Delving into the Latest Updates on Palovarotene with Synapse

RegulationOrphan Drug (United States), Orphan Drug (European Union), Orphan Drug (Japan), Orphan Drug (Australia), Rare Pediatric Disease (United States), Priority Review (United States), Breakthrough Therapy (United States)

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Structure/Sequence

Molecular FormulaC27H30N2O2

InChIKeyYTFHCXIPDIHOIA-DHZHZOJOSA-N

CAS Registry410528-02-8

The main aim of this study is to understand how moderate and severe liver impairment (based on the Child-Pugh classification) affects the body's processing of a single dose of 10 mg maximum of palovarotene, compared to healthy participants with normal liver function.
The study will also assess the safety and tolerability of the single dose of palovarotene.
Participants will be enrolled in stages and divided into three groups based on their liver function:
* Group 1: Healthy participants with normal liver function
* Group 2: Participants with moderate liver impairment
* Group 3: Participants with severe liver impairment (only enrolled if Group 2 results are safe and acceptable)
Blood samples will be taken to assess how the drug binds to proteins in the blood. Participants will undergo various safety checks and procedures. Participants will stay in the clinical unit until Day 5 for these assessments and will return on Day 10 for a final visit.

Start Date14 Mar 2025

Sponsor / Collaborator

Ipsen SA

NCT05027802

/ CompletedPhase 3

Rollover Study; Multicentre, Phase III, Open-label Study to Further Evaluate the Safety and Efficacy of Palovarotene Capsules in Male and Female Participants Aged ≥14 Years With Fibrodysplasia Ossificans Progressiva (FOP) Who Have Completed Study PVO-1A-301 or PVO-1A-202/PVO-1A-204 and May Benefit From Palovarotene Therapy.

The main objective of this study is to further evaluate the safety and efficacy of palovarotene in adult and paediatric participants with FOP.
The aim of the study is also to ensure treatment continuity to participants who have completed one of the parent studies (Study PVO-1A-301, Study PVO-1A-202 and Study PVO-1A-204) and who, in the investigator's judgement, may benefit from palovarotene therapy.

Start Date14 Mar 2022

Sponsor / Collaborator

Ipsen SA

NCT04829773

/ CompletedPhase 1

An Open-Label 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

Study to evaluate the effect of food and the effect of swallowing capsule whole versus sprinkling on apple sauce on the pharmacokinetics (PK)/bioavailability of palovarotene, and evaluate the effect of palovarotene on the PK of the CYP3A4 substrate midazolam.

Start Date03 Jan 2019

Sponsor / Collaborator

Clementia Pharmaceuticals, Inc.

100 Clinical Results associated with Palovarotene

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100 Translational Medicine associated with Palovarotene

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100 Patents (Medical) associated with Palovarotene

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62

Literatures (Medical) associated with Palovarotene

01 Jan 2026·BMJ Case Reports

Progressive ossification in an adolescent with fibrodysplasia ossificans progressiva

Article

Author: Goyal, Tarun ; Kaur, Harmeet ; Prakash, Sugandha ; Sidana, Shivani

Fibrodysplasia ossificans progressiva (FOP), also known as Munchmeyer disease or ‘stone man syndrome’, is a rare, disabling genetic disorder caused by an ACVR1/ALK2 mutation, resulting in progressive heterotopic ossification of muscles, tendons and ligaments. Even minor trauma can trigger flare-ups, causing joint fusion and severe disability.An early adolescent male presented with progressive joint stiffness, painful swellings and malnutrition. Examination revealed hallmark signs of FOP, including malformed great toes and widespread soft tissue ossification. Differential diagnoses such as progressive osseous heteroplasia and juvenile fibromatosis were excluded clinically. A biopsy was avoided due to the risk of disease exacerbation, and the diagnosis was made on clinical grounds.As no definitive cure exists, management was supportive with non-steroidal anti-inflammatory drugs for pain, nutritional supplementation and avoidance of surgery. Awareness and early recognition are crucial, while emerging therapies, such as palovarotene and gene-targeted approaches, hold promise for the future.

