United States Patent 10,076,514: Fabry Disease Treatment Claims, Scope, and Patent Landscape

U.S. Patent No. 10,076,514 protects a genotype-directed method of treating Fabry disease with 1-deoxygalactonojirimycin, also known as migalastat, or a salt. The independent claims are limited to patients carrying the G9331A mutation in the alpha-galactosidase gene relative to SEQ ID NO: 1. The patent does not broadly cover all Fabry patients, all alpha-galactosidase mutations, or all migalastat dosing regimens.

The commercially important embodiment is oral administration of approximately 150 mg of migalastat hydrochloride every other day. That dose corresponds to the FDA-approved Galafold regimen of 123 mg migalastat, equivalent to 150 mg of migalastat hydrochloride. The patent therefore overlaps directly with the approved product for patients whose mutations fall within the claimed G9331A genotype.

What does U.S. Patent 10,076,514 cover?

The patent covers treating a Fabry patient with migalastat or a salt when the patient has the specified G9331A mutation in the nucleic acid sequence encoding alpha-galactosidase.

Claim	Protected subject matter	Scope
1	Treatment of Fabry disease with 1-deoxygalactonojirimycin or salt in a patient with G9331A	Broadest independent claim
2	Dose of approximately 25 mg to 250 mg	Dependent dose range
3	Use of migalastat hydrochloride	Salt limitation
4	Approximately 150 mg every other day of migalastat hydrochloride	Commercial dosing embodiment
5	Oral or injectable administration	Route limitation
6	Oral treatment of a human patient with G9331A using approximately 150 mg every other day	Narrow independent claim
7	Approximately 150 mg migalastat hydrochloride orally every other day	Narrowest commercial claim

Claim 1 is the principal infringement claim. Claims 2 through 5 add dose, salt, and administration-route limitations. Claim 6 independently recites the human, oral, and dosing limitations. Claim 7 is narrower than claim 6 because it specifies approximately 150 mg of migalastat hydrochloride rather than an equivalent dose of another salt or free-base formulation.

What is the key limitation in the patent claims?

The key limitation is the patient's G9331A mutation.

A product or treatment can fall outside the claims if:

The patient does not have the G9331A mutation.
The mutation is identified using a different sequence reference and cannot be established as the claimed G9331A mutation relative to SEQ ID NO: 1.
The treatment does not use migalastat or a salt.
The treatment is not directed to Fabry disease.
The accused regimen does not satisfy a limitation of the asserted dependent claim.

The claims do not cover all mutations known to be amenable to migalastat. They cover one specifically identified mutation. A competitor treating patients with another amenable mutation would require a separate infringement analysis against other patents.

The mutation limitation is also relevant to induced-infringement risk. A manufacturer generally does not administer the drug directly to patients. Its exposure would depend on activities such as labeling, patient-identification programs, dosing instructions, reimbursement support, or other conduct that encourages the claimed treatment.

How does the patent relate to Galafold and migalastat?

Galafold is the U.S. trade name for migalastat hydrochloride. The FDA approved Galafold in August 2018 for adults with Fabry disease who have an amenable mutation in the alpha-galactosidase A gene. The approved dosage is one 123 mg migalastat capsule orally every other day, corresponding to 150 mg of migalastat hydrochloride on a salt-weight basis.[1]

The patent claims use the chemical name 1-deoxygalactonojirimycin. That compound is migalastat. The claims also cover salts, with specific recitation of the hydrochloride salt.

Patent terminology	Commercial terminology
1-deoxygalactonojirimycin	Migalastat
1-deoxygalactonojirimycin hydrochloride	Migalastat hydrochloride
Approximately 150 mg every other day	Galafold labeled dose on a salt-weight basis
G9331A mutation	A specific alpha-galactosidase genotype

The overlap is strongest for an oral generic or other competing product labeled for patients with the claimed mutation and dosed every other day.

What therapeutic mechanism does the patent protect?

Migalastat is a pharmacological chaperone. It binds certain mutant forms of alpha-galactosidase A and can improve trafficking of the enzyme to the lysosome. The mechanism differs from enzyme replacement therapy, which supplies recombinant alpha-galactosidase directly.

