partnership Revenue (REGENXBIO/NAVSUNLI): Under a January 2025 partnership, NS Pharma holds U.S. commercialization rights for NAVSUNLI (clemidsogene lanparvovec), a gene therapy for MPS II. NS Pharma will commercialize the therapy upon FDA approval.
- Partnership Revenue (Capricor/deramiocel): NS Pharma holds exclusive U.S. commercialization rights for deramiocel (CAP-1002), a cell therapy for DMD cardiomyopathy, under an agreement with Capricor Therapeutics.
Go-to-market motion1 record
Distribution channels1 record
Marketing channels6 records
NS Pharma product offering
Product offeringCore offering
NS Pharma is a commercial-stage biopharmaceutical company that develops, manufactures, and commercializes therapies for rare diseases, with a primary focus on Duchenne muscular dystrophy (DMD), Hunter syndrome (MPS II), and eosinophilic granulomatosis with polyangiitis (EGPA). Its marketed product is VILTEPSO (viltolarsen), an FDA-approved exon 53 skipping antisense oligonucleotide for DMD, and its pipeline includes additional exon-skipping therapies, gene therapies, a cell therapy, and a selective JAK1 inhibitor.
Product overview
NS Pharma is a biopharmaceutical company focused on rare diseases, developing a portfolio of therapies across multiple technology platforms. The company operates as a subsidiary of Nippon Shinyaku Co., Ltd. The product portfolio includes: VILTEPSO (viltolarsen), an FDA-approved exon 53 skipping therapy for DMD; Deramiocel (CAP-1002), a cell therapy for DMD cardiomyopathy under FDA review; RGX-121, a gene therapy for MPS II under FDA review; and multiple pipeline candidates including brogidirsen (exon 44 skipping), NS-229 (JAK1 inhibitor for EGPA), NS-050/NCNP-03 (exon 50 skipping), ATSN-101 (gene therapy for LCA1), RGX-111 (gene therapy for MPS I), NS-051/NCNP-04 (exon 51 skipping), and EXG-7001 (mRNA therapy for DMD). Scientific approaches include exon-skipping technology, gene therapy, cell therapy, and selective JAK1 inhibition.
Differentiator
Problem solved
Functional benefit
Brands
- VILTEPSO (viltolarsen): FDA-approved exon-skipping therapy for Duchenne muscular dystrophy patients with confirmed mutation amenable to exon 53 skipping.
- We Speak Duchenne
- NS Support
- Viltepso
Products and services
- VILTEPSO (viltolarsen) VILTEPSO is an FDA-approved exon-skipping antisense oligonucleotide therapy for Duchenne muscular dystrophy (DMD) patients with confirmed mutations amenable to exon 53 skipping, designed to enable production of a shortened but functional dystrophin protein.
- Deramiocel (CAP-1002) Deramiocel is an investigational allogeneic cardiosphere-derived cell (CDC) therapy for Duchenne muscular dystrophy cardiomyopathy, being developed with Capricor Therapeutics and under FDA BLA review with a PDUFA target date of August 22, 2026.
- NAVSUNLI / RGX-121 (clemidisogene lanparvovec)
Quantifiable outcome
- Exon-skipping therapy produces dystrophin 84-97% as long as full-length protein for common mutations
- +4 more outcomes
Companies that use NS Pharma
Customer profileNamed customers2 records
Segments3 records
Ideal customer profiles2 records
NS Pharma technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature3 records
NS Pharma partnerships and signals
Strategic signalPartnerships
Ten partnerships are on record, tiered minor and core.
- Elixirgen TherapeuticsminorNippon Shinyaku (NS Pharma parent) entered an option agreement with Elixirgen Therapeutics to obtain exclusive worldwide rights to develop and commercialize EXG-7001, an investigational mRNA-based therapeutic for DMD. EXG-7001 is in preclinical development and expresses full-length human dystrophin protein. NS Pharma intends to commercialize EXG-7001 in the U.S. following option exercise and regulatory approval.
- Boston Children's HospitalcoreNippon Shinyaku entered a Sponsored Research Agreement with Boston Children's Hospital to develop innovative therapeutic drugs in neurology. The collaboration builds on a strategic alliance initiated in July 2025 and aims to combine Boston Children's Hospital's research capabilities with Nippon Shinyaku's drug discovery expertise. Facilitated by NS Pharma's Innovation Research Partnering team in Cambridge, MA.
