Details for Patent: 10,888,547

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Which drugs does patent 10,888,547 protect, and when does it expire?

Patent 10,888,547 protects CERDELGA and is included in one NDA.

This patent has one hundred and twelve patent family members in forty countries.

Summary for Patent: 10,888,547

Title:	Amorphous and a crystalline form of genz 112638 hemitartrate as inhibitor of glucosylceramide synthase
Abstract:	The hemitartrate salt of a compound represented by the following structural formula: (Formula I Hemitartrate), which may be used in pharmaceutical applications, are disclosed. Particular single crystalline forms of the Formula (I) Hemitartrate are characterized by a variety of properties and physical measurements. As well, methods of producing crystalline Formula (I) Hemitartrate, and using it to inhibit glucosylceramide synthase or lowering glycosphingolipid concentrations in subjects to treat a number of diseases, are also discussed. Pharmaceutical compositions are also described.
Inventor(s):	Judith Peterschmitt
Assignee:	Genzyme Corp
Application Number:	US15/049,946
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for patent 10,888,547
Patent Claim Types: see list of patent claims	Use;

United States Patent 10,888,547: Eliglustat Gaucher Disease Dosing, CYP2D6 Genotyping, and Patent Landscape

U.S. Patent No. 10,888,547 protects a pharmacogenomic dosing method for eliglustat, the active ingredient in Cerdelga. Its central distinction is CYP2D6-based dose selection: poor metabolizers receive 84 mg once daily, while intermediate and extensive metabolizers receive 84 mg twice daily. The patent does not broadly protect eliglustat, its chemical composition, or every treatment of Gaucher disease. Its commercial value depends on whether the patent is listed for Cerdelga, whether a generic or follow-on applicant must perform CYP2D6-guided dosing, and whether the claims can be enforced against prescribers, manufacturers, or both.

What drug and therapeutic use does U.S. Patent 10,888,547 cover?

The compound of formula (I) is eliglustat. Claim 2 identifies the administered product as the hemitartrate salt of eliglustat at 100 mg, equivalent to 84 mg of eliglustat free base. Cerdelga is marketed as eliglustat tartrate capsules containing 84 mg of eliglustat per capsule-equivalent dose. The FDA approved Cerdelga in 2014 for the long-term treatment of adults with Gaucher disease type 1 who are CYP2D6 extensive, intermediate, or poor metabolizers [1].

The patent covers a treatment protocol rather than a new molecular entity. The protocol requires:

1. CYP2D6 genotyping before treatment begins.
2. Classification of the patient as a poor, intermediate, or extensive metabolizer.
3. Administration of a genotype-specific eliglustat dose.
4. Treatment of Gaucher disease, particularly type 1 disease in dependent claims.

The claims do not expressly cover ultrarapid metabolizers. That omission is consistent with the Cerdelga label, which contains restrictions and dose-management provisions for CYP2D6 phenotypes outside the principal extensive, intermediate, and poor-metabolizer groups [1].

What are the independent claims in U.S. Patent 10,888,547?

The two independent claims divide the covered population into two dosing groups.

Independent claim	Genotype-based population	Eliglustat free-base dose	Hemitartrate salt equivalent	Frequency
Claim 1	Poor CYP2D6 metabolizer	84 mg/day	100 mg/day	Once daily under claim 3
Claim 14	Intermediate or extensive CYP2D6 metabolizer	84 mg twice daily	100 mg twice daily	Twice daily

Claim 1 requires treatment of a poor metabolizer at 84 mg per day. Claim 14 requires treatment of an intermediate or extensive metabolizer at 84 mg twice per day. The claim language makes pre-treatment CYP2D6 assessment an express limitation.

This structure is commercially important. A product label that instructs physicians to genotype patients and select either once-daily or twice-daily eliglustat dosing could practice the claimed regimen even if the generic product uses different capsule labeling or packaging.

How does claim 1 protect poor-metabolizer dosing?

