Details for Patent: 10,849,889

Which drugs does patent 10,849,889 protect, and when does it expire?

Patent 10,849,889 protects GALAFOLD and is included in one NDA.

This patent has one hundred and thirty-seven patent family members in twenty-seven countries.

Summary for Patent: 10,849,889

Title:	Methods of treating Fabry patients having renal impairment
Abstract:	Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in α-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.
Inventor(s):	Jeff Castelli, Elfrida Benjamin
Assignee:	Bpcr LP , Amicus Therapeutics Inc
Application Number:	US16/817,877
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for patent 10,849,889
Patent Claim Types: see list of patent claims	Use; Delivery; Dosage form;

US Patent 10,849,889 Scope and Claims Analysis for Migalastat in ERT-Experienced Fabry Patients With Renal Impairment

US Patent 10,849,889 is a US-method-of-treatment patent that claims a specific clinical population (Fabry disease, ERT-experienced, renal impairment, and an “HEK assay amenable” α-galactosidase A mutation) and a specific migalastat dosing regimen (about 100–150 mg free base equivalent (FBE) once every other day). The claims are tightly tied to biomarkers and functional endpoints: reduction of GL-3 (in kidney and, in dependent claims, heart and liver) and stabilization of renal function measured via eGFR metrics, with additional numeric thresholds for proteinuria, GL-3 inclusion reduction, and α-galactosidase A activity in an HEK-293 assay.

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What is US Patent 10,849,889 and what does it claim for migalastat Fabry renal outcomes?

Core claim theme: a genotype-defined responder subgroup (HEK assay amenable α-galactosidase A mutations) treated with a specific migalastat exposure scheme to stabilize renal function in ERT-experienced Fabry patients with renal impairment.

Claim set structure in your text

The independent methods (your text shows three independent claim groupings: claims 1, 20, and 26) share these pillars:

1. Disease and treatment context

   • Fabry disease
   • Patient is ERT-experienced (explicit in claims 1 and 20; explicit in claim 26)

2. Patient selection criteria

   • Renal impairment (with eGFR ranges in dependent claims 4-6; proteinuria thresholds in dependent claims 7-9)
   • HEK assay amenable mutation in α-galactosidase A (claims 1, 16, 17, 20, 23, 26)

3. Dose, dosing frequency, and drug form

   • Migalastat administered at about 100 mg to 150 mg FBE
   • Frequency: once every other day
   • Route/formulation: solid dosage form and oral administration in dependent claims (13-14); free base vs pharmaceutically acceptable salt in dependent claims (18-19, 24-25, 27-28)

4. Mechanism/endpoint linkage via biomarkers and renal function

   • Reducing GL-3 accumulated in organs (kidney; and dependent claims extend to kidney/heart and kidney/heart/liver)
   • Stabilizing renal function, using either:
     • general “stabilize renal function” (claims 1 and 26), and/or
     • a defined quantitative renal stabilization metric in claim 20:
       • mean annualized rate of change in eGFRCKD-EPI > −1.0 mL/min/1.73 m²

Immediate claim-scope answer (featured snippet style)

US 10,849,889 claims once-every-other-day migalastat at ~100–150 mg FBE in ERT-experienced Fabry patients with renal impairment and HEK assay amenable α-galactosidase A mutations, requiring GL-3 reduction and renal function stabilization (with numeric eGFR change endpoints and/or GL-3 inclusion reduction thresholds in dependent claims).

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How broad are the independent claims (1, 20, 26) on population, dosing, and endpoints?