The patent is therefore a method-of-treatment patent based on a genotype-response relationship. Its commercial value depends on whether the G9331A variant is classified as amenable to migalastat under the FDA-approved test and labeling framework.

The claims do not require a particular clinical endpoint, biomarker threshold, or reduction in globotriaosylceramide. They require treatment of a diagnosed Fabry patient with the specified mutation using the claimed compound and, for narrower claims, the claimed dose and route.

When does U.S. Patent 10,076,514 expire?

The patent issued on September 18, 2018. Its patent family has a 2014 priority date, and the nominal 20-year patent-term endpoint is in 2034, subject to the actual U.S. nonprovisional filing date, any patent-term adjustment, terminal disclaimer, and other USPTO term calculations.[2]

Event	Date or period
Earliest reported priority period	2014
U.S. patent application prosecution	Before 2018 issuance
Patent issued	September 18, 2018
Nominal patent-term endpoint	2034, subject to USPTO adjustment
Patent term extension	No extension is established by the patent claims alone

The relevant date for generic-entry planning is the official expiration date shown in USPTO patent records and, if listed for Galafold, the FDA Orange Book. A patent's 2018 issue date is not its expiration date.

Is U.S. Patent 10,076,514 an Orange Book patent?

The patent is a method-of-use patent potentially relevant to Galafold because the claims correspond to an FDA-labeled genotype-directed use and the approved every-other-day regimen. Orange Book relevance depends on the patent's listing, use code, and the scope of the approved labeling at the time of listing.[3]

An Orange Book-listed method patent can create a Paragraph IV certification issue for an abbreviated new drug application if the generic applicant seeks approval for a use covered by the listed patent. A generic applicant may instead use a section viii statement and omit the patented use, but that strategy is viable only if the proposed labeling excludes the protected indication or patient population.

The commercial risk is highest where a proposed generic label:

Identifies G9331A as an approved or recommended migalastat-responsive mutation.
Uses the same oral every-other-day dosing.
Promotes treatment of Fabry patients with the claimed genotype.
Includes patient-support material that encourages the patented method.

What Paragraph IV challenges could affect Galafold?

A Paragraph IV certification would assert that a listed patent is invalid, unenforceable, or not infringed. For this patent, a generic challenger would likely focus on claim construction, written description, enablement, obviousness, and the boundaries of the G9331A limitation.

Potential challenge theories include:

Mutation identification. The challenger could dispute whether the proposed patient population has the claimed mutation relative to the patent's sequence reference.
Obviousness. The challenger could argue that identifying migalastat-responsive Fabry mutations and applying the established every-other-day regimen would have been obvious.
Written description and enablement. The challenger could examine whether the patent adequately supports treatment of patients carrying the specific mutation and whether the disclosure enables the full claimed dose range and salt scope.
Induced infringement. A generic could attempt to avoid liability through a narrow label that omits G9331A or does not encourage the claimed use.
Claim differentiation. Claims 6 and 7 provide narrower oral-dosing positions even if broader claim 1 is challenged.

A Paragraph IV notice would ordinarily trigger the Hatch-Waxman litigation framework if the patent were listed and the NDA holder or patent owner filed suit within the statutory period. The available claim set gives the patent owner fallback positions: a broad mutation-specific method claim, a dose-range claim, a salt claim, and a precise oral dosing claim.

What patent litigation affects U.S. Patent 10,076,514?

The principal litigation risk is an ANDA dispute involving a generic version of Galafold. The patent's claim structure is suited to a product-specific dispute because the commercial regimen is expressly recited in claims 4, 6, and 7.

A litigation assessment should distinguish among:

Issue	Relevance
Direct infringement	Usually limited because physicians or patients perform the treatment
Induced infringement	Important if the generic label promotes the claimed genotype and regimen
Literal infringement	Depends on mutation, salt, dose, route, and patient population
Doctrine of equivalents	Potentially relevant to non-hydrochloride salts or equivalent dosing
Invalidity	Likely centered on obviousness, written description, enablement, and definiteness
Regulatory stay	Relevant only if an ANDA filer makes a Paragraph IV certification and suit is timely filed

No settlement terms should be inferred merely from the existence of this patent. Any launch date, license, or non-entry agreement would depend on the actual litigation docket and settlement filings.