01 Nov 2025·PEDIATRIC DERMATOLOGY

Progressive Soft Tissue Swelling in a Pediatric Patient Leading to the Diagnosis of Fibrodysplasia Ossificans Progressiva: A Case Report

Article

Author: Fiorillo, Loretta ; Yilmaz, Orhan

ABSTRACT:

Fibrodysplasia ossificans progressiva (FOP) is a rare genetic disorder characterized by progressive heterotopic ossification (HO) and congenital malformation of the great toes. This case describes a 5‐year‐old Caucasian girl who initially presented with painless neck and back swelling as well as severe limitation of movement in the neck and shoulder girdle, who was diagnosed as FOP following tissue‐based genetic testing revealing the ACVR1 (p.Arg206His) pathogenic variant. The diagnostic process, which included imaging, skin biopsy, and a thorough infectious and autoimmune workup, emphasized the challenges in identifying FOP due to its rarity; however, bilateral congenital hallux valgus, often present in FOP, can be a clue for early diagnosis. While corticosteroids provide temporary relief, long‐term management now includes palovarotene, a novel therapy to suppress HO.

01 Apr 2025·CLINICAL DYSMORPHOLOGY

Monoallelic variants in ACVR1 in a cohort of Egyptian individuals with fibrodysplasia ossificans progressiva

Article

Author: Ibrahim Hegazy ; Engy A. Ashaat ; Doaa R. Soliman ; Mohamed A. Al Kersh ; Mona S. Aglan ; Amal I. Othman ; Sawsan Abdel-Hadi ; Khaled Hamed ; Adel M. Ashour ; Ghada M.H. Abdel-Salam ; Nehal F. Hassib ; Mennat Mehrez ; Mohamed S. Abdel-Hamid ; Hasnaa M. Elbendary ; Rasha M. Elhossini ; Hala T. El-Bassyouni

Objectives:

Fibrodysplasia ossificans progressiva (FOP) is a rare ectopic ossification disorder of connective tissue deposited in the muscles, fascia, tendons, and ligaments. The disease is an autosomal dominant pattern caused by pathogenic variants of ACVR1. Herein, we describe the largest number of affected individuals from the Middle East North Africa region who presented with FOP.

Methods:

DNA extraction and molecular studies using Sanger sequencing was done for the nine affected individuals developing bony swellings of variable severity at different ages.

Results:

Sanger sequencing identified the common ACVR1 variant (c.617G>A, p.Arg206His) in 7/9, whereas c.983G>A (p.Gly328Glu) in 2/9 affected individuals. Interestingly, the affected individuals harboring the p.Gly328Glu displayed atypical presentations involving micropenis, partial agenesis of the corpus callosum and dysmorphic brainstem, and reduction defects of fingers/toes. Moreover, they had a severe phenotype compared to affected individuals carrying the p.Arg206His variant.

Conclusions:

Our study highlights the progressive nature of the disease and the importance of early diagnosis to avoid lethal complications such as locked jaw and airway obstructions that affect swallowing and breathing. An early accurate diagnosis gives an opportunity for the affected individuals in the future to be candidates for the agonist Palovarotene drug that prevents the complications arising from ectopic ossification.

