MIGALASTAT HYDROCHLORIDE - Generic Drug Details

What are the generic drug sources for migalastat hydrochloride and what is the scope of freedom to operate?

Migalastat hydrochloride is the generic ingredient in one branded drug marketed by Amicus Therap Us and is included in one NDA. There are sixty-two patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Migalastat hydrochloride has two hundred and forty-two patent family members in thirty countries.

One supplier is listed for this compound.

Summary for MIGALASTAT HYDROCHLORIDE

International Patents:	242
US Patents:	62
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	41
Clinical Trials:	20
Patent Applications:	163
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for MIGALASTAT HYDROCHLORIDE
What excipients (inactive ingredients) are in MIGALASTAT HYDROCHLORIDE?	MIGALASTAT HYDROCHLORIDE excipients list
DailyMed Link:	MIGALASTAT HYDROCHLORIDE at DailyMed

DrugPatentWatch^® Estimated Loss of Exclusivity (LOE) Date for MIGALASTAT HYDROCHLORIDE

Constraining patent/regulatory exclusivity:

INDICATED FOR THE TREATMENT OF ADULTS WITH A CONFIRMED DIAGNOSIS OF FABRY DISEASE AND AN AMENABLE GALACTOSIDASE ALPHA GENE (GLA) VARIANT BASED ON IN VITRO ASSAY DATA

Dosage:

CAPSULE;ORAL

^*The generic entry opportunity date is the latter of the last compound-claiming patent and the last regulatory exclusivity protection. Many factors can influence early or later generic entry. This date is provided as a rough estimate of generic entry potential and should not be used as an independent source.

Recent Clinical Trials for MIGALASTAT HYDROCHLORIDE

Identify potential brand extensions & 505(b)(2) entrants

Sponsor	Phase
Genzyme, a Sanofi Company	Phase 3
Amicus Therapeutics	Phase 1
Amicus Therapeutics	Phase 3

See all MIGALASTAT HYDROCHLORIDE clinical trials

Anatomical Therapeutic Chemical (ATC) Classes for MIGALASTAT HYDROCHLORIDE

A16AX	Various alimentary tract and metabolism products
A16A	OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
A16	OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
A	Alimentary tract and metabolism

Paragraph IV (Patent) Challenges for MIGALASTAT HYDROCHLORIDE

Tradename	Dosage	Ingredient	Strength	NDA	ANDAs Submitted	Submissiondate
GALAFOLD	Capsules	migalastat hydrochloride	123 mg	208623	3	2022-08-10

US Patents and Regulatory Information for MIGALASTAT HYDROCHLORIDE

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Amicus Therap Us	GALAFOLD	migalastat hydrochloride	CAPSULE;ORAL	208623-001	Aug 10, 2018		RX	Yes	Yes
Amicus Therap Us	GALAFOLD	migalastat hydrochloride	CAPSULE;ORAL	208623-001	Aug 10, 2018		RX	Yes	Yes
Amicus Therap Us	GALAFOLD	migalastat hydrochloride	CAPSULE;ORAL	208623-001	Aug 10, 2018		RX	Yes	Yes
Amicus Therap Us	GALAFOLD	migalastat hydrochloride	CAPSULE;ORAL	208623-001	Aug 10, 2018		RX	Yes	Yes
Amicus Therap Us	GALAFOLD	migalastat hydrochloride	CAPSULE;ORAL	208623-001	Aug 10, 2018		RX	Yes	Yes
Amicus Therap Us	GALAFOLD	migalastat hydrochloride	CAPSULE;ORAL	208623-001	Aug 10, 2018		RX	Yes	Yes
Amicus Therap Us	GALAFOLD	migalastat hydrochloride	CAPSULE;ORAL	208623-001	Aug 10, 2018		RX	Yes	Yes

