Acute Porphyria – Pipeline Review, 2024

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Table of Content:

1. Introduction

1.1.Report Overview

2. Acute Porphyria - Overview

3. Executive Summary

4. Acute Porphyria : PipelineAssessment

4.1.By Stage of Development

4.2.By Route of Administration

4.3.By Drug Class

4.4.By Molecule Type

4.5.By Therapy Area / Indication

4.6.By Drug Target

4.7.By Sponsor

5. Acute Porphyria: Company & DrugProfiles

5.1.Clinical Stage

5.1.1. Panhematin - Recordati RareDiseases Inc.

5.1.1.1. Company Overview

5.1.1.2. Product Description

5.1.1.3. R&D Status & DevelopmentActivities

5.1.1.4. Mechanism of Action

5.1.1.5. Molecule Type

5.1.1.6. Stage of Development

5.1.1.7. Indications

5.1.1.8. Route of Administration

5.1.1.9. Funding

5.1.2. givosiran (ALN-AS1) - AlnylamPharmaceuticals

5.1.2.1. Company Overview

5.1.2.2. Product Description

5.1.2.3. R&D Status &Development Activities

5.1.2.4. Mechanism of Action

5.1.2.5. Molecule Type

5.1.2.6. Stage of Development

5.1.2.7. Indications

5.1.2.8. Route of Administration

5.1.2.9. Funding

5.1.3. RTX-ALAD - AmpioPharmaceuticals Inc.

5.1.3.1. Company Overview

5.1.3.2. Product Description

5.1.3.3. R&D Status &Development Activities

5.1.3.4. Mechanism of Action

5.1.3.5. Molecule Type

5.1.3.6. Stage of Development

5.1.3.7. Indications

5.1.3.8. Route of Administration

5.1.3.9. Funding

5.1.4. Others

5.2.Preclinical

5.2.1. Company Overview

5.2.2. Product Description

5.2.3. R&D Status &Development Activities

5.2.4. Mechanism of Action

5.2.5. Molecule Type

5.2.6. Indications

5.2.7. Route of Administration

5.2.8. Funding

6. Acute Porphyria: An Overview onDormant & Discontinued Pipeline Candidates

6.1.Overview

6.2.Product Description

6.3.Reason for Discontinuation

7. Acute Porphyria: Additional KeyInsights

7.1.Epidemiology Overview: Acute Porphyria

7.2.Current Market Scenario: Acute Porphyria Therapeutics

8. Acute Porphyria: News, PressReleases and Conference Details

Note:

1) This Table of Content is tentative and subject to change as the research progresses.

2) Please note that the drug candidates included as examples were in the pipeline when this TOC was designed and their status might have changed since then.