Claim 1 (individual-patient method)

Required elements

• ERT-experienced patient
• Fabry disease
• Renal impairment
• HEK assay amenable α-galactosidase A mutation
• Dosing:
  • migalastat at 100–150 mg FBE
  • once every other day
• Endpoint:
  • administration effective to “stabilize renal function”
  • and “reducing GL-3 accumulated in organs”

Dependent expansions

• Organs: kidney and heart (claim 2); kidney/heart/liver (claim 3)
• Renal impairment defined by eGFR bands (claims 4–6)
• Proteinuria thresholds at baseline (claims 7–9)
• Duration minima (claims 10–12)
• Form/route: solid dosage form; oral (claims 13–14)
• Biomarker quantification of kidney interstitial capillary GL-3 inclusions (claim 15)
• HEK assay definition and testing flow (claims 16–17)
• Drug form: free base vs salt (claims 18–19)

Practical implication: claim 1 is the broadest single-patient framework because its endpoints are expressed in functional language (“effective to stabilize renal function” and “reducing GL-3”) while still forcing the genotype and dosing regimen.

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Claim 20 (group method with quantitative renal endpoint)

Required elements

• A group of Fabry disease patients with:
  • renal impairment
  • HEK assay amenable α-galactosidase A mutation
• Dosing:
  • migalastat about 100–150 mg FBE
• Endpoint requirements expressed quantitatively as group means:
  1. mean decrease in GL-3 in kidneys
  2. mean annualized rate of change in eGFRCKD-EPI > −1.0 mL/min/1.73 m²

Dependent specificity

• Moderate vs mild renal impairment subgroup definitions and numeric GL-3 inclusion reductions (claims 21–22)
• Repeats HEK mutation definition (claim 23)
• Repeats form (claims 24–25)

Practical implication: claim 20 creates litigation leverage for population-level trial results because it pins a renal stabilization threshold to a specific statistical/clinical metric.

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Claim 26 (individual-patient method with structured baseline criterion)

Required elements

• Patient diagnosed with Fabry disease and renal impairment
• HEK assay amenable α-galactosidase A mutation
• ERT-experienced
• Proteinuria at baseline specifically 100 to 1,000 mg/24 hr
• Dosing:
  • migalastat about 100–150 mg FBE
  • once every other day
• Endpoint:
  • stabilizing renal function
  • reducing GL-3 accumulated in kidney and heart
• HEK assay definition is embedded in the claim text (claim 26)

Dependent expansions

• Free base vs salt (claims 27–28)

Practical implication: claim 26 narrows to a baseline proteinuria band. That makes it easier to prove eligibility for that subgroup but reduces coverage against proteinuria strata outside the 100–1,000 mg/24 hr window.

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What does “HEK assay amenable” mean in US 10,849,889 and how does it constrain infringement?

Both claim 16 and claim 23 define the HEK assay amenable mutation as a mutation that, when expressed in HEK-293 cells with 10 μM migalastat, shows:

1. Relative increase of at least 20% in α-galactosidase A activity (vs without migalastat)
2. Absolute increase of at least 3% of wild-type α-galactosidase A activity

Constrained infringement mechanism

These quantitative assay thresholds tie claim scope to a specific functional genotype classification rather than to a named list of mutations in the text you provided.

Where this matters

• A challenger can avoid infringement by showing the patient’s α-galactosidase A mutation does not satisfy both assay thresholds.
• Conversely, a patentee can strengthen proof by introducing assay reports, cell-based data, or validated conversion tables linking mutations to assay outcomes.

Testing/timing element

• Claim 17 adds a procedural step: determining the patient has the HEK assay amenable mutation before administering migalastat.

Litigation leverage point

Because the assay is specified by concentration and readouts, it is not purely descriptive. It can be litigated like a lab-defined eligibility test.

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What dosing scope is protected: 100–150 mg FBE and once every other day?

Dose window

All independent claim groupings require migalastat exposure at about:

• 100 mg to 150 mg FBE

Frequency

Only in claims explicitly shown as independent:

• once every other day is required in claim 1 and claim 26
• your provided text for claim 20 does not explicitly state frequency, but it does require administration of about 100–150 mg FBE and outcome thresholds

Practical implication for design-around

• Changing the dose outside 100–150 mg FBE or changing to a more frequent regimen (if it changes effective exposure and thus the “administering ... once every other day” limitation in claims 1/26) is a direct attempt at avoiding at least those independent claim paths.
• Any at-risk product profile should be evaluated versus each independent claim’s frequency requirement.