International Patents for MIGALASTAT HYDROCHLORIDE

Country	Patent Number	Title	Estimated Expiration
Canada	2715407	METHODE DE PREDICTION DE LA REPONSE DE CERTAINES MALADIES A UN TRAITEMENT PHARMACOLOGIQUE A BASE DE CHAPERON MOLECULAIRE (METHOD TO PREDICT RESPONSE TO PHARMACOLOGICAL CHAPERONE TREATMENT OF DISEASES)
Japan	2021024864	ＧＬＡ遺伝子に突然変異を有する患者のファブリー病を治療する方法 (METHOD OF TREATING FABRY DISEASE IN PATIENT HAVING A MUTATION IN THE GLA GENE)
Japan	2021097673	疾病の薬理シャペロン治療に対する応答性を予測する方法 (METHODS TO PREDICT RESPONSE TO PHARMACOLOGICAL CHAPERONE TREATMENT OF DISEASES)
South Korea	20220044560	GLA 유전자에 돌연변이를 갖는 환자에서 파브리 질병을 치료하는 방법
Israel	290365	שיטות לטיפול במחלת פברי בחולים בעלי מוטציה בגן gla (Methods of treating fabry disease in patients having a mutation in the gla gene)
China	117357524	治疗具有肾损害的法布里患者的方法 (Method of treating Fabry patients with renal injury)
Taiwan	201806597	Treatment of Fabry disease in ERT-naive and ERT-experienced patients

Supplementary Protection Certificates for MIGALASTAT HYDROCHLORIDE

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
2787345	CR 2016 00055	Denmark		PRODUCT NAME: MIGALASTAT ELLER ET SALT HERAF, HERUNDER HYDROGENKLORIDSALTET; REG. NO/DATE: EU/1/15/1082 20160531
2787345	2016/050	Ireland		PRODUCT NAME: MIGALASTAT OR A SALT THEREOF, INCLUDING THE HYDROCHLORIDE SALT; REGISTRATION NO/DATE: EU/1/15/1082 20160526
2787345	PA2016033,C2787345	Lithuania		PRODUCT NAME: MIGALASTATAS ARBA JO DRUSKA, ISKAITANT IR HIDROCHLORIDO DRUSKA; REGISTRATION NO/DATE: EU/1/15/1082 20160526
2787345	93312	Luxembourg		PRODUCT NAME: MIGALASTAT OU UN SEL DE CELUI-CI , Y COMPRIS LE SEL DE CHLORHYDRATE; AUTHORISATION NUMBER AND DATE: EU/1/15/1082 - GALAFOLD - MIGALASTAT
2787345	C 2016 042	Romania		PRODUCT NAME: MIGALASTAT SAU O SARE A ACESTUIA, INCLUSIV SAREACLORHIDRAT; NATIONAL AUTHORISATION NUMBER: EU/1/15/1082; DATE OF NATIONAL AUTHORISATION: 20160526; NUMBER OF FIRST AUTHORISATION IN EUROPEAN ECONOMIC AREA (EEA): EU/1/15/1082; DATE OF FIRST AUTHORISATION IN EEA: 20160526
2787345	16C1014	France		PRODUCT NAME: MIGALASTAT OU UN SEL DE CELUI-CI,NOTAMENT LE CHLORHYDRATE; REGISTRATION NO/DATE: EU1/15/1082 20160531
2787345	C02787345/01	Switzerland		PRODUCT NAME: MIGALASTAT; REGISTRATION NO/DATE: SWISSMEDIC-ZULASSUNG 66108 28.10.2016

MIGALASTAT HYDROCHLORIDE Market Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory for Migalastat Hydrochloride (Galafold)

Introduction

Migalastat hydrochloride, marketed under the brand name Galafold by Amicus Therapeutics, is an oral pharmacological chaperone used for the treatment of Fabry disease in adults with amenable galactosidase alpha (GLA) gene variants. Here, we delve into the market dynamics and financial trajectory of this drug.

Market Need and Patient Population

Fabry disease is a rare genetic disorder characterized by the accumulation of glycosphingolipids due to a deficiency of the alpha-Gal A enzyme. Approximately 35 to 50 percent of Fabry patients have amenable GLA variants that can be treated with migalastat, making it a significant treatment option for this patient population[4].

Regulatory Approvals and Market Access

Galafold has received regulatory approvals from major health authorities, including the FDA, EMA, and MHRA. These approvals have facilitated its global market access, enabling the treatment to reach a broader patient base[3][4].

Revenue Growth and Financial Performance

Historical Revenue

Galafold has consistently demonstrated strong revenue growth since its introduction. In 2021, global revenue for Galafold reached $306 million, representing a 17% year-over-year increase. This growth continued in 2022, with revenue guidance set between $350 million and $365 million, reflecting a 15-20% increase[3].

In 2023, the revenue for Galafold surged to $387.8 million, marking an 18% year-over-year increase, or 17% at constant exchange rates (CER). This robust growth is attributed to strong new patient accruals, sustained patient adherence, and geographic expansion[2].

Projected Growth

For 2024, Amicus Therapeutics expects double-digit revenue growth for Galafold, with a projected increase of 11-16% at CER. This growth is anticipated to be driven by continued demand from both switch and naïve patients, as well as further geographic expansion[2].