77

News (Medical) associated with Palovarotene

28 Feb 2026

·www.pharmaceutical-technology.com

Quotient and Ipsen extend partnership for ultra-rare disease therapy

Quotient Sciences has extended its commercial partnership with Ipsen, focusing on the production of Sohonos (Palovarotene) for fibrodysplasia ossificans progressiva (FOP) FOP is an ultra-rare condition, impacting fewer than 1,000 people worldwide. The collaboration involves containment, specialised handling, and sustained supply of this highly potent molecule. The agreement includes Ipsen’s investment in additional equipment at Quotient Sciences’ facility in Boothwyn, Pennsylvania, US. This comprises a Pneumatic Closed Transfer System, which maintains blend integrity during safe material transfer, and a Flexible Dispensing Isolator designed to improve operator safety and remove the need for specialised cleaning procedures. Quotient Sciences now has expanded capabilities for handling highly potent active pharmaceutical ingredients (HPAPI) and products with occupational exposure limits (OELs) at or below 1µg/m³. The new additions also allow seamless integration with existing equipment, facilitating the production process for complex drug molecules. The extended partnership aims to guarantee a reliable commercial supply of FOP therapy while expanding capacity to handle multiple highly potent compounds at commercial scale. This supports the need for flexible solutions in treating rare diseases where patient populations are small and product volumes are limited. Ipsen selected Quotient Sciences for its established operational capabilities and extensive experience in managing global shipments. The company will be responsible for ensuring the secure and timely delivery of the therapy to international markets. Quotient Sciences Philadelphia vice-president and general manager Marlene Leuenberger said: “This significant investment in advanced containment and manufacturing technology at our Boothwyn facility underscores Quotient Sciences’ unwavering commitment to supporting patients with ultra-rare diseases. “By expanding our capabilities in handling highly potent compounds, we are not only ensuring a reliable supply of critical therapies like Sohonos (Palovarotene) for the FOP community, but also reinforcing our promise to deliver flexible, high-quality solutions for our partners with specialised needs, especially for highly potent compounds.” In September 2025, Quotient Sciences entered a partnership with the UK’s Centre for Process Innovation (CPI) to expedite the creation of ribonucleic acid (RNA)-based treatments. The content above comes from the network. if any infringement, please contact us to modify.

License out/in

27 Feb 2026

·www.pharmamanufacturing.com

Quotient Sciences, Ipsen extend commercial manufacturing agreement

Quotient Sciences, an integrated contract research, development and manufacturing organization (CRDMO) headquartered in Philadelphia, has extended its commercial manufacturing partnership with Ipsen to produce a treatment for fibrodysplasia ossificans progressiva (FOP). Under the agreement, Quotient will continue manufacturing Sohonos (palovarotene), a therapy approved by the U.S. Food and Drug Administration in 2023 for the ultra-rare disease, which affects fewer than 1,000 people worldwide, according to the company. The product is a highly potent molecule requiring specialized handling and containment. As part of the partnership, Ipsen invested in new equipment at Quotient’s Boothwyn, Pennsylvania facility, including a pneumatic closed transfer system and a flexible dispensing isolator. The company upgrades are designed to ensure safe material transfer, protect operators, and eliminate certain cleaning requirements while maintaining blend integrity. Quotient said the additions expand its capabilities for manufacturing highly potent active pharmaceutical ingredients and drug products with occupational exposure limits at or below 1 µg/m³. The equipment is integrated with existing infrastructure to support commercial-scale production and global distribution, according to the announcement. The extended agreement is intended to ensure continued commercial supply of the FOP therapy and broaden the site’s ability to handle multiple highly potent compounds at commercial scale. The announcement follows additional recent investments by Quotient in technology-enabled development. In December 2025 , the company entered a multi-year partnership with Canada-based Intrepid Labs to incorporate artificial intelligence into early-stage formulation development. Under that agreement, Quotient gained access to Intrepid’s machine learning platform to support formulation design and optimization within its Translational Pharmaceutics platform.

