Genetic Disorders Therapeutics
1. Galafold patent expiration
Treatment: The treatment of fabry patients; A method of reducing left ventricular mass index (lvmi) in a fabry patient by administering migalastat
GALAFOLD's oppositions filed in EPO
| Drug Patent Number | Company | Drug Patent Title | Drug Patent Expiry | Activity Alert |
|---|---|---|---|---|
| These drug patents protects the active chemical substance. Only drug patent owner can launch products that use this active substance. | ||||
| US12280042 | AMICUS | Methods of treating fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11903938 | AMICUS | Methods of treating Fabry patients having renal impairment |
Aug, 2038
(12 years from now) | |
| US12042489 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11304940 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US12109205 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11612594 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US12042488 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11622962 | AMICUS | Methods of treating Fabry patients having renal impairment |
Mar, 2039
(13 years from now) | |
| US11357764 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11376244 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11612593 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11357765 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11426396 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11633387 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11826360 | AMICUS | Methods of treating Fabry patients having renal impairment |
Feb, 2039
(12 years from now) | |
| US11786516 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| These drug patents focus on the other aspects of the active substance like dosage, mode of administration (oral, tablet, capsules, liquids etc). | ||||
| US9000011 | AMICUS | Methods for treatment of Fabry disease |
May, 2027
(1 year, 2 months from now) | |
| US10383864 | AMICUS | Methods for treatment of Fabry disease |
May, 2027
(1 year, 2 months from now) | |
| US10471053 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US10076514 | AMICUS | Methods of treating fabry disease in patients having the G9331A mutation in the GLA gene |
Mar, 2037
(11 years from now) | |
| US8592362 | AMICUS | Method to predict response to pharmacological chaperone treatment of diseases |
Feb, 2029
(2 years from now) | |
| US11642334 | AMICUS | Methods of treating Fabry patients having renal impairment |
Feb, 2039
(13 years from now) | |
| US9095584 | AMICUS | Method to predict response to pharmacological chaperone treatment of diseases |
Feb, 2029
(2 years from now) | |
| US9999618 | AMICUS | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
Apr, 2028
(2 years from now) | |
| US10813921 | AMICUS | Method to predict response to pharmacological chaperone treatment of diseases |
Feb, 2029
(2 years from now) | |
| US10857141 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US10849890 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| USRE48608 | AMICUS | Method to predict response to pharmacological chaperone treatment of diseases |
Oct, 2031
(5 years from now) | |
| US10799491 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US10806727 | AMICUS | Methods of treating fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11458128 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US10406143 | AMICUS | Methods for treatment of fabry disease |
May, 2027
(1 year, 2 months from now) | |
| US10525045 | AMICUS | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
Apr, 2028
(2 years from now) | |
| US9987263 | AMICUS | Methods for treatment of Fabry disease |
May, 2027
(1 year, 2 months from now) | |
| US9480682 | AMICUS | Methods for treatment of Fabry disease |
May, 2027
(1 year, 2 months from now) | |
| US11234972 | AMICUS | Methods of treating fabry disease in patients having the G9331A mutation in the GLA gene |
Mar, 2037
(11 years from now) | |
| US11278538 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11278537 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11278536 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11278539 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11278540 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US10925866 | AMICUS | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
Apr, 2028
(2 years from now) | |
| US10251873 | AMICUS | Methods of treating fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11633388 | AMICUS | Methods of treating Fabry patients having renal impairment |
Mar, 2039
(13 years from now) | |
| US11033538 | AMICUS | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
Apr, 2028
(2 years from now) | |
| US12042490 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11357761 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11357762 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11357763 | AMICUS | Methods of treating fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11357784 | AMICUS | Use of migalastat for treating Fabry disease in pregnant patients |
Feb, 2039
(12 years from now) | |
| US11666564 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US10849889 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US10857142 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11389437 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11389436 | AMICUS | Methods of treating fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US10874657 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US10874655 | AMICUS | Methods of treating fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US10874656 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US10792278 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US10792279 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11833164 | AMICUS | Methods of treating Fabry disease in patients having a mutation in the GLA gene |
Jan, 2042
(15 years from now) | |
| US11813255 | AMICUS | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11241422 | AMICUS | Methods for treatment of Fabry disease |
May, 2027
(1 year, 2 months from now) | |
| Drug Exclusivity | Drug Exclusivity Expiration |
|---|---|
| New Chemical Entity Exclusivity(NCE) | Aug 10, 2023 |
| Orphan Drug Exclusivity(ODE-205) | Aug 10, 2025 |
Drugs and Companies using MIGALASTAT HYDROCHLORIDE ingredient
NCE-1 date: 10 August, 2022
Market Authorisation Date: 10 August, 2018
Dosage: CAPSULE
How can I launch a generic of GALAFOLD before it's drug patent expiration?