Competitive Landscape

Galafold competes with enzyme replacement therapies (ERTs) such as agalsidase alfa and agalsidase beta. However, its oral administration and potential cost-effectiveness make it an attractive alternative. In pharmacoeconomic analyses, migalastat has been shown to be dominant over ERT in certain scenarios, particularly when considering the disutility associated with infusion-based treatments[1].

Cost-Effectiveness and Pharmacoeconomic Analysis

The cost-utility analysis of migalastat versus ERT has shown varied results depending on several factors, including patient weight, discontinuation rates, and the price of ERT. In some scenarios, migalastat is associated with significant cost savings and improved quality-adjusted life years (QALYs). For instance, in the base case analysis, migalastat was found to be dominant, offering cost savings of $350,953 and a gain of 1.01 QALYs compared to ERT[1].

Patient Adherence and Treatment Outcomes

High patient adherence to Galafold has been a key factor in its success. The treatment's oral administration and relatively low adverse event profile contribute to its high adherence rates. This, in turn, has led to improved treatment outcomes and sustained revenue growth[2][4].

Global Launches and Market Expansion

Amicus Therapeutics has successfully launched Galafold in several major markets, including the U.S., EU, U.K., and Japan. The company continues to expand its geographic reach, which is expected to further drive revenue growth. As of 2023, over 2,400 people living with Fabry disease are on Galafold, highlighting the drug's growing market penetration[2].

Strategic Priorities and Future Outlook

For 2024, Amicus Therapeutics is focused on several key strategic priorities:

• Delivering double-digit Galafold revenue growth
• Ensuring the successful global launches of other therapies like Pombiliti + Opfolda
• Advancing ongoing studies to support medical and scientific leadership in Fabry and Pompe diseases
• Achieving full-year non-GAAP profitability[2].

Intellectual Property and Patent Protection

The company has strengthened its intellectual property portfolio with newly issued U.S. composition of matter patents for Galafold, which adds to its growing patent protection. This ensures long-term market exclusivity and protection against generic competition[5].

Conclusion

Migalastat hydrochloride, under the brand name Galafold, has established a strong market presence in the treatment of Fabry disease. Its financial trajectory is marked by consistent double-digit revenue growth, driven by high patient adherence, geographic expansion, and favorable pharmacoeconomic profiles. As Amicus Therapeutics continues to execute its strategic priorities, Galafold is poised for further growth and market dominance.

Key Takeaways

• Strong Revenue Growth: Galafold has consistently shown double-digit revenue growth.
• Global Market Access: Regulatory approvals in major markets have facilitated global reach.
• Cost-Effectiveness: Migalastat is often more cost-effective than ERT in certain scenarios.
• High Patient Adherence: Oral administration and low adverse event profile contribute to high adherence.
• Geographic Expansion: Continued expansion into new markets is expected to drive further growth.
• Intellectual Property Protection: Newly issued patents ensure long-term market exclusivity.

FAQs

Q: What is the primary use of migalastat hydrochloride? A: Migalastat hydrochloride, marketed as Galafold, is used for the treatment of adults with a confirmed diagnosis of Fabry disease and amenable galactosidase alpha (GLA) gene variants.

Q: How has the revenue for Galafold performed in recent years? A: Galafold has shown strong revenue growth, with a 17% increase in 2021 to $306 million, and an 18% increase in 2023 to $387.8 million.

Q: What are the competitive advantages of Galafold over enzyme replacement therapies (ERTs)? A: Galafold offers the advantage of oral administration and potential cost-effectiveness, particularly when considering the disutility associated with infusion-based ERTs.

Q: What are the key strategic priorities for Amicus Therapeutics in 2024? A: The priorities include delivering double-digit Galafold revenue growth, ensuring successful global launches of other therapies, advancing ongoing studies, and achieving full-year non-GAAP profitability.

Q: How does the intellectual property landscape support Galafold’s market position? A: Newly issued U.S. composition of matter patents for Galafold add to its growing patent portfolio, ensuring long-term market exclusivity and protection against generic competition.

Sources

1. CADTH Common Drug Review Pharmacoeconomic Review Report for Galafold
2. Amicus Therapeutics Reports Preliminary 2023 Revenue and Provides 2024 Strategic Outlook
3. Amicus Therapeutics Reports Preliminary 2021 Revenue and Provides 2022 Strategic Outlook
4. DrugBank Online - Migalastat: Uses, Interactions, Mechanism of Action
5. Amicus Therapeutics Announces Second Quarter 2022 Financial Results