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Development Status

Investigational

3

Designated

1

US Approved Rx

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Primary Target

Retinoic acid receptor gamma

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Highest Phase

Phase II

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Approval Year

Unknown

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2023

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Condition

Emphysema

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Fibrodysplasia ossificans progressiva

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Treatment Modality

Primary

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Originator

Roche

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Chemical

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Protein

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Code System

FDA ORPHAN DRUG

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FDA UNII

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CAS

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USAN

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NCI_THESAURUS

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MONOCLONAL ANTIBODY

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Salts, Hydrates, Esters, etc.

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Search results for "ORPHAN DRUG|Designated|Treatment of fibrodysplasia ossificans progressiva" in comments (approximate match)

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Palovarotene

28K6I5M16G

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Status:

US Approved Rx (2023)

First approved in 2023

Class (Stereo):

CHEMICAL (ACHIRAL)

Targets:

Retinoic acid receptor gamma

Conditions:

Fibrodysplasia ossificans progressiva

Palovarotene (R-667, RO-3300074) was developed by Roche Holding AG as a selective retinoic acid receptor gamma agonist for the treatment of emphysema. Phase I clinical trials of palovarotene in patients with emphysema demonstrated that the drug is we...

ll tolerated, with improvements observed in markers of emphysema progression. Unlike all-trans retinoic acid, the pharmacokinetic profile of palovarotene appears to be dose-proportional. However, those studies were discontinued. Palovarotene is also being investigated in phase II of the clinical trial in the treatment of Fibrodysplasia Ossificans Progressiva (FOP). Palovarotene received Fast Track designation from the U.S. Food and Drug Administration (FDA) and orphan designations for the treatment of FOP from both the FDA and the European Medicines Agency (EMA).

GARETOSMAB

KR9ZSKO5QE

Subunit 1 (453) Similarity Search Subunit 2 (453) Similarity Search Subunit 3 (215) Similarity Search Subunit 4 (215) Similarity Search

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Subunit 1 (453) Similarity Search Subunit 2 (453) Similarity Search Subunit 3 (215) Similarity Search Subunit 4 (215) Similarity Search

Status:

Investigational

Class:

PROTEIN

Zilurgisertib Fumarate

1R349830SB

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Status:

Investigational

Class (Stereo):

CHEMICAL (ABSOLUTE)

Fidrisertib Succinate

WU1JNY62EH

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Status:

Investigational

Class (Stereo):

CHEMICAL (ABSOLUTE)

BCX-9250

E2N83U70IX

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Status:

Designated

Class (Stereo):

CHEMICAL (ABSOLUTE)

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