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
Can you believe GALAFOLD received compensation for the extended wait time during the regulatory approval process?
| 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 THERAP US | Methods of treating fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11903938 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
Aug, 2038
(12 years from now) | |
| US12042489 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11304940 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US12109205 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11612594 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US12042488 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11357764 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11376244 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11612593 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11622962 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
Mar, 2039
(13 years from now) | |
| US11357765 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11426396 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11633387 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11826360 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
Feb, 2039
(13 years from now) | |
| US11786516 | AMICUS THERAP US | 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 THERAP US | Methods for treatment of Fabry disease |
May, 2027
(1 year, 4 months from now) | |
| US10383864 | AMICUS THERAP US | Methods for treatment of Fabry disease |
May, 2027
(1 year, 4 months from now) | |
| US10471053 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US10076514 | AMICUS THERAP US | Methods of treating fabry disease in patients having the G9331A mutation in the GLA gene |
Mar, 2037
(11 years from now) | |
| US8592362 | AMICUS THERAP US | Method to predict response to pharmacological chaperone treatment of diseases |
Feb, 2029
(3 years from now) | |
| US11642334 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
Feb, 2039
(13 years from now) | |
| US9095584 | AMICUS THERAP US | Method to predict response to pharmacological chaperone treatment of diseases |
Feb, 2029
(3 years from now) | |
| US9999618 | AMICUS THERAP US | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
Apr, 2028
(2 years from now) | |
| US11458128 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US10406143 | AMICUS THERAP US | Methods for treatment of fabry disease |
May, 2027
(1 year, 4 months from now) | |
| US10525045 | AMICUS THERAP US | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
Apr, 2028
(2 years from now) | |
| US9987263 | AMICUS THERAP US | Methods for treatment of Fabry disease |
May, 2027
(1 year, 4 months from now) | |
| US9480682 | AMICUS THERAP US | Methods for treatment of Fabry disease |
May, 2027
(1 year, 4 months from now) | |
| US10925866 | AMICUS THERAP US | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
Apr, 2028
(2 years from now) | |
| US10251873 | AMICUS THERAP US | Methods of treating fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US10813921 | AMICUS THERAP US | Method to predict response to pharmacological chaperone treatment of diseases |
Feb, 2029
(3 years from now) | |
| US11633388 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
Mar, 2039
(13 years from now) | |
| US11033538 | AMICUS THERAP US | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
Apr, 2028
(2 years from now) | |
| US12042490 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11357761 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11357762 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11357763 | AMICUS THERAP US | Methods of treating fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11357784 | AMICUS THERAP US | Use of migalastat for treating Fabry disease in pregnant patients |
Feb, 2039
(13 years from now) | |
| US11666564 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US10857141 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US10849890 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US10849889 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US10857142 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| USRE48608 | AMICUS THERAP US | Method to predict response to pharmacological chaperone treatment of diseases |
Oct, 2031
(5 years from now) | |
| US11389437 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11389436 | AMICUS THERAP US | Methods of treating fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US10874657 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US10874655 | AMICUS THERAP US | Methods of treating fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US10874656 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US10792278 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US10792279 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US10799491 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US10806727 | AMICUS THERAP US | Methods of treating fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11833164 | AMICUS THERAP US | Methods of treating Fabry disease in patients having a mutation in the GLA gene |
Jan, 2042
(16 years from now) | |
| US11813255 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11234972 | AMICUS THERAP US | Methods of treating fabry disease in patients having the G9331A mutation in the GLA gene |
Mar, 2037
(11 years from now) | |
| US11241422 | AMICUS THERAP US | Methods for treatment of Fabry disease |
May, 2027
(1 year, 4 months from now) | |
| US11278538 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11278537 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11278536 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11278539 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years from now) | |
| US11278540 | AMICUS THERAP US | Methods of treating Fabry patients having renal impairment |
May, 2038
(12 years 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
Can you believe GIVLAARI received compensation for the extended wait time during the regulatory approval process?