Drug form scope

• Free base (claims 18, 24, 27)
• Pharmaceutically acceptable salt (claims 19, 25, 28)
• Solid dosage form (claim 13)
• Oral administration (claim 14)

Practical implication Once within the dose window and regimen, the patent anticipates common commercial salt/free-base variations and oral solid formats.

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What renal impairment definitions and baseline criteria are used to limit eligibility?

eGFR ranges (dependent claims 4–6)

• Mild: 60–90 mL/min/1.73 m²
• Moderate: 30–59 mL/min/1.73 m²
• Severe: <30 mL/min/1.73 m²

These are used in dependent form, so the eGFR bands are additional limitations if asserted under those dependent claims.

Proteinuria baseline (dependent claims 7–9; embedded in claim 26)

• < 100 mg/24 hr (claim 7)
• 100–1,000 mg/24 hr (claim 8)

• 1,000 mg/24 hr (claim 9)

Claim 26 embeds 100–1,000 mg/24 hr as a hard eligibility condition.

Duration minima (dependent claims 10–12)

• at least 28 days
• at least 6 months
• at least 12 months

These are dependent claim limitations that can separate infringement theories by treatment duration evidence (protocol compliance in trials, persistence of dosing, etc.).

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What biomarker and renal endpoints are required, and where are numeric thresholds stated?

GL-3 reduction

• Claim 1: “reducing GL-3 accumulated in the patient's organs” and dependent coverage expands organs (kidney; kidney/heart; kidney/heart/liver)
• Claim 20: “mean decrease in GL-3 in the kidneys of the patients”
• Claim 26: “reducing GL-3 accumulated in the patient's kidney and heart”

Numeric kidney interstitial capillary GL-3 inclusion reductions (dependent claims 15 and 21–22)

• Claim 15: reduction in kidney interstitial capillary GL-3 inclusions of about 0.30
• Claim 21 (moderate): about 0.39
• Claim 22 (mild): about 0.30

These provide objective, quantitative readouts that can narrow debate about “reduction” vs quantified effects.

Renal function stabilization metric (claim 20)

• Mean annualized rate of change in eGFRCKD-EPI > −1.0 mL/min/1.73 m²

This is the only numeric renal threshold stated in your claim text. It is likely the strongest litigable “stabilization” criterion in the provided text.

Proteinuria is baseline-defining

Proteinuria does not appear as an outcome threshold in the text you provided. It is a selection criterion.

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What formulations and administration forms are explicitly covered?

US 10,849,889 in your text includes:

• Solid dosage form (claim 13)
• Orally administered (claim 14)
• Free base (claims 18, 24, 27)
• Pharmaceutically acceptable salt (claims 19, 25, 28)

Scope impact If an accused product uses a different route (not oral) or a non-solid form, it may attempt to avoid dependent claim limitations. However, independent claims are not route-limited in your provided text; they hinge on administration of migalastat at the dose and regimen and on biological outcomes and genotype selection.

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How does US 10,849,889 compare with typical Fabry ERT/add-on migalastat patent strategies?

This patent’s distinguishing feature is the coupling of:

1. ERT-experienced status (context limitation)
2. Genotype defined by an HEK assay functional test
3. A dosing regimen constraint (100–150 mg FBE once every other day)
4. Biomarker readouts (GL-3 inclusion reduction)
5. A renal function stabilization threshold in a group endpoint claim (claim 20)

In Fabry migalastat IP landscapes, many patents fall into one of two categories:

• genotype-response definitions and dosing for “miglatast-responsive” mutations, or
• renal/biomarker outcomes independent of ERT-exposure status.

US 10,849,889 explicitly intersects both, which increases infringement friction for generic or alternative dosing strategies.

