Drugs / Palovarotene
Regulatory milestones approvals, filings & regulatory actions · 10 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Withdrawn fibrodysplasia ossificans progressiva 8 milestones | |||||
| Refused (EMA) | EU (EMA) | Sohonos | fibrodysplasia ossificans progressiva | 2023-07-17 | “has not granted marketing authorization for palovarotene, an investigational treatment for fibrodysplasia ossificans progressiva (FOP).” ipsen.com ↗ authority europa.eu ↗ |
| Refused (EMA) | EU (EMA) | — | fibrodysplasia ossificans progressiva | 2023-01-27 | “CHMP of the European Medicines Agency (EMA) has recommended not to grant marketing authorization for investigational palovarotene” ipsen.com ↗ “In July 2023, the European Commission did not grant marketing authorization for palovarotene.” ipsen.com ↗ |
| Withdrawn | US (FDA) | — | fibrodysplasia ossificans progressiva | 2021-08-13 | “today announced, following very recent discussions with the U.S. Food and Drug Administration (FDA), withdrawal of the New Drug Application (NDA) for palovarotene.” ipsen.com ↗ |
| Filed | US (FDA) | — | fibrodysplasia ossificans progressiva | 2021-05-28 | “In June 2022, Ipsen announced that the U.S. FDA had accepted for Priority Review its resubmitted NDA for investigational palovarotene for the treatment of patients with...” ipsen.com ↗ “In May 2021, Ipsen announced that its New Drug Application for palovarotene had been accepted by the U.S. Food and Drug Administration (FDA).” ipsen.com ↗ |
| Filed | EU (EMA) | — | fibrodysplasia ossificans progressiva | 2021-05-28 | “European Medicines Agency (EMA) and Swissmedic have also validated the Marketing Authorization Application (MAA) for palovarotene” ipsen.com ↗ |
| Filed | Other | — | fibrodysplasia ossificans progressiva | 2021-05-28 | “European Medicines Agency (EMA) and Swissmedic have also validated the Marketing Authorization Application (MAA) for palovarotene” ipsen.com ↗ |
| Filed | EU (EMA) | — | fibrodysplasia ossificans progressiva | 2020-10-22 | “The regulatory filing has been submitted and validated by the EMA, thus beginning the centralized review process.” ipsen.com ↗ |
| Clinical hold | US (FDA) | — | fibrodysplasia ossificans progressiva | 2019-12-06 | “following discussions with the U.S. Food and Drug Administration (FDA), that a partial clinical hold effective immediately” ipsen.com ↗ |
| Approved Indication not stated 2 milestones | |||||
| Label expansion | US (FDA) | SOHONOS | — | 2025-03-13 | fda.gov ↗ |
| Approved | US (FDA) | SOHONOS | — | 2023-08-16 | fda.gov ↗ |
Trials 10
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 14 trials | ||||||
| Phase 1 | NCT06908954 | Mar 2025 → Feb 2026 | — | Ipsen | Completed | No outcome recorded |
| Phase 1 | NCT04829773 | Jan → Mar 2019 | fibrodysplasia ossificans progressiva | Ipsen | Completed | No outcome recorded |
| Phase 1 | NCT04762355 | Aug 2018 → Jan 2019 | dry eye syndrome | Ipsen | Completed | No outcome recorded |
| Phase 1 | NCT04829786 | Sep → Oct 2015 | fibrodysplasia ossificans progressiva | Ipsen | Completed | No outcome recorded |
| Phase 24 trials · 2 missed | ||||||
| Phase 2 | NCT03442985 | Mar 2018 → Mar 2020 | hereditary multiple osteochondromas | Ipsen | Terminated | Missed primary |
| Phase 2 | NCT02521792 | Dec 2015 → Aug 2016 | fibrodysplasia ossificans progressiva | Ipsen | Terminated | No outcome recorded Stop: Enrollment |
| Phase 2 | NCT02279095 | Oct 2014 → Sep 2022 | fibrodysplasia ossificans progressiva | Ipsen | Completed | No outcome recorded |
| Phase 2 | NCT02190747 | Jul 2014 → May 2016 | fibrodysplasia ossificans progressiva | Ipsen | Completed | Missed primary |
| Phase 32 trials | ||||||
| Phase 3 | NCT05027802 | Mar 2022 → Nov 2024 | fibrodysplasia ossificans progressiva | Ipsen | Completed | No outcome recorded |