- REGENXBIO Inc.coreUnder a January 2025 partnership, NS Pharma holds U.S. commercialization rights for NAVSUNLI (clemidsogene lanparvovec-sngl), an investigational gene therapy for Mucopolysaccharidosis II (MPS II/Hunter syndrome). REGENXBIO is responsible for development and BLA submission; NS Pharma commercializes in the U.S. upon approval. Target approximately 2,000 MPS II patients worldwide.
- MiNA TherapeuticscoreNippon Shinyaku entered a joint research agreement with MiNA Therapeutics to develop small activating RNAs (saRNAs) using RNA activation (RNAa) technology to treat rare and intractable diseases of the central nervous system. MiNA is the world leader in RNAa drugs. Initial target is a severe neurodevelopmental disorder caused by a single defective gene affecting hundreds of thousands worldwide with no approved treatments.
- Capricor TherapeuticscoreNippon Shinyaku and Capricor entered an exclusive distribution agreement for deramiocel (CAP-1002) for the U.S. in January 2022. NS Pharma is exclusively responsible for commercialization and distribution of deramiocel in the U.S. Deramiocel is a cell therapy for DMD cardiomyopathy with PDUFA target date of August 22, 2026. Note: A breach-of-contract lawsuit was filed by Capricor against NS Pharma and Nippon Shinyaku in 2026.
- National Center of Neurology and Psychiatry (NCNP)coreNCNP co-discovered brogidirsen (NS-089/NCNP-02) with Nippon Shinyaku. NCNP conducted investigator-initiated Phase 1/2 clinical trial; Nippon Shinyaku conducted extension study. Ongoing collaboration on DMD exon 44 skipping therapy.
- Atsena TherapeuticsminorNippon Shinyaku partners with Atsena Therapeutics for gene therapy development targeting GUCY2D-associated Leber congenital amaurosis (LCA1). Atsena is responsible for progress and development of this program.
- CureDuchenneminorPatient advocacy organization partnership for Duchenne muscular dystrophy awareness and support.
- Parent Project Muscular Dystrophy (PPMD)minorPatient advocacy organization partnership for Duchenne muscular dystrophy. PPMD partnered with CINRG to support expanded Duchenne Natural History Study.
- Muscular Dystrophy Association (MDA)minorPatient advocacy organization partnership. NS Pharma presents clinical data at MDA Clinical & Scientific Conference.
Scale indicators3 records
Recent moves6 records
Expansion highlights6 records
NS Pharma competitors and assessment
Company assessmentMarket position
Competitive moat5 records
Key highlights6 records
Customer concentration
NS Pharma social profiles
Digital presenceNS Pharma compliance and trust
Trust signalCompliance3 records
NS Pharma financial estimates
Financial estimateRevenue estimate
Valuation estimate
NS Pharma leadership team
Management profileNumber of profiles
Profiles13 records
NS Pharma funding detail
Funding detailFunding overview
Funding rounds
Investors
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
NS Pharma M&A and investment
M&A and investmentM&A
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about NS Pharma
What does NS Pharma do?
NS Pharma is a commercial-stage biopharmaceutical company that develops, manufactures, and commercializes therapies for rare diseases, with a primary focus on Duchenne muscular dystrophy (DMD), Hunter syndrome (MPS II), and eosinophilic granulomatosis with polyangiitis (EGPA). Its marketed product is VILTEPSO (viltolarsen), an FDA-approved exon 53 skipping antisense oligonucleotide for DMD, and its pipeline includes additional exon-skipping therapies, gene therapies, a cell therapy, and a selective JAK1 inhibitor.
Is NS Pharma a public or private company?
NS Pharma is a private company. It is classified as corporate owned and is currently operating.
When was NS Pharma founded?
NS Pharma was founded in 1999. It employs 51 to 100 people.
Where is NS Pharma based?
NS Pharma is headquartered in Paramus, United States, in the North America region.
How does NS Pharma make money?
Three revenue lines are on record. Pharmaceutical Product Sales are the primary driver. The others are partnership Revenue (REGENXBIO/NAVSUNLI) and partnership Revenue (Capricor/deramiocel).
Does NS Pharma have an API?
No public API is recorded for NS Pharma.
What industry is NS Pharma in?
NS Pharma's product category is Rare Disease Biopharmaceuticals. Its primary akta.pro industry code is HLAIACAC, Neurology & CNS Specialty Pharmaceuticals, with a secondary code of HLAIAAAF, Neurology & Psychiatry (CNS) Pharmaceuticals. Its NAICS code is 325412 and its SIC code is 2834.