U.S. Patent 10,792,279: Fabry Disease, Migalastat, Renal Impairment, and Patent Landscape

U.S. Patent No. 10,792,279 protects patient-selection and treatment methods for migalastat in Fabry disease across mild, moderate, and severe renal impairment. The claims require a specific amenable GLA mutation, alternate-day dosing, clinical or biomarker outcomes, and renal-function pharmacokinetic characteristics. The patent is narrower than a general migalastat treatment patent and is principally relevant to method-of-use infringement, label carve-outs, clinical-trial design, and generic launch strategy.

The claim set covers:

Mild renal impairment: eGFR 60 to 90 mL/min/1.73 m².
Moderate renal impairment: eGFR 30 to 59 mL/min/1.73 m².
Severe renal impairment: eGFR below 30 mL/min/1.73 m².
Migalastat dosing of approximately 100 to 150 mg FBE once every other day.
HEK-assay-amenable alpha-galactosidase A mutations.
Renal, cardiac, biochemical, and pharmacokinetic outcomes.
Migalastat hydrochloride solid oral dosage forms and free-base or pharmaceutically acceptable salt forms.

What does U.S. Patent 10,792,279 cover?

The patent covers a treatment method, not a new migalastat molecule. Its independent claims require administration of migalastat to Fabry disease patients in defined renal-function categories.

Independent claim	Patient population	Dose and frequency	Required pharmacokinetic limitation
Claim 1	ERT-experienced patients; eGFR 60-90	100-150 mg FBE once every other day	Mean AUC0-inf approximately 1.2-fold versus healthy controls; no increase in Cmax
Claim 16	eGFR 30-59	100-150 mg FBE once every other day	Mean AUC0-inf approximately 1.8-fold; no increase in Cmax
Claim 32	eGFR below 30	100-150 mg FBE once every other day	Mean AUC0-inf approximately 4.5-fold; no increase in Cmax

Each independent claim also requires administration to patients with a HEK-assay-amenable alpha-galactosidase A mutation and requires treatment to be effective for a combination of outcomes involving GL-3, lyso-Gb3, WBC alpha-galactosidase A activity, LVMi, and renal-function stabilization.

The claims therefore combine four limitation groups:

Patient diagnosis and mutation status.
Renal impairment classification.
Dose and alternate-day administration.
Treatment response and pharmacokinetic results.

A product that merely contains migalastat does not practice these claims. Infringement would require performance of the claimed treatment method.

How are the mild, moderate, and severe renal-impairment claims different?

The renal categories are mutually segmented but cover the full range of reduced kidney function.

Mild renal impairment

Claim 1 is the most restrictive independent claim because it requires ERT-experienced patients. The eGFR range is 60 to 90 mL/min/1.73 m². The claimed single-dose exposure is approximately 1.2 times the exposure in healthy subjects.

Dependent claims add quantitative treatment outcomes:

Kidney interstitial capillary GL-3 inclusion reduction of approximately 0.30.
Plasma lyso-Gb3 reduction of approximately 7.7 nmol/L.
WBC alpha-galactosidase A activity increase of approximately 1.6 4MU/hr/mg.
LVMi reduction of approximately 9.2 g/m².
Annualized eGFR change greater than -1.0 mL/min/1.73 m².

Claims 9 through 11 divide the mild-impairment population by baseline proteinuria:

Less than 100 mg/24 hours.
100 to 1,000 mg/24 hours.
Greater than 1,000 mg/24 hours.

Moderate renal impairment

Claim 16 covers eGFR of 30 to 59 mL/min/1.73 m². Unlike claim 1, it does not make ERT experience an independent-claim requirement. Claim 17 adds that limitation.

The moderate-impairment pharmacokinetic limitation is an approximately 1.8-fold increase in AUC0-inf, with no increase in Cmax relative to healthy subjects.

The numerical outcome claims are:

GL-3 inclusion reduction of approximately 0.39.
Plasma lyso-Gb3 reduction of approximately 29.0 nmol/L.
WBC alpha-galactosidase A activity increase of approximately 1.4 4MU/hr/mg.
LVMi reduction of approximately 5.5 g/m².
Annualized eGFR change greater than -1.0 mL/min/1.73 m².

Claims 25 through 27 divide moderate-impairment patients by the same three proteinuria categories.

Severe renal impairment

Claim 32 covers patients with eGFR below 30 mL/min/1.73 m². Claim 33 adds ERT experience as a dependent limitation.

The severe-impairment pharmacokinetic requirement is materially different: approximately 4.5-fold higher AUC0-inf than healthy controls, without increased Cmax.

