Details for Patent: 9,506,058

Which drugs does patent 9,506,058 protect, and when does it expire?

Patent 9,506,058 protects EXONDYS 51 and is included in one NDA.

This patent has twenty-four patent family members in thirteen countries.

Summary for Patent: 9,506,058

Title:	Compositions for treating muscular dystrophy
Abstract:	Improved compositions and methods for treating muscular dystrophy by administering antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping are described.
Inventor(s):	Edward M. Kaye
Assignee:	Biopharma Credit PLC
Application Number:	US14/214,567
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for patent 9,506,058
Patent Claim Types: see list of patent claims	Use;

US Patent 9,506,058: Eteplirsen Patent Scope, Claim Analysis, Exclusivity and Competitive Landscape

US Patent 9,506,058 protects specific long-term treatment methods for exon-51-skipping Duchenne muscular dystrophy using eteplirsen. Its core limitations are demanding: a genetically eligible DMD patient, intravenous administration, approximately 30 mg/kg once weekly, and treatment for more than 120 weeks. The patent is primarily a method-of-use asset, not a broad composition patent. Its practical value depends on whether competing products or clinical protocols meet the dosing, duration, biomarker, and outcome limitations in the claims.

The patent covers treatment, reduction of ambulation loss, restoration of the dystrophin reading frame, and preservation of respiratory function. The narrowest claims are tied to specific 120-week clinical outcomes, including 6-Minute Walk Test results and Maximum Inspiratory Pressure or Maximum Expiratory Pressure measurements.

What patent is US 9,506,058 and who owns it?

US Patent 9,506,058, titled “Methods for Treating Duchenne Muscular Dystrophy,” was issued on November 29, 2016. The patent is associated with Sarepta Therapeutics and its predecessor development organizations. It covers use of eteplirsen, marketed as Exondys 51, in DMD patients whose mutations are amenable to exon 51 skipping.

Item	Detail
Patent	US 9,506,058 B2
Title	Methods for Treating Duchenne Muscular Dystrophy
Patent type	Human-treatment method patent
Active pharmaceutical ingredient	Eteplirsen
Product	Exondys 51
Therapeutic area	Duchenne muscular dystrophy
Molecular mechanism	Antisense-mediated exon 51 skipping
Administration	Intravenous
Core dose	About 30 mg/kg weekly
Core duration	More than 120 weeks
Eligible population	DMD patients with mutations amenable to exon 51 skipping
Key clinical endpoints	Disease progression, ambulation, 6MWT, MIP, MEP, FVC
Patent holder associated with product	Sarepta Therapeutics
FDA approval	Accelerated approval in 2016

The patent does not claim every use of eteplirsen. It claims specified clinical methods using a particular dose, route, duration, population, and treatment objective.

What claims does US 9,506,058 cover?

The patent has 29 claims. Claims 1, 4, 7, 12, 13, 17, 28, and 29 are the principal independent claims.

Claim group	Protected subject matter	Principal limitations
1-3	Treatment of DMD	Eteplirsen, IV, about 30 mg/kg weekly, more than 120 weeks
4-6	Reduction in loss of ambulation	Same dosing and duration, relative to baseline
7-11	Reading-frame restoration and dystrophin production	Same dosing and duration; testing by RT-PCR, Western blot, or immunohistochemistry
12	Treatment measured by 6MWT	Disease progression delayed as measured by 6MWT
13-16	Ambulation preservation measured by 6MWT	Specific numerical and duration thresholds
17-23	Pulmonary-function preservation	MIP, MEP, or FVC; includes age limitation
24-27	Corticosteroid use and genetic confirmation	Listed steroids, timing, and confirmation of exon-51 eligibility
28	Treatment with MIP outcome	At least 14.6% improvement at 120 weeks
29	Treatment with MEP outcome	At least 15% improvement at 120 weeks

How broad is independent claim 1?