| Phase 3 | NCT03312634 | Nov 2017 → Jan 2020 | fibrodysplasia ossificans progressiva | Ipsen | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 17
| Date | Issuer | Release |
|---|---|---|
| 2023-08-16 | Ipsen | Regulatory US FDA approves Ipsen’s Sohonos™ (palovarotene) capsules, the first and only treatment for people with fibrodysplasia ossificans progressiva ipsen.com ↗
Ipsen (Euronext: IPN; ADR: IPSEY) announced today approval by the U.S. Food and Drug Administration (FDA) of Sohonos™ (palovarotene) capsules as a retinoid indicated for the reduction in volume of new heterotopic ossification in adults and pediatric patients aged 8 years and older for females and 10 years and older for males with fibrodysplasia ossificans progressiva (FOP). |
| 2023-07-19 | Ipsen | Regulatory Ipsen announces European Commission decision on palovarotene for the treatment of FOP ipsen.com ↗ |
| 2023-06-29 | Ipsen | Regulatory Ipsen announces positive outcome of FDA Advisory Committee on investigational palovarotene for fibrodysplasia ossificans progressiva ipsen.com ↗ |
| 2023-05-26 | Ipsen | Regulatory Ipsen receives CHMP negative opinion, following re-examination of potential first FOP treatment in the E.U. ipsen.com ↗ |
| 2023-03-16 | Ipsen | Regulatory Ipsen receives new FDA PDUFA date for investigational palovarotene for the treatment of people with FOP ipsen.com ↗
PDUFA date has been set for 16 August 2023 following the New Drug Application resubmission containing additional information on palovarotene clinical trial data, requested in a complete response letter to Ipsen in December 2022 |
| 2023-01-27 | Ipsen | Regulatory Ipsen receives CHMP negative opinion for palovarotene as a treatment for fibrodysplasia ossificans progressiva in E.U. ipsen.com ↗
Ipsen (Euronext: IPN; ADR: IPSEY) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended not to grant marketing authorization for investigational palovarotene as a treatment for the ultra-rare bone disease, fibrodysplasia ossificans progressiva (FOP). |
| 2022-12-23 | Ipsen | Regulatory Ipsen receives Complete Response Letter for palovarotene, an investigational treatment for fibrodysplasia ossificans progressiva ipsen.com ↗ |
| 2022-12-14 | Ipsen | Regulatory Ipsen receives Complete Response Letter for palovarotene, an investigational treatment for fibrodysplasia ossificans progressiva ipsen.com ↗
The U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) regarding the New Drug Application for palovarotene, an investigational treatment for the reduction of new abnormal bone formation (heterotopic ossification) in people living with fibrodysplasia ossificans progressiva (FOP). |
| 2022-10-25 | Ipsen | Regulatory Ipsen statement on updated timeline for palovarotene FDA Advisory Committee meeting ipsen.com ↗
Ipsen (Euronext: IPN; ADR: IPSEY) reports that the U.S. Food and Drug Administration (FDA) has today announced its decision to postpone the planned Endocrinologic and Metabolic Drugs Advisory Committee meeting for investigational palovarotene until a later date to be confirmed. |
| 2022-01-24 | Ipsen | Regulatory Health Canada approves Ipsen’s Sohonos (palovarotene capsules) as the first approved treatment for fibrodysplasia ossificans progressiva ipsen.com ↗
Ipsen (Euronext: IPN; ADR: IPSEY) today announced the Health Canada approval of SohonosTM (palovarotene capsules), an oral selective retinoic-acid receptor gamma (RARγ) agonist indicated to reduce the formation of heterotopic ossification (HO; new bone formation) in adults and children aged 8 years and above for females and 10 years and above for males with fibrodysplasia ossificans progressiva (FOP). |