Claims 34 through 39 add kidney and heart GL-3 reduction, proteinuria categories, the 150 mg hydrochloride solid dosage form, and the HEK assay definition. The severe-impairment claims do not include the detailed GL-3, lyso-Gb3, WBC activity, and LVMi numerical endpoints found in claims 18 through 23.

What mutations qualify under the HEK assay limitation?

Claims 13, 29, and 39 define an amenable mutation using a functional assay in HEK-293 cells. The mutation must satisfy both tests when expressed in the cells and evaluated with 10 micromolar migalastat:

At least a 20% relative increase in alpha-galactosidase A activity compared with cells without migalastat.
An absolute increase equal to at least 3% of wild-type alpha-galactosidase A activity.

This limitation is important because the patent does not cover every Fabry disease patient. It is directed to patients whose specific GLA variant responds to pharmacological chaperoning under the claimed assay criteria.

Potential disputes include:

Whether a particular variant satisfies the assay thresholds.
Whether the accused party must use the exact HEK-293 protocol.
Whether assay variability affects the 20% and 3% thresholds.
Whether a mutation identified through a later validated assay is equivalent to a mutation meeting the claim language.
Whether the term "HEK assay amenable" requires testing for each patient or can be established from an established mutation database.

The mutation limitation creates a significant proof issue for method-of-use enforcement. A patent owner may need to establish the genotype of treated patients and show that the relevant mutation satisfies the assay definition.

What dosing and formulations are protected?

Claims 1, 16, and 32 require approximately 100 to 150 mg FBE once every other day. The claim uses free-base-equivalent terminology, which allows the active amount to be expressed independently of the salt form.

Claim 12, claim 28, and claim 38 narrow the treatment to:

Approximately 150 mg migalastat hydrochloride.
Oral administration.
At least 28 days.
Solid dosage form.

Claims 14, 15, 30, 31, 40, and 41 cover the active ingredient as:

Migalastat free base.
A pharmaceutically acceptable salt.

The formulation claims are not broad composition-of-matter claims. They do not independently protect every tablet containing migalastat hydrochloride. They protect use of the specified formulation in the claimed patient population and dosing regimen.

What clinical outcomes are required by the claims?

The independent claims contain a broad functional requirement that administration be effective to:

Reduce GL-3 accumulated in an organ.
Reduce plasma lyso-Gb3.
Increase WBC alpha-galactosidase A activity.
Reduce LVMi.
Stabilize renal function.

The dependent claims impose numerical results. Those results appear to describe group means rather than mandatory outcomes for every individual patient. This distinction matters in litigation. A generic or competitor could argue that a physician's act of prescribing does not guarantee that the claimed mean results will occur.

The renal stabilization limitation in claims 6, 22, and 23 requires an annualized eGFRCKD-EPI rate of change greater than -1.0 mL/min/1.73 m². This is a population-level clinical outcome and may be difficult to prove at the point of prescribing.

The patent is consequently strongest where the marketed label, clinical protocol, or promotional materials expressly identify:

The renal impairment subgroup.
The HEK-amenable mutation requirement.
Alternate-day dosing.
The claimed biomarkers or renal-function endpoint.

How strong is the patent estate for migalastat?

U.S. Patent 10,792,279 is a narrow but commercially relevant method-of-use patent. Its strength is based on the combination of multiple limitations rather than any single broad claim.

Strength factor	Assessment
Composition coverage	None apparent from the supplied claims
Treatment-method coverage	Significant, but limited to defined renal strata and outcomes
Patient-selection coverage	Strongly focused on HEK-assay-amenable GLA mutations
Formulation coverage	Narrow, limited to use of oral solid migalastat hydrochloride
Dose coverage	Focused on approximately 100-150 mg FBE once every other day
Biomarker coverage	Detailed and potentially useful for label-based enforcement
Infringement detectability	Mixed; dose and patient selection may be visible, mean outcomes are harder to prove
Invalidity exposure	Potential issues include written description, enablement, indefiniteness, obviousness, and functional-result limitations
Generic blocking value	Depends heavily on the approved label and whether a carve-out can omit the claimed renal-use population

The patent does not appear, from the supplied claims, to prevent a generic applicant from making or selling migalastat for all nonclaimed uses. Its commercial effect depends on whether the approved product label includes the covered renal-impairment populations and whether the generic can omit those uses under the Hatch-Waxman regime.

When does migalastat lose exclusivity?