Claim 1 requires all of the following:

1. A patient with DMD.
2. A DMD mutation amenable to exon 51 skipping.
3. Intravenous eteplirsen administration.
4. A dose of about 30 mg/kg.
5. Weekly administration.
6. Treatment for more than 120 weeks.
7. Treatment that delays disease progression.

The claim is narrower than a general claim to exon 51 skipping or a general claim to eteplirsen. A competing protocol that uses 20 mg/kg, a different dosing interval, a different route, or treatment for 120 weeks or less may avoid literal infringement of claim 1. The phrase “about 30 mg/kg” creates a range that would be interpreted in view of the specification, prosecution history, and technical evidence rather than as an unlimited range.

The “more than 120 weeks” limitation is particularly important. A patient receiving weekly treatment for 120 weeks exactly would not literally satisfy that duration limitation. A treatment course extending beyond 120 weeks would present a stronger infringement case if the remaining limitations are met.

What does claim 4 protect?

Claim 4 covers the same core regimen where the objective is reducing loss of ambulation relative to baseline. Claims 5 and 6 narrow the claim to pediatric patients and patients who received oral corticosteroids for at least 24 weeks before the first eteplirsen dose.

This claim group is directed to a functional clinical benefit rather than merely administration. The baseline comparison creates evidentiary questions involving the definition of baseline, the timing of the measurement, patient selection, and the handling of patients who discontinue treatment.

What does claim 7 protect?

Claim 7 covers restoring the DMD mRNA reading frame to induce dystrophin production. Claims 10 and 11 provide measurement-specific limitations:

• RT-PCR;
• Western blot;
• Immunohistochemical detection; and
• North Star Ambulatory Assessment for ambulation.

The claim does not require a particular minimum dystrophin percentage in the text provided. It instead requires the claimed biological result following the specified eteplirsen regimen.

What do claims 12 through 16 protect?

These claims focus on the 6-Minute Walk Test, a common functional endpoint in DMD clinical development.

Claim 14 requires less than 5% loss of ambulation relative to baseline by 120 weeks. Claim 15 requires no greater than 13.9 meters of lost walking distance. Claim 16 requires maintenance of ambulation for more than 1.5 years following treatment.

These claims are narrower and more fact-intensive than claims 1 and 4. In an enforcement dispute, the parties would likely contest:

• the baseline date;
• whether the test was performed under a consistent protocol;
• whether the patient was ambulatory at baseline;
• whether the 13.9-meter threshold is measured per patient or as a cohort result;
• treatment interruptions; and
• the meaning of “by 120 weeks.”

What do claims 17 through 23 protect?

Claim 17 protects reducing loss of pulmonary function after weekly 30 mg/kg intravenous eteplirsen for more than 120 weeks. Claims 18 through 23 specify respiratory endpoints:

• Maximum Inspiratory Pressure;
• Maximum Expiratory Pressure;
• Forced Vital Capacity;
• at least 14.6% MIP improvement;
• at least 15% MEP improvement; and
• a patient age of seven years or older.

Claims 28 and 29 combine the general treatment method with the MIP and MEP outcome thresholds. They are outcome-dependent claims. A claimant would need reliable patient-specific or study-specific evidence showing that the required improvement occurred.

How does the patent limit infringement exposure?

The patent’s infringement risk is concentrated in protocols that reproduce the Exondys 51 regimen.

Limitation	Commercial significance
Eteplirsen	Directly targets the marketed product
IV administration	Excludes non-IV delivery unless an equivalent theory applies
About 30 mg/kg	Creates dose-range litigation risk
Weekly dosing	Distinguishes less frequent regimens
More than 120 weeks	Makes long-term continuation material
Exon-51-amenable mutation	Limits the patient population
DMD diagnosis	Excludes other dystrophinopathies
Clinical result	May require outcome evidence, not merely prescribing intent

A generic or follow-on product using the same active molecule and the FDA-approved regimen would face greater exposure than a product using a different antisense sequence, a different dose, or a different dosing schedule. A product that is chemically distinct from eteplirsen would not necessarily infringe these claims, even if it skips exon 51 and produces dystrophin.

