Fibrodysplasia Ossificans Progressiva

8
Pipeline Programs
3
Companies
9
Clinical Trials
1
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
2
0
4
0
1
1
Early DiscoveryClinical DevelopmentMarket

On Market (1)

Approved therapies currently available

Ipsen
SOHONOSApproved
palovarotene
Ipsen
Retinoid [EPC]oral2023

Competitive Landscape

3 companies ranked by most advanced pipeline stage

Ipsen
IpsenChina - Tianjin
7 programs
1
4
1
1
PalovarotenePhase 31 trial
IPN60130Phase 21 trial
PalovarotenePhase 21 trial
PalovarotenePhase 21 trial
Palovarotene dose level 1Phase 21 trial
+2 more programs
Active Trials
NCT02322255Completed114Est. Apr 2020
NCT04829773Completed48Est. Mar 2019
NCT05039515Active Not Recruiting113Est. Mar 2029
+4 more trials
DS
Daiichi SankyoChina - Shanghai
1 program
1
DS-6016aPhase 11 trial
Active Trials
NCT04818398Completed48Est. Jul 2022
Novartis
NovartisBASEL, Switzerland
1 program
Urine Sample Collection From FOP PatientsN/A1 trial
Active Trials
NCT02066324Completed25Est. Aug 2014

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
IpsenPalovarotene
IpsenIPN60130
IpsenPalovarotene
IpsenPalovarotene dose level 1
IpsenPalovarotene
Daiichi SankyoDS-6016a
IpsenPalovarotene
IpsenA Natural History Study of Fibrodysplasia Ossificans Progressiva (FOP)
NovartisUrine Sample Collection From FOP Patients

Clinical Trials (9)

Total enrollment: 559 patients across 9 trials

NCT03312634IpsenPalovarotene

An Efficacy and Safety Study of Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva.

Start: Nov 2017Est. completion: Sep 2022107 patients
Phase 3Completed

A Study to Assess the Effectiveness and Safety of 2 Dosage Regimens of Oral Fidrisertib (IPN60130) for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP).

Start: Dec 2021Est. completion: Mar 2029113 patients
Phase 2Active Not Recruiting
NCT02521792IpsenPalovarotene

In-Home Evaluation of Episodic Administration of Palovarotene in Fibrodysplasia Ossificans Progressiva (FOP) Subjects

Start: Dec 2015Est. completion: Aug 20166 patients
Phase 2Terminated
NCT02279095IpsenPalovarotene dose level 1

An Open-Label Extension Study of Palovarotene Treatment in Fibrodysplasia Ossificans Progressiva (FOP)

Start: Oct 2014Est. completion: Sep 202258 patients
Phase 2Completed
NCT02190747IpsenPalovarotene

An Efficacy and Safety Study of Palovarotene to Treat Preosseous Flare-ups in FOP Subjects

Start: Jul 2014Est. completion: May 201640 patients
Phase 2Completed

Study of Single-Ascending Doses of DS-6016a in Healthy Japanese Subjects

Start: Apr 2021Est. completion: Jul 202248 patients
Phase 1Completed
NCT04829773IpsenPalovarotene

Study Evaluating the Effect of Food on the Pharmacokinetics of Palovarotene and the Effect of Palovarotene on the Pharmacokinetics of the CYP3A4 Substrate Midazolam in Two Cohorts of Healthy Adult Subjects

Start: Jan 2019Est. completion: Mar 201948 patients
Phase 1Completed
NCT02322255IpsenA Natural History Study of Fibrodysplasia Ossificans Progressiva (FOP)

A Natural History Study of Fibrodysplasia Ossificans Progressiva (FOP)

Start: Dec 2014Est. completion: Apr 2020114 patients
N/ACompleted
NCT02066324NovartisUrine Sample Collection From FOP Patients

Urine Sample Collection From FOP Patients

Start: Nov 2013Est. completion: Aug 201425 patients
N/ACompleted

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

1 late-stage (Phase 3) programs — potential near-term approvals
3 companies competing in this space