Drugs / Cerliponase Alfa
Cerliponase Alfa
also known as
BMN 190 · Brineura · Cerliponasa alfa · Immature tripeptidyl-peptidase i · recombinant human tripeptidyl peptidase-1 · rhTPP1
Protein / enzyme biologic targets TPP1 via restoration
Regulatory milestones approvals, filings & regulatory actions · 5 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed juvenile neuronal ceroid lipofuscinosis 2 1 milestone | |||||
| Filed | EU (EMA) | Brineura | juvenile neuronal ceroid lipofuscinosis 2 | 2016-07-27 | “the European Medicines Agency (EMA) validated the Marketing Authorization Application (MAA) for Brineura™ (cerliponase alfa) to treat children with CLN2 disease” biomarin.com ↗ “BioMarin also has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for cerliponase alfa” biomarin.com ↗ |
| Filed late infantile neuronal ceroid lipofuscinosis 2 1 milestone | |||||
| Filed | US (FDA) | Brineura | late infantile neuronal ceroid lipofuscinosis 2 | 2016-07-27 | “On July 27, 2016, the U.S. Food and Drug Administration (FDA) accepted for review the submission of a Biologics License Application (BLA) for cerliponase alfa.” biomarin.com ↗ |
| Approved Indication not stated 3 milestones | |||||
| Label expansion | US (FDA) | BRINEURA | — | 2024-07-24 | fda.gov ↗ |
| Approved | EU (EMA) | Brineura | — | 2017-05-30 | europa.eu ↗ |
| Approved | US (FDA) | BRINEURA | — | 2017-04-27 | fda.gov ↗ |
Trials 5
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/23 trials · 2 met primary | ||||||
| Phase 1/2 | NCT05152914 | Nov 2021 → Mar 2026 overdue | neuronal ceroid lipofuscinosis 2 | David L Rogers, MD | Active not recruiting | No outcome recorded |
| Phase 1/2 | NCT02485899 | Feb 2015 → Dec 2020 | late infantile neuronal ceroid lipofuscinosis, late infantile neuronal ceroid lipofuscinosis 2 | BioMarin Pharmaceutical | Completed | Met primary |
| Phase 1/2 | NCT01907087 | Sep 2013 → Nov 2015 | late infantile neuronal ceroid lipofuscinosis, late infantile neuronal ceroid lipofuscinosis 2 | BioMarin Pharmaceutical | Completed | Met primary |
| Phase 21 trial · 1 met primary | ||||||
| Phase 2 | NCT02678689 | Jan 2016 → Apr 2022 | late infantile neuronal ceroid lipofuscinosis, late infantile neuronal ceroid lipofuscinosis 2 | BioMarin Pharmaceutical | Completed | Met primary |
| Phase not stated1 trial | ||||||
| — | NCT02963350 | — | late infantile neuronal ceroid lipofuscinosis 2 | BioMarin Pharmaceutical | Approved for marketing | No outcome recorded |
News releases announcing trial results or a regulatory action · 17
| Date | Issuer | Release |
|---|---|---|
| 2024-07-24 | BioMarin Pharmaceutical | Regulatory U.S. Food and Drug Administration Approves BioMarin’s BRINEURA® (cerliponase alfa) for Children Under 3 Years with CLN2 Disease biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has approved the company's supplemental Biologics License Application (sBLA) for BRINEURA ® (cerliponase alfa) to slow the loss of ambulation in children of all ages with neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as tripeptidyl peptidase 1 (TPP1) deficiency. |
| 2019-02-07 | BioMarin Pharmaceutical | Results BioMarin Announces Ongoing Study Demonstrates Durable Treatment Benefit from Brineura® (cerliponase alfa) for 3 Years biomarin.com ↗
The data presented today shows a durability of treatment effect in the primary efficacy endpoint where response to treatment was seen in 19 of 23 or 83% of treated patients after three years. |
| 2019-02-04 | BioMarin Pharmaceutical | Results BioMarin Announces 14 Presentations at 15th Annual WORLDSymposium™ 2019 biomarin.com ↗
Persistent treatment effect of cerliponase alfa in children with CLN2 disease: A 3-year update from an ongoing multicenter extension study |