What is the FDA and Orange Book status of Exondys 51?

FDA approved Exondys 51 in September 2016 under the accelerated-approval pathway. The approval is limited to DMD patients with a confirmed mutation amenable to exon 51 skipping. The approval was based primarily on dystrophin production data, with clinical benefit subject to verification in postmarketing studies. The FDA label specifies weekly intravenous administration at 30 mg/kg.[1]

The label also states that exon 51 skipping is applicable only to a subset of DMD mutations. Patient eligibility requires genetic confirmation, which is reflected in claim 27.

Exondys 51 is listed in the FDA Orange Book. The relevant patent listing and use-code position should be assessed separately from the patent’s validity and enforceability. An Orange Book listing provides notice to an abbreviated new drug application applicant and can trigger a Paragraph IV certification process. It does not establish that every listed claim is valid or infringed.[2]

Does US 9,506,058 create a generic blocking right?

It can create a regulatory and litigation barrier if:

• the patent is listed for the relevant product;
• the proposed generic seeks approval before patent expiry;
• the applicant files a Paragraph IV certification; and
• the listed claims cover the proposed labeling or use.

The patent is a method patent. A generic applicant may attempt a section viii statement, seeking approval only for non-infringing uses, if the FDA-approved label can be carved back sufficiently. That strategy is difficult where the patented method corresponds closely to the principal approved use of eteplirsen.

When does US 9,506,058 lose exclusivity?

Patent expiration depends on the patent’s effective term, including the earliest claimed nonprovisional filing date, patent-term adjustment, terminal disclaimers, and any applicable patent-term extension. The issue date alone does not determine expiration.

US 9,506,058 should be evaluated together with the FDA’s Orange Book patent-term data and the USPTO Patent Center record. The statutory framework generally provides a 20-year term measured from the earliest effective nonprovisional filing date, subject to adjustments under 35 U.S.C. §§ 154 and 156.[3][4]

FDA regulatory exclusivity is separate from patent exclusivity. Exondys 51 received orphan-drug exclusivity, which generally provides seven years of protection against approval of the same drug for the same orphan indication under the Orphan Drug Act. That period does not prevent approval of a different drug, and it does not extend the patent term.[5]

Because the patent’s commercial protection is claim- and listing-dependent, the relevant analysis is:

Exclusivity layer	Effect
US 9,506,058	Method-of-use protection for the claimed eteplirsen regimen
Other eteplirsen patents	May protect composition, sequence, formulation, or additional uses
Orphan-drug exclusivity	Regulatory protection for the designated indication
Pediatric exclusivity	Potential six-month extension of certain listed protections if granted
FDA approval	Product-specific regulatory authorization, not patent protection

Which patent types surround eteplirsen?

The broader eteplirsen estate is likely to include several distinct patent categories.

Composition and antisense-sequence patents

These patents protect antisense oligonucleotide sequences, chemical structures, backbone modifications, and exon-skipping activity. They are potentially more important against a chemically identical generic than a clinical-outcome patent.

Method-of-use patents

US 9,506,058 is in this category. It focuses on patient treatment, dosage, treatment duration, dystrophin production, ambulation, and pulmonary outcomes.

Formulation and manufacturing patents

These may cover sterile injectable formulations, concentration, excipients, purification, impurity control, oligonucleotide synthesis, and manufacturing processes. A competitor can avoid a formulation claim by changing excipients or concentration, but process claims can create manufacturing risk even when the final product is similar.

Regulatory and diagnostic barriers

Exon-51 eligibility requires a specific genotype. Patent claims that require confirmation of an amenable mutation may create a narrower risk profile than claims directed to all DMD patients. Diagnostic testing itself may be governed by separate laboratory and regulatory rules rather than by this patent.

How strong is the patent estate for eteplirsen?

US 9,506,058 has meaningful commercial strength but limited breadth.