Drug ApprovalLicense out/in

26 Feb 2026

·www.prnewswire.com

Quotient Sciences Extends Commercial Manufacturing Partnership for Ultra-Rare Disease Treatment

PHILADELPHIA , Feb. 26, 2026 /PRNewswire/ -- Quotient Sciences, a leading integrated CRDMO (Contract Research, Development and Manufacturing Organization), is pleased to announce an extended commercial supply partnership with Ipsen, a global biopharmaceutical company, to manufacture a treatment for Fibrodysplasia Ossificans Progressiva (FOP), an ultra-rare disease affecting fewer than 1,000 people worldwide. This collaboration reinforces Quotient Sciences' commitment to supporting the manufacture of niche and smaller volume commercial products often overlooked by larger CDMOs. Sohonos (Palovarotene), approved by the FDA in 2023, is a highly potent molecule which requires specialized handling and containment to protect both the facility and manufacturing operators. As part of the partnership, Ipsen has invested in new equipment at Quotient Sciences' Boothwyn, PA facility, including a Pneumatic Closed Transfer System, which ensures safe material transfer without impacting blend segregation, and a Flexible Dispensing Isolator, which enhances operator safety and eliminates special cleaning requirements. Quotient Sciences brings a world-class facility, an experienced team, reliable supply capabilities, and the ability to manage global shipments, ensuring seamless delivery and operational excellence throughout the partnership. These additions expand Quotient Sciences' capabilities for handling and manufacturing highly potent API (HPAPI) drug molecules and products, with known OELs at or below 1 µg/m³, while enabling seamless integration with existing equipment. The partnership will ensure sustained commercial supply for FOP therapy and broaden capabilities to handle multiple highly potent compounds at commercial scale, addressing critical needs in rare disease treatment. Marlene Leuenberger, VP and General Manager, Quotient Sciences, Philadelphia commented: "This significant investment in advanced containment and manufacturing technology at our Boothwyn facility underscores Quotient Sciences' unwavering commitment to supporting patients with ultra-rare diseases. By expanding our capabilities in handling highly potent compounds, we are not only ensuring a reliable supply of critical therapies like Sohonos (Palovarotene) for the FOP community, but also reinforcing our promise to deliver flexible, high-quality solutions for our partners with specialized needs especially for highly potent compounds. About Quotient Sciences : Quotient Sciences is an integrated CRDMO (contract research, development and manufacturing organization) providing services across the entire drug development and clinical pathway. Our flagship platform for drug development, Translational Pharmaceutics®, has been trusted for integrated drug product formulation development and manufacturing with clinical testing activities for over 17 years. Cutting through silos across a range of drug development capabilities, we save precious time and money in getting drugs to patients. To learn more, visit quotientsciences.com. SOURCE Quotient Sciences 21% more press release views with Request a Demo

Drug Approval

100 Deals associated with Palovarotene

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External Link

KEGG	Wiki	ATC	Drug Bank
D09365	Palovarotene	-	DB12320

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Cardiovascular Diseases	Australia	Ipsen Pty Ltd.	28 Nov 2023
Ossification, Heterotopic	Australia	Ipsen Pty Ltd.	28 Nov 2023
Myositis Ossificans	Canada	Ipsen Biopharmaceuticals, Inc.	21 Jan 2022

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Pain	Phase 3	Italy	Clementia Pharmaceuticals, Inc.	15 Mar 2018
Muscular Diseases	Phase 2	Netherlands	Clementia Pharmaceuticals, Inc.	05 Nov 2018
Multiple Hereditary Exostoses	Phase 2	United States	Clementia Pharmaceuticals, Inc.	22 Mar 2018
Multiple Hereditary Exostoses	Phase 2	Japan	Clementia Pharmaceuticals, Inc.	22 Mar 2018
Multiple Hereditary Exostoses	Phase 2	Australia	Clementia Pharmaceuticals, Inc.	22 Mar 2018
Multiple Hereditary Exostoses	Phase 2	Belgium	Clementia Pharmaceuticals, Inc.	22 Mar 2018
Multiple Hereditary Exostoses	Phase 2	Canada	Clementia Pharmaceuticals, Inc.	22 Mar 2018
Multiple Hereditary Exostoses	Phase 2	France	Clementia Pharmaceuticals, Inc.	22 Mar 2018
Multiple Hereditary Exostoses	Phase 2	Italy	Clementia Pharmaceuticals, Inc.	22 Mar 2018
Multiple Hereditary Exostoses	Phase 2	Netherlands	Clementia Pharmaceuticals, Inc.	22 Mar 2018