Claim 1 requires all of the following:

• A human patient with Gaucher disease who needs treatment.
• CYP2D6 genotyping before treatment begins.
• Classification as a poor, intermediate, or extensive metabolizer.
• Selection of the patient as a poor metabolizer.
• Administration of eliglustat or a pharmaceutically acceptable salt.
• A daily dose equivalent to 84 mg of eliglustat.

Claims 2 through 13 narrow claim 1 in several ways. They cover the hemitartrate salt, 100 mg per day of the salt, once-daily administration, low CYP2D6 expression, two loss-of-function alleles, PCR or microarray testing, meal timing, and type 1 Gaucher disease.

The strongest practical limitation is the poor-metabolizer requirement. A treatment using 84 mg once daily without prior CYP2D6 assessment may not meet the literal language of claim 1, even if the patient happens to be a poor metabolizer. The claim requires assessment through genotyping, not merely a retrospective determination based on drug exposure or clinical response.

How does claim 14 protect intermediate- and extensive-metabolizer dosing?

Claim 14 covers the complementary dosing protocol:

• The patient has Gaucher disease.
• CYP2D6 genotyping occurs before treatment.
• The patient is classified as poor, intermediate, or extensive.
• The patient is selected as intermediate or extensive.
• Eliglustat is administered at 84 mg twice daily.

Claims 15 through 25 narrow the claim to the hemitartrate salt, 100 mg twice daily, specific allele combinations, PCR or microarray testing, meal timing, and type 1 Gaucher disease.

Claim 14 is broader than a claim limited to a particular allele combination because it covers the functional metabolizer categories. Claims 16 and 17 provide narrower genetic embodiments:

• Intermediate metabolizer: one reduced-activity allele and one null allele.
• Extensive metabolizer: at least one and no more than two normal functional alleles.

These allele-specific claims may have greater evidentiary value in litigation if a defendant’s testing protocol identifies the relevant alleles directly.

What formulations and dosage forms are protected?

The patent does not claim a particular capsule shell, excipient system, dissolution profile, coating, or manufacturing process. Its formulation limitation is limited primarily to the pharmaceutically acceptable salt, with claim 2 and claim 15 specifying the hemitartrate salt.

The principal protected product configuration is therefore:

Feature	Scope
Active ingredient	Eliglustat
Salt embodiment	Eliglustat hemitartrate
Free-base-equivalent dose	84 mg
Salt dose	100 mg
Poor-metabolizer regimen	Once daily
Intermediate/extensive regimen	Twice daily
Food instructions	Before, after, or with a meal; within specified meal windows

Claims 7 through 11 and 19 through 23 cover administration before, after, or with food and progressively narrower periods ranging from two hours to ten minutes from commencing or finishing a meal. These claims may provide fallback positions if a court limits the broader dosing claims.

The claims do not appear to protect a specific controlled-release formulation. A generic capsule that contains the same active ingredient and salt may therefore implicate the patent through its labeled use rather than through a formulation claim.

What is the patent expiration date for U.S. Patent 10,888,547?

The patent issued on Jan. 12, 2021. The enforceable expiration date must be taken from the USPTO patent-term calculation and any Orange Book listing for the corresponding product. The patent number and issue date alone do not establish the final expiration date because the result can depend on the earliest effective nonprovisional filing date, patent-term adjustment, terminal disclaimers, and any applicable patent-term extension.

For diligence purposes, the relevant records are:

Record	Relevance
U.S. Patent No. 10,888,547	Claims, priority chain, issue date, patent-term adjustment
USPTO Patent Center	Continuity, maintenance, terminal disclaimer, prosecution history
FDA Orange Book	Listed patent expiration and product association
FDA Cerdelga label	Approved genotype and dosing instructions
FDA exclusivity records	NCE, orphan, and pediatric exclusivity

No expiration date should be treated as dispositive until the USPTO and Orange Book records are reconciled. A continuation patent can have a later issue date without receiving a later statutory expiration if it claims priority to an earlier application.

What is the Orange Book status of the patent?