More Information on Dosage
GALAFOLD family patents
2. Givlaari patent expiration
Treatment: Treatment of acute hepatic porphyria
GIVLAARI's oppositions filed in EPO
GIVLAARI IPR and PTAB Proceedings
| Drug Patent Number | Company | Drug Patent Title | Drug Patent Expiry | Activity Alert |
|---|---|---|---|---|
| These drug patents protects the active chemical substance. Only drug patent owner can launch products that use this active substance. | ||||
| US9708610 | ALNYLAM | Compositions comprising alternating 2′-modified nucleosides for use in gene modulation |
Jan, 2024
(2 years ago) | |
| US8546143 | ALNYLAM | Compositions and methods for inhibiting expression of a target gene |
Jan, 2022
(4 years ago) | |
| US10131907 | ALNYLAM | Glycoconjugates of RNA interference agents |
Aug, 2028
(2 years from now) | |
| US8106022 | ALNYLAM | Carbohydrate conjugates as delivery agents for oligonucleotides |
Dec, 2029
(3 years from now) | |
| US10119143 | ALNYLAM | Compositions and methods for inhibiting expression of the ALAS1 gene |
Oct, 2034
(8 years from now) | |
| US8828956 | ALNYLAM | Carbohydrate conjugates as delivery agents for oligonucleotides |
Dec, 2028
(2 years from now) | |
| US11028392 | ALNYLAM | Compositions and methods for inhibiting expression of the ALAS1 gene |
Oct, 2034
(8 years from now) | |
| US10125364 | ALNYLAM | Compositions and methods for inhibiting expression of the ALAS1 gene |
Mar, 2033
(7 years from now) | |
| US11530408 | ALNYLAM | Therapeutic compositions |
May, 2024
(1 year, 9 months ago) | |
| US9708615 | ALNYLAM | Therapeutic compositions |
Mar, 2024
(1 year, 11 months ago) | |
| US10273477 | ALNYLAM | Therapeutic compositions |
Mar, 2024
(1 year, 11 months ago) | |
| US9133461 | ALNYLAM | Compositions and methods for inhibiting expression of the ALAS1 gene |
Nov, 2033
(7 years from now) | |
| US9150605 | ALNYLAM | Compositions comprising alternating 2′-modified nucleosides for use in gene modulation |
Aug, 2025
(5 months ago) | |
| These drug patents focus on the other aspects of the active substance like dosage, mode of administration (oral, tablet, capsules, liquids etc). | ||||
| US9631193 | ALNYLAM | Compositions and methods for inhibiting expression of the ALAS1 gene |
Mar, 2033
(7 years from now) | |
| Drug Exclusivity | Drug Exclusivity Expiration |
|---|---|
| New Chemical Entity Exclusivity(NCE) | Nov 20, 2024 |
| Orphan Drug Exclusivity(ODE-273) | Nov 20, 2026 |
Drugs and Companies using GIVOSIRAN SODIUM ingredient
NCE-1 date: 21 November, 2023
Market Authorisation Date: 20 November, 2019
Dosage: SOLUTION
More Information on Dosage
GIVLAARI family patents
3. Miplyffa patent
expiration
Treatment: Use of arimoclomol, in combination with miglustat, for treatment of neurological manifestations of niemann-pick disease type c (npc)
| Drug Patent Number | Company | Drug Patent Title | Drug Patent Expiry | Activity Alert |
|---|---|---|---|---|
| These drug patents focus on the other aspects of the active substance like dosage, mode of administration (oral, tablet, capsules, liquids etc). | ||||
| US11045460 | ZEVRA | Use of Hsp70 as a regulator of enzymatic activity |
Aug, 2029
(3 years from now) | |
| US9884058 | ZEVRA | Use of Hsp70 as a regulator of enzymatic activity |
Jun, 2029
(3 years from now) | |
| US9289472 | ZEVRA | Use of HSP70 as a regulator of enzymatic activity |
Aug, 2029
(3 years from now) | |
| Drug Exclusivity | Drug Exclusivity Expiration |
|---|---|
| New Chemical Entity Exclusivity(NCE) | Sep 20, 2029 |
| Orphan Drug Exclusivity(ODE-496) | Sep 20, 2031 |
Drugs and Companies using ARIMOCLOMOL CITRATE ingredient
NCE-1 date: 20 September, 2028
Market Authorisation Date: 20 September, 2024
Dosage: CAPSULE
More Information on Dosage
MIPLYFFA family patents
4. Nulibry patent expiration
Treatment: Method of treating molybdenum cofactor deficiency type a
| Drug Patent Number | Company | Drug Patent Title | Drug Patent Expiry | Activity Alert |
|---|---|---|---|---|
| These drug patents focus on the other aspects of the active substance like dosage, mode of administration (oral, tablet, capsules, liquids etc). | ||||