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05027802 (PIVOINE, CTgov)	Phase 3	Myositis Ossificans	61	Palovarotene	enxfiglxsn = kdmbzpmqap ccqwqkhfsk (yktnfcpxlt, exqhjenibh - clbsqmigzf) View more	-	13 Jun 2025
NCT04829773 (Phase I, Pubmed \| Eur J Drug Metab Pharmacokinet)	Phase 1	-	23	Palovarotene 20 mg	dkkufvhgqx(ywvwjfckuo) = glfqbiovnp jewvcxwlxz (lclwsidzqt ) View more	Positive	01 Nov 2023
NCT03312634 (PVO-1A-301, FDA) Manual	Phase 3	Myositis Ossificans	-	SOHONOS	xrzfmxbclt(ufkucarhem) = yjfumowxjx wnhbbajnaf (eyvvhlhqcx ) View more	Positive	16 Aug 2023
				untreated group	xrzfmxbclt(ufkucarhem) = hetqkusxth wnhbbajnaf (eyvvhlhqcx )
NCT03312634 (MOVE \| PVO-1A-301, CTgov)	Phase 3	Myositis Ossificans	107	Palovarotene (Palovarotene)	lsxesypbix(uoyhwabgaw) = guiwbvctyl bvcgiedkib (vvwfvmevct, 3084.0) View more	-	14 Mar 2023
				palovarotene (Untreated (PVO-1A-001))	lsxesypbix(uoyhwabgaw) = luqcskuact bvcgiedkib (vvwfvmevct, 4850.0) View more
NCT03442985 (MO-Ped, CTgov)	Phase 2	Multiple Hereditary Exostoses	193	palovarotene+placebo (Placebo)	ugndlfztug(djupyfgxpz) = hccwqikpnn splpeyfjxn (protvstkor, jmrtgbstza - vyaojmsqwp) View more	-	01 Aug 2022
				Palovarotene (Palovarotene 2.5 mg)	ugndlfztug(djupyfgxpz) = usuyaxxkqp splpeyfjxn (protvstkor, rwestudgcr - cyyywgqffh) View more
NCT02190747 (Pubmed)	Phase 2	Myositis Ossificans	40	Palovarotene 10/5 mg	wigmzxbtbm(uoavmybcea) = yyxkctvisl cwejpykcpr (hnshnynizn )	Positive	19 Jul 2022
				Palovarotene 5/2.5 mg	wigmzxbtbm(uoavmybcea) = kohpabxazv cwejpykcpr (hnshnynizn )
NCT03312634 (MOVE, ESPE2021)	Phase 3	Myositis Ossificans	-	Palovarotene	fkomxipgie(sffxccryqo) = brfkgjhgok qwvhinwhlf (xlvslvknjh )	-	22 Sep 2021
NCT02521792 (CTgov)	Phase 2	Myositis Ossificans	6	Palovarotene	objubmpsuh = qwpwpucjkh tzgbhybjha (dydcufqkfe, kerdwhumwo - fyougwsmkp) View more	-	19 Oct 2020
NCT03312634 (MOVE, BusinessWire) Manual	Phase 3	Myositis Ossificans	107	Palovarotene	kcrdwtlmkm(mfowjziwxa) = zlukekgoqc dyafbtkivo (jroltbgjxt ) View more	Positive	25 Aug 2020
NCT02190747 (CTgov)	Phase 2	Myositis Ossificans	40	Palovarotene (Palovarotene 10/5 mg)	tvacxqnzek = romhspkngg gfdpsegwls (cthvxtulko, adddofxcwc - tmrfsajgmb) View more	-	24 Jun 2020
				Palovarotene (Palovarotene 5/2.5 mg)	tvacxqnzek = nzplzhgmzm gfdpsegwls (cthvxtulko, lsfizoixbh - zxlwpwcjpp) View more