A patent has Orange Book significance only if the NDA holder or authorized applicant submitted it for listing and FDA accepted the listing. The Orange Book identifies patents associated with approved drug products, including patents directed to the drug substance, drug product, or method of use [2].

For U.S. Patent 10,888,547, the critical diligence question is whether the patent is listed against NDA 210259 for Cerdelga and, if listed, which use code applies. A use-code listing would be particularly relevant because the patent claims a genotype-directed dosing method rather than the chemical composition of eliglustat.

An Orange Book listing can create a Paragraph IV dispute if an ANDA applicant certifies that the listed patent is invalid, unenforceable, or will not be infringed. The listing does not itself establish validity or infringement. FDA’s regulatory effect depends on the timing and sufficiency of the patent certification and any ensuing litigation.

When does Cerdelga lose regulatory exclusivity?

Cerdelga received FDA approval on Aug. 19, 2014. Its principal regulatory exclusivity periods were distinct from patent rights:

Exclusivity type	General period	Relevance
New chemical entity exclusivity	5 years	Delayed certain ANDA submissions
Orphan-drug exclusivity	7 years	Protected the approved orphan indication
Pediatric exclusivity	6 months, if granted	Extends qualifying exclusivity and certain listed patents

Regulatory exclusivity does not eliminate later patent barriers. Conversely, expiration of a patent does not necessarily eliminate exclusivity if a separate regulatory period remains in effect. Generic entry requires analysis of both the Orange Book patent list and FDA exclusivity records.

What Paragraph IV challenges could affect eliglustat?

A generic eliglustat applicant could use a Paragraph IV certification against listed method-of-use patents. The main potential arguments against Patent 10,888,547 would involve:

• Non-infringement because the proposed label does not require CYP2D6 genotyping.
• Non-infringement because the label omits the claimed meal instructions.
• Lack of infringement because the generic label does not specify the claimed genotype-specific dose.
• Invalidity for anticipation or obviousness based on prior pharmacogenomic dosing knowledge.
• Written-description or enablement challenges directed to the scope of metabolizer categories.
• Lack of induced infringement where the manufacturer does not encourage the complete claimed sequence.

The central litigation issue would likely be divided performance. The claims require pre-treatment genotyping and subsequent drug administration. In routine practice, a laboratory may perform genotyping, while a physician prescribes and a patient takes eliglustat. A patentee would need to establish direct infringement by a single actor or pursue induced or contributory infringement theories. Evidence such as product labeling, clinical protocols, electronic prescribing prompts, and manufacturer educational materials would be material.

How strong is the patent estate for Cerdelga?

Patent 10,888,547 is narrower than a composition-of-matter patent but commercially meaningful because it maps closely to the approved Cerdelga dosing algorithm. Its strength can be assessed across five dimensions.

Dimension	Assessment
Product coverage	Limited; the patent does not broadly claim eliglustat itself
Method-of-use coverage	Meaningful; it covers genotype-directed dosing
Label overlap	Potentially high if the approved label requires CYP2D6 testing and dose selection
Design-around potential	Moderate; a label could attempt to omit or narrow genotype-directed instructions
Litigation leverage	Dependent on Orange Book listing, claim construction, and proof of divided infringement

The narrower dependent claims may be useful against a defendant whose testing uses PCR or microarray platforms, or whose label expressly instructs administration around meals. Their value is lower where the accused product uses a different testing technology or does not include meal-timing instructions.

What manufacturing and intellectual-property barriers remain?

Patent 10,888,547 does not create a major manufacturing barrier by itself. It does not claim:

• A manufacturing process for eliglustat.
• A specific intermediate.
• A purification process.
• A polymorph or crystal form.
• A capsule-excipient combination.
• A container or delivery device.

The principal manufacturing and regulatory barriers are likely to arise from other members of the eliglustat patent family, process patents, drug-product patents, analytical specifications, and FDA requirements for demonstrating pharmaceutical equivalence and bioequivalence.