| US7504095 | SENTYNL | Method for obtaining precursor Z and use thereof for the production of a means for therapy of human molybdenum cofactor deficiency |
Apr, 2029
(3 years from now) | |
| Drug Exclusivity | Drug Exclusivity Expiration |
|---|---|
| M(M-286) | Oct 27, 2025 |
| New Chemical Entity Exclusivity(NCE) | Feb 26, 2026 |
| Orphan Drug Exclusivity(ODE-342) | Feb 26, 2028 |
Drugs and Companies using FOSDENOPTERIN HYDROBROMIDE ingredient
NCE-1 date: 26 February, 2025
Market Authorisation Date: 26 February, 2021
Dosage: POWDER
More Information on Dosage
NULIBRY family patents
5. Orfadin patent expiration
Treatment: Treatment of hereditary tyrosinemia type 1 (ht-1) in combination with dietary restriction of tyrosine and phenylalanine
| Drug Patent Number | Company | Drug Patent Title | Drug Patent Expiry | Activity Alert |
|---|---|---|---|---|
| These drug patents focus on the other aspects of the active substance like dosage, mode of administration (oral, tablet, capsules, liquids etc). | ||||
| US5550165 | SWEDISH ORPHAN | Pharmaceutical compositions for the treatment of hereditary tyosinemia type I |
Aug, 2013
(12 years ago) | |
| US9301932 | SWEDISH ORPHAN | Liquid pharmaceutical composition comprising nitisinone |
Feb, 2033
(7 years from now) | |
| Drug Exclusivity | Drug Exclusivity Expiration |
|---|---|
| New Dosing Schedule(D-169) | Sep 01, 2020 |
Drugs and Companies using NITISINONE ingredient
Market Authorisation Date: 18 January, 2002
Dosage: CAPSULE; SUSPENSION
More Information on Dosage
ORFADIN family patents
6. Oxlumo patent expiration
Treatment: Treatment of primary hyperoxaluria type 1 (ph1)
| Drug Patent Number | Company | Drug Patent Title | Drug Patent Expiry | Activity Alert |
|---|---|---|---|---|
| These drug patents protects the active chemical substance. Only drug patent owner can launch products that use this active substance. | ||||
| US11401517 | ALNYLAM | Modified double-stranded RNA agents |
Aug, 2035
(9 years from now) | |
| US11060093 | ALNYLAM | Methods and compositions for the specific inhibition of glycolate oxidase (HAO1) by double-stranded RNA |
Dec, 2034
(8 years from now) | |
| US10487330 | ALNYLAM | Methods and compositions for the specific inhibition of glycolate oxidase (HAO1) by double-stranded RNA |
Dec, 2034
(8 years from now) | |
| US11261447 | ALNYLAM | Methods for inhibition of HAO1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression |
Nov, 2038
(12 years from now) | |
| US9828606 | ALNYLAM | Methods and compositions for the specific inhibition of glycolate oxidase (HAO1) by double-stranded RNA |
Dec, 2034
(8 years from now) | |
| US10478500 | ALNYLAM | Compositions and methods for inhibition of HAO1 (Hydroxyacid Oxidase 1 (Glycolate Oxidase)) gene expression |
Oct, 2035
(9 years from now) | |
| US8106022 | ALNYLAM | Carbohydrate conjugates as delivery agents for oligonucleotides |
Dec, 2029
(3 years from now) | |
| US8828956 | ALNYLAM | Carbohydrate conjugates as delivery agents for oligonucleotides |
Dec, 2028
(2 years from now) | |
| US10131907 | ALNYLAM | Glycoconjugates of RNA interference agents |
Aug, 2028
(2 years from now) | |
| US10612024 | ALNYLAM | Modified double-stranded RNA agents |
Aug, 2035
(9 years from now) | |
| US10612027 | ALNYLAM | Modified double-stranded RNA agents |
Aug, 2035
(9 years from now) | |
| US10465195 | ALNYLAM | Methods and compositions for the specific inhibition of glycolate oxidase (HAO1) by double-stranded RNA |
Dec, 2034
(8 years from now) | |
| US11446380 | ALNYLAM | Compositions and methods for inhibition of HAO1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression |
Oct, 2035
(9 years from now) | |
| These drug patents focus on the other aspects of the active substance like dosage, mode of administration (oral, tablet, capsules, liquids etc). | ||||
| US10435692 | ALNYLAM | Methods and compositions for the specific inhibition of glycolate oxidase (HAO1) by double-stranded RNA |
Dec, 2034
(8 years from now) | |
| Drug Exclusivity | Drug Exclusivity Expiration |
|---|---|
| New Indication(I-901) | Oct 06, 2025 |
| New Chemical Entity Exclusivity(NCE) | Nov 23, 2025 |