Strengths

• It maps directly onto the FDA-approved 30 mg/kg weekly regimen.
• It covers long-term treatment beyond 120 weeks.
• It includes multiple clinical endpoints.
• It reaches ambulation and respiratory-function outcomes.
• It covers pediatric patients and corticosteroid-supported treatment.
• It aligns with the product’s approved exon-51-skipping population.

Vulnerabilities

• The claims contain many cumulative limitations.
• “About 30 mg/kg” may generate claim-construction disputes.
• “More than 120 weeks” narrows the claims to extended treatment.
• Several claims depend on measured clinical outcomes.
• Functional results may be difficult to prove patient by patient.
• The patent does not, on the claim language provided, broadly cover every exon-51-skipping molecule.
• A chemically different exon-skipping agent may avoid literal infringement.

Validity challenges could focus on written description, enablement, anticipation, obviousness, indefiniteness, and lack of demonstrated clinical benefit. The functional and long-duration limitations may help distinguish earlier laboratory or short-term clinical disclosures, but they can also create vulnerability if the specification does not adequately support the full breadth of the claimed patient population and outcomes.

Are there Paragraph IV challenges or patent litigation affecting Exondys 51?

A complete litigation conclusion requires current FDA Orange Book certifications, ANDA notices, district-court dockets, and USPTO records. The claims provided do not identify a Paragraph IV filer, settlement agreement, or court judgment.

The principal litigation triggers would be:

1. An ANDA applicant certifies that the listed patent is invalid, unenforceable, or not infringed.
2. Sarepta files suit within 45 days.
3. FDA approval is subject to the statutory stay period.
4. The parties negotiate a settlement governing launch timing or permitted labeling.
5. A court determines whether the proposed regimen falls within the claims.

The absence of a cited litigation record does not eliminate commercial risk. It means that patent status, Orange Book listing, and litigation status must be treated as separate data fields.

What generic launch scenarios exist for eteplirsen?

Scenario	Patent risk	Likely commercial result
Same eteplirsen, same 30 mg/kg weekly regimen	High	Paragraph IV litigation likely
Same molecule, different dose	Reduced but not eliminated	Depends on claim construction and labeling
Same molecule, less than 120 weeks on label	Potentially reduced	May face product and use-code issues
Different exon-51-skipping antisense molecule	Lower under this patent	Separate composition and method patents matter
Non-IV delivery	Lower under literal claims	Clinical and FDA approval barriers remain
Carved-out label	Potentially viable	Depends on whether unpatented use is substantial
Hospital or physician off-label use	Fact-specific	Direct and induced infringement theories may arise

A generic launch before patent expiration would be difficult if the proposed label includes the same patient population and treatment regimen. A non-infringing label strategy would need to avoid the patented method while remaining commercially and clinically viable.

How does US 9,506,058 compare with competing DMD patent estates?

Eteplirsen competes with other molecular approaches rather than only with generic eteplirsen.

Product or platform	Mechanism	Exon target	Relationship to US 9,506,058
Exondys 51	Antisense exon skipping	51	Directly covered by the claims
Vyondys 53	Antisense exon skipping	53	Different exon and product estate
Viltepso	Antisense exon skipping	53	Different active molecule and estate
Amondys 45	Antisense exon skipping	45	Different exon and product estate
Micro-dystrophin gene therapy	Gene replacement	Not exon-specific	Generally outside the literal claim scope
CRISPR-based approaches	Genome editing	Mutation-specific	Generally outside the literal claim scope

The patent is strongest against products that are therapeutically and operationally close to eteplirsen. It is less effective against gene therapy, genome editing, or a chemically distinct exon-skipping product.

What revenue exposure is linked to the patent?

Exondys 51 is one of Sarepta’s commercial DMD products. Patent exposure is therefore linked to product revenue, pricing, reimbursement, and the timing of competing exon-skipping therapies.

The highest-value claims are claims 1 and 4, because they cover the basic long-term treatment and ambulation-preservation concepts. Claims 12 through 23 and 28 through 29 provide additional positions tied to clinical measurements. They may strengthen settlement leverage, but their narrower outcome limitations could reduce their standalone value in litigation.