| 2018-04-24 | BioMarin Pharmaceutical | Results New England Journal of Medicine Published Open-label Study Showing Brineura® (cerliponase alfa) Reduced the Rate of Clinical Decline of Children with CLN2 Disease, a Form of Batten Disease biomarin.com ↗
The new data demonstrated that treatment with Brineura resulted in less decline in motor and language function compared to historical controls. |
| 2017-09-05 | BioMarin Pharmaceutical | Results BioMarin Announces 3 Platform and 15 Poster Presentations at 13th International Congress of Inborn Errors of Metabolism 2017 biomarin.com ↗
Platform presentations include preliminary safety and pharmacodynamic response data from a Phase 1/2 study of ICV BMN 250, a novel enzyme replacement therapy for the treatment of Sanfilippo B (MPS IIIB), a long-term safety and efficacy analysis of cerliponase alfa to treat children with CLN2 disease, as well as updated five-year safety and efficacy data evaluating pegvaliase to treat adults with PKU. |
| 2017-06-01 | BioMarin Pharmaceutical | Regulatory European Commission Approves Brineura™ (cerliponase alfa), the First Treatment for CLN2 Disease, a Form of Batten Disease and Ultra-Rare Brain Disorder in Children biomarin.com ↗ |
| 2017-04-27 | BioMarin Pharmaceutical | Regulatory FDA Approves Brineura™ (cerliponase alfa) for the Treatment of CLN2 Disease, a Form of Batten Disease and Ultra-Rare Pediatric Brain Disorder in Children biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the U.S. Food and Drug Administration (FDA) approved Brineura™ (cerliponase alfa) to slow the loss of ambulation in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency. |
| 2017-04-21 | BioMarin Pharmaceutical | Regulatory BioMarin Receives Positive CHMP Opinion in Europe for Brineura™ (cerliponase alfa) for First Treatment of CLN2 Disease, a Form of Batten Disease and Ultra-Rare and Fatal Brain Disorder in Children biomarin.com ↗
The CHMP's recommendation is now referred to the European Commission (EC), which is expected to render its final decision by the second quarter of 2017. |
| 2017-02-15 | BioMarin Pharmaceutical | Results BioMarin Announces 11 Presentations at 13th Annual WORLDSymposium™ 2017 biomarin.com ↗
Long-term safety and efficacy of intracerebroventricular enzyme replacement therapy with cerliponase alfa in children with CLN2 disease: interim results from an ongoing multicenter, multinational extension study |
| 2016-09-14 | BioMarin Pharmaceutical | Regulatory BioMarin Announces EMA Validation of Brineura™ (Cerliponase Alfa) Marketing Authorization Application for Treatment of CLN2 Disease, a Form of Batten Disease biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that the European Medicines Agency (EMA) validated the Marketing Authorization Application (MAA) for Brineura™ (cerliponase alfa) to treat children with CLN2 disease, a form of Batten disease. |
| 2016-09-06 | BioMarin Pharmaceutical | Regulatory BioMarin Announces Update to Brineura™ (Cerliponase Alfa) Program for Treatment of CLN2 Disease, a Form of Batten Disease biomarin.com ↗
The FDA designated this submission as a major amendment to the application, thus extending the PDUFA action date by three months to April 27, 2017. |
| 2016-07-27 | BioMarin Pharmaceutical | Regulatory FDA Accepts BLA for BioMarin’s Cerliponase Alfa for CLN2 Disease, Form of Batten Disease biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that the U.S. Food and Drug Administration (FDA) accepted for review the submission of a Biologics License Application (BLA) for cerliponase alfa, an investigational therapy to treat children with CLN2 disease, a form of Batten disease. |