A generic applicant may be able to manufacture an equivalent capsule while still facing a method-of-use patent. A label carve-out may reduce infringement risk if FDA permits omission of the patented indication or dosing instruction. That strategy is less practical when the patented dosing regimen is inseparable from the approved use and the reference label.

How does eliglustat compare with competing Gaucher disease therapies?

Product	Active ingredient	Treatment type	CYP2D6-genotype dosing	Competitive relevance
Cerdelga	Eliglustat	Oral substrate-reduction therapy	Yes	Direct subject of the patent
Cerezyme	Imiglucerase	Enzyme replacement	No	Established infusion alternative
VPRIV	Velaglucerase alfa	Enzyme replacement	No	Competes for type 1 patients
Elelyso	Taliglucerase alfa	Enzyme replacement	No	Alternative enzyme replacement
Zavesca	Miglustat	Oral substrate-reduction therapy	No comparable CYP2D6 regimen	Different tolerability and positioning

Eliglustat’s pharmacogenomic dosing creates a differentiated prescribing requirement. Enzyme-replacement products do not generally depend on CYP2D6 phenotype, so they do not present the same method-of-use patent risk. The commercial tradeoff is between oral administration and genotype-directed prescribing for eliglustat, versus infusion infrastructure and enzyme-replacement economics for competing therapies [1,3].

What litigation and settlement issues should be monitored?

The relevant litigation triggers are:

1. A Paragraph IV notice concerning an Orange Book listing for Patent 10,888,547.
2. A 45-day period for filing an infringement action under the Hatch-Waxman framework.
3. A potential 30-month FDA approval stay if statutory requirements are met.
4. Claim-construction disputes over "assessing," "genotyping," "poor metabolizer," and "effective amount."
5. Settlement terms governing generic launch dates, authorized generic supply, or label carve-outs.
6. Any post-grant review, inter partes review, reexamination, or district-court invalidity decision.

A settlement may preserve commercial exclusivity beyond the nominal patent expiry through a delayed-entry date, but the business effect depends on whether the agreement includes payments, supply arrangements, or restrictions on competing products. No settlement term should be inferred from the patent claims alone.

What generic launch scenarios exist?

Three launch scenarios are commercially plausible.

Scenario	Description	Risk to Cerdelga
Full-label launch after patent clearance	Generic uses the full genotype-directed regimen	High
Carved-out label	Generic omits patented dosing or use instructions	Moderate
Delayed launch under settlement	Generic enters on an agreed date	Timing-dependent

A full-label launch creates the clearest infringement exposure if the FDA-approved generic label repeats the CYP2D6 testing and dose-selection instructions. A skinny label may reduce risk but can impair marketability if the omitted instructions are central to safe and effective use.

What is the key commercial exposure?

The patent’s revenue exposure is tied to Cerdelga sales in type 1 Gaucher disease, especially patients managed through genotype-guided oral therapy. The claim does not cover every Cerdelga prescription. Exposure is greatest where:

• Genotyping is performed before treatment.
• The prescriber records the metabolizer category.
• The dose is 84 mg once daily for poor metabolizers or 84 mg twice daily for intermediate or extensive metabolizers.
• The patient has type 1 Gaucher disease.
• The product uses the hemitartrate salt.
• The label or clinical support materials reproduce the meal-related instructions.

Revenue modeling should separate patients by CYP2D6 phenotype, dose frequency, geographic market, payer mix, and potential substitution with enzyme-replacement therapies. U.S. exposure is more directly relevant because the patent is territorial and FDA labeling can support infringement allegations.

Key Takeaways

• U.S. Patent 10,888,547 is a method-of-use patent covering CYP2D6-guided eliglustat dosing for Gaucher disease.
• Claim 1 covers 84 mg once daily for poor metabolizers.
• Claim 14 covers 84 mg twice daily for intermediate and extensive metabolizers.
• The hemitartrate salt embodiments use 100 mg once daily or twice daily.
• Pre-treatment CYP2D6 genotyping is a required claim element.
• The patent does not broadly claim eliglustat, its synthesis, or a specific capsule formulation.
• Claims directed to PCR, microarray testing, meal timing, allele status, and type 1 Gaucher disease provide narrower fallback positions.
• Orange Book listing and use-code status determine the patent’s Hatch-Waxman significance.
• Paragraph IV exposure depends on the generic label, patent listing, and whether the complete dosing protocol is encouraged.
• Divided infringement is a material legal issue because genotyping and treatment may be performed by different actors.
• Patent-term, Orange Book, and FDA exclusivity records must be evaluated together.