Revenue risk would increase if a competitor obtains approval for the same exon-51 population and can use a label that supports the 30 mg/kg weekly regimen. Revenue risk would be lower if competition comes from products directed to different exons or from non-eteplirsen technologies.

Key Takeaways

• US 9,506,058 is a method-of-use patent for eteplirsen treatment in exon-51-amenable DMD.
• Its central regimen is intravenous eteplirsen at about 30 mg/kg weekly for more than 120 weeks.
• The claims cover treatment, ambulation preservation, dystrophin production, 6MWT outcomes, and respiratory function.
• Claims 14, 15, 19, 21, 28, and 29 contain specific clinical-performance thresholds.
• The patent does not broadly claim every exon-51-skipping molecule or every DMD treatment.
• A same-molecule generic using the approved regimen would face the highest infringement and Paragraph IV risk.
• Different exon-skipping drugs, gene therapies, and genome-editing products are generally outside the literal scope of the claims.
• Orange Book listing, FDA exclusivity, patent term, and litigation status must be analyzed separately.
• The patent’s commercial strength is high against label-matched eteplirsen competition but narrower against redesigned molecules and alternative treatment platforms.

FAQs About US Patent 9,506,058

Does US 9,506,058 cover all DMD patients?

No. The claims require a mutation amenable to exon 51 skipping. Patients with mutations requiring exon 45, exon 53, or other exon-skipping strategies fall outside the stated population.

Does the patent cover eteplirsen administered at 30 mg/kg every two weeks?

Not literally under the claims provided. The independent claims require weekly administration. A different schedule could still raise separate patent or regulatory issues, but it would not satisfy the weekly limitation as written.

Can a competing exon-skipping drug infringe this patent?

A chemically different exon-skipping drug generally would not satisfy the “administering ... eteplirsen” limitation. Separate patent claims covering the competing molecule, its sequence, or its use would control that analysis.

Why are the 120-week limitations commercially important?

They tie the claims to long-term treatment. A protocol that ends at or before 120 weeks may avoid literal infringement of claims requiring treatment for more than 120 weeks, subject to other claims and infringement theories.

Do the MIP and MEP claims require every patient to achieve the stated improvement?

Claims 28 and 29 expressly require the specified respiratory-function improvement in the treated patient. Proof would depend on the measurement protocol, baseline value, timing, and treatment records.

Sources

1. U.S. Food and Drug Administration. (2016). Exondys 51 (eteplirsen) prescribing information.
2. U.S. Food and Drug Administration. (2024). Approved drug products with therapeutic equivalence evaluations, Orange Book.
3. United States Code. 35 U.S.C. § 154. Patent term.
4. United States Code. 35 U.S.C. § 156. Extension of patent term.
5. United States Code. 21 U.S.C. § 360cc. Orphan-drug exclusivity.
6. United States Patent and Trademark Office. (2016). U.S. Patent No. 9,506,058, Methods for treating Duchenne muscular dystrophy.

Drugs Protected by US Patent 9,506,058

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Patented / Exclusive Use	Submissiondate
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-001	Sep 19, 2016		RX	Yes	Yes	9,506,058					TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-001	Sep 19, 2016		RX	Yes	Yes	9,506,058					RESTORING AN MRNA READING FRAME TO INDUCE DYSTROPHIN PROTEIN PRODUCTION IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-002	Sep 19, 2016		RX	Yes	Yes	9,506,058					TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING
Sarepta Theraps Inc	EXONDYS 51	eteplirsen	SOLUTION;INTRAVENOUS	206488-002	Sep 19, 2016		RX	Yes	Yes	9,506,058					RESTORING AN MRNA READING FRAME TO INDUCE DYSTROPHIN PROTEIN PRODUCTION IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING

International Family Members for US Patent 9,506,058

Country	Patent Number	Estimated Expiration	Supplementary Protection Certificate	SPC Country	SPC Expiration
Australia	2014233456
Australia	2019203505
Australia	2020260492
Brazil	112015022998
Canada	2906812