| 2021-08-13 | Ipsen | Regulatory Ipsen announces withdrawal of palovarotene NDA, confirming intention to re-submit following additional data analyses ipsen.com ↗
Ipsen (Euronext: IPN; ADR: IPSEY) today announced, following very recent discussions with the U.S. Food and Drug Administration (FDA), withdrawal of the New Drug Application (NDA) for palovarotene. |
| 2021-05-28 | Ipsen | Regulatory Ipsen confirms U.S. FDA accepts New Drug Application for palovarotene as the first potential treatment worldwide for fibrodysplasia ossificans progressiva (FOP) ipsen.com ↗ |
| 2020-09-10 | Ipsen | Results Ipsen to present new insights at ASBMR for potential treatment of ultra-rare disease fibrodysplasia ossificans progressiva (FOP), including global Phase III MOVE trial results ipsen.com ↗
The MOVE efficacy results were compared with data from untreated patients from Ipsen’s Natural History Study (NHS). |
| 2020-08-25 | Ipsen | Results Ipsen to present results from MOVE, the first global Phase III trial in fibrodysplasia ossificans progressiva (FOP), at ASBMR 2020 annual meeting ipsen.com ↗
Results from the MOVE trial demonstrated a 62% reduction in mean annualized new HO volume in participants treated with palovarotene (8,821 mm3) (n=97) versus untreated (23,318 mm3) (n=98) patients (nominal weighted linear mixed effects [wLME] model est. –11,611mm3, p-value = 0.0292). |
| 2020-01-24 | Ipsen | Results Ipsen’s palovarotene clinical program in fibrodysplasia ossificans progressiva reaches prespecified interim analysis futility criteria ipsen.com ↗
The results of a futility analysis indicated that the Phase III FOP trial was unlikely to meet its primary efficacy endpoint (annualized change in new HO volume as compared with Natural History Studyi) upon completion. |
| 2019-12-06 | Ipsen | Regulatory Ipsen Initiates Partial Clinical Hold for Palovarotene IND120181 and IND135403 Studies ipsen.com ↗
The FDA has placed the studies on partial clinical hold pending review of additional details regarding these events and plans to issue additional requests for information within the next 30 days. |
| 2019-04-11 | Ipsen | Regulatory Clementia Pharmaceuticals Inc. Announces Receipt of Final Court Approval for Plan of Arrangement With Ipsen S.A. ipsen.com ↗
the Québec Superior Court issued earlier today a final order approving the previously announced statutory plan of arrangement under the Canada Business Corporations Act |
All press releases naming this drug 18 releases
Evidence & citations 12 cited values
Every value below carries the sentence it was read from. 12 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | palovarotene | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04829786 ↗ |
| Known as | CLM-001 | ChEMBL registry synonym — accepted as the source's own label CHEMBL2105648 ↗ |
| Known as | IPN-60120 | ChEMBL registry synonym — accepted as the source's own label CHEMBL2105648 ↗ |
| Known as | Palovaroteno | ChEMBL registry synonym — accepted as the source's own label CHEMBL2105648 ↗ |
| Known as | R-667 | ChEMBL registry synonym — accepted as the source's own label CHEMBL2105648 ↗ |
| Known as | RG-667 | ChEMBL registry synonym — accepted as the source's own label CHEMBL2105648 ↗ |
| Known as | RO-3300074 | ChEMBL registry synonym — accepted as the source's own label CHEMBL2105648 ↗ |
| Known as | Sohonos | ChEMBL registry synonym — accepted as the source's own label CHEMBL2105648 ↗ |
| Action | Activate | “Retinoic acid receptor gamma agonist” CHEMBL2105648 ↗1“palovarotene, an RAR gamma agonist” NCT02190747 ↗ |
| Modality | Small molecule | “Palovarotene is a therapeutic retinoid” PMID 37804430 ↗ Oct 2023 |
| Route | Oral | “Palovarotene will be taken orally once daily at approximately the same time each day.” NCT02190747 ↗ |
| Target | RARG | “Retinoic acid receptor gamma agonist” CHEMBL2105648 ↗ |