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What is the likely US litigation and challenge posture for a patent with this claim construction?

Based on the claim structure you provided, the strongest defense lanes in US litigation would be:

• Mutation non-amenability: the HEK assay amenable thresholds are not met for the specific patient mutation
• Dose/frequency nonconformance: not within 100–150 mg FBE or not dosed once every other day (for claims requiring that frequency)
• Outcome nonconformance: trial or real-world outcomes do not meet:
  • eGFRCKD-EPI annualized change > −1.0 (claim 20), and/or
  • GL-3 inclusion reductions do not meet the numeric “about 0.30/0.39” in dependent claims (15, 21, 22)
• Eligibility mismatch: renal impairment category or proteinuria baseline outside the claim limitations (especially claim 26’s 100–1,000 mg/24 hr)

Settlement leverage tends to increase when a patent claims a narrow, protocol-like combination (dose, frequency, biomarker endpoints), because it reduces the set of viable noninfringing designs.

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What generic or alternative regimen entry risks does US 10,849,889 create?

For a competitor launching a generic or marketed product of migalastat:

• If the product is the same active ingredient and dose range, the key infringement question becomes whether the new regimen and patient selection match the claim elements.
• The patent is not a composition-of-matter claim in your text; it is a treatment method claim. That shifts risk to:
  • prescribing practices (dose and regimen)
  • treatment population (ERT-experienced + mutation classification)
  • and the measured biomarkers/outcomes

The design-around risk is highest when:

• formularies or labels in the US support dosing in the claimed range/frequency, and
• clinicians follow a genotype-informed approach aligned with HEK assay amenability and treat ERT-experienced patients with renal impairment.

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Key Takeaways

• US 10,849,889 claims migalastat add-on therapy for ERT-experienced Fabry patients with renal impairment, restricted to HEK assay amenable α-galactosidase A mutations defined by quantitative HEK-293 assay readouts.
• The core dosing is about 100–150 mg FBE administered once every other day (required in claims 1 and 26).
• The claimed effectiveness is tied to GL-3 reduction and renal function stabilization, with the strongest quantitative renal endpoint in claim 20: mean annualized eGFRCKD-EPI > −1.0.
• Dependent claims add numeric constraints on baseline proteinuria, eGFR categories, treatment duration, and kidney interstitial capillary GL-3 inclusion reductions.

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FAQs

1) Does US 10,849,889 require that the patient have a specific named Fabry mutation?
No. The patent text you provided defines amenability by a functional HEK-293 assay criterion (relative and absolute α-galactosidase A activity improvements in the presence of 10 μM migalastat).

2) Is the “renal stabilization” requirement objective in this patent?
It is functional in claims 1 and 26 (“stabilize renal function”) but becomes objective in claim 20 via a numeric renal endpoint: mean annualized eGFRCKD-EPI > −1.0.

3) What happens if migalastat is dosed outside 100–150 mg FBE?
That is a direct attempt to avoid the claimed dose window, which is required as an element in the independent methods in your provided text.

4) Can organ coverage expand beyond kidney?
Yes. Dependent claims specify reduction of GL-3 in kidney and heart (claim 2) and in kidney/heart/liver (claim 3).

5) Is baseline proteinuria required for all claim paths?
No. It is required in dependent claims 7–9 and embedded as an eligibility condition in claim 26 (100–1,000 mg/24 hr), but claim 1 and claim 20 do not impose a proteinuria baseline range in the portion you provided.

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References

1. User-provided claim text for US Patent 10,849,889 (claims 1–28 as included in the prompt).

Drugs Protected by US Patent 10,849,889

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	10,849,889					THE TREATMENT OF FABRY PATIENTS

International Family Members for US Patent 10,849,889

Country	Patent Number	Estimated Expiration	Supplementary Protection Certificate	SPC Country	SPC Expiration
Argentina	111971
Argentina	131106
Argentina	131107