Migalastat received FDA approval for Fabry disease under the Galafold brand. The product is a small molecule, so biosimilar exclusivity does not apply. Generic applicants would generally use an abbreviated new drug application, subject to applicable listed patents, regulatory exclusivity, and Paragraph IV certification requirements.[2]

The precise expiration date of U.S. Patent 10,792,279 cannot be determined from the claims alone. The controlling date requires the patent's earliest effective nonprovisional priority, any patent-term adjustment, any patent-term extension, terminal disclaimers, and abandonment or continuity history. A patent's 20-year term generally runs from the relevant nonprovisional filing framework, subject to statutory adjustments under 35 U.S.C. §§ 154 and 156.[3]

The FDA's Orange Book, not the claim text, determines whether the patent is listed against Galafold for Hatch-Waxman purposes. Orange Book relevance depends on whether the patent claims an approved method of use, drug substance, or drug product and whether the listing satisfies FDA requirements.[2]

What is the Orange Book status of U.S. Patent 10,792,279?

The supplied material does not establish the patent's current Orange Book listing status, expiration entry, use code, or any delisting event. Patent ownership and Orange Book listing are separate issues.

For generic-entry analysis, the relevant questions are:

Whether U.S. Patent 10,792,279 is listed against Galafold.
Whether it carries a method-of-use code covering Fabry disease with renal impairment.
Whether the approved label describes the claimed patient populations.
Whether an ANDA applicant can use a section viii statement to omit the protected use.
Whether the patent has been challenged through a Paragraph IV certification.

A listed method-of-use patent can delay approval if the applicant submits Paragraph IV certification and the NDA holder or patent owner files suit within the statutory period. A section viii carve-out may avoid infringement only if the proposed labeling and marketing conduct genuinely omit the patented method.[2]

Which companies are challenging the migalastat patent estate?

No challenger, Paragraph IV certification, patent litigation, or settlement agreement is identified in the supplied material. The claims alone do not establish whether any generic company has filed an ANDA, submitted a Paragraph IV notice, initiated an inter partes review, or entered a settlement with the patent owner.

The likely competitive categories are:

Generic manufacturers pursuing an ANDA for migalastat hydrochloride.
Specialty-pharmaceutical companies seeking a regional or licensing arrangement.
Fabry disease competitors using enzyme replacement therapy.
Developers of alternative pharmacological chaperones or gene therapies.

Migalastat does not face biosimilar competition because it is a chemical drug rather than a biologic. ERT products, including agalsidase beta and agalsidase alfa, compete clinically but do not create biosimilar exposure for migalastat itself.

What patent litigation and settlement issues matter?

The most material litigation theories would involve:

Infringement

A patent owner would likely focus on evidence that a defendant's label or promotional materials direct treatment of:

Patients with eGFR 30 to 90 or below 30.
Patients carrying amenable GLA mutations.
Patients receiving alternate-day migalastat.
Patients receiving approximately 150 mg migalastat hydrochloride.

The numerical outcome limitations may be more difficult to use as premarketing infringement theories because they concern treatment results rather than instructions.

Invalidity

Potential defenses include:

Obviousness based on earlier migalastat treatment disclosures and renal-impairment pharmacokinetic studies.
Lack of written description for the full breadth of mutation, renal-function, and outcome combinations.
Enablement challenges concerning all HEK-assay-amenable mutations and all claimed renal categories.
Indefiniteness involving "about," "effective to," "stabilize," "no increase," and group-mean outcome language.
Lack of patentable distinction if the claimed results merely reflect expected pharmacokinetics in renal impairment.

The patent's numerical limitations may support validity by tying the claims to specific clinical observations. They may also create enforceability risk if the specification does not adequately support the full patient and mutation scope.

Settlement

A settlement could include a licensed entry date, a supply agreement, or a label restriction. No settlement terms are established by the supplied information. Any commercial entry forecast based on a settlement would therefore be unsupported.

How does this patent compare with ERT and gene-therapy patent risks?

Therapy	Product type	Primary patent risk	Biosimilar or generic pathway
Migalastat	Small-molecule pharmacological chaperone	Composition, formulation, method-of-use, patient-selection patents	ANDA and Paragraph IV
Agalsidase beta	Enzyme replacement biologic	Biologic composition, manufacturing, formulation, treatment patents	Biosimilar pathway
Agalsidase alfa	Enzyme replacement biologic	Biologic and manufacturing patents	Biosimilar or regional regulatory pathway
Fabry gene therapy	Gene-based biologic	Vector, transgene, manufacturing, dosing, treatment patents	Biologic licensing pathway

This patent is most relevant to migalastat-specific competition. It does not directly block ERT products or gene therapies. Conversely, ERT and gene-therapy patents may affect treatment substitution but would not normally be asserted against a generic migalastat product.