| 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 PHARMS INC | Compositions comprising alternating 2′-modified nucleosides for use in gene modulation |
Jan, 2024
(2 years ago) | |
| US8546143 | ALNYLAM PHARMS INC | Compositions and methods for inhibiting expression of a target gene |
Jan, 2022
(4 years ago) | |
| US10131907 | ALNYLAM PHARMS INC | Glycoconjugates of RNA interference agents |
Aug, 2028
(2 years from now) | |
| US8106022 | ALNYLAM PHARMS INC | Carbohydrate conjugates as delivery agents for oligonucleotides |
Dec, 2029
(3 years from now) | |
| US10119143 | ALNYLAM PHARMS INC | Compositions and methods for inhibiting expression of the ALAS1 gene |
Oct, 2034
(8 years from now) | |
| US8828956 | ALNYLAM PHARMS INC | Carbohydrate conjugates as delivery agents for oligonucleotides |
Dec, 2028
(2 years from now) | |
| US10125364 | ALNYLAM PHARMS INC | Compositions and methods for inhibiting expression of the ALAS1 gene |
Mar, 2033
(7 years from now) | |
| US11028392 | ALNYLAM PHARMS INC | Compositions and methods for inhibiting expression of the ALAS1 gene |
Oct, 2034
(8 years from now) | |
| US11530408 | ALNYLAM PHARMS INC | Therapeutic compositions |
May, 2024
(1 year, 7 months ago) | |
| US9708615 | ALNYLAM PHARMS INC | Therapeutic compositions |
Mar, 2024
(1 year, 10 months ago) | |
| US10273477 | ALNYLAM PHARMS INC | Therapeutic compositions |
Mar, 2024
(1 year, 10 months ago) | |
| US9133461 | ALNYLAM PHARMS INC | Compositions and methods for inhibiting expression of the ALAS1 gene |
Nov, 2033
(7 years from now) | |
| US9150605 | ALNYLAM PHARMS INC | Compositions comprising alternating 2′-modified nucleosides for use in gene modulation |
Aug, 2025
(4 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 PHARMS INC | 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 DENMARK | Use of Hsp70 as a regulator of enzymatic activity |
Aug, 2029
(3 years from now) | |
| US9884058 | ZEVRA DENMARK | Use of Hsp70 as a regulator of enzymatic activity |
Jun, 2029
(3 years from now) | |
| US9289472 | ZEVRA DENMARK | 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 THERAPS INC | 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)
Can you believe OXLUMO received compensation for the extended wait time during the regulatory approval process?
| 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 PHARMS INC | Modified double-stranded RNA agents |
Aug, 2035
(9 years from now) | |
| US11060093 | ALNYLAM PHARMS INC | Methods and compositions for the specific inhibition of glycolate oxidase (HAO1) by double-stranded RNA |
Dec, 2034
(8 years from now) | |
| US10487330 | ALNYLAM PHARMS INC | Methods and compositions for the specific inhibition of glycolate oxidase (HAO1) by double-stranded RNA |
Dec, 2034
(8 years from now) | |
| US10478500 | ALNYLAM PHARMS INC | Compositions and methods for inhibition of HAO1 (Hydroxyacid Oxidase 1 (Glycolate Oxidase)) gene expression |
Oct, 2035
(9 years from now) | |
| US8106022 | ALNYLAM PHARMS INC | Carbohydrate conjugates as delivery agents for oligonucleotides |
Dec, 2029
(3 years from now) | |
| US8828956 | ALNYLAM PHARMS INC | Carbohydrate conjugates as delivery agents for oligonucleotides |
Dec, 2028
(2 years from now) | |
| US10131907 | ALNYLAM PHARMS INC | Glycoconjugates of RNA interference agents |
Aug, 2028
(2 years from now) | |
| US10612024 | ALNYLAM PHARMS INC | Modified double-stranded RNA agents |
Aug, 2035
(9 years from now) | |
| US10612027 | ALNYLAM PHARMS INC | Modified double-stranded RNA agents |
Aug, 2035
(9 years from now) | |
| US10465195 | ALNYLAM PHARMS INC | Methods and compositions for the specific inhibition of glycolate oxidase (HAO1) by double-stranded RNA |
Dec, 2034
(8 years from now) | |
| US11261447 | ALNYLAM PHARMS INC | Methods for inhibition of HAO1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression |
Nov, 2038
(12 years from now) | |
| US9828606 | ALNYLAM PHARMS INC | Methods and compositions for the specific inhibition of glycolate oxidase (HAO1) by double-stranded RNA |
Dec, 2034
(8 years from now) | |
| US11446380 | ALNYLAM PHARMS INC | 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 PHARMS INC | 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
| 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 PHARM | 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 PHARM | C-type natriuretic peptide variants to treat skeletal dysplasia in children |
Jul, 2042
(16 years from now) | |
| US10646550 | BIOMARIN PHARM | Use of C-type natriuretic peptide variants to treat skeletal dysplasia |
Aug, 2036
(10 years from now) | |
| US11911446 | BIOMARIN PHARM | Use of C-type natriuretic peptide variants to treat skeletal dysplasia |
Aug, 2036
(10 years from now) | |
| US11590204 | BIOMARIN PHARM | Use of C-type natriuretic peptide variants to treat skeletal dysplasia |
Aug, 2036
(10 years from now) | |
| USRE48267 | BIOMARIN PHARM | Variants of C-type natriuretic peptide |
May, 2030
(4 years from now) | |
| US9907834 | BIOMARIN PHARM | Use of C-type natriuretic peptide variants to treat skeletal dysplasia |
Aug, 2036
(10 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
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 THERAPS INC | Farnesyltransferase inhibitors for treatment of laminopathies, cellular aging and atherosclerosis |
Oct, 2025
(2 months ago) | |
| US7838531 | SENTYNL THERAPS INC | 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