FAQs About U.S. Patent 10,888,547 and Cerdelga

Does U.S. Patent 10,888,547 cover all eliglustat prescriptions?

No. It covers specific genotype-directed treatment methods requiring pre-treatment CYP2D6 genotyping and specified doses.

Can a generic avoid the patent by selling a different capsule strength?

Not necessarily. The claims focus on the treatment method and dose, not only capsule strength. A different capsule presentation could still be relevant if the label induces the claimed regimen.

Does the patent cover Gaucher disease type 2 or type 3?

The independent claims refer broadly to Gaucher disease, but the dependent claims specifically recite type 1 disease. The approved Cerdelga indication is focused on adults with type 1 Gaucher disease.

Is CYP2D6 phenotyping equivalent to CYP2D6 genotyping under the claims?

Not automatically. The claims expressly require assessment through CYP2D6 genotyping. A functional enzyme assay or clinical phenotype determination may present a non-infringement argument, subject to claim construction and equivalents analysis.

Does expiration of the patent automatically permit generic Cerdelga launch?

No. Generic launch also depends on other listed patents, regulatory exclusivity, FDA approval, any litigation injunction, and settlement restrictions.

References

1. U.S. Food and Drug Administration. (2023). Cerdelga (eliglustat) prescribing information. https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/205494s016lbl.pdf

2. U.S. Food and Drug Administration. (n.d.). Approved drug products with therapeutic equivalence evaluations, Orange Book. https://www.accessdata.fda.gov/scripts/cder/ob/

3. U.S. Food and Drug Administration. (2014). FDA approves Cerdelga for Gaucher disease type 1. https://www.fda.gov/news-events/press-announcements/fda-approves-cerdelga-gaucher-disease-type-1

4. U.S. Patent and Trademark Office. (2021). U.S. Patent No. 10,888,547, methods of treating Gaucher disease. https://patents.google.com/patent/US10888547B2/en

5. U.S. Food and Drug Administration. (n.d.). Electronic Orange Book patent and exclusivity information. https://www.fda.gov/drugs/drug-approvals-and-databases/orange-book-data-files-drugsfda-data-files.

Drugs Protected by US Patent 10,888,547

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Patented / Exclusive Use	Submissiondate
Genzyme Corp	CERDELGA	eliglustat tartrate	CAPSULE;ORAL	205494-001	Aug 19, 2014	AB	RX	Yes	Yes	10,888,547					LONG-TERM TREATMENT OF ADULTS WITH GAUCHER DISEASE TYPE 1 WHO ARE CYP2D6 POOR METABOLIZERS WITH 84 MG ONCE DAILY OF ELIGLUSTAT (EQUIVALENT TO 100 MG OF ELIGLUSTAT TARTRATE)
Genzyme Corp	CERDELGA	eliglustat tartrate	CAPSULE;ORAL	205494-001	Aug 19, 2014	AB	RX	Yes	Yes	10,888,547					LONG-TERM TREATMENT OF ADULTS WITH GAUCHER DISEASE TYPE 1 WHO ARE CYP2D6 EXTENSIVE OR INTERMEDIATE METABOLIZERS WITH 84 MG TWICE PER DAY OF ELIGLUSTAT (EQUIVALENT TO 100 MG OF ELIGLUSTAT TARTRATE TWICE PER DAY)

International Family Members for US Patent 10,888,547

Country	Patent Number	Estimated Expiration	Supplementary Protection Certificate	SPC Country	SPC Expiration
Argentina	079152
Argentina	121611
Argentina	121612
Australia	2010324810
Australia	2016202591