How strong is the patent estate for migalastat?

The patent estate is stronger commercially than claim 1 alone suggests because the approved product, dose, route, and mutation-directed label align with multiple claim limitations. Its weaknesses are the narrow genotype limitation and the fact that the core active ingredient and general mechanism may be addressed by other patents rather than this patent.

Strength factor	Assessment
Product overlap	High for Galafold's claimed genotype and regimen
Genotype breadth	Narrow; limited to G9331A
Dose coverage	Strong for approximately 150 mg hydrochloride every other day
Route coverage	Strong for oral use under claims 6 and 7
Salt coverage	Broad under claims 1 and 3, with equivalents addressed in claims 4 and 6
Clinical-use alignment	High because the claims track the approved dosing concept
Design-around potential	Meaningful through non-G9331A populations, label carve-outs, or different treatment instructions
Biosimilar relevance	None in the technical sense; migalastat is a small molecule
Generic litigation risk	Material if the patent is listed and the generic label includes the claimed use

The patent should be analyzed together with composition, formulation, polymorph, manufacturing, and other method-of-use patents in the Galafold family. A generic may avoid one patent while remaining blocked by another.

What other patents and technologies compete with migalastat?

Fabry disease treatment has three main technology categories:

Category	Representative therapy	IP profile
Pharmacological chaperone	Migalastat/Galafold	Mutation-specific methods, compound, formulation, and manufacturing claims
Enzyme replacement therapy	Agalsidase beta/Fabrazyme; agalsidase alfa/Replagal in certain markets	Biologic manufacturing, formulation, dosing, and regulatory exclusivity
Next-generation approaches	Gene therapy and substrate-reduction programs	Vector, transgene, delivery, manufacturing, and clinical-use patents

Migalastat does not face biosimilar substitution in the same way as Fabrazyme because it is a small molecule. A future competitor would generally pursue an ANDA or another small-molecule pathway, not a biosimilar application. Gene therapies create a different competitive threat and are more likely to rely on biologic patent estates and regulatory exclusivity.

What generic launch scenarios exist?

Scenario 1: Full-label generic launch

A generic applicant includes G9331A and the approved every-other-day regimen. This creates the clearest infringement and Paragraph IV exposure under claims 1, 4, 6, and 7.

Scenario 2: Section viii carve-out

The applicant omits the patented genotype or method from its labeling. This may reduce infringement risk, but the carve-out must be operationally credible. Promotional materials, patient-support programs, or prescribing information cannot restore the omitted use indirectly.

Scenario 3: Post-expiration launch

The applicant waits until expiration of the relevant Orange Book patents. This eliminates patent litigation risk for the expired claims but does not eliminate regulatory, manufacturing, or other patent barriers.

Scenario 4: Settlement or license

The generic may agree to a later entry date or obtain a license. The commercial value of such an agreement depends on the remaining patent term, the scope of licensed uses, and whether the agreement includes supply or authorized-generic provisions.

What manufacturing and geographic IP barriers remain?

The asserted claims are treatment claims, not manufacturing claims. A competitor that makes migalastat outside the United States may avoid U.S. method-of-treatment infringement for the manufacturing activity, but U.S. importation, sale, labeling, and use can implicate separate patent rights.

Geographic considerations include:

U.S. treatment claims apply to treatment occurring in the United States.
Foreign family members must be assessed independently.
Patent term, listing practice, and regulatory exclusivity differ by jurisdiction.
European, Japanese, and other national claims may use different mutation nomenclature.
A salt or formulation that avoids one U.S. claim may remain covered elsewhere.

A freedom-to-operate review should separate U.S. method claims from compound, formulation, process, and foreign national-phase rights.

What is the commercial exposure from this patent?

The patent's exposure is concentrated in the U.S. Galafold market and in the subset of Fabry patients carrying G9331A. Because the claims track a specific approved regimen, the patent can support meaningful protection even though it does not cover the entire Fabry population.