| Orphan Drug Exclusivity(ODE-339) | Nov 23, 2027 |
| Orphan Drug Exclusivity(ODE-415) | Oct 06, 2029 |
Drugs and Companies using LUMASIRAN SODIUM ingredient
NCE-1 date: 23 November, 2024
Market Authorisation Date: 23 November, 2020
Dosage: SOLUTION
More Information on Dosage
OXLUMO family patents
7. Voxzogo patent expiration
Treatment: Use to increase linear growth in pediatric patients with achondroplasia with open epiphyses
VOXZOGO's oppositions filed in EPO
VOXZOGO IPR and PTAB Proceedings
| Drug Patent Number | Company | Drug Patent Title | Drug Patent Expiry | Activity Alert |
|---|---|---|---|---|
| These drug patents protects the active chemical substance. Only drug patent owner can launch products that use this active substance. | ||||
| US8198242 | BIOMARIN | Variants of C-type natriuretic peptide |
Jun, 2030
(4 years from now) | |
| These drug patents focus on the other aspects of the active substance like dosage, mode of administration (oral, tablet, capsules, liquids etc). | ||||
| US12233106 | BIOMARIN | C-type natriuretic peptide variants to treat skeletal dysplasia in children |
Jul, 2042
(16 years from now) | |
| US12514906 | BIOMARIN | Use Of C-Type Natriuretic Peptide Variants To Treat Skeletal Dysplasia |
Aug, 2036
(10 years from now) | |
| US10646550 | BIOMARIN | Use of C-type natriuretic peptide variants to treat skeletal dysplasia |
Aug, 2036
(10 years from now) | |
| US11911446 | BIOMARIN | Use of C-type natriuretic peptide variants to treat skeletal dysplasia |
Aug, 2036
(10 years from now) | |
| US11590204 | BIOMARIN | Use of C-type natriuretic peptide variants to treat skeletal dysplasia |
Aug, 2036
(10 years from now) | |
| US9907834 | BIOMARIN | Use of C-type natriuretic peptide variants to treat skeletal dysplasia |
Aug, 2036
(10 years from now) | |
| USRE48267 | BIOMARIN | Variants of C-type natriuretic peptide |
May, 2030
(4 years from now) | |
| Drug Exclusivity | Drug Exclusivity Expiration |
|---|---|
| New Patient Population(NPP) | Oct 20, 2026 |
| New Chemical Entity Exclusivity(NCE) | Nov 19, 2026 |
| Orphan Drug Exclusivity(ODE-387) | Nov 19, 2028 |
| Orphan Drug Exclusivity(ODE-449) | Oct 20, 2030 |
Drugs and Companies using VOSORITIDE ingredient
NCE-1 date: 19 November, 2025
Market Authorisation Date: 19 November, 2021
Dosage: POWDER
How can I launch a generic of VOXZOGO before it's drug patent expiration?
More Information on Dosage
VOXZOGO family patents
8. Zavesca patent expiration
Treatment: NA
| Drug Patent Number | Company | Drug Patent Title | Drug Patent Expiry | Activity Alert |
|---|---|---|---|---|
| These drug patents focus on the other aspects of the active substance like dosage, mode of administration (oral, tablet, capsules, liquids etc). | ||||
| US5525616 | ACTELION | Method of inhibiting glycolipid synthesis |
Jun, 2013
(12 years ago) | |
| US5472969 | ACTELION | Method of inhibiting glycolipid synthesis |
May, 2013
(12 years ago) | |
Drugs and Companies using MIGLUSTAT ingredient
Market Authorisation Date: 31 July, 2003
Dosage: CAPSULE
More Information on Dosage
ZAVESCA family patents
9. Zokinvy patent expiration
Treatment: Reducing the risk of mortality in hutchinson-gilford progeria syndrome (hgps)
| Drug Patent Number | Company | Drug Patent Title | Drug Patent Expiry | Activity Alert |
|---|---|---|---|---|
| These drug patents focus on the other aspects of the active substance like dosage, mode of administration (oral, tablet, capsules, liquids etc). | ||||
| US8828356 | SENTYNL | Farnesyltransferase inhibitors for treatment of laminopathies, cellular aging and atherosclerosis |
Oct, 2025
(4 months ago) | |
| US7838531 | SENTYNL | Farnesyltransferase inhibitors for treatment of laminopathies, cellular aging and atherosclerosis |
Jul, 2029
(3 years from now) | |
| Drug Exclusivity | Drug Exclusivity Expiration |
|---|---|
| New Chemical Entity Exclusivity(NCE) | Nov 20, 2025 |
| Orphan Drug Exclusivity(ODE-324) | Nov 20, 2027 |
Drugs and Companies using LONAFARNIB ingredient
NCE-1 date: 20 November, 2024
Market Authorisation Date: 20 November, 2020
Dosage: CAPSULE
More Information on Dosage
ZOKINVY family patents