Opfolda

(Miglustat)

Phase 3Small molecule

Opfolda is a small molecule that works as a Glucosylceramide Synthase Inhibitor.[2] It is being investigated across 1 indication, with its most advanced program in Phase 3.[1]

Opfolda is one of 39 small molecule programs targeting Immunogenicity. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

FDA Applications

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[3][1]

Indication

ImmunogenicityRecruiting

ImmunogenicityPhase 3

Recruiting

Competition

Competitive Landscape

87 competitors in Immunogenicity

#DrugCompanyPhaseTrials

Perjeta

Roche

Phase 3

2 2

Rabies Vaccine

Novartis

Phase 3

12 3

Prolia

OOrganon and Co

Phase 3

2 4

HPV Vaccine

SShanghai Zerun Biotechnology Co.,Ltd

Phase 3

3 5

Gardasil-9

BBeijing Health Guard Biotechnology, Inc

Phase 3

2 6

Rsvpref

Pfizer

Phase 3

5 7

Bexsero

GSK

Phase 3

8 8

Gardasil

Merck & Co.

Phase 3

7 9

PCV21

Sanofi

Phase 3

1 10

Pentavalent Vaccine

GSK

Phase 3

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q3 2027

Phase 3Recruiting

Immunogenicity

NCT04808505

Phase 3Active, not recruiting

Pompe Disease (late-Onset)

NCT03911505

Q4 2034

Recruiting

Pompe Disease

NCT06121011

Clinical

Trials by Indication[1]

Pompe Disease3 trials

A Global Prospective Observational Registry of Patients With Pompe Disease

Recruiting·AAmicus Therapeutics··NCT06121011·500 enrolled

Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies

Completed·UUniversity of Florida··NCT01451879·11 enrolled

A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease

Completed·AAmicus Therapeutics·Phase 3·NCT03729362·125 enrolled

Cystic Fibrosis3 trials

Does a Nasal Instillation of Miglustat Normalize the Nasal Potential Difference in Cystic Fibrosis Patients ?

Completed·CCliniques universitaires Saint-Luc- Université Catholique de Louvain·Phase 2·NCT00945347·10 enrolled

Miglustat in Cystic Fibrosis

Completed· Johnson & Johnson logo

Johnson & Johnson·Phase 2·NCT00742092·11 enrolled

Miglustat / OGT 918 in the Treatment of Cystic Fibrosis

Terminated· Johnson & Johnson logo

Johnson & Johnson·Phase 2·NCT00537602·6 enrolled

Gaucher Disease2 trials

Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease

Completed· Johnson & Johnson logo

Johnson & Johnson·Phase 3·NCT00319046·42 enrolled

Miglustat on Gaucher Disease Type IIIB

Unknown status·NNational Taiwan University Hospital·N/A·NCT02520934·19 enrolled

Spastic paraplegia1 trial

Testing Miglustat Administration in Subjects With Spastic Paraplegia 11

Completed·IIRCCS Fondazione Stella Maris·Phase 2·NCT04768166·10 enrolled

Safety and Efficacy1 trial

Safety and Efficacy of Miglustat in Chinese NPC Patients

Completed· Johnson & Johnson logo

Johnson & Johnson·Phase 4·NCT03910621·17 enrolled

7 more indications

GM2 gangliosidosis1 trial

Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis

Completed·TThe Hospital for Sick Children·Phase 2·NCT00418847·5 enrolled

Pilot Study1 trial

Glycosphingolipid Inhibition and Spermatogenesis in Man: A Pilot Study (MIG 2)

Completed·UUniversity of Washington·Phase 4·NCT00194649·8 enrolled

Niemann-Pick Type C Disease1 trial

Miglustat in Niemann-Pick Type C Disease

Completed· Johnson & Johnson logo

Johnson & Johnson·Phase 2·NCT00517153·29 enrolled

Immunogenicity1 trial

A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18

Recruiting·AAmicus Therapeutics·Phase 3·NCT04808505·36 enrolled

Treatment1 trial

Synergistic Enteral Regimen for Treatment of the Gangliosidoses

Terminated·UUniversity of Minnesota·Phase 4·NCT02030015·16 enrolled

Pompe Disease (Late-onset)1 trial

An Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Pediatric Subjects Aged 0 to < 18 Years With Late-onset Pompe Disease

Active, not recruiting·AAmicus Therapeutics·Phase 3·NCT03911505·21 enrolled

Unspecified2 trials

Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD)

Terminated·TTehran University of Medical Sciences·Phase 3·NCT03822013·30 enrolled

Application of Miglustat in Patients With Niemann-Pick Type C

Completed·NNational Taiwan University Hospital·Phase 3·NCT01760564·5 enrolled

Regulatory

FDA Approval Timeline[4]

Jul 31, 2003

ZAVESCA

NDA NDA021348

Sep 28, 2023

OPFOLDA

NDA NDA215211

Sep 28, 2023

OPFOLDA

NDA NDA215211

Jul 25, 2024

OPFOLDA

NDA NDA215211

Sources

References

[1]
ClinicalTrials.gov — 18 trial records for Miglustat. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Miglustat compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Miglustat. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (NDA NDA215211). U.S. Food & Drug Administration.
[5]
FDA Orange Book — Patent and exclusivity data for Opfolda. U.S. Food & Drug Administration.
[6]
SEC EDGAR — Johnson & Johnson company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: Glucosylceramide Synthase Inhibitor[2]
Modality: Small molecule[2]
Highest Phase: Phase 3[1]
Company: Johnson & Johnson

Patent Landscape[5]

Patent Cliff

Mar 7, 2033

Earliest patent expiration — generic entry possible after this date

Patent #Expires

12419937Mar 7, 2033

11278599Mar 7, 2033

10512677Mar 7, 2033

10961522Sep 30, 2035

10208299Sep 30, 2035

5 more patents

11753632Sep 30, 2035

11278601Dec 29, 2036

12414985Dec 29, 2036

10857212Aug 12, 2037

12246062Sep 16, 2038

Opfolda

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q3 2027

Q4 2034

Trials by Indication[1]

Pompe Disease3 trials

Cystic Fibrosis3 trials

Gaucher Disease2 trials

Spastic paraplegia1 trial

Safety and Efficacy1 trial

GM2 gangliosidosis1 trial

Pilot Study1 trial

Niemann-Pick Type C Disease1 trial

Immunogenicity1 trial

Treatment1 trial

Pompe Disease (Late-onset)1 trial

Unspecified2 trials

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

Patent Landscape[5]

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