Revenue exposure depends on:

U.S. Galafold sales before relevant patent expiry.
The prevalence of G9331A among U.S. Fabry patients.
Whether the FDA label identifies or enables treatment of the claimed genotype.
The number and timing of ANDA filings.
The availability of label carve-outs.
Patent litigation outcomes and settlement terms.
Substitution from enzyme replacement therapy or emerging gene therapies.

The patent is therefore best characterized as a targeted lifecycle and indication patent, not a standalone composition patent.

Key Takeaways

U.S. Patent 10,076,514 claims treatment of Fabry disease with migalastat in patients carrying the G9331A alpha-galactosidase mutation.
Claims 4, 6, and 7 closely track the commercial Galafold regimen of approximately 150 mg migalastat hydrochloride every other day.
The patent does not cover all Fabry patients or all migalastat-amenable mutations.
The core legal risks are mutation identification, induced infringement, obviousness, written description, enablement, and claim construction.
Migalastat is a small molecule, so generic-drug risk is more relevant than biosimilar risk.
The nominal patent-term endpoint is in 2034, subject to the official USPTO term calculation and any applicable disclaimers or adjustments.
A generic label omitting G9331A or the claimed dosing instructions could reduce infringement exposure, but associated promotional activity would remain relevant.
The patent should be evaluated with the complete Galafold patent family, Orange Book listings, use codes, and any later-filed formulation, manufacturing, or method-of-use patents.

FAQs

Does Patent 10,076,514 cover all patients with Fabry disease?

No. The claims require the specific G9331A mutation relative to SEQ ID NO: 1.

Does the patent cover migalastat free base?

Claim 1 covers 1-deoxygalactonojirimycin or a salt. Claims 3, 4, 6, and 7 focus more specifically on migalastat hydrochloride and equivalent dosing.

Can a generic launch for a different Fabry mutation avoid this patent?

Potentially. A treatment directed solely to a different mutation would not satisfy the express G9331A limitation, although other patents could still apply.

Is Galafold subject to biosimilar competition?

No. Galafold contains migalastat, a small molecule. The relevant pathway for a competing chemically equivalent product is generally the ANDA pathway, not biosimilar approval.

Does the 150 mg dose mean 150 mg of active migalastat?

The patent claims 150 mg of migalastat hydrochloride. The FDA Galafold label expresses the dose as 123 mg of migalastat, equivalent to 150 mg of the hydrochloride salt.[1]

References

U.S. Food and Drug Administration. (2018). Galafold (migalastat) prescribing information. FDA.
United States Patent and Trademark Office. (2018). U.S. Patent No. 10,076,514, methods of treating Fabry disease. USPTO.
U.S. Food and Drug Administration. (2024). Approved drug products with therapeutic equivalence evaluations: Orange Book. FDA.

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Patented / Exclusive Use	Submissiondate
Amicus Therap Us	GALAFOLD	migalastat hydrochloride	CAPSULE;ORAL	208623-001	Aug 10, 2018		RX	Yes	Yes	⤷ Start Trial	⤷ Start Trial				THE TREATMENT OF FABRY PATIENTS	⤷ Start Trial
>Applicant	>Tradename	>Generic Name	>Dosage	>NDA	>Approval Date	>TE	>Type	>RLD	>RS	>Patent No.	>Patent Expiration	>Product	>Substance	>Delist Req.	>Patented / Exclusive Use	>Submissiondate

Country	Patent Number	Estimated Expiration	Supplementary Protection Certificate	SPC Country	SPC Expiration
China	109069494	⤷ Start Trial
China	114159436	⤷ Start Trial
European Patent Office	3432882	⤷ Start Trial
Japan	2019510030	⤷ Start Trial
Japan	2022130589	⤷ Start Trial
Japan	2024122992	⤷ Start Trial
>Country	>Patent Number	>Estimated Expiration	>Supplementary Protection Certificate	>SPC Country	>SPC Expiration

Details for Patent: 10,076,514

Which drugs does patent 10,076,514 protect, and when does it expire?

Summary for Patent: 10,076,514