| 2016-05-03 | BioMarin Pharmaceutical | Regulatory BioMarin Announces EMA Grants Accelerated Assessment for Cerliponase Alfa, Experimental Treatment for a Form of Batten Disease biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the European Medicines Agency (EMA) has granted BioMarin's request for accelerated assessment for the planned cerliponase alfa Marketing Authorization Application (MAA). |
| 2016-03-02 | BioMarin Pharmaceutical | Results BioMarin Announces Positive Data From Cerliponase Alfa Program for Treatment of CLN2 Disease, a Form of Batten Disease, at 12th Annual WORLDSymposium(TM) 2016 biomarin.com ↗
Treatment with 300 mg cerliponase alfa administered via intracerebroventricular (ICV) infusion every other week was generally safe and well-tolerated in 24 patients and resulted in disease stabilization in 65% (15 of 23) of patients treated over a 48-week period, based on the Hamburg Motor + Language CLN2 rating. |
| 2015-01-12 | BioMarin Pharmaceutical | Results BioMarin Provides Preliminary Data From Ongoing Phase 1/2 Pivotal Study of BMN 190 for Treatment of CLN2 Disorder, a Form of Batten Disease biomarin.com ↗
In the nine BMN 190 patients treated for more than six months and up to 15 months, six patients showed no net change in their CLN2 rating scale score, while the other three showed a decline of one point. |
| 2014-02-13 | BioMarin Pharmaceutical | Results BioMarin Presents 15 Abstracts From Basic Research to Clinical Trials at Lysosomal Disease Network’s 10th Annual WORLDSymposium(TM) biomarin.com ↗
Preliminary Clinical Efficacy and Safety of BMN 701, GILT-tagged Recombinant Human Acid Alpha Glucosidase (rhGAA), in Late Onset Pompe Disease: Results of an Extension Study |
| 2013-04-01 | BioMarin Pharmaceutical | Regulatory BioMarin Submits CTA for BMN-190 for Batten Disease biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that it has submitted a Clinical Trial Application (CTA) with the Medicines and Healthcare Products Regulatory Agency (MHRA) in the U.K. for BMN-190, a recombinant human tripeptidyl peptidase 1 (rhTPP1) for the treatment of patients with neuronal ceroid lipofuscinosis type 2 (NCL-2), a form of Batten disease. |
All press releases naming this drug 20 releases
Evidence & citations 11 cited values
Every value below carries the sentence it was read from. 8 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Cerliponase Alfa | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05152914 ↗ |
| Known as | BMN 190 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT01907087 ↗ |
| Known as | Brineura | ChEMBL registry synonym — accepted as the source's own label CHEMBL3544921 ↗ |
| Known as | Cerliponasa alfa | ChEMBL registry synonym — accepted as the source's own label CHEMBL3544921 ↗ |
| Known as | Immature tripeptidyl-peptidase i | ChEMBL registry synonym — accepted as the source's own label CHEMBL3544921 ↗ |
| Known as | recombinant human tripeptidyl peptidase-1 | “BMN190, recombinant human tripeptidyl peptidase-1 (rhTPP1)” NCT02963350 ↗ |
| Known as | rhTPP1 | “BMN190, recombinant human tripeptidyl peptidase-1 (rhTPP1)” NCT02963350 ↗ |
| Action | Restore | “As an enzyme replacement therapy (ERT), BMN 190 is designed to help restore TPP1 enzyme activity” NCT02485899 ↗ |
| Modality | Protein / enzyme biologic | “Recombinant human tripeptidyl peptidase 1 (cerliponase alfa) is an enzyme-replacement therapy” PMID 29688815 ↗ Apr 2018 |
| Route | Other | “Treatment was intracerebroventricular infusion of 300 mg cerliponase alfa every 2 weeks.” PMID 38101904 ↗ Jan 2024 |
| Target | TPP1 | “BMN 190 is designed to help restore TPP1 enzyme activity” NCT02485899 ↗ |