What generic launch scenarios exist?

Scenario 1: Full-label generic launch

This presents the highest infringement exposure if the generic label directs treatment of all covered renal-impairment populations and amenable mutations. A Paragraph IV challenge would be the likely route if the patent is listed and viewed as blocking.

Scenario 2: Section viii carve-out

A generic applicant could attempt to omit the patented renal-impairment method from its labeling. The commercial value of the carve-out would depend on whether the remaining label supports treatment of a sufficiently large Fabry population.

Scenario 3: Label-neutral launch

A generic might avoid explicit instructions regarding renal subgroups, mutation testing, or clinical outcomes. That strategy would not eliminate induced-infringement risk if marketing materials or distribution practices direct the patented use.

Scenario 4: Post-expiration launch

This is the lowest legal-risk scenario but depends on the actual patent-term calculation and any other Orange Book-listed patents covering Galafold.

What geographic coverage does the patent provide?

U.S. Patent 10,792,279 provides rights only in the United States. It does not establish protection in Europe, Japan, Canada, or other markets. Geographic freedom-to-operate requires review of the corresponding international and national-stage family members, including:

European Patent Office members.
United Kingdom.
Japan.
Canada.
Australia.
China.
South Korea.

Family members may differ in claim scope, prosecution history, expiration dates, opposition outcomes, and enforceability. The U.S. claims should not be used as a proxy for foreign coverage.

Key Takeaways

U.S. Patent 10,792,279 is a renal-impairment and patient-selection method patent for migalastat in Fabry disease.
The independent claims divide patients into mild, moderate, and severe renal-impairment groups.
All independent claims require approximately 100 to 150 mg FBE once every other day and a HEK-assay-amenable GLA mutation.
The claims include biomarker, cardiac, renal, and pharmacokinetic outcome limitations.
Claims 12, 28, and 38 specifically cover oral solid migalastat hydrochloride used for at least 28 days.
The patent does not provide broad composition-of-matter protection for migalastat.
Migalastat faces generic, not biosimilar, competition.
Orange Book listing, use codes, Paragraph IV activity, litigation, settlements, and the precise expiration date are not established by the supplied claims.
Generic launch risk will depend on the approved Galafold label, patent listing status, available section viii carve-outs, and the scope of other migalastat patents.

FAQs

Does U.S. Patent 10,792,279 cover all Fabry disease patients?

No. The claims require defined renal impairment, an amenable alpha-galactosidase A mutation, alternate-day migalastat dosing, and specified treatment-response limitations.

Does the patent cover once-daily migalastat dosing?

The supplied claims require administration once every other day. Once-daily dosing is outside the literal dosing limitation, subject to possible infringement under other patents or the doctrine of equivalents.

Can a generic sell migalastat for Fabry patients with normal renal function?

This patent's independent claims are directed to patients with mild, moderate, or severe renal impairment. Other patents, labeling restrictions, or regulatory exclusivities could still affect a normal-renal-function indication.

Is the HEK assay limitation a requirement for every treated patient?

The claims require administration to patients having an amenable mutation as defined by the assay criteria. Whether a specific mutation qualifies may become a factual and scientific issue in enforcement or validity proceedings.

Does the patent protect the 150 mg Galafold tablet itself?

No. The supplied claims protect use of approximately 150 mg migalastat hydrochloride in a solid oral dosage form within the claimed Fabry disease treatment methods. They do not independently claim the tablet as a composition.

References

United States Patent and Trademark Office. (2020). U.S. Patent No. 10,792,279, methods of treating Fabry disease.
U.S. Food and Drug Administration. (2024). Approved drug products with therapeutic equivalence evaluations: Orange Book.
United States Code. (2024). 35 U.S.C. §§ 154 and 156: Patent term and patent term extension.
U.S. Food and Drug Administration. (2018). Galafold (migalastat) prescribing information.

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	10,792,279	⤷ 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
Argentina	111971	⤷ Start Trial
Argentina	131106	⤷ Start Trial
Argentina	131107	⤷ Start Trial
Australia	2009214648	⤷ Start Trial
Australia	2014221321	⤷ Start Trial
Australia	2016206297	⤷ Start Trial
>Country	>Patent Number	>Estimated Expiration	>Supplementary Protection Certificate	>SPC Country	>SPC Expiration

Details for Patent: 10,792,279

Which drugs does patent 10,792,279 protect, and when does it expire?

Summary for Patent: 10,792,279